HG005

HG005 is an investigational gene therapy developed by HuidaGene Therapeutics for the treatment of Stargardt disease type 1 (STGD1), a rare inherited retinal disorder that leads to progressive vision loss. The therapy uses two adeno-associated virus (AAV) vectors to deliver a functional copy of the ABCA4 gene—too large to fit into a single AAV vector—into retinal cells. The expressed ABCA4 protein is intended to restore normal waste removal processes in the retina, protect photoreceptor cells, and slow or halt disease progression. HG005 is currently undergoing early phase 1 clinical evaluation in pediatric patients with STGD1 caused by biallelic ABCA4 mutations[1][3].

Other names

HG005HG-005HG 005