Cell and Gene Therapy Weekly News – September 18th 2025

LucidQuest

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From regulatory shifts to global partnerships and technological breakthroughs, this week’s digest highlights the innovations redefining gene and cell therapy. Whether your focus is rare disease, CNS, or manufacturing efficiency, these updates bring you the latest advances and market drivers.

In Today’s Newsletter

🩹 FDA okays Vyjuvek (beremagene geperpavec-svdt) label expansion & at-home use [1] [US • 15 Sep 2025]

https://www.hcplive.com/view/fda-approves-beremagene-geperpavec-svdt-vyjuvek-label-update-in-deb

Context: Vyjuvek is a redosable topical COL7A1 gene therapy for DEB; prior GEM-1/2 and GEM-3 supported approval; update informed by RWD and OLE.
Key point: Label now includes DEB patients from birth and permits caregiver application and flexible dressing changes at home.
Implication: Access programs: May expand screening, initiation, and follow-up at scale.

🧠 Oligodendrocyte-targeted AAV for Canavan shows early benefit (Phase 1/2) [2] [16 Sep 2025]

https://www.nature.com/articles/s41591-025-03919-w
Context: MYR-101 (rAAV-Olig001-ASPA) intracranial dosing in 8 children; 12-mo interim: AEs common, SAEs resolved; none deemed treatment-related.
Key point: Reduced CSF NAA (P=0.0008), increased myelination (P=0.0137), and MSEL gains (P=0.0171) suggest target engagement and early functional effect.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🤖 “CRISPR-GPT” AI copilot aims to speed gene-editing workflows [3] [US • 16 Sep 2025]

https://med.stanford.edu/news/all-news/2025/09/ai-crispr-gene-therapy.html
Context: Stanford team reports an LLM that designs, refines, and QC’s CRISPR experiments; modes for beginners/experts; guardrails for misuse.
Key point: Tool draws on 11 years of community/published data to predict off-targets and troubleshoot, with lab anecdotes of first-try success.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

⚠️ Capsida pauses CAP-002 after pediatric death (STXBP1) [4] [12 Sep 2025]

https://www.clinicaltrialsarena.com/news/capsida-biotherapeutics-gene-therapy-patient-death/?cf-view
Context: Phase I/IIa IV AAV study initiated ~2 months prior; FDA notified; sponsor-initiated pause pending root-cause analysis.
Key point: First dosed patient died; company had aimed to detarget liver/DRG vs class toxicities; next steps under review.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.

👁️ Ocugen licenses OCU400 to Kwangdong for Korea [5] [South Korea • 15 Sep 2025]

https://www.ophthalmologytimes.com/view/ocugen-and-kwangdong-complete-licensing-agreement-for-ocu400-gene-therapy-in-korea
Context: Modifier gene therapy for RP; Phase 1/2 update showed LLVA gains; Phase 3 liMeliGhT underway targeting mid-2026 BLA.
Key point: Deal terms: up to $7.5M near-term; $1.5M per $15M sales milestones; 25% royalty; Ocugen to supply commercial product.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

🧪 FDA curbs ex-US processing of US patients’ cells [6] [US • 15 Sep 2025]

https://www.expresspharma.in/fdas-curb-on-overseas-cell-processing-to-hit-smaller-cell-and-gene-therapy-companies-globaldata
Context: Report cites linkage to executive orders; impacts trials shipping live cells abroad for editing then reinfusion.
Key point: Scope unspecified; smaller CGT firms and programs using overseas CAR-T/manufacturing steps may be most affected.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.

🗾 Japan opens JEAP to accelerate RM/CGT entry [7] [Japan • 16 Sep 2025]

https://www.openpr.com/news/4185051/japan-accelerates-regenerative-medicine-cell-therapy
Context: JEAP invites RM/CGT startups; offers mentorship, market insights, and connections to Japanese CDMOs/CROs and pharma.
Key point: Part of broader reforms to cut drug lag and modernize post-approval processes in Japan.
Implication: Access programs: May expand screening, initiation, and follow-up at scale.

🇰🇷 CHA Biotech targets 10× CGT cost reduction [8] [South Korea • 15 Sep 2025]

https://www.kedglobal.com/kiw-2025/newsView/ked202509160007
Context: KIW 2025 keynote: “K-cell sovereignty,” local supply chains, and IP strategy; cites 96-hospital network and GMP capacity.
Key point: Goal to slash CGT costs to one-tenth to broaden access; positions Korea firms for top-10 global status.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

🚺 Cook MyoSite + Veristat hit Phase III milestone in women’s health [9] [US • 16 Sep 2025]

https://www.biospace.com/press-releases/cook-myosite-and-veristat-partner-in-complex-phase-iii-adaptive-clinical-study-of-investigational-cell-therapy-for-unmet-need-in-womens-health
Context: DigniFI: two-stage, randomized, controlled Phase III of iltamiocel vs placebo in chronic fecal incontinence with OASI history.
Key point: >100 participants enrolled; 26 sites active; >95% adherence; milestone achieved.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🧬 Stem cells restore function post-stroke in mice (UZH) [10] [Switzerland • 16 Sep 2025]

https://www.news-medical.net/news/20250916/Stem-cell-therapy-restores-brain-function-after-stroke.aspx
Context: Human iPSC-derived neural stem cells transplanted 1 week after permanent stroke in mice; 5-week analyses.
Key point: Cells survived and became neurons, with vessel growth, reduced inflammation, tighter BBB, and motor recovery; delivery and safety-switch work ongoing.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

Why it matters

Label flexibility (Vyjuvek) shows regulators embracing RWD to widen at-home genetic medicine use.
Early CNS gene therapy signals (Canavan) and AI design tools (CRISPR-GPT) highlight a maturing toolchain for complex indications.
Safety events (Capsida) and policy shifts (FDA overseas processing) will shape CGT development geographies and CMC choices.
Regional momentum (Korea, Japan) is building new hubs for RM/CGT access, manufacturing, and cost innovation.

📢 Stay Ahead in Cell and Gene Therapy!
✅ Contact LucidQuest at 📧 info@lqventures.com for strategic guidance on gene therapy innovations and clinical researchbreakthroughs.

FAQ

What changed in Vyjuvek’s label and why does it matter?

FDA now allows use from birth and caregiver at-home application with flexible dressings, supported by real-world and OLE data—potentially improving adherence and access for DEB families [1].

How strong are the Canavan gene therapy signals?

In 8 children, intracranial MYR-101 showed statistically significant NAA reductions and imaging/functional improvements at 12 months with no treatment-related SAEs reported; durability and larger cohorts remain to be shown [2].

What exactly is “CRISPR-GPT”?

A Stanford-built LLM that designs and troubleshoots CRISPR experiments, predicts off-targets, and offers beginner/expert modes with embedded safety guardrails—intended to shorten experiment cycles [3].

What is known about the Capsida trial death?

The first dosed pediatric patient in the CAP-002 STXBP1 study died; the company paused the trial to investigate causality, has notified FDA, and will determine next steps after review [4].

How will the FDA’s overseas-processing curb affect sponsors?

Trials that ship US patients’ cells abroad for processing/editing and reinfusion may face holds; smaller CGT firms relying on ex-US capacity could be disproportionately impacted (scope not quantified) [6].

What are the key terms of Ocugen’s Korea deal?

Kwangdong pays up to $7.5M near-term plus $1.5M per $15M sales milestones and a 25% royalty; Ocugen supplies commercial product, and Korea filings will leverage global data packages [5].

Entities / Keywords

Krystal Biotech (Vyjuvek; beremagene geperpavec-svdt); Dystrophic epidermolysis bullosa; rAAV-Olig001-ASPA (MYR-101); Canavan disease; Stanford “CRISPR-GPT”; Capsida Biotherapeutics (CAP-002; STXBP1); Ocugen (OCU400; modifier gene therapy); Kwangdong Pharmaceutical; FDA overseas cell processing; JEAP (Japan); CHA Biotech; Cook MyoSite (iltamiocel); Veristat; University of Zurich (iPSC neural stem cells).

References