US Market News
2週前
Neurogene Announces Successful Completion of Dosing in Embolden™ Registrational Trial of NGN-401 for Rett SyndromeJune 8, 2026 7:30 AM
Business Wire Exceeded target by dosing a total of 25 participants with NGN-401 in registrational trial due to strong demand from Rett syndrome community NGN-401 has been generally well-tolerated, with no cases of HLH at the 1E15 vg dose; additional interim Phase 1/2 data expected mid-2026 Topline data from Embolden anticipated in 2H 2027 Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the successful completion of dosing in the Embolden™ registrational trial of NGN-401, an investigational gene therapy designed to be a potential best-in-class, one-time treatment for Rett syndrome. While the initial enrollment target was 20 participants, the statistical analysis plan pre-specified an intent-to-treat (ITT) population of up to 24 participants, providing flexibility to support robust trial execution. Due to strong demand from the Rett syndrome community and to ensure timely completion of dosing, the Company elected to overenroll the trial and dose all eligible participants already in screening for a total of 25. “We are pleased to have completed dosing in the Embolden trial of NGN-401 within our original timeline while exceeding our enrollment target, reflecting both the significant unmet need and strong demand from the Rett syndrome community,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “This milestone brings us one step closer to delivering a potential best-in-class, one-time treatment for Rett syndrome. We are deeply grateful to the participants, their families and the investigators for their trust, partnership and ongoing support of the development of NGN-401. We look forward to sharing topline results from Embolden in the second half of 2027 and advancing NGN-401 towards a planned BLA submission.” Embolden is a multi-center, single-arm, open-label, baseline-controlled registrational trial designed to evaluate the efficacy, safety and tolerability of a one-time dose of NGN-401 (1E15 vg) in females with Rett syndrome ages three and older. The primary endpoint is a composite of a Clinical Global Impression-Improvement (CGI-I) score of ≤ 3 and a gain from baseline of any one developmental milestone from a pre-specified list. The primary analysis to support the planned Biologics License Application (BLA) submission is expected to occur after the first 24 participants (pre-specified ITT) have completed 12 months of follow-up, with topline data expected in the second half of 2027. The threshold for success required for the registrational trial is a 33% (or 8 of 24 participants) minimum response rate. Data from the additional participant is expected to supplement the overall safety and durability dataset. As of June 7, 2026, NGN-401 at the 1E15 vg dose (n=35) continues to be generally well-tolerated. There were no cases of hemophagocytic lymphohistiocytosis (HLH) at this dose level in the Phase 1/2 trial or the Embolden trial. The Embolden trial builds on positive interim data from the Phase 1/2 trial of NGN-401, which demonstrated multidomain, durable gains with continued developmental milestone acquisitions as of the most recently disclosed safety and efficacy data, with a cutoff date of October 30, 2025. Neurogene plans to present updated interim safety and efficacy data from the Phase 1/2 trial, including at least 12 months of follow-up for all 10 participants, in mid-2026. About Neurogene
Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn. About NGN-401
NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene’s EXACT™ transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Interim data from the Phase 1/2 trial (as of October 30, 2025) have shown that participants experienced multidomain, durable gains with continued skill acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency. Cautionary Note Regarding Forward-Looking Statements
Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; the applicability of reported interim results from the NGN-401 Phase 1/2 clinical trial to other participants or potential participants, including adolescent or adult patients; the potential for NGN-401 to be a best-in-class gene therapy for Rett syndrome; trial designs and clinical development plans for the Company’s Embolden™ registrational clinical trial of NGN-401 for Rett syndrome, including expectations to supplement the overall safety and durability set with data from the additional participant; the response rate, expected durability and deepening of clinical data results from our NGN-401 clinical trial; the potential for future approval for commercialization of NGN-401 as a treatment for Rett syndrome; expected timing for release of additional data from Neurogene’s Phase 1/2 clinical trial of NGN-401 and topline data from the Company’s Embolden registrational trial of NGN-401; the potential for success of the Embolden registrational clinical trial of NGN-401 for Rett syndrome; the timing and potential for success of a BLA submission to the FDA for NGN-401; and expected future interactions with or positions of the FDA, including the timing and outcome of any such interactions and anticipated benefits of any regulatory designation for NGN-401, including the FDA’s Breakthrough Therapy designation, Rare Pediatric Disease designation, RMAT designation and participation in the FDA’s START program. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things, the risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, filed with the Securities and Exchange Commission (“SEC”) on May 12, 2026, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. View source version on businesswire.com: https://www.businesswire.com/news/home/20260608913151/en/ Media Contact:
Mike Devine
Executive Director, Corporate Communications
michael.devine@neurogene.com Investor Contact:
Lina Li
Executive Director, Investor Relations
lina.li@neurogene.com Original: Neurogene Announces Successful Completion of Dosing in Embolden™ Registrational Trial of NGN-401 for Rett Syndrome
US Market News
1月前
Neurogene Reports First Quarter 2026 Financial Results and Highlights Recent UpdatesMay 12, 2026 4:05 PM
Business Wire Dosed ~90% of participants in Embolden™ registrational trial of NGN-401 for Rett syndrome; on track to complete dosing in the second quarter of 2026 NGN-401 has been generally well-tolerated, with no cases of HLH at the 1E15 vg dose; additional interim Phase 1/2 data expected mid-2026 Presentation at ASGCT Meeting highlighted therapeutic rationale for ICV delivery in CNS-targeted gene therapy, including NGN-401 Strong cash position provides runway through first quarter of 2028 Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced first quarter 2026 financial results and highlighted recent corporate updates. “The significant unmet need in Rett syndrome and strong interest among physicians and caregivers continue to drive momentum for NGN-401, with approximately 90% of participants dosed in the Embolden™ registrational trial,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “We continue to be encouraged that NGN-401 at the 1E15 vg dose has been generally well-tolerated. We are laser-focused on completing dosing in Embolden in the coming weeks and look forward to providing an update on this important clinical development milestone. In parallel, we are continuing to strengthen the organization and execute on early commercial-readiness activities to support a successful potential launch of NGN-401.” First Quarter 2026 and Recent Highlights NGN-401 Gene Therapy for the Treatment of Rett Syndrome Announced today that ~90% of participants have been dosed in the Embolden registrational trial, and the Company is on track to complete dosing in the second quarter of 2026 NGN-401 at the 1E15 vg dose has been generally well-tolerated, with no cases of hemophagocytic lymphohistiocytosis (HLH) in the Phase 1/2 trial or Embolden as of May 11, 2026 Presented on the therapeutic rationale for intracerebroventricular (ICV) administration of central nervous system (CNS)-targeted gene therapy, including NGN-401, at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting Presentation highlighted the biological need to deliver gene therapy to the key regions of the brain underlying disease, supported by preclinical data demonstrating broader CNS biodistribution to brain regions central to Rett syndrome pathophysiology, as compared with intrathecal lumbar (IT-L) administration Additional Corporate Updates Strengthened the leadership team with the appointment of Christy Shafer as Chief Commercial Officer and Christine Mikail, J.D., President and CFO of Neurogene, to the Board of Directors Ms. Shafer brings more than 20 years of industry experience building and leading high-performing commercial organizations and launching therapies for rare neurological diseases Ms. Mikail brings to the Board deep expertise in strategic transactions, capital formation, and corporate development, with a track record of building and positioning companies for long-term value creation Key Anticipated NGN-401 Milestones in 2026 Complete dosing of participants in the Embolden registrational trial in the second quarter of 2026 Present updated interim safety and efficacy data on the pediatric and adolescent/adult cohorts from the Phase 1/2 trial, including at least 12 months of follow-up for all 10 participants, in mid-2026 Initiate Process Performance Qualification (PPQ) campaign in mid-2026 Continue early commercial-readiness activities Upcoming Events H.C. Wainwright 4th Annual BioConnect Investor Conference: Management will participate in a fireside chat at 11:30 a.m. ET on May 19 and participate in investor meetings Goldman Sachs 47th Annual Global Healthcare Conference: Management will participate in a fireside chat at 2:40 p.m. ET on June 8 and participate in investor meetings First Quarter 2026 Financial Results Cash, Cash Equivalents and Short-Term Investments: Cash, cash equivalents and short-term investments as of March 31, 2026 were $243.2 million and are expected to fund planned operations through the first quarter of 2028. Research & Development (R&D) Expenses: R&D expenses were $25.2 million for the three months ended March 31, 2026, compared to $17.8 million for the three months ended March 31, 2025. The increase in R&D expenses for the three months ended March 31, 2026 was primarily driven by an increase in Rett syndrome clinical trial costs of NGN-401, chemistry, manufacturing and controls (CMC) costs to support NGN-401 and employee-related expenses due to an increase in R&D headcount. The increase was partially offset by decreases in spending on the CLN5 Batten disease program and early discovery. General & Administrative (G&A) Expenses: G&A expenses were $8.2 million for the three months ended March 31, 2026 and the three months ended March 31, 2025. Higher costs related to corporate and pre-commercial activities, along with increased employee-related expenses and professional fees, were offset by a decrease in non-cash stock-based compensation expense. Net Loss: Net loss was $30.9 million for the three months ended March 31, 2026, compared to $22.6 million for the three months ended March 31, 2025. About Neurogene
Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn. About NGN-401
NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene’s EXACT™ transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Interim data from the Phase 1/2 trial (as of October 30, 2025) have shown that participants experienced multidomain, durable gains with continued skill acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency. Cautionary Note Regarding Forward-Looking Statements
Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law. Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; the applicability of reported interim results from the NGN-401 Phase 1/2 clinical trial to other participants or potential participants, including adolescent or adult patients; the potential for NGN-401 to be a best-in-class gene therapy for Rett syndrome; trial designs and clinical development plans for the Company’s Embolden registrational clinical trial of NGN-401 for Rett syndrome, including timing of anticipated dosing and completion of participant dosing in the Company’s clinical trial and the expected timeline of its PPQ activities for its CMC requirements; the response rate, expected durability and deepening of clinical data results from our NGN-401 clinical trial; the potential for future approval for commercialization of NGN-401 as a treatment for Rett syndrome; commercial launch readiness for NGN-401; expected timing for release of additional data from the Company’s Phase 1/2 clinical trial of NGN-401; the potential for success of the Embolden registrational clinical trial of NGN-401 for Rett syndrome; the clinical benefit of delivering NGN-401 via ICV administration; expected future interactions with or positions of the FDA, including the timing and outcome of any such interactions and anticipated benefits of any regulatory designation for NGN-401, including the FDA’s Breakthrough Therapy designation, Rare Pediatric Disease designation, RMAT designation and participation in the FDA’s START program; and the time period over which existing cash resources may be sufficient to fund the Company’s operations. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things: the potential for negative impacts to participants in the Phase 1/2 clinical trial of NGN-401 for the treatment of Rett syndrome; the risk that the Company may not be able to report data on the predicted timeline; risks related to the Company’s ability to obtain regulatory approval for, and ultimately commercialize, its product candidates, including NGN-401; risks related to timing of completing enrollment in the Embolden trial of NGN-401 for Rett syndrome; and other risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission (“SEC”) on March 24, 2026, the Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, filed with the SEC on May 12, 2026, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. This communication contains hyperlinks to information that is not deemed to be incorporated by reference into this communication. - Financial Tables Follow - Neurogene Inc. Unaudited Condensed Consolidated Balance Sheet Data (In thousands of U.S. dollars) March 31, 2026 December 31, 2025 Assets Cash and cash equivalents $ 124,161 $ 103,845 Short-term investments 119,021 165,168 Other current assets 3,565 2,757 Non-current assets 16,394 16,834 Total assets $ 263,141 $ 288,604 Liabilities Current liabilities $ 18,866 $ 16,411 Non-current liabilities 6,492 7,306 Total liabilities 25,358 23,717 Stockholders' equity 237,783 264,887 Total liabilities and stockholders' equity $ 263,141 $ 288,604 Neurogene Inc. Unaudited Condensed Consolidated Statements of Operations (In thousands of U.S. dollars, except per share information) Three Months Ended March 31, 2026 2025 Operating expenses: Research and development expenses $ 25,150 $ 17,760 General and administrative expenses 8,199 8,159 Total operating expenses 33,349 25,919 Loss from operations (33,349 ) (25,919 ) Other income, net 2,415 3,272 Net loss $ (30,934 ) $ (22,647 ) Per share information: Net loss per share, basic and diluted $ (1.39 ) $ (1.08 ) Weighted-average shares of common stock outstanding, basic and diluted 22,305,734 20,996,287 View source version on businesswire.com: https://www.businesswire.com/news/home/20260512420099/en/ Media Contact:
Mike Devine
Executive Director, Corporate Communications
michael.devine@neurogene.com Investor Contact:
Lina Li
Executive Director, Investor Relations
lina.li@neurogene.com Original: Neurogene Reports First Quarter 2026 Financial Results and Highlights Recent Updates
US Market News
2月前
Neurogene Announces Upcoming Presentation on the Therapeutic Rationale for ICV Delivery in CNS-Targeted Gene TherapyApril 28, 2026 7:30 AM
Business Wire
Presentation at ASGCT Meeting will feature ICV as a routine neurosurgical procedure that provides broad biodistribution of gene therapy to the brain and nervous system
Safety and efficacy of NGN-401 for Rett syndrome, delivered via ICV administration, to be highlighted
Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced an upcoming poster presentation that will demystify the intracerebroventricular (ICV) route of administration as a well-established, routinely performed neurosurgical procedure and a deliberate delivery strategy designed to maximize efficacy for central nervous system (CNS) gene therapies at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, taking place May 11-15, 2026.
The presentation will highlight the biological rationale to deliver gene therapy broadly to key regions of the brain, leading to the selection of ICV administration of NGN-401 gene therapy for Rett syndrome. Preclinical data will show that ICV administration resulted in greater biodistribution to brain regions central to Rett syndrome pathophysiology, compared to intrathecal-lumbar administration. The presentation will review that clinical safety in CNS gene therapy is shown to be driven by the product itself, and not the delivery route, with ICV administration showing a generally well-tolerated profile.
“Our selection of ICV delivery for NGN-401 was informed by robust preclinical data that showed broader brain and nervous system biodistribution compared to IT-L, critical for a CNS-mediated disease such as Rett syndrome,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “ICV administration is a well-established neurosurgical approach that has been used across multiple therapeutic areas and represents a deliberate choice to enable effective gene delivery. We believe the data we have reported from our ongoing Phase 1/2 trial reinforce our choice of ICV delivery, with participants demonstrating durable, multidomain developmental milestone gains over time and a safety profile at the 1E15 vg dose that supports continued advancement of NGN-401 in our Embolden™ registrational trial.”
Poster Presentation Details
Title: Gene Therapy Targeting CNS Diseases: ICV Administration as a Growing Standard for Delivery
Date: Tuesday, May 12
Presentation Time: 5-6:30 p.m. ET
Presenter: Daniel J. Curry, M.D., Director of Functional Neurosurgery and Epilepsy Surgery at Texas Children's Hospital, Professor of Neurological Surgery at Baylor College of Medicine
Location: MCEC Exhibit and Poster Hall
The abstract is available on ASGCT’s website.
About Neurogene
Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn.
About NGN-401
NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene’s EXACT™ transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden™ registrational clinical trial. Interim data from the Phase 1/2 trial (as of October 30, 2025) have shown that participants experienced multidomain, durable gains with continued skill acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; the applicability of reported interim results from the NGN-401 Phase 1/2 clinical trial to other participants or potential participants, including adolescent or adult participants; the potential for NGN-401 to be a best-in-class gene therapy for Rett syndrome; trial designs and clinical development plans for Neurogene’s Embolden™ registrational clinical trial of NGN-401 for Rett Syndrome; the response rate, expected durability and deepening of clinical data results from our NGN-401 clinical trials; the potential for success of the Embolden registrational clinical trial of NGN-401 for Rett Syndrome; the clinical benefit of delivering NGN-401 via ICV administration; and expected or anticipated benefits of any regulatory designation for NGN-401, including the FDA's Breakthrough Therapy designation, Rare Pediatric Disease designation, RMAT designation, and participation in the FDA's START Pilot Program. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include, among other things, the risks and uncertainties identified under the heading "Risk Factors" included in Neurogene’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission (SEC) on March 24, 2026, and other filings that Neurogene has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260428694916/en/
Media Contact:
Mike Devine
Executive Director, Corporate Communications
michael.devine@neurogene.com
Investor Contact:
Lina Li
Executive Director, Investor Relations
lina.li@neurogene.com
Original: Neurogene Announces Upcoming Presentation on the Therapeutic Rationale for ICV Delivery in CNS-Targeted Gene Therapy
US Market News
2月前
Neurogene Appoints Christy Shafer as Chief Commercial Officer and Christine Mikail, J.D., to Board of DirectorsApril 20, 2026 7:30 AM
Business Wire
Ms. Shafer, a proven commercial leader in rare neurological diseases, will lead Neurogene’s commercial strategy and launch readiness for NGN-401 gene therapy for Rett syndrome
Ms. Mikail, President and CFO of Neurogene, adds strategic and operational leadership to the Board as Company advances key priorities
Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the appointment of Christy Shafer as Chief Commercial Officer and the appointment of Christine Mikail, J.D., to its Board of Directors. Ms. Shafer brings more than 20 years of industry experience building and leading high-performing commercial organizations and launching therapies for rare neurological diseases. Ms. Mikail brings to the Board deep expertise in strategic transactions, capital formation, and corporate development, with a track record of building and positioning companies for long-term value creation.
“With the addition of Christy Shafer to Neurogene’s experienced executive team, we welcome a proven commercial leader with deep expertise in building organizations and executing rare neurological disease product launches to guide our commercial strategy and launch readiness for NGN-401, our gene therapy product for Rett syndrome,” stated Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene. “Christine Mikail has deep strategic, financial, transactional, and operational expertise, along with a rigorous understanding of Neurogene’s business and long-term objectives. Her perspective and judgment will strengthen the Board as Neurogene continues to advance NGN-401 and prepares for its next phase of growth.”
Since joining Neurogene, Ms. Mikail has played a central role in shaping the Company’s long-term strategy, strengthening its financial foundation, and supporting advancement of its clinical and corporate objectives.
“I am honored to join Neurogene’s Board of Directors at this important stage in the Company’s evolution,” said Ms. Mikail. “I look forward to contributing my experience and perspective to Neurogene’s long-term strategy as a member of the Board.”
Christy Shafer most recently served as Senior Vice President and General Manager, North America, of Avidity Biosciences, a late-stage biotech developing RNA therapeutics for rare neuromuscular diseases that was acquired by Novartis. She was responsible for building and leading Avidity’s North America commercial organization in advance of its first U.S. product launches. Prior to Avidity, Ms. Shafer served as Chief Commercial Officer at Marinus Pharmaceuticals, where she built and led the commercial organization and launched Ztalmy®, the first approved therapy for seizures associated with CDKL5 deficiency disorder, which led to Marinus Pharmaceuticals’ acquisition by Immedica Pharma. Previously, Ms. Shafer held leadership roles of increasing responsibility at Alexion Pharmaceuticals, where she supported multiple neurology launches, including Soliris® for generalized myasthenia gravis and neuromyelitis optica spectrum disorder, as well as earlier commercial leadership positions at Pacira Pharmaceuticals and Sanofi Biosurgery (formerly Genzyme). She holds a Bachelor of Life Science degree in Cell and Molecular Biology and Genetics from the University of Maryland, College Park, and completed postbaccalaureate studies in immunology and pharmacology.
“Neurogene’s differentiated approach to gene therapy represents a meaningful opportunity to transform outcomes for patients with devastating neurological diseases,” said Ms. Shafer. “I am thrilled to join this team at this inflection point and look forward to partnering across the organization to help deliver the capabilities, strategy, and infrastructure needed to support a successful launch and commercialization of NGN-401, if approved.”
About Neurogene
Neurogene (NASDAQ: NGNE) is a clinical-stage biotechnology company focused on developing life-changing genetic medicines for people and their families impacted by devastating neurological diseases. The Company is using a biology-first approach paired with optimized delivery to develop purpose-built genetic medicines, including programs powered by its novel and proprietary EXACT™ transgene regulation technology. Neurogene is advancing its lead gene therapy program, NGN-401, as a potential best-in-class, one-time treatment for Rett syndrome. For more information, visit neurogene.com or follow on LinkedIn.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release are made as of the date of this press release. Neurogene does not undertake any obligation to make any updates to these statements to reflect events that occur or circumstances that arise after the date of this press release, except as may be required under applicable U.S. securities law.
Statements in this press release which are not historical in nature are intended to be, and hereby are identified as, forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding: the therapeutic potential and utility, efficacy and clinical benefits of NGN-401; the safety and tolerability profile of NGN-401; the potential for NGN-401 to be a best-in-class gene therapy for Rett syndrome; the potential for future approval for commercialization of NGN-401 as a treatment for Rett syndrome; commercial launch readiness for NGN-401; and the potential for success of the Embolden registrational clinical trial of NGN-401 for Rett syndrome. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “on track,” and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks, uncertainties and assumptions that are difficult to predict with regard to timing, extent, likelihood, and degree of occurrence, which could cause actual results to differ materially from anticipated results and many of which are outside of Neurogene’s control. Such risks, uncertainties and assumptions include those identified under the heading "Risk Factors" included in Neurogene’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission (“SEC”) on March 17, 2026, and other filings that the Company has made and may make with the SEC in the future. Nothing in this communication should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260420578021/en/
Company Contact:
Mike Devine
Executive Director, Corporate Communications
michael.devine@neurogene.com
Investor Contact:
Lina Li
Executive Director, Investor Relations
lina.li@neurogene.com
Original: Neurogene Appoints Christy Shafer as Chief Commercial Officer and Christine Mikail, J.D., to Board of Directors
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