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VGX-0111
- Development stage
- Preclinical
- Lead developer
- Visgenx
- Modality
- Lipid-mediated Delivery → Gene Addition/Replacement → Gene Therapies, Electrical: Electroporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Magnetic: Magnetofection → Physical Methods → Gene Addition/Replacement → Gene Therapies, Mechanical: Microinjection, microfluidics, gene gun → Physical Methods → Gene Addition/Replacement → Gene Therapies, Acoustic/Optical: Sonoporation, laser photoporation → Physical Methods → Gene Addition/Replacement → Gene Therapies, Pressure-driven: Hydrodynamic injection → Physical Methods → Gene Addition/Replacement → Gene Therapies
- Administration
- Subretinal, Intraocular, Ophthalmic
01
Overview
VGX-0111 is an investigational gene therapy developed by Visgenx designed to increase expression of the ELOVL2 gene, which plays a key role in the biosynthesis of long-chain and very-long-chain polyunsaturated fatty acids (LC and VLC PUFAs) essential for retinal health. The therapy uses a fully synthetic adeno-associated virus (AAV)-based vector platform for non-viral gene delivery. Declining expression of ELOVL2 has been implicated in aging-related disorders, particularly dry age-related macular degeneration (dry AMD), where it may contribute to photoreceptor cell loss and visual decline. By restoring physiological levels of ELOVL2 in target tissues such as the retina, VGX-0111 aims to slow or halt disease progression by increasing LC and VLC PUFA levels. The approach is positioned as a safer alternative to CRISPR-based methods due to its synthetic AAV genome platform[1][3][4][5].
- Other names
- Synthetic AAV (Non-viral) ELOVL2 Gene Therapy
02
Targets
ELOVL2
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