Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

NCTID	NCT05203679 (View at clinicaltrials.gov)
Description	This is a multi-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per deciliter (IU/dl) residual factor IX (FIX) levels. BBM-H901 is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene and raises circulating levels of endogenous FIX. (Show More)
Development Status	Active
Indication	Hemophilia B
Disease Ontology Term	DOID:12259
Compound Name	BBM-H901
Sponsor	Shanghai Belief-Delivery BioMed Co., Ltd
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	32
Results Posted	Not Available

Target Gene/Variant	F9
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV843
Editor Type	none
Dose 1	5E12 vg/kg (IIT and Phase 1 dose)
Dose 2	1E13 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	9
Locations	China

Has US IND	False
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Dosing completed of all Phase III subjects