A Study of FVIII Gene Therapy for Hemophilia A

Summary

This is a single-arm, open-label, clinical study to evaluate the safety, tolerability of BBM 002 injection in Hemophilia A subjects with residual factor VIII (FVIII) levels ≤2 International unit per deciliter (IU/dl) . BBM 002 injection is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene and raises circulating levels of endogenous FVIII.

Timeline

Start

2022-07-19

Primary completion

2024-12-15

Completion

2027-09-15

Drugs

Indications