Gene Therapy for Chinese Hemophilia B

Completed 10 enrolled Institute of Hematology & Blood Diseases Hospital, China East China University of Science and Technology · collab

NaSingle-groupOpen-labelTreatment

Summary

GT2019001 is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.

Timeline

Start: 2019-10-16
Primary completion: 2022-01-07
Completion: 2022-01-07

Publications

Xue F, Li H, Wu X, Liu W, Zhang F, Tang D, Chen Y, Wang W, Chi Y, Zheng J, Du Z, Jiang W, Zhong C, Wei J, Zhu P, Fu R, Liu X, Chen L, Pei X, Sun J, Cheng T, Yang R, Xiao X, Zhang L. Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. Lancet Haematol. 2022 Jul;9(7):e504-e513. doi: 10.1016/S2352-3026(22)00113-2. Epub 2022 May 19.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	BBM-H901	Gene therapy (AAV / viral vector)	5e+12 vg/kg	Intravenous

Gene Therapy for Chinese Hemophilia B

Summary

Timeline

Publications

Drugs

Indications