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

NCTID	NCT06111638 (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-H803 injection in heavy 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. (Show More)
Development Status	Active
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	BBM-H803
Sponsor	Shanghai Belief-Delivery BioMed Co., Ltd
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	12
Results Posted	Not Available

Target Gene/Variant	F8
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	AAV
Editor Type	none
Dose 1	Undisclosed dose 1
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China