🧬 This Week in Gene & Cell Therapy: Diabetes Deals, Vision Breakthroughs, FDA Pushback and More!
From immune tolerance strategies for type 1 diabetes to FDA rejections over manufacturing flaws, this week’s episode explores the major moves, milestones, and regulatory shakeups shaping the future of gene and cell therapy. Whether you’re tracking progress in retinitis pigmentosa, hemophilia B, or metastatic cancer, this is your biotech briefing—decoded. 🔬🚀
🔍 Highlights in this episode:
🩸 Amarna & NorthX join forces to advance type 1 diabetes gene therapy Nimvec AM510—human trials set for 2027
🧪 Bio-Techne’s Simple Western platform powers ZEVASKYN’s FDA approval for RDEB
🇷🇺 Biocad aims to launch Russia’s first hemophilia B gene therapy by 2026
👁️ Atsena Therapeutics accelerates XLRS gene therapy pathway with continuous Phase 1/2/3 trial
🌟 Nanoscope begins FDA submission for gene-agnostic RP therapy—eligible for priority review
🚀 Sanofi’s macular degeneration gene therapy SAR446597 earns Fast Track designation
🎯 MiNK’s iNKT cell therapy achieves remission in a patient with metastatic solid tumors
🔁 Pierre Fabre takes over the Tabelecleucel IND from Atara in pivotal EBV-specific T-cell program
⛔ FDA rejects Capricor’s Duchenne therapy due to insufficient efficacy data
🛑 Ultragenyx’s UX111 for Sanfilippo syndrome denied over manufacturing flaws, despite positive clinical data
💸 JCR licenses its JUST-AAV platform to AstraZeneca in a deal worth up to $825M
This isn’t just a recap—it’s your weekly intelligence drop on the science transforming modern medicine. 🎯
📢 Stay Ahead in Gene and Cell Therapy Research!
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