To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A

Summary

This study is a single-arm, open-label, multicenter study evaluating the efficacy and safety of GS1191-0445 injection as a single dose in Chinese subjects with hemophilia A. GS1191-0445 is an AAV8-based gene therapy vector designed to express B-domain deleted human factor VIII (FVIII) under the regulation of a human liver-specific promoter. Following a single intravenous administration, AAV8 targets hepatocytes and facilitates the specific expression and secretion of FVIII into the bloodstream.

Timeline

Start

2025-03-31

Primary completion

2026-11-30

Completion

2030-11-30

Drugs

Indications