Cell and Gene Therapy Weekly News - May 29th 2025

This Week in Gene & Cell Therapy: Clinical Trial Expansions, Rare Disease Designations, and Breakthrough Manufacturing Approvals! 🧬

From Immix Biopharma’s rapid expansion of clinical sites for AL amyloidosis to Ocugen’s Rare Pediatric Disease Designation for Stargardt disease, this week’s roundup is packed with exciting developments in hematology, ophthalmology, oncology, and neurodegeneration.

💡 Top Highlights This Week:

🚀 Immix Biopharma expands NEXICART-2 trial to 14 U.S. sites with enrollment exceeding expectations and interim data coming at ASCO 2025.

🎗️ Iovance’s Amtagvi shows durable five-year survival in advanced melanoma—first one-time T cell therapy for solid tumors with long-term safety.

👁️ Porton Advanced and TongEYE join forces to accelerate iPSC-derived retinal pigment epithelium therapies targeting blinding diseases.

🤝 RHEACELL and AOP Health form a strategic partnership to commercialize somatic cell therapies across Europe, Turkey, Israel, and MENA.

🌟 Ocugen’s OCU410ST gene therapy receives FDA Rare Pediatric Disease Designation for Stargardt disease affecting 100,000 patients in the US and Europe.

⚠️ Rocket Pharmaceuticals pauses Danon disease gene therapy trial after a fatal patient event; investigation underway on immune suppression agents.

🧬 Reduced-intensity lentiviral gene therapy for sickle cell disease shows over 80 percent reduction in crises with promising safety profile.

🧠 AskBio reports positive Phase 1b Parkinson’s trial results with stable motor and non-motor symptoms, advancing Phase 2 REGENERATE-PD.

👁️ Vitalgen completes Phase III enrollment for VGR-R01 gene therapy targeting Bietti crystalline dystrophy, aiming for 2026 BLA submission.

🏭 Procella expands GMP manufacturing capabilities with Swedish regulatory approval, supporting cardiac and Parkinson’s cell therapies.

🥇 CARsgen’s Satri-cel granted Priority Review by China’s NMPA for advanced gastric cancer with ongoing trials in China and North America.

Whether you’re a researcher, clinician, or investor, these insights keep you at the forefront of gene and cell therapy innovation.

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