In this biweekly edition, we cover the most significant developments shaping the future of Gene & Cell Therapy — from scientific advances to industry and regulatory milestones.
Watch Our Video Summary Capturing Top Cell and Gene News from the Last Two Weeks
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Top Stories Covered in This Video
💊 Astellas passes on TSHA-102 option as Taysha preps pivotal [1] [17 Oct 2025]
https://www.fiercebiotech.com/biotech/astellas-walks-away-gene-therapy-pact-taysha-pivotal-trial-launch
Context: 2022 equity/option deal covered TSHA-102 for Rett and other CNS assets. Taysha shared adult and pediatric Phase 1/2 data in mid-2025.
Key point: Astellas declined to license TSHA-102; Taysha retains full rights and plans a single-arm, open-label pivotal in Rett with a milestone-based primary endpoint.
Implication: Signals pipeline investment and modality expansion.
❤️ Lexeo’s LX2006 gets FDA openness to accelerated approval, plus interim positives [2] [17 Oct 2025]
https://finance.yahoo.com/news/lexeo-therapeutics-announces-fda-support-132708869.html
Context: Ongoing Phase 1/2 in FA cardiomyopathy; switch to Sf9-baculo manufacturing planned for pivotal.
Key point: FDA indicated openness to an accelerated approval BLA using pooled Phase 1/2 plus pivotal data, with earlier LV mass index endpoint; added CMC and nonclinical asks.
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 Immusoft’s engineered B-cell therapy ISP-001 earns Fast Track for MPS I [3] [21 Oct 2025]
https://www.morningstar.com/news/pr-newswire/20251021cl02855/immusoft-receives-fda-fast-track-designation-for-isp-001-a-first-in-class-engineered-b-cell-therapy-for-mps-i
Context: Phase 1/2; early data show favorable safety and first re-dosing with pharmacodynamic and functional gains.
Key point: FDA granted Fast Track to ISP-001, which programs autologous B cells to secrete IDUA for MPS I.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 Galapagos to wind down cell therapy unit after failed sale process [4] [21 Oct 2025]
https://www.fiercebiotech.com/biotech/galapagos-wind-down-cell-therapy-unit-threatening-365-jobs-and-5-facilities
Context: Strategic review since May; limited non-binding offers; 365 roles and 5 sites affected across regions.
Key point: Company will cease investing in the unit and pursue broader BD to rebuild pipeline, while considering any viable acquisition proposals during wind-down.
Implication: Signals pipeline investment and modality expansion.
🧬 Intellia pauses Phase 3 MAGNITUDE trials of nex-z in ATTR after Grade 4 LFTs [5] [27 Oct 2025]
https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-provides-update-magnitude-clinical-trials
Context: MAGNITUDE (ATTR-CM) and MAGNITUDE-2 (ATTR-PN); >650 enrolled and ~450+ dosed in total.
Key point: Temporary pause to dosing/screening after one patient had Grade 4 transaminase elevation with hyperbilirubinemia; company engaging regulators on risk mitigation.
Implication: May influence prescriber choice and payer reviews pending full data.
👁️ Lilly to acquire Adverum, bringing Phase 3 Ixo-vec for wAMD [6] [24 Oct 2025]
https://investor.lilly.com/news-releases/news-release-details/lilly-acquire-adverum-biotechnologies
Context: Ixo-vec is a single-dose intravitreal gene therapy in Phase 3 ARTEMIS; RMAT, Fast Track, PRIME and UK Innovation Passport.
Key point: Deal includes cash plus CVR tied to U.S. approval and $1B sales milestone; bridge financing via secured promissory note supports ongoing trials pre-close.
Implication: Signals pipeline investment and modality expansion.
🧠 BridgeBio’s BBP-418 hits Phase 3 interim in LGMD2I/R9, NDA planned H1 2026 [7] [27 Oct 2025]
https://www.biospace.com/drug-development/bridgebio-pops-on-late-stage-limb-girdle-muscular-dystrophy-data
Context: FORTIFY trial; biomarker αDG increase and CK reduction; functional gains at 12 months reported.
Key point: Interim met primary and secondary endpoints with no new safety signals; company guiding to U.S. filing in H1 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
🩸 BioMarin to divest Roctavian after limited sales, keeps supply in 3 markets [8] [28 Oct 2025]
https://www.fiercepharma.com/pharma/biomarin-resigned-ending-journey-hemophilia-gene-therapy-roctavian-seeking-divestiture-amid
Context: Q3 sales were modest; prior commercial narrowing to U.S., Germany and Italy with idle manufacturing.
Key point: Company will pursue out-licensing or divestiture while maintaining access in current reimbursed markets.
Implication: Signals pipeline investment and modality expansion.
Why it matters
- Clinical momentum continued, with one late-stage win (BridgeBio) alongside a gene-editing safety pause (Intellia).
- Regulatory pathfinding remained active, from Fast Track (Immusoft) to accelerated approval dialogue (Lexeo).
- Strategic reshaping accelerated, with one acquisition (Lilly–Adverum) and multiple exits or wind-downs (Astellas/Taysha option lapse, Galapagos, BioMarin).
- Manufacturing choices surfaced as gating factors for acceleration and scaling (Lexeo’s Sf9-baculo comparability).
- Market realities in one-time therapies continued to pressure commercial viability in crowded categories (Roctavian vs factor and non-factor options).
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FAQ
What is TSHA-102 and why did Astellas pass?
TSHA-102 is Taysha’s AAV gene therapy for Rett syndrome. After reviewing Phase 1/2 data, Astellas declined its option; Taysha will advance a single-arm pivotal on its own [1].
How could Lexeo’s LX2006 reach the market faster?
FDA indicated openness to an accelerated approval BLA combining ongoing Phase 1/2 with pivotal data, contingent on added CMC and nonclinical work, and with an earlier LV mass index endpoint [2].
What makes Immusoft’s ISP-001 different from typical gene therapy?
It engineers a patient’s B cells to act as “living biofactories” secreting IDUA for MPS I, with Fast Track granted and early safety plus re-dosing experience reported [3].
Why did Intellia pause the MAGNITUDE trials?
A patient in MAGNITUDE had Grade 4 liver enzyme elevations with increased bilirubin, triggering a protocol-defined pause while the company consults regulators on next steps [5].
What is Lilly acquiring in the Adverum deal?
Ixo-vec, a Phase 3 intravitreal gene therapy for wAMD, plus a platform of single-administration ocular programs; consideration includes cash and a CVR tied to approval and sales [6].
Why is BioMarin divesting Roctavian now?
Persistent uptake challenges and modest sales led BioMarin to seek an out-license or sale while maintaining access in reimbursed markets during the process [8].
Entities / Keywords
Astellas; Taysha Gene Therapies; TSHA-102; Rett syndrome; Lexeo Therapeutics; LX2006; Friedreich ataxia cardiomyopathy; Immusoft; ISP-001; MPS I; Galapagos; cell therapy unit; Intellia Therapeutics; nex-z; MAGNITUDE; ATTR-CM; ATTR-PN; Eli Lilly; Adverum Biotechnologies; Ixo-vec; wAMD; BridgeBio Pharma; BBP-418; LGMD2I/R9; BioMarin; Roctavian.
References
- https://www.fiercebiotech.com/biotech/astellas-walks-away-gene-therapy-pact-taysha-pivotal-trial-launch
- https://finance.yahoo.com/news/lexeo-therapeutics-announces-fda-support-132708869.html
- https://www.morningstar.com/news/pr-newswire/20251021cl02855/immusoft-receives-fda-fast-track-designation-for-isp-001-a-first-in-class-engineered-b-cell-therapy-for-mps-i
- https://www.fiercebiotech.com/biotech/galapagos-wind-down-cell-therapy-unit-threatening-365-jobs-and-5-facilities
- https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-provides-update-magnitude-clinical-trials
- https://investor.lilly.com/news-releases/news-release-details/lilly-acquire-adverum-biotechnologies
- https://www.biospace.com/drug-development/bridgebio-pops-on-late-stage-limb-girdle-muscular-dystrophy-data
- https://www.fiercepharma.com/pharma/biomarin-resigned-ending-journey-hemophilia-gene-therapy-roctavian-seeking-divestiture-amid