To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A

This study is a single-arm, open-label study evaluating the safety and efficacy of GS1191-0445 injection as a single dose in Chinese subjects with severe hemophilia A. GS1191-0445 is an adeno-associated virus 8 (AAV8)-delivered gene therapy 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 gene expression cassette, which transfects hepatocytes and facilitates the specific expression and secretion of FVIII into the blood.

NCT Number	NCT07548411
Lead Sponsor	Gritgen Therapeutics Co., Ltd.
Conditions	Severe Hemophilia A
Enrollment	7 participants
Start Date	2023-07-10
Primary Completion	2024-12-31 (estimated)
Study Completion	2029-12-23 (estimated)
Updated on ClinicalTrials.gov	2026-05-06