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iqDNA factor VIII
- Development stage
- Preclinical
- Lead developer
- Generation Bio
- Modality
- Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
iqDNA factor VIII is an investigational non-viral gene therapy developed by Generation Bio for the treatment of hemophilia A. It utilizes the company's proprietary immune-quiet DNA (iqDNA) technology, which consists of modified, structured, partially single-stranded DNA designed to evade innate immune detection while maintaining high transcriptional activity. The therapy is delivered via a lipid nanoparticle (LNP) system and is produced using a rapid enzymatic synthesis (RES) manufacturing process. By delivering a functional gene encoding Factor VIII, the therapy aims to provide sustained production of the essential blood clotting factor in patients who are deficient. As of 2024, the program is in preclinical development, with a focus on second-generation formulations and scaling for non-human primate studies.
- Other names
- immune-quiet DNA factor VIII
02
Targets
F8 (Coagulation Factor VIIIa)
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