This weekly Cell & Gene Therapy update spans regulatory actions, durable long-term outcomes, early clinical starts, IP moves, manufacturing scale-outs, and preclinical advances—shaping the CGT landscape.
In Today’s Newsletter
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🧬 CHMP positive for Waskyra in WAS [1] [EU • 14 Nov 2025]
Key point: CHMP recommended EU marketing authorisation for the ex vivo gene therapy Waskyra.
Context: Developed at SR-Tiget; to be offered at IRCCS Ospedale San Raffaele; US BLA under FDA review.
Implication: Could affect pricing and formulary access.
🧠 RNA approach halts ALS degeneration in models [2] [Israel • 17 Nov 2025]
Key point: microRNA therapy restored neuronal health and halted degeneration in ALS models.
Context: Nature Neuroscience publication; Israeli–European collaboration.
Implication: Signals modality expansion.
⚠️ FDA restricts Elevidys label after deaths [3] [US • 14 Nov 2025]
Key point: Indication limited to ambulatory DMD ≥4; boxed warning for serious liver injury.
Context: Follows reported deaths and acute liver injury; added monitoring and post-marketing requirements.
Implication: May shift payer and site-of-care decisions.
👁️ Coave unveils CoTx-10 for retinal vascular disease [4] [18 Nov 2025]
https://www.ophthalmologytimes.com/view/coave-therapeutics-reveals-lead-gene-therapy-program-cotx-10
Key point: coAAV-SCS vector aims for sustained dual-biologic expression via suprachoroidal delivery.
Context: NHP studies through 2026; IND readiness targeted 2027.
Implication: Pipeline investment signal.
🛡️ ADA-SCID lentiviral therapy shows durable outcomes [5] [16 Nov 2025]
Key point: 100% OS with robust immune/metabolic correction; no vector-related leukoproliferative events.
Context: 62-patient multicenter cohort, median 7.5 years follow-up.
Implication: Supports real-world durability narratives.
📜 USPTO grants REQORSA+PD-L1 combo patent [6] [US • 18 Nov 2025]
Key point: Patent through 2037; ties to Acclaim-3 SCLC with atezolizumab.
Context: Related filings across regions.
Implication: Ecosystem and IP positioning.
👓 Opus doses first in OPGx-BEST1 Phase 1/2 [7] [US • 13 Nov 2025]
Key point: One-time subretinal BEST1 gene therapy; initial data expected Q1 2026.
Context: With Retina Foundation of the Southwest and Retina Consultants of Texas.
Implication: Modality expansion.
🤝 Solid licenses AAV-SLB101 to Andelyn CDMO [8] [US • 17 Nov 2025]
https://finance.yahoo.com/news/solid-biosciences-announces-licensing-agreement-130000832.html
Key point: Andelyn to offer SLB101 capsid via AAV Curator platform.
Context: SLB101 used in SGT-003; 23 pediatric participants dosed in Phase 1/2.
Implication: Manufacturing and platform scale-out.
✅ FDA approves Arrowhead’s Redempio (plozasiran) for FCS [9] [US • 18 Nov 2025]
Key point: siRNA reduces triglycerides in FCS; first FDA approval for Arrowhead.
Context: Phase 3 PALISADE met primary and key secondary endpoints.
Implication: Pricing and access dynamics to watch.
🏷️ WHO assigns INN to Anixa FSHR CAR-T [10] [17 Nov 2025]
Key point: INN “liraltagene autoleucel” approved; shorthand “lira-cel.”
Context: Ongoing Phase 1 with Moffitt Cancer Center.
Implication: Readies for global recognition and potential commercialization.
🧒 ARTEMIS CAR T shows preclinical edge in solid tumors [11] [US • 13 Nov 2025]
https://finance.yahoo.com/news/eureka-artemis-car-t-cells-130000744.html
Key point: Outperformed conventional CAR T in neuroblastoma models.
Context: NCI-led study in Cell Reports Medicine; ongoing trials in liver cancer and B-cell lymphoma.
Implication: Modality innovation in solid tumors.
🦶 ENCell wins Korea Phase 2a OK in CMT1A [12] [South Korea • 17 Nov 2025]
https://biz.chosun.com/en/en-science/2025/11/17/FC2IA63O6NGQTIE6YWPLAQDSW4/
Key point: MFDS cleared EN001 (umbilical cord–derived MSC) Phase 2a; eyeing accelerated routes.
Context: Prior Phase 1 safety/signals; regenerative medicine sandbox considered.
Implication: Regional pathway exploration.
🧪 T-knife files CTA for TK-6302 PRAME TCR-T [13] [17 Nov 2025]
Key point: CTA filed for 2026 start of “supercharged” PRAME TCR-T (ATLAS).
Context: Non-viral editing; enhanced fitness and persistence in preclinical data.
Implication: Early clinical pipeline build-out.
Why it matters
- Regulators advanced both gene and RNA modalities (WAS, FCS) [1][9].
- Safety governance tightened for AAV (Elevidys) [3].
- Ophthalmology remains a hotbed for in-office/subretinal delivery [4][7].
- Platform/IP moves (capsids, patents, INNs) prepare assets for scale [6][8][10].
- Innovative cell architectures target solid tumors; rare neuropathies progress regionally [11][12][13].
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FAQ
What did EMA say about Waskyra?
CHMP issued a positive opinion recommending EU marketing authorisation; US BLA remains under FDA review [1].
How did the Elevidys label change?
Limited to ambulatory DMD ≥4 years; non-ambulatory removed; boxed warning for liver injury; added monitoring and post-marketing study [3].
What is Arrowhead’s new approval?
Redempio (plozasiran), an siRNA for FCS, based on PALISADE Phase 3 success; first FDA-approved Arrowhead therapy [9].
Which ophthalmic gene therapy moves occurred?
Opus dosed first in OPGx-BEST1 with data planned Q1 2026 [7]; Coave unveiled CoTx-10 aiming for 2027 IND after NHP work [4].
What IP/naming milestones landed?
Genprex patent for REQORSA+PD-L1 combos through 2037 [6]; WHO approved INN “liraltagene autoleucel” for an FSHR CAR-T [10].
Any notable cell-therapy advances?
Preclinical superiority for ARTEMIS CAR T in neuroblastoma [11]; CTA filed for PRAME TCR-T [13]; ENCell’s EN001 cleared for CMT1A Phase 2a in Korea [12].
Entities / Keywords
Waskyra; Wiskott–Aldrich; SR-Tiget; EMA CHMP
Elevidys; DMD; AAV; boxed warning
CoTx-10; coAAV-SCS; wAMD; DME
ADA-SCID; lentiviral gene therapy; long-term follow-up
REQORSA; PD-L1; Tecentriq; Acclaim-3
OPGx-BEST1; Best disease; subretinal
AAV-SLB101; Andelyn; capsid licensing
Redempio; plozasiran; FCS; siRNA
liraltagene autoleucel; INN; CAR-T
ARTEMIS CAR T; neuroblastoma; NCI
EN001; CMT1A; MFDS Korea
TK-6302; PRAME; TCR-T
References
- https://www.fondazionetelethon.it/en/stories-and-news/news/from-telethon-foundation/fondazione-telethon-announces-chmp-positive-opinion-for-waskyra-a-gene-therapy-for-the-treatment-of-wiskott-aldrich-syndrome/
- https://www.timesofisrael.com/in-world-first-israeli-scientists-use-rna-based-gene-therapy-to-stop-als-deterioration/
- https://www.fiercepharma.com/pharma/fda-restricts-sarepta-elevidys-gene-therapy-use-after-duchenne-patient-deaths
- https://www.ophthalmologytimes.com/view/coave-therapeutics-reveals-lead-gene-therapy-program-cotx-10
- https://www.contemporarypediatrics.com/view/long-term-outcomes-of-lentiviral-gene-therapy-for-ada-deficient-severe-combined-immunodeficiency
- https://www.prnewswire.com/news-releases/genprex-announces-us-patent-for-reqorsa-gene-therapy-in-combination-with-pd-l1-antibodies-to-treat-cancers-302617555.html
- https://www.globenewswire.com/news-release/2025/11/13/3187171/0/en/Opus-Genetics-Announces-Dosing-of-First-Participant-in-OPGx-BEST1-Phase-1-2-Gene-Therapy-Clinical-Trial-for-Best-Disease.html
- https://finance.yahoo.com/news/solid-biosciences-announces-licensing-agreement-130000832.html
- https://www.biospace.com/fda/arrowhead-secures-maiden-approval-first-fda-nod-for-rare-genetic-metabolic-disorder
- https://www.prnewswire.com/news-releases/anixa-biosciences-announces-world-health-organization-who-approval-of-international-non-proprietary-name-for-its-car-t-therapy-marking-a-key-milestone-toward-global-recognition-and-future-commercialization-302616557.html
- https://finance.yahoo.com/news/eureka-artemis-car-t-cells-130000744.html
- https://biz.chosun.com/en/en-science/2025/11/17/FC2IA63O6NGQTIE6YWPLAQDSW4/
- https://www.globenewswire.com/news-release/2025/11/17/3189058/0/en/T-knife-Therapeutics-Files-Clinical-Trial-Application-for-Phase-1-ATLAS-Trial-for-TK-6302-a-Supercharged-PRAME-targeted-T-Cell-Receptor-Therapy.html