Cell and Gene Therapy Today—November 13, 2025

This week’s cell and gene therapy update covers key developments including safety signals, trial initiations, and early efficacy readouts. It also highlights regulatory decisions, platform advances, and combination strategies that are shaping the evolving cell and gene therapy landscape.

In Today’s Newsletter

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👁️ Lilly licenses MeiraGTx’s LCA4 gene therapy for up to $475M [1] [10 Nov 2025]

https://www.fiercebiotech.com/biotech/lilly-bags-eye-catching-retinal-disease-gene-therapy-meiragtx-475m-deal
Context: MeiraGTx’s AAV-AIPL1 restored vision in 11 children with LCA4, per company; UK and US filings planned.
Key point: Lilly gains global rights to AAV-AIPL1 and access to MeiraGTx capsids, AI promoters, and riboswitch tech.
Implication: Signals pipeline investment and modality expansion.

⚠️ FDA places hold on Tenaya’s TN-201 HCM gene therapy trial [2] [10 Nov 2025]

https://www.fiercebiotech.com/biotech/fda-puts-tenaya-heart-disease-gene-therapy-clinical-trial-hold
Context: Phase 1b/2a in MYBPC3-associated HCM; DSMB okayed expansion in July; immunosuppression course shortened in Cohort 2.
Key point: Hold tied to standardizing immunosuppression monitoring and management across sites; company sees no new meaningful safety events.
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 Patient death after Intellia’s CRISPR ATTR therapy event [3] [07 Nov 2025]

https://www.biospace.com/drug-development/patient-dies-after-receiving-intellias-crispr-gene-therapy-candidate
Context: Grade 4 liver toxicity in Phase 3 MAGNITUDE; MAGNITUDE and MAGNITUDE-2 paused and on FDA hold.
Key point: Death attributed to complicating comorbidities by physician; company developing a risk mitigation plan.
Implication: May influence prescriber choice and payer reviews pending full data.

🧠 First gene therapy trial for Angelman syndrome doses patient [4] [06 Nov 2025]

https://finance.yahoo.com/news/mavrix-bio-announces-first-patient-122700027.html
Context: Phase 1/2 ASCEND-AS of MVX-220 using AAV to restore UBE3A in CNS; adult and pediatric genotypes included.
Key point: First patient dosed; FDA Orphan Drug Designation granted for MVX-220.
Implication: Signals pipeline investment and modality expansion.

❤️ YAP101 heart-failure gene therapy clears first safety check [5] [11 Nov 2025]

https://www.biospace.com/press-releases/first-clinical-investigation-of-a-gene-therapy-yap101-to-induce-cardiac-regeneration-to-treat-heart-failure-passes-initial-safety-hurdle
Context: Phase 1 SALVADOR-HF, first cohort (n=3) completed; AAV shRNA transiently lifts Hippo brake to activate YAP.
Key point: Independent Safety Review Team recommended escalation to next dose.
Implication: May influence prescriber choice and payer reviews pending full data.

💪 Dyno-bn8 AAV claims best-in-class muscle delivery in NHPs [6] [11 Nov 2025]

https://www.biospace.com/press-releases/dyno-therapeutics-unveils-best-in-class-dyno-bn8-aav-vector-for-muscle-gene-therapies-achieving-therapeutic-delivery-with-improved-safety-potential
Context: NHP data: systemic 5.2e12 vg/kg, broad skeletal and cardiac uptake with liver detargeting; manufacturable with AAV9 processes.
Key point: Company positions bn8 as low-dose, safety-improved vector and opens licensing.
Implication: Signals pipeline investment and modality expansion.

👁️ Opus LCA5 program aligned with FDA on pivotal path [7] [06 Nov 2025]

https://www.globenewswire.com/news-release/2025/11/06/3182378/0/en/Opus-Genetics-Announces-Successful-FDA-Meeting-Supporting-Advancement-of-OPGx-LCA5-Toward-Pivotal-Trial-for-LCA5-Related-Inherited-Retinal-Disease.html
Context: RMAT Type B meeting; six late-stage participants showed clinically meaningful vision gains in Phase 1/2.
Key point: Adaptive Phase 3 envisioned with small single-arm design and run-in; intent to use new RDEP review process.
Implication: May influence prescriber choice and payer reviews pending full data.

🇦🇺 CRISPR CTX310 halves LDL and triglycerides in early trial [8] [Australia • 11 Nov 2025]

https://madhyamamonline.com/amp/lifestyle/health/australia-runs-historic-gene-editing-therapy-to-halve-bad-cholesterol-1466309
Context: First-in-human trial across AU/NZ/UK; lipid nanoparticle CRISPR targets ANGPTL3; NEJM-reported per article.
Key point: Up to ~50% LDL and ~55% triglyceride reductions at highest dose at 60 days; mild short-term side effects reported.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🐱 FDA-CVM safety section complete for feline stem cell therapy [9] [10 Nov 2025]

https://www.prnewswire.com/news-releases/fda-completes-safety-for-gallants-first-ready-to-use-stem-cell-therapy-for-refractory-fcgs-302610213.html
Context: Sonruvetcel, uterine-derived allogeneic MSCs for refractory feline chronic gingivostomatitis.
Key point: Target Animal Safety “Technical Section Complete,” supporting 365-day safety; conditional approval targeted early 2026.
Implication: Introduces competition that may affect pricing and formulary access.

🤝 Cue Biopharma–ImmunoScape “Seed-and-Boost” cell therapy deal [10] [06 Nov 2025]

https://finance.yahoo.com/news/cue-biopharma-immunoscape-announce-strategic-130000615.html
Context: Combines TCR-T “Seed” with Immuno-STAT IL-2 “Boost” for solid tumors; preclinical data reported.
Key point: Upfront $15M and equity; IND-enabling studies targeted for 2027 submission.
Implication: Signals pipeline investment and modality expansion.

🧫 Indapta allogeneic g-NK shows activity in R/R myeloma [11] [07 Nov 2025]

https://www.businesswire.com/news/home/20251107598785/en/Indapta-Therapeutics-Presents-Data-Demonstrating-Clinical-Activity-of-its-Allogeneic-Natural-Killer-Cell-Therapy-in-Patients-with-RelapsedRefractory-Multiple-Myeloma
Context: SITC presentation, Phase 1/2 safety run-in plus combo cohort with isatuximab; 10B cells/dose level noted.
Key point: Confirmed responses in 4/5 evaluable patients at stated combo dose; treatment generally well tolerated.
Implication: May influence prescriber choice and payer reviews pending full data.

🌟 MiNK’s allo-iNKT agenT-797 in PD-1–refractory tumors [12] [07 Nov 2025]

https://www.globenewswire.com/news-release/2025/11/07/3183675/0/en/MiNK-Therapeutics-Reports-Durable-Responses-and-Immune-Reactivation-with-Allo-iNKT-Cell-Therapy-agenT-797-in-PD-1-Refractory-Solid-Tumors-at-SITC-2025.html
Context: SITC late-breaking abstract; single-agent and combo with anti-PD-1 across multiple solid tumors.
Key point: Reports durable survival and deep remissions with immune reactivation and favorable safety profile.
Implication: May influence prescriber choice and payer reviews pending full data.

🧾 Lyell acquires GCC CAR T LYL273 for mCRC, early ORR 67% at higher dose [13] [US • 10 Nov 2025]

https://ir.lyell.com/news-releases/news-release-details/lyell-immunopharma-acquires-exclusive-global-rights-next
Context: US Phase 1 data cutoff 28 Oct 2025; dose-dependent activity with manageable safety after protocol optimization.
Key point: Exclusive rights outside Greater China; Fast Track in US; upfront $40M plus stock and milestones.
Implication: Signals pipeline investment and modality expansion.

Why it matters

Vision-restoring retinal gene therapies are moving toward registrational paths in ultra-rare LCA subtypes [1][7].
Cardiac gene therapy remains promising but operational rigor around immunosuppression is front-and-center [2][5].
Safety governance in in vivo editing and ATTR remains critical as programs scale to late-phase studies [3][8].
Next-gen delivery and off-the-shelf cell platforms aim to lower dose, improve manufacturability, and broaden access [6][10][11][12].
Oncology cell therapy momentum extends into solid tumors with targeted CARs and immune-restorative iNKT approaches [12][13].

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FAQ

What did Lilly obtain from MeiraGTx and for which disease?

Lilly licensed global rights to AAV-AIPL1 for Leber congenital amaurosis type 4 and gained access to certain MeiraGTx ophthalmology platforms, with $75M upfront and milestones [1].

Why is Tenaya’s TN-201 trial on FDA hold?

The FDA requested a protocol amendment to standardize immunosuppression monitoring and management across sites in the Phase 1b/2a study in MYBPC3-HCM [2].

What happened in Intellia’s ATTR program?

A patient who experienced grade 4 liver toxicity later died, attributed to complicating comorbidities by the physician. Studies are paused and on FDA hold while a risk plan is developed [3].

What is CTX310 and what were early outcomes?

CTX310 is a CRISPR-Cas9 therapy delivered via lipid nanoparticles to knock out ANGPTL3. Early participants saw roughly 50% LDL and 55% triglyceride reductions at highest dose, with mild short-term side effects, per report [8] (endpoint specifics and duration limited).

How is Opus advancing OPGx-LCA5?

After an RMAT meeting, Opus plans an adaptive Phase 3 with a run-in period and intends to apply under the Rare Disease Evidence Principles process [7].

What is Lyell’s newly acquired LYL273 showing so far?

In a US Phase 1, LYL273 (GCC CAR T) showed dose-dependent activity with 67% ORR at the higher dose level and a manageable safety profile under optimized management, per company update [13].

Entities / Keywords

Eli Lilly; MeiraGTx; AAV-AIPL1; LCA4; Tenaya Therapeutics; TN-201; MYBPC3-HCM; Intellia Therapeutics; nexiguran ziclumeran; ATTR; MavriX Bio; MVX-220; UBE3A; Angelman syndrome; Medley Therapeutics; YAP101; Hippo pathway; Dyno Therapeutics; Dyno-bn8; AAV capsid; Opus Genetics; OPGx-LCA5; RMAT; RDEP; CTX310; ANGPTL3; Gallant; sonruvetcel; FCGS; Cue Biopharma; ImmunoScape; Immuno-STAT; TCR-T; Indapta Therapeutics; IDP-023; g-NK; isatuximab; MiNK Therapeutics; agenT-797; iNKT; Lyell Immunopharma; LYL273; GCC CAR T; mCRC.