This week’s Cell and Gene therapy update highlights regulatory momentum, dealmaking and manufacturing scale-up alongside mixed commercial traction and early clinical signals across multiple indications.
In Today’s Newsletter
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🏭 Johnson & Johnson expands US cell-therapy manufacturing in Pennsylvania [US • 18 Feb 2026]
Content: Next-generation cell therapy manufacturing facility planned for Montgomery County, Pennsylvania, job creation claims included.
Key Point: Johnson & Johnson announced a more than $1B investment to expand US cell-therapy manufacturing capacity.
Implication: Signals pipeline investment and modality expansion.
🧭 FDA draft guidance outlines “plausible mechanism” pathway for bespoke therapies [US • 23 Feb 2026
https://www.fiercebiotech.com/biotech/fda-illuminates-new-approval-pathway-bespoke-gene-therapies
Content: Draft focuses on genome editing and RNA-based approaches for rare diseases, with post-market data collection emphasized.
Key Point: FDA described a framework that could allow very small patient datasets, plus strong mechanistic rationale and natural history data, to support approvals (criteria-based, case-by-case).
Implication: May influence prescriber choice and payer reviews pending full data.
🧬 Genprex strengthens REQORSA combo IP in Japan and Europe [EU • 23 Feb 2026]
Content: Patents relate to combining REQORSA (Genprex) with PD-1 or PD-L1 antibodies, Acclaim-3 trial referenced (details not provided here).
Key Point: Genprex said Japan and EU patent decisions strengthen protection for REQORSA plus checkpoint inhibitor combinations.
Implication: Signals pipeline investment and modality expansion.
⚖️ REGENXBIO wins appeal in AAV patent dispute involving Sarepta [US • 23 Feb 2026]
https://www.biospace.com/policy/regenxbio-secures-legal-win-in-gene-therapy-patent-battle-vs-sarepta
Content: Appeals court reversed a January 2024 lower court decision that had found REGENXBIO’s patent invalid (University of Pennsylvania owns, licenses to REGENXBIO).
Key Point: Appeals court sided with REGENXBIO, sending the case back for reconsideration and rejecting a narrow “product of nature” view.
Implication: Introduces competition that may affect pricing and formulary access.
🩸 Casgevy and Lyfgenia adoption remains constrained two years post-approval [US • 23 Feb 2026]
Content: Article highlights conditioning burden (busulfan), time intensity, fertility concerns, provider familiarity, and operational complexity.
Key Point: BioSpace reports limited treated patient counts to date and continued access barriers for Vertex, CRISPR Therapeutics (Casgevy) and Genetix Biotherapeutics, formerly bluebird (Lyfgenia).
Implication: Could inform practice and payer discussions, interpretation depends on study design and confounding control.
🗂️ Ultragenyx DTX401 gene therapy gets Priority Review, PDUFA 23 Aug 2026 [US • 23 Feb 2026]
Content: Company cites 52 treated patients and up to six years follow-up across its program, Phase 3 GlucoGene trial mentioned.
Key Point: Ultragenyx said FDA accepted the DTX401 (pariglasgene brecaparvovec) BLA for GSDIa and granted Priority Review, with a 23 Aug 2026 action date.
Implication: May influence prescriber choice and payer reviews pending full data.
🧠 iRegene hits US and China clinical milestones for iPSC-derived cell therapies [US • 23 Feb 2026]
Content: Sponsored article describes iPSC-derived, “off-the-shelf” programs and cross-border development.
Key Point: iRegene reported first US patient dosed in an open-label Phase IIa study of NouvNeu001 (Parkinson’s) and first patient enrolled in China in a randomized study of NouvNeu004 (MSA).
Implication: Signals pipeline investment and modality expansion.
🤝 Gilead to acquire Arcellx, seeks full control of anito-cel [US • 23 Feb 2026]
Content: Builds on Kite–Arcellx collaboration (2022); anito-cel is a BCMA-directed CAR T, BLA already accepted per release.
Key Point: Gilead announced a definitive agreement to buy Arcellx for $115 per share plus a CVR, aiming to accelerate anito-cel development and commercialization.
Implication: Signals pipeline investment and modality expansion.
🧯 BioMarin withdraws Roctavian after buyer search fails [US • 24 Feb 2026]
Content: Article cites prior scale-backs and a divestiture plan, plus Q4 2025 charges including inventory write-off.
Key Point: BioMarin pulled Roctavian (hemophilia A gene therapy) from the market after unsuccessful divestiture efforts, recording significant charges (components described in the source).
Implication: Introduces competition that may affect pricing and formulary access.
🇯🇵 Sarepta and Chugai launch ELEVIDYS in Japan [Japan • 24 Feb 2026]
Content: Launch follows Japan reimbursement listing; conditional and time-limited approval noted; Chugai handles postmarketing obligations per release.
Key Point: Sarepta said ELEVIDYS launched commercially in Japan via Chugai for eligible ambulatory children (criteria specified in the release).
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 Iovance reports early lifileucel activity in UPS and DDLPS sarcoma pilot
Content: Pilot trial led by Memorial Sloan Kettering Cancer Center; lifileucel monotherapy; first six evaluable patients.
Key Point: Iovance reported a 50% confirmed ORR (RECIST v1.1) in the first six evaluable patients with advanced UPS or DDLPS, and said it plans a single-arm registrational trial starting Q2 2026.
Implication: May influence prescriber choice and payer reviews pending full data.
Why It Matters
- FDA’s bespoke-therapy draft guidance could reshape evidence expectations for ultra-rare genome editing and RNA-based programs, especially where natural history datasets are central.
- Manufacturing investments (JNJ, Ultragenyx) highlight continued US-based capacity build-out as advanced-therapy pipelines mature.
- M&A (Gilead–Arcellx) reinforces strategic value of late-stage CAR T assets with accepted BLAs.
- Commercial reality check — Roctavian withdrawal and slow SCD gene-therapy uptake — keeps the spotlight on access, conditioning burden, and delivery infrastructure.
- Early solid-tumor cell therapy signals (Iovance in sarcoma) add momentum for TIL approaches beyond melanoma.
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FAQ
What is the FDA’s “plausible mechanism” framework, and what modalities are covered in the draft?
FDA describes a pathway focused on genome editing and RNA-based approaches for rare diseases, using criteria tied to mechanism and supporting data, with post-market follow-up.
What is Ultragenyx seeking approval for with DTX401, and what is the PDUFA date?
Ultragenyx said FDA accepted the DTX401 (pariglasgene brecaparvovec) BLA for GSDIa with Priority Review, action date 23 Aug 2026.
What is Gilead buying from Arcellx, and why does anito-cel matter to the deal?
Gilead announced an acquisition of Arcellx to obtain full control of anito-cel, a BCMA-directed CAR T, and to remove profit-share, milestones, and royalties under the prior collaboration.
Why did BioMarin withdraw Roctavian from the market?
Fierce Pharma reports BioMarin ended commercialization after failing to find a qualified buyer for Roctavian, citing charges tied to the decision (details in the source).
What did Sarepta announce about ELEVIDYS in Japan, and who is commercializing it?
Sarepta said ELEVIDYS launched commercially in Japan via Chugai after reimbursement listing, under Japan’s conditional and time-limited approval framework referenced in the release.
What is Iovance’s lifileucel signal in soft-tissue sarcomas?
Iovance reported a 50% confirmed ORR in the first six evaluable patients in a pilot trial in UPS and DDLPS, and plans a registrational study in 2026 (per release).
Entities / Keywords
Johnson & Johnson (JNJ), cell therapy manufacturing, Pennsylvania, Montgomery County FDA, CBER, “plausible mechanism” framework, bespoke therapies, CRISPR, antisense oligonucleotides Genprex, REQORSA, PD-1, PD-L1, Tecentriq (Genentech) REGENXBIO, Sarepta, AAV vectors, University of Pennsylvania, patent litigation, ELEVIDYS Vertex, CRISPR Therapeutics, Casgevy, Genetix Biotherapeutics (bluebird bio legacy), Lyfgenia, sickle cell disease Ultragenyx, DTX401, pariglasgene brecaparvovec, glycogen storage disease type Ia (GSDIa), BLA, Priority Review, PDUFA iRegene, NouvNeu001 (Parkinson’s), NouvNeu004 (MSA), iPSC-derived cell therapy Gilead, Kite, Arcellx, anito-cel (anitocabtagene autoleucel), BCMA CAR T, acquisition, CVR BioMarin, Roctavian, hemophilia A, withdrawal Sarepta, Chugai, Roche Group, Japan NHI, MHLW Iovance, lifileucel, TIL therapy, MSKCC, undifferentiated pleomorphic sarcoma (UPS), dedifferentiated liposarcoma (DDLPS)
