AMT-191 is an investigational AAV5 gene therapy candidate from uniQure for Fabry disease. It is designed to deliver a GLA transgene to the liver to drive GLA protein production as a potential one-time treatment approach. Clinical trial enrollment began in 2024 and the program remains in early-phase clinical development.
uniQure disclosed the FDA will not accept Phase I/II data alone for AMT-130 approval, causing shares to drop 49%. The company held regulatory meetings and reported $622.5 million in cash while investors face an April 13 class action deadline.
uniQure received FDA feedback stating current AMT-130 data unlikely to support accelerated approval for Huntington's disease, while securities fraud lawsuits challenge prior disclosures. Fresh Fabry disease trial data showed elevated enzyme activity but prompted a dosing pause for safety review.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06270316 |
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease |
RECRUITING | PHASE1/PHASE2 |
