Drug intelligence / Profile preview

FVIII-containing platelets

Development stage
Unknown
Lead developer
Medical College of Wisconsin
Modality
Recombinant Proteins and Enzymes, Gene Therapies
Administration
Intravenous
01

Overview

**FVIII-containing platelets** refer to platelets genetically engineered to express **Factor VIII (FVIII)**, a critical clotting protein deficient in **hemophilia A (HA)**, using platelet-specific promoters like αIIb. This approach delivers FVIII directly to the site of vascular injury via platelet aggregation, bypassing circulating inhibitors and providing effective hemostasis even in patients with anti-FVIII antibodies. Developed as a **gene therapy** strategy, it has demonstrated superior efficacy over plasma-derived FVIII in preclinical models, including resistance to antiplatelet drugs like clopidogrel, and is currently in **Phase I clinical trials**.[1][2]

Other names
platelet-derived FVIIIplatelet-FVIIIplatelet-targeted FVIII
02

Targets

BB-031 (Von Willebrand factor)Coagulation Factor IXaPS (Phosphatidylserine)F10 (Factor Xa)

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