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 senescense 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:
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.
Non-viral ELOVL2 Gene Therapy
As part of our ELOVL2 gene therapy program, Visgenx is developing synthetic vector constructs that may overcome limitations to viral mediated gene transfer. Advantages of our synthetic vector constructs include:
- The ability to deliver genes to large for current viral systems
- They are unaffected by pre-existing anti-capsid antibodies
- They are less immunogenic. There is no capsid protein, thus no anti-capsid antibodies.
Mission In Focus.
Learn more about our goal to develop ELOVL2 based therapeutics to slow or halt aging-related disorders.