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
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