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

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By John Carroll | Endpoints News | December 19, 2017

Spark Therapeutics 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 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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