AAV8-hCocH for Cocaine Use Disorder

计划入组 10 人 · 首次发布于 2021 年 5 月 13 日

Hemophilia B Gene Therapy With AAV8 Vector

计划入组 4 人 · 首次发布于 2012 年 6 月 15 日

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients

计划入组 22 人 · 首次发布于 2022 年 11 月 15 日

CTx-ABCA4

ABCA4 gene therapy (dual AAV vector, Stargardt disease)

ABCA4 DNA nanoparticles

argenx SE

Belgium

Orion Corporation

Finland

Zai Lab Limited

China

AAVantgarde to Present Promising Gene Therapy Data for Usher 1B and Stargardt Disease at ARVO 2025

SpliceBio Initiates First-in-Human Trial of Dual AAV Gene Therapy for Stargardt Disease

Ocugen's OCU410ST Receives Orphan Drug Designation from EMA for ABCA4 Retinopathies