Cell and Gene Therapy Video Recap—November 13, 2025

LucidQuest

5 days ago

This biweekly Cell and Gene Therapy video spotlights the most important trends driving the future of cell and gene therapy, covering science, industry, and regulatory milestones.

🎯 Watch Our Video Summary Capturing Cell and Gene Therapy News from the Last Two Weeks

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Week 30 October–5 November 2025
Week 6–12 November 2025

📚 See the full Cell and Gene Therapy archive on our research hub page.

Transcript

Welcome to the latest edition of Gene & Cell Therapy Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.

Sarepta Therapeutics ran a confirmatory study for Amondys 45 and Vyondys 53 in Duchenne muscular dystrophy, but the trial failed, and the outlook for Elevidys was softened after prior safety scrutiny. The company plans FDA discussions on converting accelerated to full approvals despite the negative trial, which may shape prescriber decisions and payer reviews as full data emerge.

Caribou Biosciences reported Phase 1 ANTLER results for allogeneic vispa-cel, which included a confirmatory cohort of 22 patients and an optimized cohort of 35 patients. The overall response, complete response, and 12-month progression-free survival were on par with autologous CAR-T, alongside outpatient-amenable safety. The FDA recommended a randomized Phase 3 in second-line LBCL for CD19-naïve patients, with about 250 participants and progression-free survival as the primary endpoint.

uniQure announced that the FDA reversed course on Huntington’s disease program AMT-130, indicating that the data are insufficient for an approval filing after earlier expedited signals. The timing for submission is now unclear, and investor sentiment has turned negative.

Eli Lilly and MeiraGTx entered into a licensing agreement for AAV-AIPL1, a gene therapy for LCA4, with $75 million upfront and a deal valued up to $475 million plus royalties. Lilly gained access to capsids, AI-guided promoters, and riboswitch technology. The therapy reportedly restored vision in 11 children with LCA4, and filings are planned in both the United Kingdom and the United States.

Tenaya Therapeutics announced that the FDA placed a clinical hold on the TN-201 study in MYBPC3-hypertrophic cardiomyopathy, a Phase 1b 2a program. Although the DSMB had cleared expansion and cohort 2 showed higher transduction with shorter steroid courses, the hold aims to standardize immunosuppression monitoring and management across sites, with no new meaningful safety events reported.

Intellia Therapeutics reported a patient death following grade 4 elevations of transaminases and bilirubin in the Phase 3 MAGNITUDE program for ATTR. Both MAGNITUDE and MAGNITUDE-2 are on FDA hold, with the treating physician attributing the death to complicating comorbidities. The company is now developing a risk-mitigation plan.

Dyno Therapeutics unveiled the Dyno-bn8 AAV capsid, which showed nonhuman primate data at 5.2e12 vector genomes per kilogram. This indicated broad skeletal and cardiac muscle transduction with liver detargeting and manufacturability compatible with AAV9 processes. The platform is available for licensing and for Frontiers partners.

Opus Genetics reported that an RMAT meeting supported an adaptive pivotal path for OPGx-LCA5. Six late-stage participants in Phase 1 2 had clinically meaningful vision gains, and a run-in for Phase 3 has started. The plan is for a small, single-arm adaptive Phase 3 with an application under the Rare Pediatric Disease Priority Review Voucher pathway.

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Why It Matters

FDA unpredictability is reshaping late-stage gene therapy paths and investor risk calculations [1][3][5][6].
Off-the-shelf cell therapies are approaching autologous performance with simpler logistics and outpatient potential [2].
Ocular gene therapy momentum continues with both BD and regulatory alignment toward pivotal studies [4][8].
Next-gen delivery vectors aim to cut dose and off-target exposure, a key safety lever for systemic gene therapies [7].

🗓️ Explore weekly details and sources

Week 30 October–5 November 2025
Week 6–12 November 2025

📚 See the full Cell and Gene Therapy archive on our research hub page.

FAQ

What failed for Sarepta and does it affect market availability?

The confirmatory DMD trial for Amondys 45 and Vyondys 53 missed. Sarepta says availability is unchanged while it engages FDA on conversion to full approvals [1].

How close is Caribou’s vispa-cel to autologous CAR-T efficacy?

In Phase 1 LBCL, ORR, CR, and 12-month PFS were on par with approved autologous CAR-T in confirmatory and optimized cohorts, with outpatient-amenable safety; a randomized Phase 3 is planned in 2L LBCL [2].

Why did the FDA shift on uniQure’s AMT-130?

After earlier expedited signals, FDA now views current data as inadequate for filing, delaying timelines despite positive mid-stage readouts [3].

What exactly did Lilly obtain from MeiraGTx?

Exclusive global rights to AAV-AIPL1 for LCA4 plus access to ophthalmology gene therapy technologies, for $75M upfront and milestones [4].

What triggered Tenaya’s clinical hold?

FDA requested protocol changes to standardize immunosuppression monitoring and management across sites in the TN-201 MYBPC3-HCM study [5].

What is Dyno-bn8 and why is it notable?

An AAV capsid with NHP data showing low-dose systemic muscle delivery and liver detargeting, compatible with AAV9 manufacturing, and available to license [7].

Entities / Keywords

Sarepta Therapeutics; Amondys 45; Vyondys 53; Elevidys; Caribou Biosciences; vispa-cel; ANTLER; LBCL; uniQure; AMT-130; Huntington’s disease; Eli Lilly; MeiraGTx; AAV-AIPL1; LCA4; Tenaya Therapeutics; TN-201; MYBPC3-HCM; Intellia Therapeutics; nexiguran ziclumeran; ATTR; Dyno Therapeutics; Dyno-bn8; AAV capsid; Opus Genetics; OPGx-LCA5; RMAT; RDEP.