Vision Medicines has a promising drug candidate to treat Stargardt disease, a rare genetic retinal degenerative disease, and other rare diseases caused by inborn errors of aldehyde metabolism. Vision Medicines has recruited a world-class scientific advisory board that includes leading scientists and clinicians specializing in retinal biology and genetics.
VM200 is a preclinical-stage oral small molecule drug candidate for the treatment of Stargardt disease, a rare disease characterized by early-onset macular degeneration that leads to legal blindness. Stargardt disease is caused by mutations in a key transporter called ABCA4, which lead to the buildup of toxic vitamin A aldehydes and derivatives in the retina to cause blindness. VM200 neutralizes the toxic vitamin A aldehyde and has the potential to be the first treatment for the disease.
VM200 has the potential to be applicable to other rare diseases caused by inborn errors of aldehyde metabolism. Sjogren-Larsson-Syndrome (SLS) is an orphan genetic disease caused by mutations in the FALDH gene, which leads to accumulation of fatty alcohols and inflammatory aldehydes. SLS is characterized by severe retinal, neurological, and dermal disorders. Succinic Semialdehyde Dehydrogenase (SSADH) deficiency is a rare genetic disease caused by mutations in the SSADH gene, which leads to accumulation of toxic aldehydes. SSADH deficiency is characterized by seizures, cognitive delay, ataxia, and other severe disorders. There are no current treatments for SLS and SSADH. In addition to SLS and SSADH, there are many other rare genetic diseases that lead to accumulation of toxic aldehydes where VM200, as an aldehyde trap, may be applicable.