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
🧬 Astellas exits Taysha option on TSHA-102; Taysha moves to pivotal [1] [17 Oct 2025]
Context: Option window followed Taysha’s mid-2025 TSHA-102 data package and FDA Breakthrough designation.
Key point: Astellas declined to license TSHA-102; Taysha will run a single-arm pivotal with FDA-aligned protocol and SAP.
Implication: May influence prescriber choice and payer reviews pending full data.
👁️ AAVantgarde’s AAVB-039 wins FDA ODD and UK CTA clearance [2] [21 Oct 2025]
Context: Dual-AAV intein approach to deliver ABCA4; CELESTE phase 1/2 underway, STELLA natural history supports design.
Key point: FDA Orphan Drug designation and MHRA CTA clearance add to prior IND and Fast Track.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🤖 MeiraGTx touts AI-optimized promoters at ASHG 2025 [3] [17 Oct 2025]
https://tradersunion.com/news/market-voices/show/690350-gene-therapy-innovation/
Context: Presentation highlights AI-driven promoter design to enhance AAV potency; prior RMAT in Parkinson’s noted.
Key point: Claims up to 6× promoter activity and up to 20% higher protein expression (as presented; not peer-reviewed here).
Implication: Signals pipeline investment and modality expansion.
🧡 Lexeo gets FDA support for accelerated path for LX2006 in FA cardiomyopathy [4] [17 Oct 2025]
https://finance.yahoo.com/news/lexeo-therapeutics-announces-fda-support-132708869.html
Context: Shift to Sf9-baculovirus manufacturing requires comparability; co-primary LV mass index at earlier time point.
Key point: FDA open to accelerated approval BLA pooling phase 1/2 with pivotal data, plus additional nonclinical item before pivotal start.
Implication: May influence prescriber choice and payer reviews pending full data.
❤️ AskBio’s AB-1002 CHF phase 1, 12-month data published in Nature Medicine [5] [21 Oct 2025]
Context: Non-randomized, sequential dose-escalation in NYHA III non-ischemic HFrEF; single intracoronary dose.
Key point: No AEs deemed related to AB-1002 and clinically meaningful improvements reported at 12 months.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 Immusoft’s engineered B-cell therapy ISP-001 gets FDA Fast Track in MPS I [6] [21 Oct 2025]
Context: Autologous B cells engineered to secrete IDUA; early phase 1/2 data include first reported safe re-dosing (n not stated).
Key point: FDA Fast Track granted; functional gains reported in one patient alongside biomarker changes.
Implication: Signals pipeline investment and modality expansion.
🧯 Galapagos to wind down cell therapy unit after failed sale process [7] [21 Oct 2025]
Context: Strategic review since May; limited bids, evolving market dynamics and capital needs cited.
Key point: Plan to close five facilities and affect ~365 roles, subject to worker consultations; open to last-minute proposals.
Implication: Introduces competition that may affect pricing and formulary access.
🧲 ME Therapeutics licenses NRC nanobody-based CD22 binder for next-gen in vivo CAR [8] [16 Oct 2025]
Context: CD22 target relevant in post-CD19 relapse; binder in investigator phase 1 (NCT06208735).
Key point: Exclusive commercial rights across major regions for CAR-T and CAR-M, with sub-licensing and milestone/royalty terms.
Implication: Signals pipeline investment and modality expansion.
Why it matters
- Rett, Stargardt and FA cardiomyopathy advance via designations and regulatory feedback.
- Engineered B cells and in vivo CAR approaches diversify beyond AAV vectors.
- AI-assisted regulatory element design could raise vector efficiency across pipelines.
- Peer-reviewed CHF gene therapy data support cardiovascular use cases for AAV.
- Market consolidation continues as Galapagos exits cell therapy operations.
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FAQ
Who controls TSHA-102 for Rett syndrome now and what is next?
Astellas declined its option. Taysha will proceed with a single-arm pivotal trial under an FDA-aligned protocol and SAP ([1]
What regulatory steps did LX2006 receive from FDA?
FDA indicated openness to an accelerated approval BLA that pools phase 1/2 with pivotal data, contingent on manufacturing comparability and an added nonclinical item; LV mass index can be assessed earlier than 12 months [4]
What do the AB-1002 data show?
In a phase 1 dose-escalation study, no events were deemed related to AB-1002 and clinically meaningful improvements were observed at 12 months; a randomized phase 2 is enrolling to confirm findings [5]
What makes ISP-001 different from typical gene therapy?
It uses engineered autologous B cells as “living biofactories” to secrete IDUA for MPS I, with FDA Fast Track designation and early re-dosing experience reported (n not stated) ([6]
How is AAVB-039 addressing ABCA4’s size limit?
It uses a dual-AAV intein strategy to deliver the large ABCA4 gene; the program now holds FDA Orphan, FDA Fast Track, and UK CTA with CELESTE phase 1/2 active [2]
What is happening at Galapagos’ cell therapy unit?
After limited bids and a review of market dynamics, Galapagos plans to wind down the unit, impacting ~365 roles and five facilities, pending consultations [7]
Entities / Keywords
Taysha Gene Therapies; TSHA-102; Astellas; Rett syndrome; AAVantgarde Bio; AAVB-039; ABCA4; Stargardt disease; MeiraGTx; AI promoter design; AskBio; AB-1002; congestive heart failure; HFrEF; Lexeo Therapeutics; LX2006; Friedreich ataxia cardiomyopathy; Immusoft; ISP-001; engineered B cells; MPS I; Galapagos; cell therapy wind-down; ME Therapeutics; National Research Council of Canada; nanobody; CD22; in vivo CAR-T; CAR-M.
References
- https://www.fiercebiotech.com/biotech/astellas-walks-away-gene-therapy-pact-taysha-pivotal-trial-launch
- https://www.cgtlive.com/view/aavantgarde-stargardt-gene-therapy-aavb-039-garners-fda-orphan-drug-designation-cleared-trial-uk
- https://tradersunion.com/news/market-voices/show/690350-gene-therapy-innovation/
- https://finance.yahoo.com/news/lexeo-therapeutics-announces-fda-support-132708869.html
- https://www.globenewswire.com/news-release/2025/10/21/3170137/0/en/AskBio-Announces-Publication-in-Nature-Medicine-of-12-month-data-from-Phase-1-Trial-of-AB-1002-Gene-Therapy-in-Participants-with-Congestive-Heart-Failure.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://www.businesswire.com/news/home/20251016721353/en/ME-Therapeutics-Granted-Licence-for-CD22-Nanobody-Asset-to-Expand-Next-Gen-In-Vivo-CAR-Cell-Therapy-Program