FDA rolls out an early and historic OK for Spark’s pioneering gene therapy

  Spark Therapeutics $ONCE  has scored an historic FDA approval of Luxturna, the world’s first such AAV-delivered gene therapy designed to cure a rare eye disease triggered by a genetic mutation. The drug is OK’d for RPE65 mutation linked retinal dystrophy. The treatment uses a viral vector to insert the correct copy of a gene retinal cells need to create a protein that turns light into electric signals which can restore vision lost to the disease. As with the earlier pioneering approval of the world’s first CAR-T, FDA commissioner Scott Gottlieb did the honors in recognizing the importance of this approval. And he says the agency will make sure that the regulatory path is straight and clear for the rest of the field looking to following Spark’s footsteps. “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing ...

• Source: drugdu
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• December 20, 2017