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LipidA-LNP encapsulating FVIII plasmid
- Development stage
- Preclinical
- Lead developer
- Seattle Children's Research Institute
- Modality
- Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
LipidA-LNP encapsulating FVIII plasmid is a non-viral gene therapy candidate being developed for the treatment of Hemophilia A. It utilizes a proprietary lipid nanoparticle (LNP) platform featuring a novel lipid, LipidA (and its optimized version LipidAopt), to encapsulate a DNA plasmid (pCAG-FVIIIX10) encoding a secreted version of Coagulation Factor VIII. Unlike traditional viral vectors, this LNP-based DNA delivery system is designed to be re-dosable and avoid the constraints of pre-existing immunity to viral capsids. Preclinical studies in Hemophilia A mouse models have demonstrated sustained Factor VIII expression and the feasibility of re-administration to maintain therapeutic levels.
- Other names
- LipidA-LNP-pDNALipidA-LNP-pCAG-FVIIIX10LipidA-LNP-pCAG-FVIIIX-10LipidA-LNP-pCAG-FVIIIX 10
02
Targets
Coagulation Factor IXaF10 (Factor Xa)BB-031 (Von Willebrand factor)F8 (Coagulation Factor VIIIa)
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