AMT-180

AMT-180 is an experimental gene therapy developed by uniQure for the treatment of hemophilia A. It utilizes an adeno-associated virus serotype 5 (AAV5) vector to deliver a novel, proprietary variant of coagulation factor IX (FIX). Unlike traditional hemophilia A gene therapies that aim to replace factor VIII (FVIII), AMT-180 is designed to bypass the need for FVIII by expressing a FIX variant that can promote blood clotting independently of FVIII levels. This mechanism makes it potentially suitable for all hemophilia A patients, including those who have developed inhibitors (neutralizing antibodies) against FVIII. Preclinical studies in mouse and primate models have demonstrated that a single dose of AMT-180 can promote clinically meaningful blood-clotting activity independent of FVIII levels.