This week’s Cell & Gene Therapy update highlights major gene-editing partnerships, regulatory designations, clinical progress in neurological and ophthalmic disorders, CAR-T approvals, and next-gen manufacturing advances shaping the rare-disease and cell/gene therapy landscape.
In Today’s Newsletter
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🤝 Regeneron backs Tessera’s gene writing in AATD [1] [US • 01 Dec 2025]
Context: Collaboration on TSRA-196, an in vivo gene-writing candidate for AATD, preclinical data in mice and NHPs.
Key point: Regeneron to pay $150M upfront and equity, split global costs and profits, with up to $125M milestones, Tessera to lead first-in-human.
Implication: Signals pipeline investment and modality expansion.
🧠 Imvax’s GBM cell therapy pivots to OS after PFS miss [2] [US • 02 Dec 2025]
Context: Phase 2b in newly diagnosed GBM, 99 patients, personalized chamber implant plus SOC.
Key point: Primary PFS endpoint missed, reported longer median OS versus placebo (statistical details not stated), planning FDA pathway meeting.
Implication: May influence prescriber choice and payer reviews pending full data.
🧬 Solid Biosciences SGT-212 wins FDA RPD, Fast Track in FA [3] [US • 01 Dec 2025]
Context: SGT-212 uses dual-route delivery, intradentate nucleus and IV, to restore frataxin; FALCON Phase 1b screening underway.
Key point: FDA granted Rare Pediatric Disease and Fast Track; potential PRV upon approval per program rules.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 PBS Biotech debuts MiniPRO process dev platform [4] [US • 02 Dec 2025]
Context: MiniPRO supports up to 24 independent cultures with closed-system control, based on Vertical-Wheel technology.
Key point: Aims to mirror GMP bioreactor performance at small scale to speed optimization and scale-up.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧫 China approves Juventas CD19 CAR-T for r/r LBCL [5] [China • 28 Nov 2025]
Context: Ina-cel (YORWIDA) gains NMPA approval in r/r LBCL after 2+ prior lines; second indication after adult r/r B-ALL.
Key point: Single-arm Phase II supported approval; source highlights safety and durability in study and real-world settings.
Implication: Introduces competition that may affect pricing and formulary access.
🩸 KBR: Hemophilia care trends, durability, and new modalities [6] [South Korea • 28 Nov 2025]
https://www.koreabiomed.com/news/articleView.html?idxno=29797
Context: Expert talk highlights evolving prophylaxis targets, subcutaneous agents, rebalancing therapies, and gene therapy considerations.
Key point: Commentary cites earlier cohorts with gene therapy durability up to 13 years (products not specified), and movement toward oral approaches in preclinical stages.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧒 Latus Bio IND cleared for CLN2 gene therapy LTS-101 [7] [US • 02 Dec 2025]
Context: One-time AAV gene therapy aiming for CNS expression of TPP1 in CLN2 disease.
Key point: FDA cleared IND and granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations.
Implication: Signals pipeline investment and modality expansion.
👁️ GenSight’s GS010/LUMEVOQ REVISE study approved in France [8] [France • 02 Dec 2025]
Context: ANSM and Ethics Committee authorized open-label Phase II dose-ranging in ND4-LHON, n=14 target.
Key point: Primary endpoint is BCVA change at 1.5 years; supports review of French named patient early access program.
Implication: May influence prescriber choice and payer reviews pending full data.
⚡ Encoded to share interim ETX101 Dravet data at AES [9] [US • 02 Dec 2025]
Context: POLARIS Phase 1/2 open-label in SCN1A+ Dravet, assessing seizures and neurodevelopmental scales through 7 months.
Key point: Interim safety and early efficacy to be presented in oral and poster sessions at AES 2025.
Implication: May influence prescriber choice and payer reviews pending full data.
Why it matters
- Partnerships and designations de-risk early programs and can pull forward timelines.
- Manufacturing tools that mirror GMP at bench scale can compress CMC cycles.
- China’s second CD19 CAR-T indication broadens market dynamics across leukemia–lymphoma.
- Regulatory green lights for exploratory studies keep pipeline optionality alive while access routes are evaluated.
- Emerging data readouts at major meetings set expectations for 2026 pivotal plans.
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FAQ
What is TSRA-196 and who is leading development? [1]
TSRA-196 is Tessera’s in vivo gene-writing candidate for AATD. Tessera will lead the first-in-human study, with Regeneron leading later global development and commercialization. The deal includes $150M upfront and equity, plus milestones.
Did Imvax’s GBM program meet its endpoints? [2]
The Phase 2b missed its primary PFS endpoint. Imvax reported longer median OS versus placebo, with no statistical details stated, and plans to discuss a regulatory path with FDA.
What designations did SGT-212 receive, and what is the route? [3]
SGT-212 for Friedreich’s ataxia received FDA Rare Pediatric Disease and Fast Track designations. It uses dual-route administration, intradentate nucleus and IV, with Phase 1b screening underway.
Which CAR-T just gained a second indication in China? [5]
Inaticabtagene autoleucel (Ina-cel, YORWIDA) by Juventas was approved by NMPA for r/r LBCL after 2+ lines, adding to its adult r/r B-ALL approval.
What is LTS-101 targeting and what FDA statuses apply? [7]
LTS-101 targets CLN2 disease, aiming for CNS TPP1 expression with one-time AAV dosing. FDA cleared the IND and granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations.
What will Encoded’s ETX101 AES presentations cover? [9]
Interim Phase 1/2 data in SCN1A+ Dravet children, including safety, seizure frequency to 7 months, and neurodevelopmental assessments, will be presented at AES 2025.
Entities / Keywords
Regeneron; Tessera Therapeutics; TSRA-196; AATD; Imvax; IGV-001; glioblastoma; Solid Biosciences; SGT-212; Friedreich’s ataxia; PBS Biotech; MiniPRO; Vertical-Wheel; Juventas; Inaticabtagene autoleucel; Ina-cel; YORWIDA; LBCL; B-ALL; Korea Biomedical Review; hemophilia; rebalancing agents; Latus Bio; LTS-101; CLN2; TPP1; GenSight Biologics; GS010; LUMEVOQ; ND4-LHON; Encoded Therapeutics; ETX101; Dravet syndrome; AES.
References
- https://www.fiercebiotech.com/biotech/regeneron-makes-150m-bet-tesseras-rare-disease-gene-writing-prospect
- https://www.fiercebiotech.com/biotech/imvax-takes-brain-cancer-cell-therapy-fda-betting-survival-hit-will-overshadow-primary-miss
- https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-receives-fda-rare-pediatric-disease
- https://www.prnewswire.com/news-releases/pbs-biotech-unveils-a-breakthrough-product-for-cell-therapy-process-development-at-isscr-boston-302630109.html
- https://www.manilatimes.net/2025/11/28/tmt-newswire/pr-newswire/inaticabtagene-autoleucel-yorwidatm-new-drug-application-for-lymphoma-approved-chinas-innovative-car-t-enters-a-new-era-of-dual-indications/2233239
- https://www.koreabiomed.com/news/articleView.html?idxno=29797
- https://www.businesswire.com/news/home/20251202268130/en/Latus-Bio-Announces-IND-Clearance-of-LTS-101-for-CLN2-Disease-and-Receipt-of-Fast-Track-Orphan-Drug-and-Rare-Pediatric-Disease-Designations
- https://www.gensight-biologics.com/2025/12/02/gensight-biologics-announces-regulatory-approval-for-gs010-lumevoq-revise-dose-ranging-study-in-france/
- https://www.biospace.com/press-releases/encoded-therapeutics-to-present-interim-phase-1-2-clinical-data-on-etx101-the-first-one-time-gene-therapy-in-development-for-dravet-syndrome-at-the-2025-american-epilepsy-society-annual-meeting