🧬 This Week in Gene & Cell Therapy: Elevidys Safety Data, CAR-T Updates, EMA Approvals, and More
From Sarepta’s Elevidys safety data to Beam Therapeutics’ exciting RMAT designation, this week’s episode brings you the latest advancements shaping the world of gene and cell therapy. Whether you’re following the progress of gene therapies for sickle cell disease, Stargardt disease, or exploring pivotal updates on gene therapies for rare conditions, this episode has you covered with the must-know news in biotech. 🔬💡
🔍 Highlights in this episode:
🌍 Sarepta shares additional safety data for Duchenne muscular dystrophy gene therapy, Elevidys
🧬 Taysha Gene Therapies reports strong Q2 2025 results, advancing TSHA-102 for Rett syndrome
🇪🇺 Ocugen receives positive EMA advice for OCU410ST gene therapy in Stargardt disease
🧬 Apertura licenses blood-brain barrier-penetrant AAV capsid for CNS gene therapies
💡 Nanoscope Therapeutics publishes STARLIGHT Phase 2 data for MCO-010 in Stargardt disease
🧠 Klotho Neurosciences partners with AAVnerGene for KLTO-202 manufacturing
🚨 Breakthrough T1D paper paves the way for beta cell replacement therapies
⚠️ FDA delays PDUFA date for Regenxbio’s RGX-121 in Hunter syndrome
🌟 Beam Therapeutics receives FDA RMAT designation for BEAM-101 in sickle cell disease
💰 Abeona Therapeutics reports positive Q2 2025 results, ZEVASKYN launch updates
🩺 ImmunityBio’s CAR-NK therapy shows complete responses in lymphoma patients
🇰🇷 Korea approves Gilead’s Yescarta CAR-T therapy for B-cell lymphomas
💼 Genentech walks away from $2B partnership with Adaptive Biotechnologies
This isn’t just a news roundup—it’s your essential look at the trailblazing research and innovations transforming gene and cell therapy today. 🎯
📢 Stay Ahead in Gene & Cell Therapy Research!
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