Pre-BLA feedback says Phase I/II external-control data may not suffice as primary evidence. AMT-130 has Breakthrough (Apr 2025) and RMAT (May 2024); minutes due in 30 days.
uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease
11/03/2025 - 07:05 AM
LEXINGTON, Mass. and AMSTERDAM, Nov. 03, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therap... [7258 chars]