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ceDNA Factor VIII
- Development stage
- Discontinued
- Lead developer
- Generation Bio
- Modality
- Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
ceDNA Factor VIII is a non-viral gene therapy candidate developed by Generation Bio for the treatment of hemophilia A. The therapy utilizes closed-ended DNA (ceDNA) constructs designed to encode the Factor VIII protein, delivered to the liver via cell-targeted lipid nanoparticles (ctLNP). This approach aims to provide a redosable, durable treatment that enables the patient's own liver to produce functional Factor VIII. The ceDNA is manufactured using a proprietary cell-free rapid enzymatic synthesis (RES) process. In 2023, Generation Bio announced it was shifting its focus to a next-generation variant called immune-quiet DNA (iqDNA) for its hemophilia A program, effectively discontinuing the development of the initial ceDNA Factor VIII constructs.
- Other names
- ceDNA Factor 8
02
Targets
F8 (Coagulation Factor VIIIa)
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