This technology comprises inhibitors of retinal pigment epithelium 65 (RPE65) to treat retinal degeneration including atrophic forms of age-related macular degeneration and Stargardt disease.
Age-related macular degeneration, particularly for people older than 60, is the most common cause of blindness in developed countries. In the Caucasian population alone, the frequency of all forms of AMD is 90% in males and 16.4% in females for those over 80. Stargardt disease (STGD1) is the most common form of inherited macular dystrophy. There are no proven treatments for either Stargardt disease or dry age-related macular degeneration. Although retinal pigment epithelium 65 (RPE65) is a well-validated drug target for the inhibition of the pathologic increase in bisretinoid synthesis, it proves to be a very difficult drug target as it catalyzes a complex, poorly understood enzymatic reaction.
These biological compounds inhibit retinal pigment epithelium 65 (RPE65) isomerohydrolase via an uncompetitive mechanism to treat ocular diseases characterized by excessive lipofuscin accumulation in the retina. Assessment of this technology’s selectivity confirms general specificity of the compound for RPE65. Moreover, these compounds exhibit significant potency in the RPE65 inhibition assay. These compounds have been identified to have improved drug-like characteristics compared to existing compounds. Furthermore, these compounds have an improved ocular safety profile due to a reduction in mechanism-based ocular toxicity. These compounds have significant therapeutic potential as a therapy for retinal degeneration, including age related macular degeneration (AMD) and Stargardt’s disease (STGD).
These compounds have been tested in vivo in the mouse model of light-induced retinal degeneration.
Patent Pending(WO/2022/266394)
IR CU21263, CU22365
Licensing Contact: Kristin Neuman