Great news for UniQure and the Huntington's Disease patient community. Hopefully this paves the road to more flexibility (and coherent guidelines) from the regulatory perspective for trials where a traditional design presents feasibility or ethical concerns.

so the FDA just reversed their stance on AMT-130...following a hotly anticipated type B meeting, ph I/II data will indeed be acceptable for potential accelerated approval. BLA filing to follow in Q3'26.

uniQure receives positive FDA feedback for accelerated approval of its one-time gene therapy AMT-130 for Huntington’s disease. BLA submission planned for Q3 2026. https://lnkd.in/e7YDVdwa

REGENXBIO has announced that it has dosed the last participant in the confirmatory study of RGX-202, its investigational gene therapy for Duchenne, and plans to begin a U.S. FDA application later in 2026: https://bit.ly/4p25wm0

A potential accelerated approval pathway is taking shape for AMT-130, uniQure's investigational gene therapy for Huntington disease. The FDA has greenlit 3-year clinical data as the primary basis for a BLA submission🔬👇 Our coverage: https://hubs.li/Q04mc_2f0

Today we announced that we have aligned with the U.S. FDA on a path forward toward potential accelerated approval of NAVSUNLI™ (RGX-121), our investigational one-time gene therapy for MPS II (Hunter syndrome). Learn more: https://lnkd.in/eRVqtsgs

Researchers have successfully demonstrated a groundbreaking approach to Parkinson's disease treatment through BBM-P002, a dual-target gene therapy that addresses two critical bottlenecks in dopamine production. https://lnkd.in/d9Xp8n2B

Happy to share CBER/OTP’s latest webinar with LCDR Jessica Dunn and I explaining the FDA’s CGT CMC Flexibility guidance. We hope this consolidates useful information from across the regs and FDA guidances to help you implement a phase appropriate development strategy for cellular and gene therapies.

UniQure to seek FDA approval for Huntington's disease gene therapy after previous clash with agency https://ift.tt/fRwUtYM The announcement comes months after UniQure became embroiled in a public debate with FDA leaders over the clinical trial data supporting its application.

uniQure’s planned third-quarter submission for its Huntington’s disease gene therapy may be a harbinger of a more flexible FDA under acting commissioner Kyle Diamantas—but how long will it last? And how can companies be sure these positive decisions won’t just be reversed? https://hubs.li/Q04lS_6-0