AAVLK03hOTC clinical trials

Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

This study is testing a new gene therapy for children with a rare liver condition called Ornithine Transcarbamylase Deficiency (OTCD). It aims to help their livers produce a missing enzyme, reduce harmful ammonia build-up, and potentially act as a 'bridge' to a liver transplant, improving their health.