Gene therapy flunks PhII/III study, but former Sarepta partner sees a path forward — if it can find the cash
The development path for Lysogene’s gene therapy for MPS IIIA has been a rocky one. After the FDA slapped a partial clinical hold on a Phase II/III study, a patient already dosed in the trial died, although it was deemed unrelated to treatment. Then earlier this year, Sarepta pulled out of their three-year partnership due to disagreements on who will handle commercial supply.
And now, Lysogene reported the trial has failed its primary endpoint.
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