REGENXBIO Inc (RGNX)

REGENXBIO Announces First Patient Dosed in Phase IIb/III NAAVIGATE Trial of Surabgene Lomparvovec in Diabetic Retinopathy; Company to Receive $100 Million MilestoneJune 29, 2026 7:05 AM
PR Newswire (US) New long-term data from diabetic retinopathy and wet AMD programs to be presented at American Society of Retina Specialists (ASRS) 2026ROCKVILLE, Md., June 29, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the first patient has been dosed in the Phase IIb/III NAAVIGATE clinical trial of investigational surabgene lomparvovec (sura-vec, ABBV-RGX-314) in diabetic retinopathy (DR) using suprachoroidal delivery. REGENXBIO will receive $100 million from AbbVie for this milestone."Dosing the first patient in the Phase IIb/III study is a significant milestone in our commitment with AbbVie to bring a one-time gene therapy to patients who continue to face progressive and vision-threatening complications across multiple chronic retinal diseases," said Steve Pakola, M.D., Chief Medical Officer, REGENXBIO. "We are excited to advance sura-vec for DR, following the two-year data that reinforced the durable and disease-modifying impact that sura-vec has the potential to deliver with a single administration. The opportunity to prevent disease progression before vision damage occurs is critical for this population, and we are collaborating closely with the investigators and AbbVie to progress this innovative treatment option.""Diabetic retinopathy is a leading cause of vision loss among working adults, and the goal as a physician is always to prevent the disease from progressing and treat patients before they lose their vision. Current treatment options often require ongoing, repeated interventions, which can lead to undertreatment and progressive vision-threatening complications," said Arshad Khanani, M.D., M.A., FASRS, Director of Clinical Research at Sierra Eye Associates, Reno, NV. "A one-time, in-office treatment has the potential to transform DR management by improving long-term outcomes while making earlier intervention both practical and scalable for patients worldwide."NAAVIGATE is a Phase IIb/III multicenter, randomized, masked, sham-controlled study to evaluate the safety and efficacy of sura-vec in subjects with non-proliferative DR (NPDR) without center-involved diabetic macular edema (CI-DME). Subjects will receive sura-vec at 1.0x1012 genome copies (GC)/eye, which was evaluated as dose level 3 in the Phase II ALTITUDE® trial of sura-vec, and short-course topical prophylactic steroids. The primary endpoint is > 2-step improvement on the diabetic retinopathy severity scale (DRSS) at one year. The Phase IIb portion, operationalized by REGENXBIO, is expected to enroll approximately 135 participants in the United States.REGENXBIO plans to present two and a half-year data from the long term follow up study of the ALTITUDE trial at the ASRS 44th Annual Meeting in July 2026. In two-year dose level 3 data from the ALTITUDE trial, sura-vec demonstrated a durable efficacy profile and was well tolerated with no intraocular inflammation observed (n=15), supporting the potential of an in-office gene therapy approach.Five-year long-term follow-up data from the Phase I/IIa study of subretinal sura-vec for wet AMD will also be presented at ASRS. REGENXBIO expects to announce topline data with AbbVie from the ATMOSPHERE® and ASCENT® pivotal trials of sura-vec using subretinal delivery in Q4 2026.About Surabgene Lomparvovec (sura-vec, ABBV-RGX-314)
Sura-vec is a one-time investigational gene therapy designed to deliver sustained treatment effect in wet AMD, diabetic retinopathy and other chronic retinal conditions. Sura-vec uses the NAV® AAV8 vector to encode an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). Sura-vec is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.1About Diabetic Retinopathy
Diabetic retinopathy (DR) is the leading cause of vision loss in adults between 24 and 75 years of age worldwide.2 DR affects nearly 10 million people in the United States alone.3 The spectrum of DR severity ranges from non-proliferative diabetic retinopathy (NPDR) to proliferative diabetic retinopathy (PDR).4 As DR progresses, a large proportion of patients develop vision threatening complications, including diabetic macular edema (DME) and neovascularization that can lead to blindness.4 Current treatment options for patients with NPDR typically include "watchful waiting" or anti-VEGF treatment. For patients with PDR, current treatment options include anti-VEGF treatment or retinal laser; surgical treatment may be required for advanced PDR.2ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie, and NAVSUNLI™ (clemidsogene lanparvovec-sngl, RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.REGENXBIO.com.FORWARD-LOOKING STATEMENTS
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials, the timing, availability and interpretation of clinical data. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including risks related to the FDA's review process, the timing of enrollment, commencement, completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of any milestone payments, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2025, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at WWW.SEC.GOV. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.Zolgensma® is a registered trademark of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO.Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.comInvestors:
George E. MacDougall
Investor Relations
IR@regenxbio.com _______________________________
*These are interim results from analyses performed by REGENXBIO for an ongoing trial.
1 Penn JS, Madan A, Caldwell RB, et al. Vascular endothelial growth factor in eye disease. Prog Retin Eye Res. 2008;27(4):331-71.
2 Cheung N, Mitchell P, Wong TY. Diabetic retinopathy. Lancet. 2010;376(9735):124–36.
3 Lundeen EA, Burke-Conte Z, Rein DB, Wittenborn JS, Saaddine J, Lee AY, Flaxman AD. Prevalence of Diabetic Retinopathy in the US in 2021. JAMA Ophthalmology. 2023;141(8):747-754.
4 Berrocal MD, Alexandra Acabá. Current Management of Diabetic Retinopathy, 2018  View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-first-patient-dosed-in-phase-iibiii-naavigate-trial-of-surabgene-lomparvovec-in-diabetic-retinopathy-company-to-receive-100-million-milestone-302812721.htmlSOURCE REGENXBIO Inc. Original: REGENXBIO Announces First Patient Dosed in Phase IIb/III NAAVIGATE Trial of Surabgene Lomparvovec in Diabetic Retinopathy; Company to Receive $100 Million Milestone

REGENXBIO Completes Dosing in Confirmatory Study of RGX-202, Marking Completion of Registrational Development Program and Supporting Planned BLA Submission in Q3 2026June 24, 2026 7:05 AM
PR Newswire (US) Confirmatory study completed ahead of schedule due to strong patient demand and robust investigator interestOn track to initiate BLA in Q3 2026 under the accelerated approval pathway supporting potential approval in 2H 2027ROCKVILLE, Md., June 24, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the successful completion of dosing in the confirmatory study of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy. This milestone positions REGENXBIO to initiate a Biologics License Application (BLA) under the accelerated approval pathway in Q3 2026 for a potential approval by the U.S. Food and Drug Administration (FDA) in the second half of 2027.The BLA submission will include a substantial safety dataset from the AFFINITY DUCHENNE® study of RGX-202 pivotal and confirmatory studies (n=63) and efficacy data from the pivotal portion (n=30). The BLA is expected to include 12-month functional data for at least half of the total participants in the pivotal study."Completing the enrollment of our confirmatory study ahead of schedule is a significant milestone that underscores the urgent unmet need in Duchenne and brings us another step closer to advancing RGX-202 towards approval via the accelerated approval pathway," said Curran Simpson, President and Chief Executive Officer of REGENXBIO. "We are encouraged by the recent FDA trends in rare disease development, including our collaborative discussion with FDA regarding our Hunter Syndrome program, demonstrating that the accelerated approval pathway remains available and supported for rare diseases. The RGX-202 pivotal dataset directly aligns with the established accelerated approval criteria: the magnitude of clinical effect seen in functional improvement from baseline, correlation between the biomarker and functional outcomes, and a differentiated safety profile. For all of these reasons, we believe we are well positioned to deliver RGX-202 as the next approved gene therapy for Duchenne patients."In the recent topline pivotal dataset, RGX 202 met the primary endpoint of 10% microdystrophin expression in >93% of patients at Week 12. Additionally,Functional data from patients who reached 12-month assessments (n=9) showed a large magnitude of effect in multiple timed function tests and the North Star Ambulatory Assessment (NSAA).RGX-202 was well tolerated and demonstrated a favorable safety profile.RGX-202 demonstrated strong correlation between microdystrophin expression at Week 12 and interim functional improvement at one year, supporting the use of RGX-202 microdystrophin as a surrogate endpoint reasonably likely to predict clinical benefit.In anticipation of potential FDA approval, the Company is preparing for launch, leveraging its fully end-to-end, commercial-ready in-house manufacturing at the REGENXBIO Manufacturing Innovation Center in Rockville, Md., where production intended for commercial supply was initiated last year. About RGX-202
RGX-202 is designed to address the underlying cause of Duchenne by enabling targeted expression of a novel microdystrophin that is closest to naturally occurring dystrophin. It is the only microdystrophin that includes the C-Terminal domain, which has been shown to protect and preserve muscle function. The differentiated therapeutic approach behind RGX-202 includes a novel construct, a proactive immune suppression regimen, and a suspension-based manufacturing process that delivers industry-leading product purity levels. These factors enable RGX-202 to be delivered at the highest dose among gene therapy programs, with the goal of providing maximum functional benefit and durability while maintaining a favorable safety profile for patients.  About Duchenne Muscular Dystrophy
Duchenne is a severe, progressive, degenerative muscle disease, affecting 1 in 3,500 to 5,000 boys born each year worldwide. Duchenne is caused by mutations in the Duchenne gene which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy and premature death.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie, and NAVSUNLI™ (clemidsogene lanparvovec-sngl, RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.REGENXBIO.com.FORWARD-LOOKING STATEMENTS
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials, the timing, availability and interpretation of clinical data. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including risks related to the FDA's review process, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2025, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at WWW.SEC.GOV. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.Zolgensma® is a registered trademark of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO.Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.comInvestors:
George E. MacDougall
Investor Relations
IR@regenxbio.com  View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-completes-dosing-in-confirmatory-study-of-rgx-202-marking-completion-of-registrational-development-program-and-supporting-planned-bla-submission-in-q3-2026-302808616.htmlSOURCE REGENXBIO Inc. Original: REGENXBIO Completes Dosing in Confirmatory Study of RGX-202, Marking Completion of Registrational Development Program and Supporting Planned BLA Submission in Q3 2026

REGENXBIO to Participate in Upcoming Investor ConferencesMay 18, 2026 4:05 PM
PR Newswire (US) ROCKVILLE, Md., May 18, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following investor conferences:2026 RBC Capital Markets Global Healthcare Conference
Fireside Chat: Wednesday, May 20 at 9:30am ET
Location: New York, NYStifel 2026 Virtual Ophthalmology Forum
Fireside Chat: Tuesday, May 26 at 9:30am ET
Location: VirtualLive webcasts of select events can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. Archived replays of the webcasts will be available for approximately 30 days following the events.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit WWW.REGENXBIO.COM.Contacts:Dana Cormack
Corporate Communications
dcormack@regenxbio.comInvestors:
George E. MacDougall
Investor Relations
IR@regenxbio.com   View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-to-participate-in-upcoming-investor-conferences-302775147.htmlSOURCE REGENXBIO Inc. Original: REGENXBIO to Participate in Upcoming Investor Conferences

Regenxbio Shares Rise After Positive Duchenne Trial ResultsMay 14, 2026 10:09 AM
IH Market News Regenxbio (NASDAQ:RGNX) shares gained more than 4% in premarket trading Thursday after investors focused on encouraging clinical trial results for the company’s Duchenne muscular dystrophy therapy, despite weaker-than-expected quarterly financial results. Earnings and Revenue Miss Expectations Regenxbio reported a first-quarter loss of $1.72 per share, wider than analyst expectations of a $1.34 loss per share.Revenue totaled $6.39 million, well below the consensus estimate of $25.8 million.The company’s revenue declined 93% year-on-year from $89.0 million in the first quarter of 2025.Regenxbio said the drop was largely due to a $70 million upfront license payment from Nippon Shinyaku recognized in the prior-year period, as well as a $12.2 million reduction in ZOLGENSMA royalty revenue after certain licensed U.S. patents expired in January 2026. Duchenne Trial Meets Primary Endpoint Investor sentiment improved after the company announced positive topline results from the pivotal Phase III AFFINITY DUCHENNE trial evaluating RGX-202.According to Regenxbio, the study achieved its primary endpoint with high statistical significance.The company said 93% of patients reached RGX-202 microdystrophin expression levels above 10%, with a reported p-value of less than 0.0001.The treatment also demonstrated a statistically significant relationship between microdystrophin expression and functional improvement. CEO Highlights Transformational Year President and chief executive Curran Simpson said the company continues to make progress across its late-stage pipeline.“REGENXBIO enters a transformative year with positive momentum, reaching significant late-stage milestones to support our potential first- and best-in-class gene therapies,” Simpson said. Research Spending Continues to Increase Research and development expenses rose to $57.3 million from $53.1 million in the prior-year quarter.The increase was mainly linked to clinical trial costs associated with RGX-202, along with higher personnel expenses.General and administrative expenses also increased modestly to $21.3 million from $20.3 million a year earlier. Cash Position Expected to Support Operations Into 2027 As of March 31, 2026, Regenxbio held $150.5 million in cash, cash equivalents and marketable securities.The company said its current liquidity is expected to fund operations into early 2027.This outlook excludes any potential future milestone payments from partners.Regenxbio also said it expects to receive a $100 million milestone payment from AbbVie once the first patient is dosed in the Phase IIb portion of the NAAVIGATE study, which is expected during the second quarter of 2026.Regenxbio stock price Original: Regenxbio Shares Rise After Positive Duchenne Trial Results

REGENXBIO Announces Positive Topline Results from Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202May 14, 2026 7:00 AM
PR Newswire (US) Achieved primary endpoint with high statistical significance; 93% of patients achieved microdystrophin expression above 10% (p

REGENXBIO Reports First Quarter 2026 Financial Results and Operational HighlightsMay 14, 2026 7:10 AM
PR Newswire (US) Company announced positive topline results from pivotal Phase III AFFINITY DUCHENNE® study of RGX-202Primary endpoint achieved with high statistical significanceStatistically significant correlation between RGX-202 microdystrophin expression and functional improvement (NSAA, n=9), supporting validity of surrogate endpoint Surabgene lomparvovec (sura-vec, ABBV-RGX-314) on track toward key catalysts Sites activated in pivotal Phase IIb/III study for diabetic retinopathy; first patient dosed expected Q2 2026Subretinal wet AMD topline pivotal data expected in Q4 2026Clinical hold lifted for RGX-121Webcast today at 8:00 a.m. ET to discuss RGX-202 topline pivotal data and Q1 2026 earnings ROCKVILLE, Md., May 14, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the first quarter ended March 31, 2026."REGENXBIO enters a transformative year with positive momentum, reaching significant late-stage milestones to support our potential first- and best-in-class gene therapies," said Curran Simpson, President and Chief Executive Officer, REGENXBIO. "On our mission to bring meaningful new therapies to rare disease communities facing limited options, we remain focused on advancing RGX-202 for Duchenne toward potential BLA submission and are excited to share topline pivotal data in our webcast today; we also continue engaging with the FDA regarding a potential path forward for RGX-121 for Hunter syndrome. In chronic retinal diseases, we are executing against our multi-indication strategy and preparing for the Phase 3 wet AMD readout with partner AbbVie. Additionally, we continue harnessing internal manufacturing capabilities to support anticipated commercial needs. We remain well-positioned to capitalize on several near-term, high-value opportunities and bring these highly differentiated treatments to patients." PROGRAM HIGHLIGHTS AND MILESTONESNeuromuscular Disease: RGX-202 is a potential best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to address the underlying cause of Duchenne by enabling targeted expression of a novel microdystrophin that is closest to naturally occurring dystrophin. It is the only microdystrophin that includes the C-Terminal domain, which has been shown to protect and preserve muscle function. The differentiated therapeutic approach behind RGX-202 includes a novel construct, a proactive immune suppression regimen, and a suspension-based manufacturing process that delivers industry-leading product purity levels. RGX-202 is designed for improved muscle function, durability and positive safety outcomes and is being evaluated in the Phase I/II/III AFFINITY DUCHENNE® trial in ambulatory patients aged 1+.REGENXBIO today announced positive topline results from the pivotal Phase III AFFINITY DUCHENNE trial of RGX-202, including primary endpoint (n=30 at Week 12), interim safety (n=31), and interim functional data (n=9 at 12 months):[1]Achieved primary endpoint with high statistical significance; 93% of patients achieved RGX-202 microdystrophin expression above 10% (p

REGENXBIO Announces Presentations at the American Society of Gene & Cell Therapy 2026 Annual MeetingMay 7, 2026 7:05 AM
PR Newswire (US) ROCKVILLE, Md., May 7, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the 2026 American Society of Gene & Cell Therapy Annual Meeting ("ASGCT 2026") taking place May 11-15, 2026, in Boston, Massachusetts. Oral Presentations:
The oral presentations include an overview of recent advancements in production titer and product quality achieved through REGENXBIO's NAVXpress® manufacturing platform and an encore presentation of topline pivotal data from RGX-202, the company's next generation gene therapy program for Duchenne muscular dystrophy.Title: Advancing AAV manufacturability toolbox for early programs and beyond
Presenter: Metewo Selase Kosi Enuameh, Ph.D., Associate Director, Vector Core at REGENXBIO
Presentation: 266
Session: AAV Critical Quality Attributes
Date/Time: Thursday, May 14, 8:45 – 9:00am ET
Location: MCEC Room 204AB (Level 2)Title: RGX-202: Investigational gene therapy for Duchenne Muscular Dystrophy
Presenter: Aravindhan Veerapandiyan, M.D., Director of the Comprehensive Neuromuscular Program, PPMD Certified Duchenne Care Center, and Co-Director of the Muscular Dystrophy Association Care Center at Arkansas Children's Hospital
Presentation: 506
Session: In vivo clinical trials in eye and muscle disorders
Date/Time: Friday, May 15, 4:30 – 4:45pm ET
Location: MCEC Room 210ABC (Level 2)Poster Presentations:
These presentations demonstrate REGENXBIO's continued leadership in the discovery and advancement of next-generation gene therapies for rare and retinal diseases, and highlight the company's unique capsid engineering and translational capabilities.Title: NVG82, a capsid engineered for enhanced outer retinal gene transfer when administered in the suprachoroidal space, demonstrates superior on-target activity compared to AAV8
Poster: 1031
Presenter: Brendan Lilley, Ph.D., Director of Ophthalmology Research, REGENXBIO
Location: MCEC Exhibit and Poster Hall (Halls B2-C, Exhibit level)
Date/Time: Tuesday, May 12, 5:00 – 6:30pm ETTitle: Examination of Route-Dependent Ocular Transduction in Rodent Models by the Novel AAV Capsid NVG82
Poster: 3031
Presenter: Huzzatul Mursalin, Ph.D., Scientist II, Gene Therapy Research, REGENXBIO
Location: MCEC Exhibit and Poster Hall (Hall B2-C, Exhibit level)
Date/Time: Thursday, May 14, 5:00 – 6:30pm ETTitle: Development of an engineered hybrid AAV with reduced liver and DRG transduction and high productivity
Poster: 3030
Presenter: Samantha Yost, Ph.D., Senior Scientist, Gene Therapy Research, REGENXBIO
Location: MCEC Exhibit and Poster Hall (Hall B2-C, Exhibit level)
Date/Time: Thursday, May 14, 5:00 – 6:30pm ETTitle: Single-nuclei transcriptomic analysis of skeletal muscle mRNA in mdx mice treated with RGX-202, an AAV vector encoding micro-dystrophin
Poster: 3499
Presenter: Justin Glenn, Ph.D., Principal Scientist, Gene Therapy Research, REGENXBIO
Location: MCEC Exhibit and Poster Hall (Hall B2-C, Exhibit level)
Date/Time: Thursday, May 14, 5:00 – 6:30pm ETAll presentations will be available on the Publications page of REGENXBIO's website, www.regenxbio.com.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit WWW.REGENXBIO.COM.Contacts:Dana Cormack
Corporate Communications
dcormack@regenxbio.comInvestors:
George E. MacDougall
Investor Relations
IR@regenxbio.com   View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-presentations-at-the-american-society-of-gene--cell-therapy-2026-annual-meeting-302765129.htmlSOURCE REGENXBIO Inc. Original: REGENXBIO Announces Presentations at the American Society of Gene & Cell Therapy 2026 Annual Meeting

REGENXBIO to Host Webcast on May 14 to Discuss Topline Results from Pivotal Trial of RGX-202 for Duchenne Muscular DystrophyMay 6, 2026 7:05 AM
PR Newswire (US) -- Company will also report first quarter 2026 financial results and operational highlights --ROCKVILLE, Md., May 6, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast on Thursday, May 14, 2026, at 8:00 a.m. ET to discuss topline results from the pivotal trial of RGX-202, the company's next-generation investigational gene therapy for the treatment of Duchenne muscular dystrophy.The webcast will feature leading Duchenne physicians including Aravindhan Veerapandiyan, M.D., Director of the Comprehensive Neuromuscular Program and Co-Director of the Muscular Dystrophy Association Care Center at Arkansas Children's Hospital, Carolina Tesi-Rocha, M.D., Clinical Professor, Neurology, Stanford School of Medicine, Stanford Children's Health, and Diana Castro, M.D., Board Certified Neurologist and Neuromuscular Physician, Founder and Director Neurology and Neuromuscular Care Center and Founder and Director Neurology Rare Disease Center.The company will also report financial results and operational highlights for the first quarter ended March 31, 2026 pre-market Thursday, May 14.The live webcast can be accessed here and in the Investor section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit WWW.REGENXBIO.COM.Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.comInvestors:
George E. MacDougall
Investor Relations
IR@regenxbio.com  View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-to-host-webcast-on-may-14-to-discuss-topline-results-from-pivotal-trial-of-rgx-202-for-duchenne-muscular-dystrophy-302763647.htmlSOURCE REGENXBIO Inc. Original: REGENXBIO to Host Webcast on May 14 to Discuss Topline Results from Pivotal Trial of RGX-202 for Duchenne Muscular Dystrophy

REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNXApril 14, 2026 8:05 PM
PR Newswire (US)

LOS ANGELES, April 14, 2026 /PRNewswire/ -- The DJS Law Group reminds investors of a class action lawsuit against REGENXBIO Inc. ("Regenxbio" or "the Company") (NASDAQ: RGNX) for violations of §§10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by the U.S. Securities and Exchange Commission.Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointments. Appointment as lead plaintiff is not required to partake in any recovery.CLASS PERIOD: February 9, 2022 to January 27, 2026DEADLINE: April 14, 2026CASE DETAILS: According to the Complaint, the Company made false and misleading statements to the market. Regenxbio made consistently positive statements to the market about the safety and efficacy of its RGX-111 product candidate. Meanwhile, it was concealing adverse information on safety and efficacy from investors. The FDA placed a clinical hold on RGX-111 when it was revealed a clinical trial participant had developed a tumor. Based on these facts, Regenxbio's public statements were false and materially misleading throughout the class period.If you are a shareholder who suffered a loss, contact us to participate.WHY DJS LAW GROUP? DJS Law Group's primary focus is to enhance investor return through balanced counseling and aggressive advocacy. We specialize in securities class actions, corporate governance litigation, and domestic/international M&A appraisals. Our clients are some of the largest and most sophisticated hedge funds and alternative asset managers in the world. The litigation claims of our clients are extraordinarily valuable assets that demand respect, focus, and results.Join the case to recover your losses.This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.CONTACT:David J. Schwartz
DJS Law Group
274 White Plains Road, Suite 1
Eastchester, NY 10709
Phone: 914-206-9742
Email: David@djslawllp.com



View original content:https://www.prnewswire.com/news-releases/regenxbio-inc-sued-for-securities-law-violations---contact-the-djs-law-group-to-discuss-your-rights--rgnx-302740174.htmlSOURCE DJS Law Group LLP

Original: REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNX

REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNXApril 6, 2026 2:57 AM
PR Newswire (US)

LOS ANGELES, April 6, 2026 /PRNewswire/ -- The DJS Law Group reminds investors of a class action lawsuit against REGENXBIO Inc. ("Regenxbio" or "the Company") (NASDAQ: RGNX) for violations of §§10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by the U.S. Securities and Exchange Commission.Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointments. Appointment as lead plaintiff is not required to partake in any recovery.CLASS PERIOD: February 9, 2022 to January 27, 2026DEADLINE: April 14, 2026CASE DETAILS: According to the Complaint, the Company made false and misleading statements to the market. Regenxbio made consistently positive statements to the market about the safety and efficacy of its RGX-111 product candidate. Meanwhile, it was concealing adverse information on safety and efficacy from investors. The FDA placed a clinical hold on RGX-111 when it was revealed a clinical trial participant had developed a tumor. Based on these facts, Regenxbio's public statements were false and materially misleading throughout the class period.If you are a shareholder who suffered a loss, contact us to participate.WHY DJS LAW GROUP? DJS Law Group's primary focus is to enhance investor return through balanced counseling and aggressive advocacy. We specialize in securities class actions, corporate governance litigation, and domestic/international M&A appraisals. Our clients are some of the largest and most sophisticated hedge funds and alternative asset managers in the world. The litigation claims of our clients are extraordinarily valuable assets that demand respect, focus, and results.Join the case to recover your losses.This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.CONTACT:David J. SchwartzDJS Law Group274 White Plains Road, Suite 1Eastchester, NY 10709Phone: 914-206-9742Email: David@djslawllp.com



View original content:https://www.prnewswire.com/news-releases/regenxbio-inc-sued-for-securities-law-violations---contact-the-djs-law-group-to-discuss-your-rights--rgnx-302734479.htmlSOURCE DJS Law Group LLP

Original: REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNX

REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNXMarch 30, 2026 4:06 AM
PR Newswire (US)

LOS ANGELES, March 30, 2026 /PRNewswire/ -- The DJS Law Group reminds investors of a class action lawsuit against REGENXBIO Inc. ("Regenxbio" or "the Company") (NASDAQ: RGNX) for violations of §§10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by the U.S. Securities and Exchange Commission.Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointments. Appointment as lead plaintiff is not required to partake in any recovery.CLASS PERIOD: February 9, 2022 to January 27, 2026DEADLINE: April 14, 2026CASE DETAILS: According to the Complaint, the Company made false and misleading statements to the market. Regenxbio made consistently positive statements to the market about the safety and efficacy of its RGX-111 product candidate. Meanwhile, it was concealing adverse information on safety and efficacy from investors. The FDA placed a clinical hold on RGX-111 when it was revealed a clinical trial participant had developed a tumor. Based on these facts, Regenxbio's public statements were false and materially misleading throughout the class period.If you are a shareholder who suffered a loss, contact us to participate.WHY DJS LAW GROUP? DJS Law Group's primary focus is to enhance investor return through balanced counseling and aggressive advocacy. We specialize in securities class actions, corporate governance litigation, and domestic/international M&A appraisals. Our clients are some of the largest and most sophisticated hedge funds and alternative asset managers in the world. The litigation claims of our clients are extraordinarily valuable assets that demand respect, focus, and results.Join the case to recover your losses.This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.CONTACT:David J. Schwartz
DJS Law Group
274 White Plains Road, Suite 1
Eastchester, NY 10709
Phone: 914-206-9742
Email: David@djslawllp.com



View original content:https://www.prnewswire.com/news-releases/regenxbio-inc-sued-for-securities-law-violations---contact-the-djs-law-group-to-discuss-your-rights--rgnx-302728307.htmlSOURCE DJS Law Group LLP

Original: REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNX

REGENXBIO REPORTS NEW POSITIVE INTERIM DATA FROM PHASE I/II AFFINITY DUCHENNE® TRIAL OF RGX-202March 11, 2026 11:45 AM
PR Newswire (US)

Investigational RGX-202 continues to demonstrate evidence of positively changing disease trajectory for Duchenne Pivotal dose participants exceeded external controls across functional measures at 1 year, including participants aged 8+Cardiac MRI data for pivotal dose patients demonstrated stability at 1 yearFavorable safety profile continued with no serious adverse events or adverse events of special interest observed in Phase I/II studyNew data from multiple measures supported liver safety in pivotal patientsPivotal topline data expected Q2 2026ROCKVILLE, Md., March 11, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy. Trial investigator Carolina Tesi-Rocha, M.D., Clinical Professor, Neurology, Stanford School of Medicine, Stanford Children's Health, is presenting this data, including new functional, safety, biomarker, and cardiac MRI measures, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference."Today's new Phase I/II interim data demonstrates continued positive impact on function, stable cardiac health, and a favorable safety profile, highlighting the potential of RGX-202 to be a differentiated gene therapy option for Duchenne," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "Our proactive, comprehensive approach to safety combined with our novel microdystrophin construct are supported by the sustained safety and durable functional outcomes through two years post-treatment. As we approach our topline pivotal data readout in early Q2, we are very encouraged by this thorough new dataset and the opportunity to advance RGX-202 as a potential meaningful treatment option for patients.""Duchenne is a devastating, degenerative disease that robs children of muscle strength and independence over time, and I'm pleased to see the continued positive safety and encouraging efficacy profile of RGX-202," said Dr. Tesi-Rocha. "These positive Phase I/II data, including biomarker results, functional improvement, cardiac stability, and liver safety provide a clearer picture of RGX-202's potential impact across key measures of health in Duchenne."AFFINITY DUCHENNE Phase I/II Interim Data Updates (data cut: January 5, 2026)Functional Data
In the interim functional results from seven participants treated at the pivotal dose level (2x1014 GC/kg), aged approximately 6 to 12 years at dosing, RGX-202 continues to demonstrate evidence of positively impacting disease trajectory on North Star Ambulatory Assessment (NSAA) and all timed function tests (Time to Stand, 10 Meter Walk-run, Time to Climb) at one year.Functional outcomes were analyzed using multiple validated methods to estimate expected disease progression without treatment, including the cTAP disease progression model and external control comparisons using coarsened exact matching and propensity score weighting. Propensity score weighting is the primary analysis method specified in the SAP for the pivotal trial.The pivotal dose participants demonstrated improved performance across all timed function tests and NSAA when compared to external control using propensity score weighting. The 95% confidence interval demonstrates favorability to RGX-202.  [Figure 1]On NSAA, pivotal dose participants exceeded expected disease trajectory and external controls. Notably, five of the seven participants were aged 8+ at dosing, when functional decline is expected. At one year, participants (n=7) improved an average of +4.9 points compared to cTAP. The older participants (n=5) improved an average of +5.2 points compared to cTAP. [Figure 2]Additionally, dose level 1 (1x1014 GC/kg) participants (n=3) exceeded expected disease trajectory and improved an average of +5.6 points compared to cTAP at two years. [Figure 3]Cardiac Function
Pivotal dose participants demonstrated cardiac stability at one year post-treatment as measured by MRI endpoints, including mean left ventricular ejection fraction, global circumferential strain, and fibrosis assessed by late gadolinium enhancement.
Baseline12 MonthsSubjects (N)77AgeMean (range)8.7*(5.8-12.1)**9.7(6.8-13.1)Left Ventricular Ejection FractionMean (range)Median61.7%(54-72)**60%61.6%(57-74)60%Global Circumferential StrainMean (range)Median-20.4%(-22% to -19%)-20.4%-20.9%(-23% to -17%)-21.5%Late Gadolinium Enhancement (LGE)1 participant with
fibrosisNo change from baselineBiomarker Data
Biomarker data from the Phase I/II study continues to support consistent, high expression and transduction of RGX-202 microdystrophin (n=13). New data from an additional patient, aged 3.6 at dosing, had a microdystrophin expression level of 51.2% at Week 12. The primary endpoint in the pivotal phase of AFFINITY DUCHENNE is the proportion of participants whose RGX-202 microdystrophin expression is >10% at Week 12.RGX-202 was appropriately localized to the sarcolemma, demonstrating that the differentiated construct with the inclusion of the C-Terminal (CT) domain is appropriately targeting the muscle.Safety and Tolerability Data
New interim safety data from all Phase I/II pivotal dose participants aged 1 to

uniQure shares jump 36% after news of FDA gene therapy chief’s departureMarch 9, 2026 10:14 AM
IH Market News
Shares of uniQure BV (NASDAQ:QURE) surged 36% on Monday after reports emerged that the head of the U.S. Food and Drug Administration’s vaccines and gene therapies division will step down, triggering several analyst upgrades across the biotechnology sector. Other companies in the space also rallied, with REGENXBIO (NASDAQ:RGNX) rising 13% and Biohaven (NYSE:BHVN) gaining 23%.Vinay Prasad, who currently leads the FDA’s Center for Biologics Evaluation and Research division overseeing vaccines and gene therapies, is expected to leave the agency next month to return to academia. His tenure has been viewed by some industry observers as a period of heightened regulatory scrutiny for gene therapy developers.RBC Capital analyst Luca Issi upgraded uniQure from Sector Perform to Outperform and raised the firm’s price target to $35.00 from $11.00. The company had recently faced regulatory setbacks after the FDA indicated it would not approve its Huntington’s disease therapy based solely on comparisons to natural history data, a development uniQure described as a “key shift” from earlier feedback given before changes in FDA leadership.“We believe that Prasad’s departure is likely to open up a more balanced discussion on risk/reward for HD,” Issi wrote, adding that he now estimates a 50% probability that the therapy could eventually gain approval.Stifel analyst Paul Matteis also reacted positively to the news, calling Prasad’s exit “a big win for biotech, especially for companies in the rare disease space.” Matteis added that several gene therapy and rare disease developers had been under pressure as part of what he described as the “FDA risk off” basket, with Prasad reportedly overriding review teams to recommend negative outcomes in certain cases.An analyst at Truist noted that Prasad’s leadership “marked a sharp departure from the more flexible regulatory approach for rare and serious diseases” seen under his predecessor Peter Marks. According to the analyst, several companies encountered evolving regulatory expectations during development discussions while Prasad was in charge.uniQure is scheduled to hold a Type B meeting with the FDA in the second quarter.uniQure stock price

Original: uniQure shares jump 36% after news of FDA gene therapy chief’s departure

RGNX'S "TRANSFORMATIONAL" PROMISE BECAME A $2.40 LOSS FOR INVESTORS: SUEWALLSTMarch 5, 2026 2:25 PM
PR Newswire (US)

Promise vs. Reality: The REGENXBIO Gene Therapy Performance GapNEW YORK, March 5, 2026 /PRNewswire/ -- "Very promising results." That is how REGENXBIO, Inc. (NASDAQ: RGNX) characterized its RGX-111 gene therapy candidate as recently as January 2025. By January 28, 2026, the FDA had placed a clinical hold on the program after a CNS tumor was discovered in a trial participant, and RGNX shareholders lost $2.40 per share in a single trading session. Find out if you can recover your investment losses or contact Joseph E. Levi, Esq. at jlevi@SueWallSt.com or (888) SueWallSt.Shares fell 17.8%, declining from $13.41 to $11.01, after the company disclosed the intraventricular CNS tumor and FDA clinical hold. The lead plaintiff deadline is April 14, 2026.The PromiseThroughout the Class Period from February 9, 2022 to January 27, 2026, REGENXBIO projected confidence at every turn. The company described RGX-111 as its "second-most advanced clinical candidate" and a cornerstone of its "5x'25" strategy to have five gene therapies on the market or in late-stage development by 2025. In January 2025, management called RGX-111 and its partner therapy "potentially transformational medicines" when announcing a strategic partnership with Nippon Shinyaku Co., Ltd.The RealityOn January 28, 2026, REGENXBIO revealed that a routine brain MRI identified an intraventricular CNS tumor in an asymptomatic five-year-old participant who had received RGX-111 four years earlier. Preliminary genetic analysis detected an AAV vector genome integration event linked to overexpression of a proto-oncogene (PLAG1). The FDA placed clinical holds on both RGX-111 and the related RGX-121 program.The Numbers: Promised vs. ActualPromised: "Well tolerated with no drug-related serious adverse events" (repeated across multiple earnings calls and press releases from 2022 through 2023)Actual: A CNS tumor discovered in a pediatric trial participant, triggering an FDA clinical holdPromised: RGX-111 positioned as a "potentially transformational medicine" with "very promising results" (January 2025 partnership announcement)Actual: The FDA halted clinical development of both RGX-111 and RGX-121 just twelve months laterPromised: Part of a five-therapy commercialization strategy ("5x'25")Actual: De-prioritized in November 2023 under circumstances the lawsuit contends reflected awareness of underlying safety problemsPromised: Fast Track FDA designation and a clear path to regulatory advancementActual: Full clinical hold with causality investigation ongoingWhat the Lawsuit Alleges About the GapThe complaint contends that the abrupt November 2023 decision to de-prioritize RGX-111 and seek "strategic alternatives" reflected internal awareness of safety problems that were not disclosed to investors. Despite this de-prioritization, management continued to describe the therapy's results as "very promising" when announcing the Nippon Shinyaku partnership over a year later."Companies that make specific promises to investors about future performance have an obligation to disclose known risks to those projections. When a gene therapy candidate is repeatedly described as transformational while serious safety signals allegedly go undisclosed, the gap between promise and reality can be measured in shareholder losses." -- Joseph E. Levi, Esq.Speak with an attorney about recovering your RGNX losses or call Joseph E. Levi, Esq. at (212) 363-7500.LEAD PLAINTIFF DEADLINE: April 14, 2026Levi & Korsinsky, LLP is a nationally recognized shareholder rights firm. Over the past 20 years, the firm has secured hundreds of millions of dollars for aggrieved shareholders. Ranked in ISS Top 50 for seven consecutive years.CONTACT:Levi & Korsinsky, LLPJoseph E. Levi, Esq.33 Whitehall Street, 27th FloorNew York, NY 10004jlevi @Icons1



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Original: RGNX'S "TRANSFORMATIONAL" PROMISE BECAME A $2.40 LOSS FOR INVESTORS: SUEWALLST

REGENXBIO Reports Fourth Quarter and Full Year 2025 Financial Results and Operational HighlightsMarch 5, 2026 7:05 AM
PR Newswire (US)

Late-stage gene therapy pipeline for rare and retinal diseases advancing toward key catalysts RGX-202 for Duchenne muscular dystrophy: New Phase I/II data at MDA, topline pivotal results expected early Q2 2026Robust patient enrollment continues in confirmatory trialSurabgene lomparvovec (sura-vec, ABBV-RGX-314) subretinal wet AMD topline pivotal data expected in Q4 2026; first patient dosed in pivotal Phase IIb/III trial in diabetic retinopathy expected in Q2 2026Company to engage FDA on potential path forward for RGX-121Conference call today at 8:00 a.m. ETROCKVILLE, Md., March 5, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and operational highlights for the fourth quarter and year ended December 31, 2025."We are rapidly advancing our late-stage pipeline of gene therapies to treat rare and retinal diseases with significant unmet need, with multiple near-term catalysts in 2026," said Curran Simpson, President and Chief Executive Officer, REGENXBIO. "We will drive continued momentum across our programs – powered by our fully in-house, end-to-end capabilities, commercial-ready manufacturing, and global partners. We remain focused on executing on our mission to deliver meaningful new medicines to patients in need through the curative potential of gene therapy."PROGRAM HIGHLIGHTS AND MILESTONESNeuromuscular Disease: RGX-202 is a potential best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to address the underlying cause of Duchenne by enabling targeted expression of a novel microdystrophin that is closest to naturally occurring dystrophin. It is the only microdystrophin that includes the C-Terminal domain, which has been shown to protect and preserve muscle function. The differentiated therapeutic approach behind RGX-202 includes a novel construct, a proactive immune suppression regimen, and a suspension-based manufacturing process that delivers industry-leading product purity levels. RGX-202 is designed for improved muscle function, durability and safety outcomes for patients.Positive 18-month functional data from patients treated with the pivotal dose in the Phase I/II portion of the AFFINITY DUCHENNE® trial (n=4) were reported in January 2026. All patients exceeded expected disease trajectory on the North Star Ambulatory Assessment (NSAA) using the established cTAP disease progression model. RGX-202 recipients improved an average of 7.4 points compared to cTAP. These same patients improved an average of 6.6 points compared to cTAP at 12 months post-treatment.The Company plans to share additional Phase I/II safety, biomarker, and functional data at the MDA Clinical and Scientific Conference on March 11, 2026.REGENXBIO continues to manufacture RGX-202 intended for commercial supply at its in-house Manufacturing Innovation Center.REGENXBIO expects to share pivotal topline data in early Q2 2026.REGENXBIO plans to request a pre-BLA meeting in mid-2026 to align with FDA on the BLA submission. Additional regulatory interactions with the FDA and European Medical Association (EMA) are planned for 1H 2026.Following the completion of enrollment in the pivotal trial (n=30) in October 2025, the Company continues to enroll in the confirmatory trial (n=30) and expects to have the majority of this trial enrolled at the time of BLA submission.Retinal Disease: Surabgene lomparvovec (sura-vec, ABBV-RGX-314), developed in collaboration with AbbVie, is potentially the first-in-class treatment for wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR).Sura-vec for the Treatment of Wet AMD (Subretinal Delivery)REGENXBIO expects to share topline data with AbbVie from ATMOSPHERE® and ASCENT® pivotal trials of sura-vec using subretinal delivery in Q4 2026.Global regulatory submissions are expected in 2027.Sura-vec for the Treatment of DR (Suprachoroidal Delivery)REGENXBIO is activating U.S. clinical sites in the pivotal Phase IIb/III NAAVIGATE study. The Company will receive a $100 million milestone payment from AbbVie upon first patient dosed in the Phase IIb portion, expected Q2 2026.NAAVIGATE is a Phase IIb/III multicenter, randomized, masked, sham-controlled study to evaluate the safety and efficacy of sura-vec in subjects with non-proliferative DR (NPDR) without center-involved diabetic macular edema (CI-DME). The primary endpoint is ≥2-step improvement on the diabetic retinopathy severity scale (DRSS) at one year. Following an interim analysis, REGENXBIO and AbbVie will initiate a Phase III expansion, which will include two Phase III trials, including a U.S. trial and a parallel global trial, led by AbbVie.All subjects will receive sura-vec at 1.0x1012 genome copies (GC)/eye, which was evaluated as Dose Level 3 in the Phase II ALTITUDE® trial of sura-vec, and short-course topical prophylactic steroids.At two years post treatment with Dose Level 3 and short-course prophylactic topical steroids in ALTITUDE, no intraocular inflammation was observed (n = 15). The majority of subjects achieved DRSS improvement, with 50% achieving ≥2-step improvement without additional DR treatment. Sura-vec at Dose Level 3 also reduced the risk of disease progression, demonstrating a ≥70% risk reduction in vision-threatening complications compared to historical controls.Sura-vec for the Treatment of Wet AMD (Suprachoroidal Delivery)Enrollment is complete in the Phase II AAVIATE® trial.Neurodegenerative Disease: Clemidsogene lanparvovec (RGX-121) is a potential first-in-class treatment for Mucopolysaccharidosis (MPS) Type II, also known as Hunter syndrome. RGX-111 is an investigational one-time treatment for severe MPS I, also known as Hurler syndrome. These programs are partnered with Nippon Shinyaku.In January 2026, the FDA placed a clinical hold on RGX-111 following preliminary analysis of a single case of neoplasm (intraventricular CNS tumor) in a participant treated in its Phase I/II study.The FDA also placed a clinical hold on RGX-121 citing similarities to RGX-111, study populations, and shared risk between the clinical studies.In February 2026, the FDA issued a complete response letter (CRL) for the RGX-121 BLA. REGENXBIO is working to address concerns in the CRL with the goal of resubmitting the BLA.FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable securities were $240.9 million as of December 31, 2025, compared to $244.9 million as of December 31, 2024. The decrease was primarily driven by cash used to fund operating activities during the year ended December 31, 2025, and was partially offset by the $110.0 million upfront payment received under the Nippon Shinyaku partnership in March 2025 and $144.5 million in net proceeds received from the royalty monetization with HCRx in May 2025.Revenues: Revenues were $30.3 million and $170.4 million for the three months and full year ended December 31, 2025, respectively, compared to $21.2 million and $83.3 million for the three months and full year ended December 31, 2024, respectively. The increases were primarily attributable to revenues recognized under our collaboration with Nippon Shinyaku, including $72.9 million of up-front license revenue and $11.8 million of service revenue recognized in 2025, as well as an increase in royalty revenues for Zolgensma and Itvisma.Research and Development Expenses: Research and development expenses were $59.6 million and $228.3 million for the three months and full year ended December 31, 2025, respectively, compared to $50.4 million and $208.5 million for the three months and full year ended December 31, 2024, respectively. The increases were primarily attributable to personnel-related costs and clinical trial expenses, largely driven by RGX-202 pivotal trials, for the fourth quarter and full year 2025, as well as an increase in manufacturing-related expenses Sura-vec, RGX-202 and RGX-121 for the full year 2025.General and Administrative Expenses: General and administrative expenses were $22.4 million and $82.9 million for the three months and full year ended December 31, 2025, respectively, compared to $20.1 million and $76.6 million for the three months and full year ended December 31, 2024, respectively. The increases were largely driven by professional services, consulting and other corporate advisory services.Net Loss: Net loss was $67.1 million, or $1.30 basic and diluted net loss per share, for the three months ended December 31, 2025, compared to a net loss of $51.2 million, or $1.01 basic and diluted net loss per share, for the three months ended December 31, 2024. Net loss was $193.9 million, or $3.76 basic and diluted net loss per share, for the year ended December 31, 2025, compared to a net loss of $227.1 million, or $4.59 basic and diluted net loss per share, for the year ended December 31, 2024.FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and marketable securities of $240.9 million as of December 31, 2025 to fund its operations into early 2027. This cash runway guidance is based on the Company's current operational plans and excludes the impact of any material payments that may potentially be received from partners or licensees upon the achievement of development or regulatory milestones, or upon the approval or commercialization of product candidates, and excludes any additional potential non-dilutive funding opportunities.CONFERENCE CALL
In connection with this announcement, REGENXBIO will host a conference call and webcast at 8:00 a.m. ET today. Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.REGENXBIO.com.FORWARD-LOOKING STATEMENTS
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, regulatory interactions following the complete response letter, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie or Nippon Shinyaku, the monetization of any priority review voucher, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2025, which will be filed with the U.S. Securities and Exchange Commission (SEC) in the first quarter of 2026 and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at WWW.SEC.GOV. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.Zolgensma® and Itvisma® are registered trademarks of Novartis Gene Therapies, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.CONTACTS: 
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com George E. MacDougall
Investor Relations
IR@regenxbio.com REGENXBIO INC.CONSOLIDATED BALANCE SHEETS(unaudited)(in thousands) 


As of December 31,


2025

2024
Assets





Current assets





Cash and cash equivalents
$34,466

$57,526
Marketable securities

195,604


177,161
Accounts receivable

26,379


20,473
Prepaid expenses

11,927


9,067
Other current assets

12,905


13,774
Total current assets

281,281


278,001
Marketable securities

10,785


10,179
Accounts receivable

2,312


474
Property and equipment, net

104,855


117,589
Operating lease right-of-use assets

47,156


53,716
Restricted cash

2,030


2,030
Other assets

4,613


4,000
Total assets
$453,032

$465,989
Liabilities and Stockholders' Equity





Current liabilities





Accounts payable
$21,358

$22,798
Accrued expenses and other current liabilities

38,390


38,070
Deferred revenue

10,452


115
Operating lease liabilities

8,286


7,902
Royalty monetization liabilities

39,609


34,309
Total current liabilities

118,095


103,194
Deferred revenue

18,943


—
Operating lease liabilities

65,215


74,131
Royalty monetization liabilities

147,408


25,378
Other liabilities

638


3,635
Total liabilities

350,299


206,338
Stockholders' equity





Preferred stock; no shares issued and outstanding
   at December 31, 2025 and 2024

—


—
Common stock; 50,892 and 49,549 shares issued
   and outstanding at December 31, 2025 and
   2024, respectively

5


5
Additional paid-in capital

1,229,442


1,192,536
Accumulated other comprehensive loss

(687)


(741)
Accumulated deficit

(1,126,027)


(932,149)
Total stockholders' equity

102,733


259,651
Total liabilities and stockholders' equity
$453,032

$465,989
 REGENXBIO INC.CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(unaudited)(in thousands, except per share data) 


Three Months

Years


Ended December 31,

Ended December 31,


2025

2024

2025

2024
Revenues











License and royalty revenue
$27,148

$20,788

$156,267

$81,960
Service revenue

3,189


426


14,174


1,368
Total revenues

30,337


21,214


170,441


83,328
Operating Expenses











Cost of license and royalty revenues

5,928


6,318


20,298


33,567
Research and development

59,611


50,380


228,299


208,522
General and administrative

22,380


20,051


82,863


76,619
Credit losses (recoveries)

—


(5,000)


—


(5,000)
Impairment of long-lived assets

—


—


—


2,101
Other operating expenses

54


833


179


865
Total operating expenses

87,973


72,582


331,639


316,674
Loss from operations

(57,636)


(51,368)


(161,198)


(233,346)
Other Income (Expense)











Interest income from licensing

18


83


83


174
Investment income

2,745


9,516


12,245


18,729
Interest expense

(12,276)


(9,417)


(45,008)


(12,659)
Total other income (expense)

(9,513)


182


(32,680)


6,244
Net loss
$(67,149)

$(51,186)

$(193,878)

$(227,102)
Other Comprehensive Income (Loss)











Unrealized gain (loss) on available-for-sale securities, net

(35)


(159)


54


3,688
Total other comprehensive income (loss)

(35)


(159)


54


3,688
Comprehensive loss
$(67,184)

$(51,345)

$(193,824)

$(223,414)













Net loss per share, basic and diluted
$(1.30)

$(1.01)

$(3.76)

$(4.59)
Weighted-average common shares outstanding, basic and diluted

51,752


50,871


51,573


49,509
 





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Original: REGENXBIO Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights

REGENXBIO Announces Presentations at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific ConferenceMarch 4, 2026 7:05 AM
PR Newswire (US)

ROCKVILLE, Md., March 4, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its RGX-202 investigational gene therapy for Duchenne muscular dystrophy at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Orlando, FL March 8-11, 2026.Presentations include preclinical and Phase I/II clinical safety, biomarker, and functional data.Podium Presentation:
Title: RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Phase I/II Clinical Data (P432 O)
Session: Clinical Trial Updates
Date/Time: March 11, 2026; 11:45 a.m. ET
Presenter: Carolina Tesi Rocha, M.D., Clinical Professor, Neurology, Stanford School of Medicine, Stanford Children's HealthPoster Presentation:
Title: Microdystrophin with an extended C-Terminal domain protects against pharmacologically induced cardiac damage and remodeling in mdx mice (P173 M)
Session: Pre-Clinical Research
Presenter: Steven Foltz, Ph.D., Principal Scientist, Gene Therapy Research at REGENXBIOThe presentations will be available in the Publications section of REGENXBIO's website.REGENXBIO will also host a symposium titled, "Advancing Duchenne Gene Therapy Trials in a New Era: Optimizing Design and Interpretation." The event will feature leading experts and take place March 9, 2026 at 12 p.m. ET.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit WWW.REGENXBIO.COM.Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com 





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Original: REGENXBIO Announces Presentations at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

REGENXBIO to Participate in Upcoming Investor ConferencesMarch 2, 2026 7:05 AM
PR Newswire (US)

ROCKVILLE, Md., March 2, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following investor conferences:Leerink Partners Global Healthcare Conference
Fireside Chat: Monday, March 9 at 9:20am ET
Location: Miami, FLBarclays 28th Annual Global Healthcare Conference
Fireside Chat: Tuesday, March 10 at 1:30pm ET
Location: Miami, FLRBC Inaugural Virtual Ophthalmology Conference 
Fireside Chat: Tuesday, March 24 at 8:45am ET
Location: VirtualLive webcasts of select events can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. Archived replays of the webcasts will be available for approximately 30 days following the events.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit WWW.REGENXBIO.COM.Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com  





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Original: REGENXBIO to Participate in Upcoming Investor Conferences

REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNXFebruary 27, 2026 1:10 AM
PR Newswire (US)

LOS ANGELES, Feb. 27, 2026 /PRNewswire/ --  The DJS Law Group  reminds investors of a class action lawsuit against  REGENXBIO Inc. ("Regenxbio " or "the Company") (NASDAQ: RGNX ) for violations of §§10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by the U.S. Securities and Exchange Commission.Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointments. Appointment as lead plaintiff is not required to partake in any recovery. CLASS PERIOD:  February 9, 2022 to January 27, 2026 DEADLINE: April 14, 2026 CASE DETAILS: According to the Complaint, the Company made false and misleading statements to the market. Regenxbio made consistently positive statements to the market about the safety and efficacy of its RGX-111 product candidate. Meanwhile, it was concealing adverse information on safety and efficacy from investors. The FDA placed a clinical hold on RGX-111 when it was revealed a clinical trial participant had developed a tumor. Based on these facts, Regenxbio's public statements were false and materially misleading throughout the class period.If you are a shareholder who suffered a loss, contact us to participate . WHY DJS LAW GROUP?  DJS Law Group's primary focus is to enhance investor return through balanced counseling and aggressive advocacy. We specialize in securities class actions, corporate governance litigation, and domestic/international M&A appraisals. Our clients are some of the largest and most sophisticated hedge funds and alternative asset managers in the world. The litigation claims of our clients are extraordinarily valuable assets that demand respect, focus, and results.Join the case to recover your losses.This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics. CONTACT: David J. Schwartz
DJS Law Group
274 White Plains Road, Suite 1
Eastchester, NY 10709
Phone: 914-206-9742
Email: David@djslawllp.com



View original content:https://www.prnewswire.com/news-releases/regenxbio-inc-sued-for-securities-law-violations---contact-the-djs-law-group-to-discuss-your-rights--rgnx-302699314.htmlSOURCE DJS Law Group LLP

Original: REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNX

REGENXBIO to Host Conference Call on March 5 to Discuss Fourth Quarter and Full Year 2025 Financial Results and Operational HighlightsFebruary 25, 2026 7:05 AM
PR Newswire (US)

ROCKVILLE, Md., Feb. 25, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, March 5, at 8:00 a.m. ET to discuss its financial results for the fourth quarter and full year ended December 31, 2025, and operational highlights.Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit WWW.REGENXBIO.COM.Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com 





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Original: REGENXBIO to Host Conference Call on March 5 to Discuss Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights

REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNXFebruary 23, 2026 3:15 AM
PR Newswire (US)

LOS ANGELES, Feb. 23, 2026 /PRNewswire/ -- The DJS Law Group  reminds investors of a class action lawsuit against  REGENXBIO Inc. ("Regenxbio " or "the Company") (NASDAQ: RGNX ) for violations of §§10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by the U.S. Securities and Exchange Commission.Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointments. Appointment as lead plaintiff is not required to partake in any recovery. CLASS PERIOD:  February 9, 2022 to January 27, 2026 DEADLINE: April 14, 2026 CASE DETAILS: According to the Complaint, the Company made false and misleading statements to the market. Regenxbio made consistently positive statements to the market about the safety and efficacy of its RGX-111 product candidate. Meanwhile, it was concealing adverse information on safety and efficacy from investors. The FDA placed a clinical hold on RGX-111 when it was revealed a clinical trial participant had developed a tumor. Based on these facts, Regenxbio's public statements were false and materially misleading throughout the class period.If you are a shareholder who suffered a loss, contact us to participate . WHY DJS LAW GROUP?  DJS Law Group's primary focus is to enhance investor return through balanced counseling and aggressive advocacy. We specialize in securities class actions, corporate governance litigation, and domestic/international M&A appraisals. Our clients are some of the largest and most sophisticated hedge funds and alternative asset managers in the world. The litigation claims of our clients are extraordinarily valuable assets that demand respect, focus, and results.Join the case to recover your losses.This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics. CONTACT: David J. SchwartzDJS Law Group274 White Plains Road, Suite 1 Eastchester, NY 10709Phone: 914-206-9742Email: David@djslawllp.com



View original content:https://www.prnewswire.com/news-releases/regenxbio-inc-sued-for-securities-law-violations---contact-the-djs-law-group-to-discuss-your-rights--rgnx-302694287.htmlSOURCE DJS Law Group LLP

Original: REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNX

REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNXFebruary 20, 2026 4:19 AM
PR Newswire (US)

LOS ANGELES, Feb. 20, 2026 /PRNewswire/ -- The DJS Law Group  reminds investors of a class action lawsuit against  REGENXBIO Inc. ("Regenxbio " or "the Company") (NASDAQ: RGNX ) for violations of §§10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by the U.S. Securities and Exchange Commission.Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointments. Appointment as lead plaintiff is not required to partake in any recovery. CLASS PERIOD:  February 9, 2022 to January 27, 2026 DEADLINE: April 14, 2026 CASE DETAILS: According to the Complaint, the Company made false and misleading statements to the market. Regenxbio made consistently positive statements to the market about the safety and efficacy of its RGX-111 product candidate. Meanwhile, it was concealing adverse information on safety and efficacy from investors. The FDA placed a clinical hold on RGX-111 when it was revealed a clinical trial participant had developed a tumor. Based on these facts, Regenxbio's public statements were false and materially misleading throughout the class period.If you are a shareholder who suffered a loss, contact us to participate . WHY DJS LAW GROUP?  DJS Law Group's primary focus is to enhance investor return through balanced counseling and aggressive advocacy. We specialize in securities class actions, corporate governance litigation, and domestic/international M&A appraisals. Our clients are some of the largest and most sophisticated hedge funds and alternative asset managers in the world. The litigation claims of our clients are extraordinarily valuable assets that demand respect, focus, and results.Join the case to recover your losses.This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics. CONTACT:
David J. Schwartz
DJS Law Group
274 White Plains Road, Suite 1
Eastchester, NY 10709
Phone: 914-206-9742
Email: David@djslawllp.com



View original content:https://www.prnewswire.com/news-releases/regenxbio-inc-sued-for-securities-law-violations---contact-the-djs-law-group-to-discuss-your-rights--rgnx-302693373.htmlSOURCE DJS Law Group LLP

Original: REGENXBIO Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RGNX

The Gross Law Firm Notifies REGENXBIO Inc. Investors of a Class Action Lawsuit and Upcoming Deadline - RGNXFebruary 19, 2026 10:15 AM
PR Newswire (US)

NEW YORK, Feb. 19, 2026 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of REGENXBIO Inc. (NASDAQ: RGNX).







Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery.CONTACT US HERE:https://securitiesclasslaw.com/securities/regenxbio-inc-loss-submission-form/?id=183652&from=4CLASS PERIOD: February 9, 2022 to January 27, 2026ALLEGATIONS: According to the complaint, defendants provided investors with material information concerning REGENXBIO's plan to develop and commercialize its product candidate RGX-111, a one-time gene therapy for the treatment of severe Mucopolysaccharidosis Type I, also known as Hurler syndrome. Defendants' statements included, among other things, REGENXBIO's positive assertions of RGX-111's future trial success based on continuing positive biomarker and safety data from the ongoing PhaseI/II study. 3. Defendants provided these overwhelmingly positive statements to investors while, at the same time, disseminating false and misleading statements and/or concealing material adverse facts concerning the efficacy and safety of its RGX-111 trial study.   On January 28, 2026, REGENXBIO issued a press release announcing that the FDA placed a clinical hold on its investigational gene therapy RGX-111. Defendants announced that an intraventricular CNS tumor was found in a participant treated in its RGX-111 Phase I/II study. Following this news, the price of REGENXBIO's common stock declined from a closing market price of $13.41 per share on January 27, 2026, REGENXBIO's stock price fell to $11.01 per share on January 28, 2026, a decline of 17.8% in the span of just a single day.DEADLINE: April 14, 2026 Shareholders should not delay in registering for this class action. Register your information here: https://securitiesclasslaw.com/securities/regenxbio-inc-loss-submission-form/?id=183652&from=4NEXT STEPS FOR SHAREHOLDERS: Once you register as a shareholder who purchased shares of RGNX during the timeframe listed above, you will be enrolled in a portfolio monitoring software to provide you with status updates throughout the lifecycle of the case. The deadline to seek to be a lead plaintiff is April 14, 2026. There is no cost or obligation to you to participate in this case.WHY GROSS LAW FIRM? The Gross Law Firm is a nationally recognized class action law firm, and our mission is to protect the rights of all investors who have suffered as a result of deceit, fraud, and illegal business practices. The Gross Law Firm is committed to ensuring that companies adhere to responsible business practices and engage in good corporate citizenship. The firm seeks recovery on behalf of investors who incurred losses when false and/or misleading statements or the omission of material information by a company lead to artificial inflation of the company's stock. Attorney advertising. Prior results do not guarantee similar outcomes.CONTACT:
The Gross Law Firm
15 West 38th Street, 12th floor
New York, NY, 10018
Email: dg @bback



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Original: The Gross Law Firm Notifies REGENXBIO Inc. Investors of a Class Action Lawsuit and Upcoming Deadline - RGNX

REGENXBIO Inc. Sued for Securities Law Violations – Contact the DJS Law Group to Discuss Your Rights – RGNXFebruary 17, 2026 7:30 AM
Business Wire
The DJS Law Group reminds investors of a class action lawsuit against REGENXBIO Inc. (“Regenxbio” or “the Company”) (NASDAQ: RGNX) for violations of §§10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder by the U.S. Securities and Exchange Commission.


Shareholders who purchased shares of RGNX during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointments. Appointment as lead plaintiff is not required to partake in any recovery.


CLASS PERIOD: February 9, 2022 to January 27, 2026


DEADLINE: April 14, 2026


CASE DETAILS: According to the Complaint, the Company made false and misleading statements to the market. Regenxbio made consistently positive statements to the market about the safety and efficacy of its RGX-111 product candidate. Meanwhile, it was concealing adverse information on safety and efficacy from investors. The FDA placed a clinical hold on RGX-111 when it was revealed a clinical trial participant had developed a tumor. Based on these facts, Regenxbio’s public statements were false and materially misleading throughout the class period.


If you are a shareholder who suffered a loss, contact us to participate.


WHY DJS LAW GROUP? DJS Law Group’s primary focus is to enhance investor return through balanced counseling and aggressive advocacy. We specialize in securities class actions, corporate governance litigation, and domestic/international M&A appraisals. Our clients are some of the largest and most sophisticated hedge funds and alternative asset managers in the world. The litigation claims of our clients are extraordinarily valuable assets that demand respect, focus, and results.


Join the case to recover your losses.


This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and rules of ethics.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260217862316/en/
David J. Schwartz

DJS Law Group

274 White Plains Road, Suite 1

Eastchester, NY 10709

Phone: 914-206-9742

Email: David@djslawllp.com


Original: REGENXBIO Inc. Sued for Securities Law Violations – Contact the DJS Law Group to Discuss Your Rights – RGNX

REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS IIFebruary 9, 2026 4:05 PM
PR Newswire (US)

FDA issues Complete Response Letter for RGX-121 (clemidsogene lanparvovec) for treatment of Mucopolysaccharidosis II (MPS II) REGENXBIO plans to work with FDA on a path forward with the goal of resubmitting the BLAROCKVILLE, Md., Feb. 9, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding its Biologics License Application (BLA) for RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II), an ultra-rare neurodegenerative disease also known as Hunter syndrome.In May 2025, the FDA accepted the RGX-121 BLA under the accelerated approval pathway. In the February 7, 2026 CRL, the FDA stated that it had agreed to the study protocol in principle and outlined several reasons for not approving the gene therapy, including uncertainty regarding the study eligibility criteria to adequately define a population with neuronopathic disease (vs. attenuated disease), the comparability of the natural history external control to the study population, and the appropriateness of CSF HS D2S6 as a surrogate endpoint reasonably likely to predict clinical benefit. The CRL lists several potential paths forward, including a new study, treating additional patients and conducting longer-term follow up, and using an untreated control arm, all of which would be challenging in an ultra-rare disease population, like MPS II."This decision is devastating for the families of boys living with this progressive, life-threatening disease," said Curran Simpson, President and CEO of REGENXBIO. "We are concerned about FDA's feedback regarding the overall development path and evaluation of the data in the context of the urgent need for this irreversible ultra-rare disease. We remain confident in the quality and volume of evidence demonstrating the long-term potential of RGX-121 to positively change the trajectory of Hunter syndrome. This program has been in development for over 10 years. We are incredibly grateful to all the patients, their families, investigators, and site staff who have supported this program and our continued efforts to bring a much-needed new treatment option to the Hunter syndrome community. We will continue those efforts."Throughout active discussions during the BLA process, REGENXBIO believed it had addressed the points raised in the CRL through the submission of additional data and responses to numerous information requests. Independent, leading global MPS and biomarker experts conducted analyses and reviews with the FDA, as well. Ultimately, the FDA did not agree the data set provided substantial evidence of effectiveness to support approval of RGX-121 for the treatment of MPS II.REGENXBIO plans to request a Type A meeting to discuss the CRL, as well as the planned BLA resubmission to provide additional evidence from global MPS II experts to further clarify the neuronopathic patient population and additional longer-term clinical data to support evidence of effectiveness. REGENXBIO intends to find a path forward as quickly as possible with the goal of resubmitting the BLA."MPS II is a very complex disease, but its impact is well established, resulting in irreversible brain damage for the majority of patients; without appropriate treatments stopping this neurocognitive decline, the neuronopathic MPS II child will die prematurely, usually in their mid-teens," said Joseph Muenzer, M.D., Ph.D., Director, Muenzer MPS Research and Treatment Center, Bryson Distinguished Professor in the Division of Genetics and Metabolism, Department of Pediatrics Genetics, University of North Carolina at Chapel Hill. "I remain encouraged by the clinical data behind RGX-121. New innovations like gene therapy could make a significant impact for these patients, and time is precious for these families.""I've seen the severe impact of MPS II on patients and their families firsthand and am extremely disheartened by today's news," said Terri Klein, President and CEO, National MPS Society. "Families know the devastating trajectory of this disease all too well and have waited 20 years for new treatment options. They cannot wait any longer. Drug development for ultra-rare disease must be streamlined to allow new medicines to reach patients. We urge the FDA to find a swift path forward so that boys living with MPS II and their families have the chance for a better life."About RGX-121 (clemidsogene lanparvovec)
RGX-121 is a potential one-time gene therapy for the treatment of boys with MPS II, designed to deliver the iduronate-2-sulfatase (IDS) gene to the CNS. Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted iduronate-2-sulfatase (I2S) protein beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 expressed protein is structurally identical to normal I2S.The BLA for RGX-121 for MPS II was supported by positive biomarker, functional and safety data from the CAMPSIITE I/II/III trial, including out to 12 months. RGX-121 has been well tolerated in all patients dosed across all phases of the CAMPSIITE trial.RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA and advanced therapy medicinal products (ATMP) classification from the European Medicines Agency. About Mucopolysaccharidosis Type II (MPS II)
MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme I2S leading to an accumulation of glycosaminoglycans (GAGs), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the central nervous system (CNS). In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate CSF HS D2S6, which has been shown to correlate with neurocognitive manifestations of the disorder.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.REGENXBIO.com.FORWARD-LOOKING STATEMENTS
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie or Nippon Shinyaku, the monetization of any priority review voucher, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2024, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at WWW.SEC.GOV. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com George E. MacDougall
Investor Relations
IR@regenxbio.com 





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Original: REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS II

REGENXBIO Announces Regulatory Update on Ultra Rare MPS ProgramsJanuary 28, 2026 12:30 PM
PR Newswire (US)

ROCKVILLE, Md., Jan. 28, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) placed a clinical hold on its investigational gene therapy, RGX-111, for the treatment of MPS I, also known as Hurler syndrome, following preliminary analysis of a single case of neoplasm (intraventricular CNS tumor) in a participant treated in its Phase I/II study. The FDA also placed a clinical hold on RGX-121, for the treatment of MPS II, also known as Hunter Syndrome, citing the similarities in products, study populations, and shared risk between the clinical studies.The case was identified during a routine brain MRI of an asymptomatic five-year-old participant who received intracisternal RGX-111 four years prior. Preliminary genetic analysis of the resected tumor detected an AAV vector genome integration event associated with overexpression of a proto-oncogene (PLAG1), which is known to be susceptible to chromosomal rearrangements. The investigation to determine if this SAE is drug related is ongoing. The causality has not been established. The participant continues to be asymptomatic, with positive developmental advancements noted by the treating physician. No evidence of neoplasm has been reported in the nine other participants treated with RGX-111 nor in the 32 participants treated with RGX-121."We are surprised by FDA's decision to place our RGX-121 program on hold while the investigation of this single, inconclusive incident in RGX-111 continues," said Curran Simpson, President and CEO of REGENXBIO. "These are separate therapies, and the positive safety profile of RGX-121 in more than 30 patients treated, including those dosed nearly seven years ago, remains unchanged. Patient safety is our top priority, and we, our investigators, and the patient community remain confident in the benefit-risk ratio of RGX-121 and are highly encouraged by the meaningful efficacy profile demonstrated in the pivotal trial. RGX-121 presents an opportunity to address the urgent, significant unmet medical need in this ultra-rare disease community, and continued delay means continued neurodevelopmental decline in boys with MPS II."REGENXBIO has not yet received the full clinical hold letter and awaits additional details from the FDA.About RGX-121 (clemidsogene lanparvovec)
RGX-121 is a potential one-time gene therapy for the treatment of boys with MPS II, designed to deliver the iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted iduronate-2-sulfatase (I2S) protein beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 expressed protein is structurally identical to normal I2S.RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA and advanced therapy medicinal products (ATMP) classification from the European Medicines Agency. About Mucopolysaccharidosis Type II (MPS II)
MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme I2S leading to an accumulation of glycosaminoglycans (GAGs), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the central nervous system (CNS). In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate CSF HS D2S6, which has been shown to correlate with neurocognitive manifestations of the disorder.About RGX-111
RGX-111 is designed to use the AAV9 vector to deliver the a-l-iduronidase (IDUA) gene to the central nervous system (CNS). Delivery of the IDUA gene within the cells in the central nervous system (CNS) could provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross-correction of cells throughout the CNS. By providing rapid IDUA delivery to the brain, RGX-111 could potentially help prevent the progression of cognitive deficits that otherwise occurs in MPS I patients. RGX-111 has received orphan drug product, rare pediatric disease and Fast Track designations from the FDA.About Mucopolysaccharidosis Type I (MPS I)
MPS I is a rare autosomal recessive genetic disease caused by a deficiency in the lysosomal enzyme alpha-L-iduronidase (IDUA), leading to an accumulation of glycosaminoglycans (GAGs) including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the central nervous system (CNS). This can include excessive accumulation of fluid in the brain, spinal cord compression, and cognitive impairment. MPS I is estimated to occur in 1 in 100,000 births. Current disease modifying therapies for MPS I include hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy with a recombinant form of human IDUA administered intravenously. However, intravenous enzyme therapy does not treat the CNS manifestations of MPS I, and HSCT can be associated with clinically significant morbidity and mortality. Key biomarkers of IDUA enzymatic activity in MPS I patients include its substrate heparan sulfate (HS), which has been shown to correlate with neurocognitive manifestations of the disorder.ABOUT REGENXBIO Inc.
REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; NAVSUNLI for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.REGENXBIO.com.FORWARD-LOOKING STATEMENTS
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, costs and cash flow. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timing or likelihood of payments from AbbVie or Nippon Shinyaku, the monetization of any priority review voucher, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2024, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at WWW.SEC.GOV. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.CONTACTS: Dana Cormack
Corporate Communications
Dcormack@regenxbio.comGeorge E. MacDougall
Investor Relations
IR@regenxbio.com 





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Original: REGENXBIO Announces Regulatory Update on Ultra Rare MPS Programs

RGNX, under $8

RGNX under $10

36M share trading float.

RGNX

AbbVie And REGENXBIO Announce Eye Care Collaboration; AbbVie To Pay REGENXBIO $370M Upfront Payment And Up To $1.38B In milestones
6:31 am ET September 13, 2021 (Benzinga) Print
AbbVie (NYSE:ABBV) and REGENXBIO Inc. (NASDAQ:RGNX) today announced a partnership to develop and commercialize RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal diseases. RGX-314 is currently being evaluated in patients with wet AMD in a pivotal trial utilizing subretinal delivery, and in patients with wet AMD and DR in two separate Phase II clinical trials utilizing in-office suprachoroidal delivery.

Under the collaboration, REGENXBIO will be responsible for completion of the ongoing trials of RGX-314. AbbVie and REGENXBIO will collaborate and share costs on additional trials of RGX-314, including the planned second pivotal trial evaluating subretinal delivery for the treatment of wet AMD and future trials. AbbVie will lead the clinical development and commercialization of RGX-314 globally. REGENXBIO shall participate in U.S. commercialization efforts as provided under a mutually agreed upon commercialization plan.

"We are committed to finding solutions for patients living with difficult-to-treat retinal diseases and to helping preserve and protect our patients from visual impairment and devastating vision loss," said Tom Hudson, MD, senior vice president, R&D, chief scientific officer, AbbVie. "In collaboration with REGENXBIO, we aim to make a remarkable impact for the millions of patients suffering from vision loss associated with retinal diseases."

"AbbVie is a strong, complementary partner for REGENXBIO. We expect to leverage AbbVie's global developmental and commercial infrastructure within eye care with our expertise in AAV gene therapy clinical development and deep in-house knowledge of manufacturing and production to continue the development of RGX-314," said Kenneth T. Mills, president and chief executive officer of REGENXBIO.

Under the terms of the agreement, AbbVie will pay REGENXBIO a $370 million upfront payment with the potential for REGENXBIO to receive up to $1.38 billion in additional development, regulatory and commercial milestones. REGENXBIO and AbbVie will share equally in profits from net sales of RGX-314 in the U.S. AbbVie will pay REGENXBIO tiered royalties on net sales of RGX-314 outside the U.S. In addition, REGENXBIO will lead the manufacturing of RGX-314 for clinical development and U.S. commercial supply, and AbbVie will lead manufacturing of RGX-314 for commercial supply outside the U.S.

https://www.google.com/amp/s/www.nasdaq.com/article/4-gene-therapy-players-likely-to-become-buyout-targets-in-2019-cm1125990/amp

I believe the buyout is in a distance future. JUNO has the same trading behavior before buyout was announced!

is there an argument for a BO? https://msmoneymoves.com/2019/05/28/could-this-biotech-stock-get-bought-for-5-5-billion-either-way-im-buying-it/

Strong buy @ 50!!!!

RGNX buy 66.90























normal chart




log chart

Hope no more form 4s for insider selling

Let’s see how tomorrow will be

Why insiders sold at $34 and $37 while the target price is $90? Anybody has any clue?

$45 soon! Yummy

RGNX bullish 14.39



normal chart


log chart

Agreed, would like to to see the stock come down to single digits before buying though. Still only in preclinical trials and burning a ton of cash.

Interesting play here for the long term. Very good technology with a lot of prospects for them and licensing.

RGNX no bid support, too early still, listed VC...