Safety and Tolerability of ZVS101e in Patients With Bietti 's Crystalline Dystrophy

Summary

The purpose of the study is to evaluate the safety and tolerability of an adeno-associated virus vector expressing CYP4V2 in patients with Bietti's crystalline dystrophy (BCD).

Timeline

Start

2022-09-23

Primary completion

2025-03-31

Completion

2025-03-31

Publications

• Background Jia R, Meng X, Chen S, Zhang F, Du J, Liu X, Yang L. AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice. Hum Mol Genet. 2023 Jan 1;32(1):122-138. doi: 10.1093/hmg/ddac181.

Drugs

Indications