🧬 Gene & Cell Therapy Weekly Update: FDA Firsts, Alzheimer’s Breakthroughs, $135M Raised & More!
In this episode of Gene and Cell Therapy Updates, we unpack the most critical headlines shaping the future of genetic medicine. From FDA milestones to global academic alliances and funding breakthroughs.
💡 Top Highlights This Week:
📢 Regulatory Reform: RFK Jr. advocates for flexible gene therapy regulations—backed by leaders from the FDA, NIH, and academia
🏆 Historic FDA Win: Sarepta’s rAAVrh74 vector secures the first-ever FDA platform tech designation—cutting red tape across therapies
🌍 Global Alliance: Seoul National University Hospital & UMass Chan join forces to fight rare neurogenetic diseases
🧠 Alzheimer’s Innovation: UCSD shifts focus from plaques to cell behavior—a bold new therapeutic strategy
💪 Duchenne Breakthrough: REGENXBIO’s RGX-202 boosts function with strong safety—BLA planned for 2026
🧬 Parkinson’s Progress: Casma’s CSM-101 restores lysosomal function—IND filing expected early 2026
🔬 Oncology Tech Leap: Mission Bio’s new single-cell platform analyzes 10K+ cells and cuts cost by 60%
👁️ Stargardt Hope: VeonGen earns FDA RPDD for dual-AAV gene therapy delivering the full ABCA4 gene
🇰🇷 Korean Licensing Deal: Ocugen out-licenses OCU400 for Retinitis Pigmentosa—targeting ~15,000 patients
🧫 AML Therapy Setback: TCR therapy weakened post-transplant due to NK-like shift—Azacitidine shows promise
🫁 Lung Cancer Advances:
• MAIA’s ateganosine combo shows 17.8-month OS in CPI-resistant NSCLC
• AbilityPharma’s Ibrilatazar doubles OS to 22.5 months in squamous NSCLC
💰 $135M Raised: SpliceBio secures major funding for Stargardt therapy—backed by Sanofi, EQT, Roche & more
⚙️ Manufacturing Alliance: MaxCyte + Ori Biotech team up to scale CRISPR-engineered CD19 CAR-T production
🚀 From neurogenetics to oncology, this week’s news showcases the next wave of breakthroughs in gene and cell therapy.
Whether you’re a researcher, clinician, or biotech leader, these updates keep you plugged into the future of gene and cell therapy.
📢 Stay Ahead in Gene & Cell Therapy Research!
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