Posted by Michael Wonder on 17 Jun 2018
Nightstar Therapeutics receives regenerative medicine advanced therapy designation for NSR-REP1 in choroideremia
14 June 2018 - First gene therapy RMAT designation for an inherited retinal disease.
Nightstar Therapeutics today announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to NSR-REP1, the company’s lead product candidate currently in Phase 3 development for the treatment of choroideremia, a rare, degenerative, genetic retinal disorder that leads to blindness.
RMAT designation for NSR-REP1 was based on clinical data supporting the maintenance and improvement of visual acuity from completed Phase 1/2 trials in choroideremia patients treated with NSR-REP1 and disease progression in untreated patients in the ongoing NIGHT natural history observational study.
