Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

Summary

This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects. BBM-H803 is an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene and raises circulating levels of endogenous FVIII.

Timeline

Start

2024-01-03

Primary completion

2027-05-30

Completion

2031-05-30

Drugs

Indications