CRISPR Therapeutics AG (CRSP)

I think the criticism is partially valid in general biotech investing, but it doesn't apply to programs like CTX320/CTX321.

For something like CRISPR-based therapies, the situation is very different. Dose escalation is not ''greed'' - it's safety-critical. This isn't altering the dose of an oral pill - it's a one-time intervention with lifelong effects.

CTX320 is clear proof-of-concept and CTX321 is an improved version. To me this is a rational pipeline evolution, not stalling. It suggests that they have learned from CTX320 and optimised before pushing ahead. That's often smarter than pushing a suboptimal therapy (when looking at the data from some of the other therapies in development) into larger and more expensive trials.

Good summary on the Needham conference https://finance.yahoo.com/sectors/healthcare/articles/crispr-therapeutics-touts-second-phase-193416515.html

Dose escalation can be a good thing, once in a while. When companies, however, refuse to advance to the next level of testing because of dose escalation greed, valuable time (years) is lost bringing a new product to market. Once a good product is approved, scaling up higher dose testing makes sense if the company really believes in the potential.. Publicly traded companies have ruined themselves and their shareholders by focusing waaaay too much on dose escalation instead of going for approval. What happens? Cash drains, stock price collapses (no revenues), reverse splits, and the eventual destruction of the shareholder takes place, the entity that made testing possible in the first place.

Building on insights from CTX320, which demonstrated up to 73% reduction in Lp(a), in dose escalation phase of the clinical trial, the company is advancing its next-generation program, CTX321. CTX321 incorporates an updated guide RNA that demonstrates approximately two-fold greater potency while utilising the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies, with an Lp(a) program update expected this year.

https://www.fiercebiotech.com/biotech/its-going-be-huge-diabolical-molecule-poised-become-biotechs-next-gold-rush

Anti-Aging Protein Research Takes a Step Forward with Cell CloningMarch 24, 2026 11:05 AM
PR Newswire (Canada)

Issued on behalf of Avaí Bio, Inc.VANCOUVER, BC, March 24, 2026 /CNW/ -- Equity-Insider.com — A protein called a-Klotho circulating in the bloodstream protects the brain, heart, kidneys, and the immune system. Peer-reviewed research has linked higher Klotho levels to reduced risk of Alzheimer's, cardiovascular disease, and certain cancers. Mayo Clinic research connects declining Klotho to arterial stiffness and vascular calcification.
The challenge is that the body cuts production of this protein by approximately 50% after the age of 40. The molecule that guards against the deadliest age-related diseases starts to decline just as the risk for these conditions starts to rises. Market projections highlight the scale of the issue: Alzheimer's alone is projected to reach $32.8 billion by 2033, cardiovascular disease remains the leading global cause of death, and kidney disease affects 850 million people worldwide.The global cell therapy market has surpassed $8.2 billion in 2026. The broader cell and gene therapy sector is forecast to surge from $10.4 billion to more than $45 billion by 2035, with more than 40 FDA-approved products now on the market. Regenerative medicine alone is projected to reach $578 billion by 2033. The science of reversing biological decline is no longer theoretical, it is an industry. And the companies building the cellular foundations for these therapies are at the forefront of market attention.Vertex Pharmaceuticals (NASDAQ: VRTX) demonstrated what that looks like at scale. The company's gene-edited cell therapy Casgevy — developed with CRISPR Therapeutics (NASDAQ: CRSP) — became the world's first approved CRISPR-based treatment, now available for sickle cell disease and transfusion-dependent beta-thalassemia. Vertex expects to file regulatory submissions for the 5–11 age group in the first half of 2026. CRISPR Therapeutics, meanwhile, reported Phase 1 data showing its in vivo gene-editing therapy CTX310 achieved mean reductions of 73% in ANGPTL3, 55% in triglycerides, and 49% in LDL cholesterol after a single intravenous infusion — a one-dose cardiovascular intervention that could reshape metabolic disease treatment.Altimmune (NASDAQ: ALT) is advancing pemvidutide, a GLP-1/glucagon dual receptor agonist for metabolic disease that targets the $65 billion metabolic syndrome opportunity through hormonal regulation of fat metabolism. Arrowhead Pharmaceuticals (NASDAQ: ARWR) dosed the first patients in a Phase 1/2a trial of ARO-DIMER-PA, the first clinical candidate designed to silence two genes simultaneously for atherosclerotic cardiovascular disease. These are platform-level interventions pulling institutional capital into longevity and regenerative medicine at unprecedented speed.But before any cell therapy can reach patients, it needs a starting point. And that's the step one this company just completed.Avaí Bio (OTCQB: AVAI) recently announced a critical early-stage milestone alongside joint venture partner Austrianova: beginning the creation of a Master Cell Bank (MCB) of genetically modified cells that overexpress the a-Klotho protein. An MCB is the process of taking a single genetically engineered cell and cloning it into tens of millions of identical copies, creating a standardized, GMP-compliant bank of cellular starting material. It's the essential foundation from which all future working cell banks and therapy development will proceed — the step that ensures consistency, quality, and scalability before any therapeutic product can be developed."We are excited to enter the first step in the production phase of a-Klotho producing cells as part of our commitment to deliver safe, effective treatments for aging associated diseases," said Chris Winter, CEO of Avaí Bio.The cells banked in the MCB will be used in conjunction with Austrianova's proprietary Cell-in-a-Box® encapsulation technology, which protects therapeutic cells inside a biocompatible shell to allow continuous protein secretion without triggering immune rejection. The technology is backed by over 50 peer-reviewed publications and decades of development. Avaí Bio's dual-program approach targets both the Klothonova anti-aging platform and the Insulinova diabetes program, each leveraging this same encapsulation technology. The company participated in the 15th European Pancreas and Islet Transplantation Association Symposium in January 2026, where Dr. Eva Maria Lilli Brandtner evaluated advanced cells for potential application in diabetes therapy.The protein that protects against the most serious age-related diseases is steadily disappearing from the bloodstream. The science to potentially restore it exists. And the company that just created the standardized cellular foundation to advance that science — Avaí Bio (OTCQB: AVAI) — is doing so in a market where every platform capable of developing cell-based therapeutics is being re-evaluated.For more information on Avaí Bio (OTCQB: AVAI) and its Klothonova and Insulinova programs, visit Equity-Insider.comRead this and more news for Avaí Bio at: Equity-Insider.comArticle Source: https://usanewsgroup.com/avai-profile/ CONTACT:
EQUITY INSIDER
info @acblanke1DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. Equity Insider is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Avaí Bio, Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Avaí Bio, Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Avaí Bio, Inc. which were purchased in the open market. MIQ reserves the right to buy and sell, and will buy and sell shares of Avaí Bio, Inc. at any time thereafter without any further notice. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material disseminated by MIQ has been approved by the above mentioned company; this is a paid advertisement, and we own shares of the mentioned company that we will sell, and we also reserve the right to buy shares of the company in the open market, or through other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment.SOURCES:Straits Research, Global Cell Therapy Market 2026 — https://straitsresearch.com/report/cell-therapy-marketPrecedence Research, Cell and Gene Therapy Market Forecast — https://www.precedenceresearch.com/cell-and-gene-therapy-marketGrand View Research, CAR T-Cell Therapy Market — https://www.grandviewresearch.com/industry-analysis/car-t-cell-therapy-market-reportAstute Analytica, Regenerative Medicine Market 2025–2033 — https://www.globenewswire.com/news-release/2026/01/27/3226653/0/en/Regenerative-Medicine-Market-Review-2020-2024-and-Forecast-2025-2033-A-578-29-Bn-Opportunity-Says-Astute-Analytica.html Logo: https://mma.prnewswire.com/media/2840019/Equity_Insider_Logo.jpg  



View original content:https://www.prnewswire.com/news-releases/anti-aging-protein-research-takes-a-step-forward-with-cell-cloning-302723584.html

Original: Anti-Aging Protein Research Takes a Step Forward with Cell Cloning

CRISPR Therapeutics shares drop after announcing convertible notes saleMarch 10, 2026 8:46 AM
IH Market News
CRISPR Therapeutics AG (NASDAQ:CRSP) shares declined 6.75% on Tuesday after the company revealed plans to raise $350 million through a convertible senior notes offering.The gene-editing firm said it plans to issue convertible senior notes due in 2031 through a private placement aimed at qualified institutional buyers under Rule 144A of the Securities Act. The company also expects to grant the initial purchasers the option to buy up to an additional $52.5 million in notes.The securities will be senior unsecured obligations of the company and will accrue interest, payable twice a year in arrears on March 1 and September 1, starting on September 1, 2026. The notes are scheduled to mature on March 1, 2031, unless they are converted, redeemed or repurchased before that date.If the notes are converted, CRISPR Therapeutics will issue common shares with a nominal value of CHF 0.03 per share. The final interest rate, initial conversion rate and other key terms will be determined when the offering is priced.The company said it plans to use the net proceeds from the offering for general corporate purposes, though it did not provide further details on how the funds will be allocated.CRISPR Therapeutics stock price

Original: CRISPR Therapeutics shares drop after announcing convertible notes sale

Immuno-Oncology Investments Accelerate as Pipeline Catalysts Multiply in 2026February 25, 2026 9:00 AM
PR Newswire (Canada)

Issued on behalf of Oncolytics Biotech Inc.VANCOUVER, BC, Feb. 25, 2026 /CNW/ -- Equity Insider News Commentary, The immuno-oncology market is forecast to grow from $65.22 billion in 2025 to $170.19 billion by 2032, fueled by checkpoint inhibitors, CAR-T cell therapy, and cancer vaccines[1]. Five companies positioned at the forefront of this expansion include Oncolytics Biotech (NASDAQ: ONCY), ImmunityBio (NASDAQ: IBRX), CRISPR Therapeutics (NASDAQ: CRSP), BioNTech (NASDAQ: BNTX), and Novocure (NASDAQ: NVCR).







The broader oncology market is projected to nearly triple from $279.98 billion to $748.17 billion by 2035, with North America commanding a 43% market share[2]. Targeted therapy remains the fastest-growing segment within the $335.2 billion cancer drugs market projected for 2033, as personalized medicine reshapes treatment paradigms[3].Oncolytics Biotech Inc. (NASDAQ: ONCY) recently announced its decision to focus on registrational programs in anal and colorectal cancer, concluding enrollment in the GOBLET gastrointestinal study after generating sufficient clinical and translational data to chart a clear path toward FDA approval.The promising efficacy signal in GOBLET Cohort 4 has defined a clear registrational path for pelareorep in second-line and later squamous cell anal cancer, a setting where available therapies offer only limited benefit to patients. Oncolytics expects to meet with the FDA in mid-April to align on study design, and believes a clinical trial of well under 100 subjects will be sufficient to secure approval in this rare cancer indication.With sufficient cash on hand to execute near-term milestones, the company expects to avoid immediate material dilution, redirecting capital from the GOBLET cohorts toward its highest-conviction registration programs."GOBLET has done its job successfully. We now know where pelareorep can make the greatest impact for patients and where we can pursue approval most efficiently," said Jared Kelly, CEO of Oncolytics Biotech. "Our disciplined strategy is to run registrational or registration-enabling studies with ruthless efficiency that can create maximum shareholder value without unnecessary dilution."The registration push builds on pelareorep's recent Fast Track Designation from the FDA for second-line KRAS-mutant microsatellite-stable (MSS) metastatic colorectal cancer. Clinical data showed pelareorep combined with standard chemotherapy and Avastin® achieved a 33% response rate versus roughly 10% with chemotherapy and Avastin®, while median overall survival reached 27 months versus 11.2 months with standard treatment.KRAS-mutant MSS colorectal cancer represents one of the hardest-to-treat populations, with limited options after first-line treatment fails. The global market for second-line treatment in this patient group runs between $3 billion and $5 billion annually. The company plans to launch a controlled study with the first clinical site activating in March and interim data expected by year-end 2026.Pelareorep is also delivering strong results in anal cancer, where third-line patients achieved a 29% response rate with the median duration of response lasting around 17 months in a setting with no FDA-approved treatments. In second-line or later patients, the 30% response rate more than doubled the benchmark for the FDA-approved immunotherapy.The company continues strengthening its leadership, having recently appointed John McAdory as EVP of Strategy and Operations and Yujun Wu as Head of Biostatistics. Kelly and Chief Business Officer Andrew Aromando both joined from Ambrx Biopharma, which sold to Johnson & Johnson for $2 billion in 2024.CONTINUED… Read this and more news for Oncolytics Biotech at:https://equity-insider.com/2025/03/18/is-oncolytics-biotech-the-markets-most-undervalued-cancer-opportunity/In other industry developments:ImmunityBio (NASDAQ: IBRX) recently announced a partnership with Accord Healthcare to expand European access to ANKTIVA for bladder cancer patients across 33 countries. ANKTIVA achieved a 71% complete response rate in BCG-unresponsive non-muscle invasive bladder cancer, with approvals spanning the U.S., EU, UK, and Saudi Arabia."Our partnership with Accord marks a significant step in our European growth strategy and our mission to redefine cancer care," said Richard Adcock, President and CEO of ImmunityBio.The company continues to advance its IL-15 receptor agonist platform.CRISPR Therapeutics (NASDAQ: CRSP) provided a business update reporting encouraging clinical data from its allogeneic CAR-T candidate, zugocabtagene geleucel (zugo-cel), in both autoimmune disease and oncology indications. CASGEVY generated $116 million in full-year 2025 revenue, with patient initiations increasing nearly three-fold year-over-year."We made meaningful advances across multiple clinical and preclinical programs, including encouraging data from zugo-cel in autoimmune disease and oncology, continued global uptake of CASGEVY, and important developments across our in vivo liver editing portfolio," said Samarth Kulkarni, Ph.D., Chairman and CEO of CRISPR Therapeutics.The company is building a differentiated oncology platform with its allogeneic CAR-T approach, which could enable off-the-shelf cancer therapies without patient-specific manufacturing.BioNTech (NASDAQ: BNTX) outlined its 2026 strategy at the J.P. Morgan Healthcare Conference, detailing plans to initiate six additional Phase 3 clinical trials this year. The company now has more than 25 ongoing Phase 2 or 3 oncology trials spanning immunomodulators, antibody-drug conjugates, and mRNA cancer immunotherapies."We see 2026 as a year when science translates into tangible results," said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech.The company held approximately 17.2 billion euros in cash as of December 2025, positioning it to fund at least 17 late-stage data readouts expected by 2030.Novocure (NASDAQ: NVCR) received FDA approval for its Optune Pax system in locally advanced pancreatic cancer, the first new treatment approved for this indication in approximately 30 years. The Phase 3 PANOVA-3 trial demonstrated a statistically significant improvement in overall survival."Systemic therapies have shown poor bioavailability in pancreatic tumors, limiting their effectiveness. Optune Pax is a fundamentally different treatment, utilizing a biophysical approach that targets the unique electrical properties of cancer cells," said Frank Leonard, CEO, Novocure.The Tumor Treating Fields platform is also approved in certain countries for glioblastoma and malignant pleural mesothelioma, with additional pipeline candidates in development.Article Source: https://equity-insider.com/2025/03/18/is-oncolytics-biotech-the-markets-most-undervalued-cancer-opportunity/CONTACT:
EQUITY INSIDER
info @acblanke1DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. Equity Insider is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Oncolytics Biotech Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Oncolytics Biotech Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Oncolytics Biotech Inc. which were purchased in the open market, and reserve the right to buy and sell, and will buy and sell shares of Oncolytics Biotech Inc. at any time without any further notice commencing immediately and ongoing. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material, including this article, which is disseminated by MIQ has been approved by Oncolytics Biotech Inc.; this is a paid advertisement, we currently own shares of Oncolytics Biotech Inc. and will buy and sell shares of the company in the open market, or through private placements, and/or other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between the any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment.SOURCES:https://www.openpr.com/news/4395526/immuno-oncology-market-set-for-remarkable-expansion-ashttps://www.pharmiweb.com/press-release/2026-02-19/oncology-market-value-to-more-than-double-reaching-usd-74817-billion-by-2035https://www.prnewswire.com/news-releases/oncologycancer-drugs-market-to-reach-335-2-billion-by-2033-globally-at-7-2-cagr-allied-market-research-302683974.htmlLogo - https://mma.prnewswire.com/media/2840019/5821165/Equity_Insider_Logo.jpg



View original content to download multimedia:https://www.prnewswire.com/news-releases/immuno-oncology-investments-accelerate-as-pipeline-catalysts-multiply-in-2026-302695977.html

Original: Immuno-Oncology Investments Accelerate as Pipeline Catalysts Multiply in 2026

CRISPR Therapeutics touts zugo-cel CAR-T gains, eyes autoimmune expansion at Citi oncology summit
https://finance.yahoo.com/news/crispr-therapeutics-touts-zugo-cel-121608154.html

Strategic Priorities and Anticipated 2026 Milestones https://www.globenewswire.com/news-release/2026/01/12/3216929/0/en/crispr-therapeutics-highlights-strategic-priorities-and-anticipated-2026-milestones.html

J.P. Morgan Healthcare Conference webcast https://jpmorgan.metameetings.net/events/healthcare26/sessions/317279-crispr-therapeutics-ag/webcast/general_signin?gpu_only=true&kiosk=true

Slides https://ir.crisprtx.com/static-files/da9ff8dc-7afc-47f5-9920-8efe55cb24d4

I think we're seeing "Sell on the News" here.

OAG

''Phase 1 clinical data for CTX310® demonstrate robust, dose-dependent reductions in circulating ANGPTL3 with a mean reduction from baseline of -73% (maximum -89%), a mean reduction in triglycerides (TG) of -55% (maximum -84%), and a mean reduction of low-density lipoprotein (LDL) of -49% (maximum -87%) at the highest dose-

Among participants with elevated baseline TG (>150 mg/dL), a mean reduction of 60% in TG were observed at therapeutic doses

CTX310 was well tolerated with no treatment-related serious adverse events and no ≥Grade 3 changes in liver transaminases''

https://uk.finance.yahoo.com/news/crispr-therapeutics-announces-positive-phase-145600051.html

Slides https://ir.crisprtx.com/static-files/71bae76f-e18f-4b2a-bafd-86c5a774f20d

NEJM https://www.nejm.org/doi/full/10.1056/NEJMoa2511778

A CRISPR spokesperson told Endpoints that the company is striving to restore AAT levels toward ''the normal range (>20 µM),'' while others have set the goal of partially restoring AAT levels in the blood to a level of 11 µM. Also that CRISPR is discussing a potential licensing deal with Prime Medicine. https://endpoints.news/crispr-therapeutics-says-its-new-gene-editing-tool-is-a-significant-advance-over-prime-editing-prime-medicine-disagrees/

''The AATD program, CTX460, is the first investigational candidate to emerge from the SyNTase editing platform and the Company expects to initiate a clinical trial with CTX460 in mid-2026.'' https://finance.yahoo.com/news/crispr-therapeutics-presents-preclinical-data-120000376.html

''CRISPR Therapeutics has developed SyNTase editing, a proprietary, next-generation, site-specific gene correction platform. SyNTase editors represent a significant advance over currently described prime editing systems by combining compact Cas9 proteins with a novel class of engineered polymerases. Together, these components enable gene editing with greater efficiency and precision, while also supporting scalable manufacturing.

Using AI-guided structural modeling and large-scale screening, the polymerase was optimized to support gene correction activity based on synthetic nucleotide templates. When integrated with Cas9, SyNTase editors can utilize engineered templates with improved serum stability, enabling higher target correction efficiency.'' https://finance.yahoo.com/news/crispr-therapeutics-present-preclinical-data-120000429.html

CRSP signed a collaboration in June 2021 with them (originally, for ALS and FA) https://www.fiercebiotech.com/biotech/capsida-pauses-phase-1-gene-therapy-trial-after-child-dies



AAVs do have known risks https://www.nature.com/articles/s41392-024-01780-w

A late-breaking oral presentation highlighting the company's phase one clinical data of its investigational in vivo gene editing therapy, CTX310, targeting angiopoietin-related protein 3 (ANGPTL3) for cardiovascular and cardiometabolic disease, will be presented at the American Heart Association (AHA) Scientific Sessions 2025, taking place November 7-10, 2025, in New Orleans, Louisiana.

Additionally, the company will present a poster presentation on CTX340, its in vivo preclinical program targeting angiotensinogen (AGT) for the treatment of refractory hypertension.

Apparently another director plopped down 1 million bucks and bought shares on Aug 6.  

https://investorshub.advfn.com/boards/read_msg.aspx?message_id=176550768 $CRSP

Cathie Wood: “This Will 15X by 2030”



CRSP starts at 2 minutes.

Nice increase in volume and OBV has turned up.
Weekly Chart

My CRSP position is in my retirement account and smiled with last Friday's nice gain.
Thanks for the news,
OAG Tom

director Simeon George purchased 989,812 shares for $51.5 million at $52.03, increasing his stake by 134% to 1.73 million shares, valued at approximately $110 million

It appears that CRSP has had some accumulation activity recently when looking at this histogram from StockCharts:

https://schrts.co/qKzkRRgT

My first target price for selling some inventory in CRSP doesn't occur for another $10/share. I need around $68 for it to trigger. Even so, the improvement in price is much appreciated.

Best wishes,
OAG

An update https://www.globenewswire.com/news-release/2025/06/26/3105698/0/en/CRISPR-Therapeutics-Reports-Positive-Additional-Phase-1-Data-for-CTX310-Targeting-ANGPTL3-and-Provides-Update-on-In-Vivo-Cardiovascular-Pipeline.html

''New Phase 1 clinical data for CTX310™ continues to demonstrate dose-dependent reductions in triglycerides (TG) and low-density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 86% in LDL, with a well-tolerated safety profile

Complete Phase 1 data presentation for CTX310 anticipated at a medical meeting in the second half of 2025

Data update for CTX320™, targeting the LPA gene, now expected in the first half of 2026

Preclinical in vivo cardiovascular program CTX340™ advancing toward IND / CTA filings targeting refractory hypertension.''

Slightly off-topic, but VRTX just published this (on an iPSC-derived islet-cell therapy) https://www.nejm.org/doi/full/10.1056/NEJMoa2506549

After twelve months, out of the twelve, ten no longer needed to inject insulin.

VRTX paid $100 million upfront for non-exclusive rights to CRSP's tech https://www.fiercebiotech.com/biotech/vertex-goes-back-crispr-well-paying-100m-upfront-exa-cel-partner-help-diabetes-work

CRSP is also working on its own iPSC-derived islet-cell therapy. Along with the core edits for immune evasion (MHC-I KO, PD-L1 KI and HLA-E KI) and cell fitness (TXNIP KO, TNFAIP3 KI and MANF KI) there will be additional ones (undisclosed).

TXNIP KO protects from oxidative and ER stress https://faseb.onlinelibrary.wiley.com/doi/10.1096/fj.08-111690

TNFAIP3 KI induces graft acceptance and protection from cytokine induced apoptosis (programmed cell death) https://insight.jci.org/articles/view/131028

MANF KI enhances proliferation and protection against inflammatory stress https://www.cell.com/cell-reports/fulltext/S2211-1247(14)00201-0

New data from Dr. Liu's group. It is the result of more than three years of continuously improving CRISPR-associated transposases. A version, dubbed evoCAST, improves the editing efficiency from less than 0.1% of human cells to between ~10%-30% of cells.

One major hurdle of turning the evoCAST into a therapeutic solution that is able to insert large genes is the size and collective complexity of the seven proteins that comprise the system. But Dr. Liu said they are working on streamlining the system further.

https://www.science.org/doi/10.1126/science.adt5199

Good news seems to be on the long-term horizon. The problem is the time it takes to adequately test dosage. Ten patients isn't going to cut the mustard and get this product into Phase III for several years. I have seen this scenario happen with other companies. Stocks get ruined because the companies are more interested in trying to perfect dosage than to take a positive dosage and navigate it through advanced trials. APTOF is a prime example of that type of history.

Based on the CTX310 data, it looks to have a clean safety profile. There were no SAEs, no grade =3 AEs, and no clinically significant changes in either liver enzymes (ALT, AST and/or bilirubin) or platelets across all dose levels. However, just ten patients across all four dose cohorts were treated. The data shows minimal activity at DL1 and DL2, but meaningful responses at DL3 and DL4. A single patient at the highest dose level achieved the most impressive reductions, indicating they could take this into the phase one(b).

Q1 financial results and positive top-line data from phase one trial of CTX310 https://www.globenewswire.com/news-release/2025/05/06/3075498/0/en/CRISPR-Therapeutics-Provides-First-Quarter-2025-Financial-Results-and-Announces-Positive-Top-Line-Data-from-Phase-1-Clinical-Trial-of-CTX310-Targeting-ANGPTL3.html

CRSP recent 52 week low

CRSP, new 52 week low

CRISPR-X is working on CRISPR-associated transposases, base editors, prime editors, epigenetic editors and novel editing. All have (multiple) advantages over oligonucleotides.

Sold my CRSP shares and BOUGHT MORE WVE WAVE THERAPEUTICS

RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; WVE-007 for the treatment of obesity and other metabolic disorders; WVE-N531, an exon skipping oligonucleotide for the treatment of duchenne muscular dystrophy; and WVE-003, an antisense silencing oligonucleotide for the treatment of Huntington's disease (HD). It also engages in the programs, for discovery stage research of PNPLA3, LDLR, and APOB. The company has collaboration agreements with GlaxoSmithKline for the research, development, and commercialization of oligonucleotide therapeutics; Takeda Pharmaceutical Company Limited for the research, development, and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System; and Asuragen, Inc. for the development and potential commercialization of companion diagnostics for investigational allele-selective therapeutic programs targeting HD. Wave Life Sciences Ltd. was incorporated in 2012 and is based in Singapore.

Looks pretty bullish for Casgevy https://www.businesswire.com/news/home/20250221469479/en/bluebird-bio-Announces-Definitive-Agreement-to-be-Acquired-by-Carlyle-and-SK-Capital

Added a chunk today to my stash average is now 42.00

$CRSP

Nice pre market action!

Very deep pipeline with a mega partner VRTX

$CRSP

Starting to turn the corner now!

Glad I re-loaded the low 40's

$CRSP

Anticipated 2025 milestones https://www.globenewswire.com/news-release/2025/01/13/3008308/0/en/CRISPR-Therapeutics-Highlights-Strategic-Priorities-and-Anticipated-2025-Milestones.html

Link to the webcast https://jpmorgan.metameetings.net/events/healthcare25/sessions/58421-crispr-therapeutics/webcast/general_signin?gpu_only=true&kiosk=true+%5Bjpmorgan.metameetings.net%5D

They will present at the 43rd Annual J.P. Morgan Healthcare Conference (Jan 14, at 2:15 PM PST).

cheap cheap

The PR https://www.globenewswire.com/news-release/2024/12/09/2993974/0/en/CRISPR-Therapeutics-Presents-Data-at-the-2024-American-Society-of-Hematology-ASH-Annual-Meeting.html

Poster https://ir.crisprtx.com/static-files/12114290-21a6-4544-9cb7-de4fe2852e94

Neglected ugly duckling. $NTLA, $VERV & $CRBU now do trials outside of the US.... Maybe sentiment change is on the horizon -- see below

"The CBER director also apologized for “mistakes” in how the FDA handled early work for in vivo gene therapies, which “led to offshoring of some of the trials.”"
https://endpts.com/fda-vaccine-chief-peter-marks-pledges-to-stay-as-long-as-hes-welcome/#:~:text=The%20CBER%20director%20also%20apologized,%E2%80%9Cit%20is%20really%20important.%E2%80%9D

CHEAP CHEAP
SRPT and BMS announcing nice CRISPR deals with ARWR and PRME should help this too!

CTX130 data in R/R T-cell lymphomas (peripheral T-cell lymphoma or cutaneous T-cell lymphoma) https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(24)00508-4/abstract

The ORR was 46.2%. Of those treated at DL3 and higher, the ORR was 51.6%.

so so so undervalued and misunderstood i believe...... frankly just cheap
#FDA CBER's Marks apparently apologizing (!) for holding back in vivo #CRISPR development in the US:

h/t @endpts pic.twitter.com/AZi8R30F7Y— Dirk Haussecker (@RNAiAnalyst) November 22, 2024

They now have the ability to put in eight to nine edits. So it is a single cassette that expresses the CAR, can express a cytokine and has an shRNA-like approach to knock down genes. So there's no theoretical limit as to the number of edits, but there may be marginal utility beyond a certain point.

Hopefully, they look to improve donor selection, cell selection and manufacturing, as all could increase efficacy.

ASH abstract https://ash.confex.com/ash/2024/webprogram/Paper203563.html

Q3 business update and financial results https://www.globenewswire.com/news-release/2024/11/05/2974996/0/en/CRISPR-Therapeutics-Provides-Business-Update-and-Reports-Third-Quarter-2024-Financial-Results.html

CRSP 10Q 11/5

CRSP under $50