Vision Restoration with Photoswitch Technology

DESCRIPTION provided by applicant The goal this project is to restore vision to persons with degenerative diseases of the retina The target patient population are currently living with very limited or no sight and include persons with retinitis pigmentosa RP and age related macular degeneration AMD RP is an inherited disorder affecting approximately people in the US that is typically diagnosed in adolescents and young adults Approximately one quarter of all persons with RP become legally blind in both eyes AMD is a relatively common affliction affecting approximately ten million Americans and it is the leading cause of blindness in Americans years and older Early detection and treatment of macular degeneration may help reduce vision loss but does not restore vision There are no treatments that can restore moderate to high acuity vision to these persons The present proposal comprises candidate selection experiments for a small molecule therapeutic Specifically we propose making high purity batches of our candidate photoswitch compounds formulate those compounds for delivery to the back of the eye and complete a series of operant conditioning vision restoration efficacy studies in rat models of RP Based on those results we will select a single compound and formulation for thorough characterization in pharmacokinetic PK studies in rat and rabbit and in in vitro and in vivo safety studies Successful completion of the presen project will allow us to have a fully informed pre IND meeting with the FDA raise monies from private sources to fund the IND enabling GLP safety studies file an IND and initiate a Phase clinical trial in the target patient population Such a clinical trial will provide a clear indiction of the efficacy of the compound in man PUBLIC HEALTH RELEVANCE The goal of the present project is to develop a therapeutic that would restore vision to persons with advanced retinitis pigmentosa RP and age related macular degeneration AMD RP and AMD are progressive degenerative diseases of the retina that result in low vision and blindness There are currently no treatments that can restore moderate or high acuity vision to these persons