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HEM-8
- Development stage
- Preclinical
- Lead developer
- Copernicus Therapeutics
- Modality
- Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
HEM-8 is a non-viral gene therapy candidate being developed by Copernicus Therapeutics for the treatment of Hemophilia A. The program utilizes the company's proprietary DNA nanoparticle platform, which involves compacting single molecules of plasmid DNA encoding the Coagulation Factor VIII (FVIII) gene into stable nanoparticles using poly-L-lysine-polyethylene glycol (PEG) polymers. Unlike traditional viral vectors such as AAV, these non-viral nanoparticles are designed to be non-immunogenic and non-inflammatory, which may allow for repeat dosing and avoids the limitations of pre-existing immunity. The therapy aims to achieve sustained, therapeutic levels of FVIII expression in the liver to restore normal blood clotting and prevent spontaneous bleeding episodes. It is currently in the preclinical stage of development.
- Other names
- Copernicus Hemophilia A gene therapynon-viral DNA nanoparticle gene therapy for Hemophilia A
02
Targets
F8 (Coagulation Factor VIIIa)
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