UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms
While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
uniQure held a Type A meeting with the FDA at the end of January and, in a statement released today, said the agency "cannot agree that data from the Phase I/II studies, compared to an external ...
Qure (NASDAQ:QURE) used its fourth-quarter and full-year 2025 earnings call to outline next steps for its lead Huntington’s ...
~ Supraphysiological expression of α-Gal A activity maintained for over a year in longest treated patient as of data cutoff date ~ ~ Stable Lyso-Gb3 levels maintained post-dosing, regardless of enzyme ...
SOUTH SAN FRANCISCO, Calif., March 03, 2023 (GLOBE NEWSWIRE) -- Applied Molecular Transport Inc. (AMTI) (AMT) today announced additional Phase 2 data for oral AMT-101 in chronic pouchitis patients.
AMT-130 Huntington’s gene therapy data, FDA Phase III sham-trial push, cash runway, and 2026 catalysts—read now.
LEXINGTON, Mass. and AMSTERDAM, Feb. 06, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, ...
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