To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
Status:
RECRUITING
Trial end date:
2030-11-30
Target enrollment:
Participant gender:
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.