Cell and Gene Therapy Weekly News – September 25th 2025

LucidQuest

1 month ago

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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

💉 Deramiocel (CAP-1002) Phase 3 HOPE-3 in DMD; BLA path after FDA CRL [US • 22 Sep 2025]

https://www.cgtlive.com/view/capricor-therapeutics-testing-dmd-cell-therapy-deramiocel-mettle-phase-hope-3-clinical-trial
Context: Randomized, double-blind, placebo-controlled Phase 3 (n=104); primary endpoint PUL 2.0 change at 12 months; multicenter US sites.
Key point: FDA issued a CRL (Jul 2025) to BLA based on HOPE-2/HOPE-2-OLE; Capricor plans to submit HOPE-3 data (topline expected Q4 2025) to address effectiveness.
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 Kinea Bio nabs funding + AAV capsid license for dysferlinopathy program [US • 23 Sep 2025]

https://www.businesswire.com/news/home/20250923556795/en/Kinea-Bio-Secures-%241.1M-Jain-Foundation-Investment-and-Solid-Biosciences-Capsid-License-to-Advance-Novel-Gene-Therapy-for-Dysferlinopathy-a-Rare-Neuromuscular-Disease
Context: Up to $1.1M milestone-based support from Jain Foundation; non-exclusive license to Solid’s myotropic AAV-SLB101 for dual-AAV KNA-155.
Key point: Funds IND-enabling dose-finding and GLP tox in dysferlin-deficient models to define window and biodistribution.
Implication: Partnerships/BD — Signals pipeline investment and modality expansion.

🧠 FDA declines higher-dose Spinraza filing [US • 23 Sep 2025]

https://pharma.economictimes.indiatimes.com/news/pharma-industry/fda-rejects-biogens-higher-dose-sma-treatment-amid-rising-competition/124085007
Context: Application sought a higher-dose regimen for SMA; higher dose approved in Japan; EU review ongoing.
Key point: FDA requested revisions to manufacturing section; Biogen plans resubmission (clinical endpoint not specified).
Implication: Regulatory/generics — Introduces competition that may affect pricing and formulary access.

🚀 Fast Track for MVX-220 in Angelman syndrome [US • 22 Sep 2025]

https://www.prnewswire.com/news-releases/mavrix-bio-receives-fda-fast-track-designation-for-mvx-220-for-treatment-of-angelman-syndrome-302562593.html
Context: IND cleared May 2025; Phase 1/2 ASCEND-AS first-in-human planned; developer GEMMABio cited as vector originator.
Key point: FDA granted Fast Track to AAV MVX-220 to facilitate development and review for a serious unmet need.
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 UC Davis advancing HSC gene therapy for Angelman [US • 18 Sep 2025]

https://health.ucdavis.edu/news/headlines/uc-davis-scientists-pioneer-stem-cell-gene-therapy-for-angelman-syndrome/2025/09
Context: CIRM $4.5M grant; autologous HSCs engineered to deliver functional UBE3A; prior mouse data reversed/prevented phenotype.
Key point: Current study focuses on safety and expression control (“Goldilocks” dosing) to enable human trials.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧠 Bayer advancing dual CGT strategy in Parkinson’s (cell + gene) [DE • 22 Sep 2025]

https://www.bayer.com/media/en-us/bayer-first-company-to-advance-cell-therapy-as-well-as-gene-therapy-against-parkinsons-disease/
Context: Phase 3 exPDite-2 starts for bemdaneprocel (BlueRock cell therapy); Phase 2 REGENERATE-PD randomizing for AB-1005 (AskBio) in EU; US ongoing.
Key point: First pivotal Phase 3 for an allogeneic pluripotent stem-cell therapy in PD; AB-1005 delivers GDNF via AAV2 with CED.
Implication: May influence prescriber choice and payer reviews pending full data.

🛡️ CHMP backs Uplizna for IgG4-RD—first EU therapy for the disease [EU • 23 Sep 2025]

https://www.medscape.com/viewarticle/europe-b-cell-therapy-moves-igg4-related-disease-2025a1000pdk?form=fpf
Context: Recommendation based on Phase 3 MITIGATE (n=135) with IV inebilizumab vs placebo plus steroid taper.
Key point: Marked flare reduction and higher steroid-free complete remission at 52 weeks; safety was consistent with prior indications.
Implication: May influence prescriber choice and payer reviews pending full data.

👁️ First ENCELTO retinal gene therapy in the US Northeast [US • 23 Sep 2025]

https://www.geneonline.com/ocli-vision-performs-first-encelto-gene-therapy-procedure-in-northeast-us/
Context: OCLI Vision (NY) performed procedure Sept 22; Spectrum Vision Partners network involvement.
Key point: ENCELTO targets inherited retinal disease with limited options; milestone for regional adoption.
Implication: Access programs — May expand screening, initiation, and follow-up at scale.

🧫 Earlier use of cilta-cel in myeloma; monitoring burdens easing [US • 23 Sep 2025]

https://www.targetedonc.com/view/treating-multiple-myeloma-with-earlier-car-t-cell-therapy
Context: Expert discussion cites CARTITUDE-4 outcomes and reduced REMS requirements (monitoring/drive restrictions).
Key point: Cilta-cel shows strong PFS vs SOC in 1–3L len-refractory setting; logistics trending toward outpatient feasibility.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🌏 eXmoor × Siam Bioscience to build CGT hub in Thailand [TH • 23 Sep 2025]

https://www.contractpharma.com/breaking-news/exmoor-siam-bioscience-partner-to-establish-cgt-services-in-southeast-asia/
Context: Joint CGT development + GMP manufacturing center near Bangkok; consultancy through commercial scale.
Key point: Platform aims to localize global CGT manufacturing, reduce logistics, and support regional access.
Implication: Partnerships/BD — Signals pipeline investment and modality expansion.

Why it matters

Parkinson’s field gets both restorative cell therapy (Phase 3) and neurotrophic gene therapy (Phase 2), diversifying CGT modalities in CNS.
Angelman programs span AAV Fast Track and HSC-based approaches, broadening shots on goal for a high-need population.
EMA’s positive opinion for IgG4-RD may set first-in-class EU standard and catalyze steroid-sparing care pathways.
Manufacturing capacity and regional hubs (Thailand) could de-bottleneck CGT access across SE Asia.
Regulatory dynamics (Spinraza supplement) underscore CMC rigor as rivals and new modalities intensify SMA competition.

📢 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 is deramiocel (CAP-1002) and where does HOPE-3 stand?

Deramiocel is an allogeneic cardiosphere-derived cell therapy for DMD cardiomyopathy. HOPE-3 is a randomized, double-blind Phase 3 (n=104) with PUL 2.0 at 12 months as primary endpoint; Capricor plans to use HOPE-3 data after a July 2025 FDA CRL to its BLA based on HOPE-2/OLE.

What did the CHMP say about Uplizna (inebilizumab) for IgG4-RD?

The CHMP recommended expanding Uplizna’s label to active IgG4-RD based on Phase 3 MITIGATE (n=135), showing fewer flares and more steroid-free remission vs placebo; safety was consistent with prior indications.

How is Bayer advancing CGT in Parkinson’s?

BlueRock’s bemdaneprocel (cell therapy) began the pivotal Phase 3 exPDite-2, while AskBio’s AB-1005 (AAV2-GDNF) is randomizing in REGENERATE-PD across EU sites, with US activity ongoing.

What’s the regulatory update on Spinraza’s higher dose?

FDA declined Biogen’s US application citing manufacturing-section revisions; Biogen plans to resubmit. Japan has approved the higher dose; EU review is ongoing per reporting (endpoints not specified).

What’s moving in Angelman syndrome gene therapy?

MavriX’s MVX-220 received Fast Track and is preparing a Ph1/2 trial; UC Davis is advancing an autologous HSC gene therapy with CIRM support toward first-in-human after safety work.

What is Kinea Bio developing for dysferlinopathy?

KNA-155 is a dual-AAV full-length DYSF program. New funding from Jain Foundation and a license to Solid’s AAV-SLB101 will support IND-enabling tox and biodistribution studies.

Entities / Keywords

Capricor Therapeutics; deramiocel; CAP-1002; HOPE-3; PUL 2.0 • Kinea Bio; KNA-155; dysferlinopathy; DYSF; AAV-SLB101 (Solid Biosciences) • Biogen; Spinraza; SMA • MavriX Bio; MVX-220; Angelman syndrome; GEMMABio • UC Davis; CIRM; UBE3A; hematopoietic stem cell gene therapy • Bayer; BlueRock; bemdaneprocel (BRT-DA01); AskBio; AB-1005; GDNF; Parkinson’s • Amgen; Uplizna; inebilizumab; IgG4-RD; CHMP; MITIGATE • OCLI Vision; ENCELTO; inherited retinal disease • Janssen/Legend (cilta-cel); CARTITUDE-4; REMS; multiple myeloma • eXmoor Pharma; Siam Bioscience; CGT manufacturing; Thailand.