Gene Therapy for Chinese Hemophilia A

Summary

IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \<1 IU/dl residual FVIII levels.

Timeline

Start

2021-03-04

Primary completion

2028-07-31

Completion

2028-07-31

Publications

• Liu W, Pei X, Yu T, Xu B, Dai X, Xue F, Chen L, Wang X, Wang Y, Chen Y, Liu X, Fu R, Wang W, Chi Y, Li HY, Yang R, Zhang L. Phase 1 pilot study for hemophilia-A: AAV8 vector with prophylactic tacrolimus-glucocorticoid achieves therapeutic FVIII activity. Signal Transduct Target Ther. 2026 Mar 11;11(1):88. doi: 10.1038/s41392-026-02599-3.

Drugs

Indications