Can progenitor cells go further than gene therapy in retinal disorders? A biotech's answer shines in PhIIb
By the time the FDA approved Luxturna — the pioneering gene therapy for an inherited retinal disease — Henry Klassen had been researching and treating the group of diseases known as retinitis pigmentosa for more than 20 years.
Spark Therapeutics’ success there had not just inspired a $4.3 billion takeover by Roche, it had also emboldened other biotechs pursuing a therapy that would deliver one of the genes tied to different variations of the disorder. Nightstar Therapeutics subsequently scored its own buyout with Biogen, and MeiraGTx recently posted early but “exciting data” on its J&J-partnered program.
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