Therapeutic Approach
Our strategy is to slow cellular senescence in macular degeneration and other age-related disorders by “turning back the clock” to a more youthful level of ELOVL2 expression.
Our Pipeline
Our pipeline includes candidate therapeutics for dry AMD based on gene therapy and epigenetic approaches. Longer term programs target ELOVL2 gene therapy for other aging-related disorders.
Rescue From Age-Related Loss of ELOVL2
Our strategy is to slow cellular senescence in macular degeneration and other age related disorders by “turning back the clock” to a more youthful level of ELOVL2 expression. Our therapeutics are based on:
1. Gene Therapy
2. Epigenetic Therapy
Supplement ELOVL2 expression by adding additional copies of the gene to photoreceptor and RPE cells.
Therapeutic Candidates
ELOVL2 Retinal Gene Therapy: Suprachoroidal and Subretinal Administration
Visgenx has developed proprietary ELOVL2 gene constructs that are well tolerated and express in the targeted cells in the eye (photoreceptors and RPE).
Studies in aged mice have demonstrated a single subretinal administration resulted in:
- Enhanced photoreceptor and Muller cell function
- Protection from aging induced photoreceptor loss
Epigenetic Therapy: Restore Endogeneous ELOVL2 Expression by Demethylation
Visgenx has developed proprietary decitabine microparticles for demethylating hypermethylated ELOVL2 gene promoters to rescue ELOVL2 expression in photoreceptors and RPE.
Preliminary non clinical studies suggest single and repeat intravitreal administration of our proprietary decitabine microparticles is well tolerated and increases the levels of the targeted lipids in the retina.