Cell and Gene Therapy Today—November 6, 2025

LucidQuest

2 days ago

This week’s cell and gene therapy update spotlights key developments, regulatory milestones, and the latest progress shaping the biopharma landscape.

In Today’s Newsletter

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👁️ Otsuka licenses 4D-150 ophthalmic gene therapy rights from 4DMT [1] [31 Oct 2025]

https://www.otsuka.co.jp/en/company/newsreleases/2025/20251031_1.html
Context: 4D-150 targets four VEGF family members via AAV; reported durability to 130 weeks (source figure). 4DMT retains global Phase 3 lead, with APAC sites planned end-2025 and Japan in Jan 2026.
Key point: Otsuka gains exclusive Asia–Oceania rights to 4D-150 for nAMD and DME with $85M upfront, milestones, and tiered royalties.
Implication: Signals pipeline investment and modality expansion.

🧬 Sarepta slumps on DMD trial miss and weak Elevidys outlook [2] [04 Nov 2025]

https://www.reuters.com/business/healthcare-pharmaceuticals/sarepta-slumps-gene-therapy-setback-adds-drug-pipeline-woes-2025-11-04/
Context: Company cites pandemic disruptions; analysts question full-approval prospects though drugs remain marketed.
Key point: Shares fell nearly 30% after confirmatory trial failure for Amondys 45 and Vyondys 53 and softer near-term Elevidys volumes.
Implication: May influence prescriber choice and payer reviews pending full data.

🤖 Latus Bio unveils AI/ML engine for AAV capsids and payloads, adds SAB members [3] [03 Nov 2025]

https://www.businesswire.com/news/home/20251103349850/en/Latus-Bio-Launches-AIML-Strategy-to-Transform-AAV-Gene-Therapy-Adds-Two-Leading-Experts-in-Peptide-and-Protein-Modeling-to-Scientific-Advisory-Board
Context: Focus spans capsid tropism, regulatory elements, and payload design to speed discovery.
Key point: Company will train models on nine NHP campaigns and 100M+ “delivery zip codes” and adds Drs. Chatterjee and Kim to the SAB.
Implication: Signals pipeline investment and modality expansion.

🧪 Helex raises $3.5M seed for kidney-targeted non-viral gene editing in ADPKD [4] [30 Oct 2025]

https://www.businesswire.com/news/home/20251030697943/en/Helex-Raises-%243.5-Million-in-Seed-Round-to-Advance-Non-Viral-Gene-Therapies-for-Kidney-Diseases
Context: Platform aims single-dose, non-viral gene-editing therapy for PKD1/PKD2-driven disease.
Key point: Financing led by pi Ventures supports kidney-tropic LNP platform and lead ADPKD program toward IND-enabling studies.
Implication: Signals pipeline investment and modality expansion.

🧫 Caribou’s vispa-cel shows autologous-like outcomes in LBCL Phase 1 ANTLER [5] [03 Nov 2025]

https://investingnews.com/caribou-biosciences-announces-positive-data-from-antler-phase-1-trial-demonstrating-efficacy-and-durability-of-vispa-cel-an-allogeneic-car-t-cell-therapy-on-par-with-autologous-car-t-cell-therapies/
Context: Partial HLA matching and young donors linked to outcomes; safety generally manageable with outpatient feasibility.
Key point: Confirmatory cohort (N=22) reported 82% ORR, 64% CR, 51% 12-mo PFS; optimized profile cohort (N=35) reported 86% ORR, 63% CR, 53% 12-mo PFS.
Implication: May influence prescriber choice and payer reviews pending full data.

❤️ BioCardia enrolls first UW patient in Phase 3 CardiAMP HF II [6] [30 Oct 2025]

https://www.manilatimes.net/2025/10/30/tmt-newswire/globenewswire/biocardia-announces-university-of-wisconsin-enrolls-their-first-patient-in-phase-3-cardiamp-hf-ii-cell-therapy-pivotal-trial/2212140
Context: Trial targets patients with elevated cardiac stress markers on optimized therapy.
Key point: University of Wisconsin site enrolls its first ischemic HF patient in ongoing pivotal study.
Implication: May influence prescriber choice and payer reviews pending full data.

🩸 Beam to present updated BEAM-101 BEACON SCD data at ASH 2025 [7] [03 Nov 2025]

https://www.globenewswire.com/news-release/2025/11/03/3179359/0/en/Beam-Therapeutics-to-Present-Updated-Data-from-BEACON-Phase-1-2-Trial-of-BEAM-101-in-Sickle-Cell-Disease-at-American-Society-of-Hematology-ASH-Annual-Meeting.html
Context: Presentations include HbF induction outcomes and mobilization approaches in ongoing Phase 1/2.
Key point: Company to share safety and efficacy updates on ex vivo base-edited HSPC therapy BEAM-101.
Implication: May influence prescriber choice and payer reviews pending full data.

🧠 uniQure hit by FDA shift on AMT-130 for Huntington’s disease [8] [03 Nov 2025]

https://www.biopharmadive.com/news/uniqure-fda-huntingtons-gene-therapy-reversal-approval-application/804516/
Context: Shares fell after update; article notes broader uncertainty around FDA review dynamics.
Key point: Company now believes current mid-stage data may be insufficient for approval filing, reversing earlier alignment signals.
Implication: Introduces competition that may affect pricing and formulary access.

👓 AAVantgarde raises $141M series B for retinal gene therapy trials [9] [03 Nov 2025]

https://www.fiercebiotech.com/biotech/italian-biotech-aavantgarde-eyes-143m-series-b-fund-retinal-disease-gene-therapy-trials
Context: Early AAVB-081 data showed signals of benefit per company at a retinal meeting.
Key point: Financing will fund Phase 1/2 programs AAVB-039 (Stargardt) and AAVB-081 (Usher 1B RP).
Implication: Signals pipeline investment and modality expansion.

🛡️ Genprex gets EPO intent to grant patent on REQORSA + PD-1 combos [10] [04 Nov 2025]

https://www.genprex.com/2025/11/04/genprex-announces-european-patent-offices-intent-to-grant-a-patent-for-the-combination-of-reqorsa-gene-therapy-and-pd-1-antibodies-to-treat-cancer/
Context: Company positions REQORSA for combinations in lung cancer, citing preclinical complementarity.
Key point: European patent to cover REQORSA with PD-1 antibodies, adding to grants in multiple countries.
Implication: Introduces competition that may affect pricing and formulary access.

Why it matters

Ophthalmology continues to attract capital and partners, with Otsuka–4DMT and AAVantgarde advancing clinical portfolios.
Allogeneic cell therapy momentum grows as Caribou reports autologous-like outcomes with manageable safety.
FDA unpredictability remains a core risk for gene therapy timelines, per uniQure coverage.
Kidney-targeted delivery and AI-designed AAVs highlight platform innovation beyond liver and eye.
Upcoming hematology readouts, including Beam’s BEACON, may shape 2026 competitive positioning.

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FAQ

What did Otsuka license from 4DMT, and where? [1]

Otsuka licensed exclusive development and commercialization rights to 4D-150 for nAMD and DME in Asia–Oceania, including Japan. 4DMT keeps global Phase 3 leadership and plans APAC site openings by end-2025 and in Japan in Jan 2026.

Why did Sarepta’s stock drop? [2]

A confirmatory trial for Amondys 45 and Vyondys 53 failed, and management guided to flat or slightly lower Elevidys infusions for Q4. Reuters reports a nearly 30% share decline on Nov 4, 2025.

How strong are Caribou’s ANTLER data for vispa-cel? [5]

In LBCL, confirmatory cohort ORR/CR were 82%/64% with 51% 12-mo PFS. An optimized cohort showed 86% ORR, 63% CR, and 53% 12-mo PFS. Safety was generally manageable with outpatient use noted.

What is Latus Bio’s AI/ML initiative aiming to do? [3]

Leverage a large in vivo NHP dataset to train models for AAV capsid tropism and payload design, guided by new SAB members Drs. Chatterjee and Kim, to accelerate discovery and delivery specificity.

What changed for uniQure’s AMT-130 with FDA? [8]

After a recent meeting, uniQure now believes current mid-stage data may not support an approval application, a notable shift from prior signals. The article frames broader FDA unpredictability for gene therapies.

What IP did Genprex secure in Europe? [10]

EPO’s intent to grant covers using REQORSA with PD-1 antibodies for cancer, adding to similar patents in several countries, supporting combination strategies in lung cancer.

Entities / Keywords

Otsuka Pharmaceutical; 4D Molecular Therapeutics; 4D-150; nAMD; DME; AAV; Sarepta Therapeutics; Amondys 45; Vyondys 53; Elevidys; Latus Bio; AI/ML; AAV capsid engineering; Helex; LNP; ADPKD; Caribou Biosciences; vispa-cel; LBCL; ANTLER; BioCardia; CardiAMP HF II; Beam Therapeutics; BEAM-101; SCD; BEACON; uniQure; AMT-130; Huntington’s disease; AAVantgarde Bio; AAVB-039; AAVB-081; Stargardt; Usher 1B; Genprex; REQORSA; PD-1.