Post by Dr. Timos Papagatsias

Name: Trending in Gene and Cell Therapy: NAVSUNLI BLA Path, AMT-130 BLA and more This biweekly Gene and C
Uploaded: 2026-06-26T12:56:38.959Z
Channel: Dr. Timos Papagatsias
Description: Trending in Gene and Cell Therapy: NAVSUNLI BLA Path, AMT-130 BLA and more This biweekly Gene and Cell Therapy update highlights regulatory and clinical milestones across rare diseases, oncology, and

Scaling Strategy, Insights & Wargames with AI | Founder, LucidQuest & NexusBrain | Advisor to BioPharma, HealthTech & Investors

Trending in Gene and Cell Therapy: NAVSUNLI BLA Path, AMT-130 BLA and more This biweekly Gene and Cell Therapy update highlights regulatory and clinical milestones across rare diseases, oncology, and neurodegenerative disorders. Key developments include FDA alignment on REGENXBIO’s NAVSUNLI pathway, uniQure’s planned AMT-130 BLA submission, Lexeo’s pivotal trial design for LX2006, and new inherited retinal disease programs from Opus Genetics. Additional updates cover in vivo CAR-T data from Legend Biotech, a first treated Cockayne syndrome patient, China approval of satri-cel, and expanded FDA approval for TECELRA in synovial sarcoma.

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