FDA rolls out an early and historic OK for Spark's pioneering gene therapy -- now let's talk price
Spark Therapeutics CSO Katherine High and CEO Jeff Marrazzo. Spark
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.
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