The FDA has granted regenerative medicine advanced therapy designation to choroideremia treatment NSR-REP1, Nightstar Therapeutics announced in a press release.
Currently in phase 3 development, NSR-REP1 is comprised of an AAV2 vector containing recombinant human complementary DNA designed to produce the protein REP1 in the eye, the release said.
The designation allows for an “expedited program for the advancement and approval of regenerative medicine products.” It was based on clinical data from phase 1/2 trials in which NSR-REP1 maintained and improved visual function.
“Receiving RMAT designation for NSR-REP1 highlights the potential of this gene therapy to maintain and improve visual acuity in choroideremia,” Dave Fellows, CEO of Nightstar, said in the release. “This designation further underscores a recognition of the serious nature of choroideremia and the urgent need to develop new treatments for those affected by inherited retinal diseases that would otherwise lead to blindness. We look forward to working closely with the FDA to discuss the NSR-REP1 development program and to determine how we can accelerate the pathway for making NSR-REP1 available to choroideremia patients.”