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Nightstar gene therapy wins US FDA designation for regenerative medicine

Nightstar Therapeutics PLC said the U.S. Food and Drug Administration granted regenerative medicine advanced therapy designation to its gene therapy NSR-REP1 for a rare genetic disorder that leads to blindness.

The London-based gene therapy company said that NSR-REP1 is currently being evaluated in a phase 3 trial to treat choroideremia, a genetic retinal disorder characterized by progressive vision loss.

Nightstar said the U.S. regulator's designation was based on clinical data from phase 1/2 trials supporting the maintenance and improvement of visual acuity in choroideremia patients.

A regenerative medicine is eligible for the designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition.

The designation includes all the benefits of a breakthrough therapy designation, including eligibility for priority and rolling reviews and accelerated approval from the FDA.