ZVS101e

ZVS101e is an investigational gene therapy designed for the treatment of Bietti's crystalline dystrophy (BCD), a rare inherited retinal degenerative disease. It utilizes a recombinant adeno-associated virus (AAV) vector to deliver the human CYP4V2 gene via subretinal injection. The therapy aims to replace defective or missing CYP4V2 function in retinal cells, addressing the underlying cause of BCD associated with biallelic mutations in the CYP4V2 gene. Preclinical and early clinical studies have demonstrated good safety and significant improvement in visual function among treated patients. ZVS101e has received orphan drug status from both the FDA and Chinese regulatory authorities[1][5][6].

Other names

ZVS101eZVS-101eZVS 101eVGR-R01VGR-R-01VGR-R 01