Cell and Gene Therapy Weekly News – April 24th 2025

🔬 This Week in Gene & Cell Therapy: Key Breakthroughs in Lupus, Huntington’s, and Pediatric Brain Tumors | Global Trials & RMAT Milestones  🧬

From Korea’s first CAR-T trial for lupus to FDA Breakthrough Therapy designations in Huntington’s and pediatric brain cancer — this week’s update highlights pivotal advances across autoimmune disease, neurodegeneration, pediatric oncology, and regenerative medicine.

In this episode:
💉 Korea launches its first-ever CAR-T trial for systemic lupus with promising early results
🧠 uniQure’s AMT-130 earns Breakthrough Therapy status for Huntington’s after strong Phase I/II data
🧬 Sarepta moves closer to BLA submissions in LGMD gene therapy trials
👶 BrainChild Bio’s CAR-T therapy gains FDA Breakthrough status for DIPG, a rare pediatric brain tumor
🧓 CHOP reports 3–6 year follow-up data for Hemophilia B gene therapy with durable clinical outcomes
🧠 Kyoto University’s iPSC transplant shows early success in Parkinson’s with no major side effects
🧠 BlueRock’s Parkinson’s therapy confirms 18-month safety and dopaminergic cell engraftment
🗣️ UC Davis earns RMAT designation for its cell therapy targeting dysphagia post cancer treatment
👁️ Atsena’s XLRS gene therapy secures RMAT status using novel retinal targeting capsid
🧪 XellSmart’s iPSC therapies approved for trials in both Parkinson’s and ALS across the U.S. and China
🔬 NeuroScientific acquires Isopogen, boosting its MSC platform for inflammatory diseases
💼 Longeveron shares 2025 roadmap for frailty, neurodegenerative, and pediatric heart disease trials
🔬 DAAN and GC Cell announce CAR-T collaboration targeting solid tumors like lung and colorectal cancer
💉 Ferring’s gene therapy for bladder cancer achieves a 75 percent complete response rate in Japan

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