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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