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A Study of FVIII Gene Therapy for Hemophilia A

Institute of Hematology & Blood Diseases Hospital, China · 2022
ClinicalTrials.gov · Datasets · License: Public Domain · 2022
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adeno-associated-virusgene-therapy
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.
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