Orphagen Pharmaceutical was awarded a $446,635 NIH National Center for Advancing Translational Sciences (NCATS) Phase I SBIR grant to Investigate Novel Small Molecules to treat Retina Pigmentosa (RP). SDEE member Scott Thacher, Orphagen's founder and CEO, is a Principal Investigator of the grant.
Each year thousands of Americans are diagnosed with RP, a complex neurodegenerative disease caused by mutations in more than 90 different genes. RP initially presents as loss of night vision, but progresses to loss of central vision and eventual blindness. There is no cure. Orphagen has identified small molecule compounds that could significantly delay degeneration in as many as 40% of RP patients.