This Week in Gene & Cell Therapy: Breakthrough Trials, Fast Track Designations, and Global Access Expansions! 🧬
From uniQure’s seizure breakthrough in drug-resistant epilepsy to ART001 making history as China’s first FDA RMAT-designated gene edit therapy, this week’s roundup delivers transformative headlines across CNS disorders, oncology, and advanced manufacturing.
đź’ˇ Top Highlights This Week:
🧬 uniQure’s AMT-260 achieves a 92 percent seizure reduction in drug-resistant epilepsy, while AMT-130 advances toward a 2026 BLA with FDA support for accelerated approval in Huntington’s disease.
🚀 Capsida’s CAP-002 receives FDA Fast Track designation—the first IV-administered AAV gene therapy for STXBP1-DEE, setting the stage for the upcoming SYNRGY trial.
🧠Cartesian’s Descartes-08 begins Phase 3 as a potential chemo-free, single-course treatment for AChR-positive myasthenia gravis.
🏥 Yescarta shows comparable safety in outpatient lymphoma treatment, with nearly half of patients avoiding hospital admission within 3 days.
💡 Estrella’s EB103 expands its B-cell NHL CAR-T trial, delivering early safety and complete responses using the ARTEMIS® T-cell platform.
🔬 OBX-115 reaches a 67 percent overall response rate in ICI-resistant melanoma, with no ICU admissions or severe toxicities—highlighting its IL2-sparing design.
🌬️ Genprex’s Reqorsa aims to extend progression-free survival in SCLC as it enters Phase 2, targeting TUSC2 loss and combining with Tecentriq.
🧫 Immix Biopharma reports a 70 percent complete response rate in AL amyloidosis patients treated with NXC-201—no relapses or safety concerns observed.
🎌 ART001 earns FDA RMAT status with transthyretin reductions lasting 72 weeks and zero off-target effects—marking a global milestone for Chinese gene editing.
🧬 Korea’s Assembly prepares to debate legislative changes to expand access to in-vivo gene therapies following a 50,000+ signature petition from rare disease families.
🎯 The CAR T Coalition announces a bold plan to double CAR T access by 2030, tackling cost, awareness, and referral bottlenecks with backing from MSKCC and ASTCT leaders.
🏠Cellares & Mitsui Fudosan launch a Smart Factory in Japan to automate CAR-T manufacturing, cut costs by 50 percent, and employ 350 professionals.
🧪 Bionique & Cellipont team up to fast-track mycoplasma QC testing, helping cell therapy manufacturers achieve faster, compliant batch release.
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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