BBM-H901 is an innovative gene therapy product developed for the treatment of Hemophilia B, an inherited bleeding disorder caused by a deficiency of coagulation factor IX (FIX). Utilizing a recombinant adeno-associated virus (rAAV) vector, BBM-H901 delivers an optimized human FIX gene directly to the liver cells of patients, enabling continuous, long-term expression of functional FIX protein through the host’s natural gene transcription system.
This one-time intravenous infusion is designed to restore and maintain FIX activity at therapeutic levels, significantly reducing the risk of spontaneous bleeding, joint damage, and the need for frequent factor replacement infusions. BBM-H901 addresses the limitations of conventional therapies, offering patients greater convenience, improved quality of life, and reduced long-term healthcare burdens.
Key Features:
- Vector Type: Recombinant Adeno-Associated Virus (rAAV)
- Gene Target: Optimized human coagulation factor IX (FIX)
- Administration: Single intravenous infusion
- Mechanism: Gene transfer to liver cells for continuous endogenous FIX production
- Indication: Moderate to severe Hemophilia B
Clinical Highlights:
- Achieved mean FIX activity of 55.08 IU/dL in Phase III trials (52-week follow-up)
- Reduced annualized bleeding rate (ABR) to 0.6
- 80.8% of patients experienced no bleeding events post-treatment
- Significant decrease in FIX infusions from 58.2 times/year to 2.9 times/year
- No serious adverse events (SAEs) reported related to the therapy
Regulatory Status:
- Breakthrough Therapy Designation — Center for Drug Evaluation, NMPA (China)
- Orphan Drug Designation (ODD) — U.S. Food and Drug Administration (FDA)
- Rare Pediatric Disease Designation (RPDD) — U.S. FDA
- Advanced Therapy Medicinal Product (ATMP) — European Medicines Agency (EMA)
Intended Use:
BBM-H901 is intended for adult and pediatric patients with moderate to severe Hemophilia B, providing a durable therapeutic option through a single administration, significantly reducing bleeding events and improving long-term joint health.
