Prime Medicine's PM359, the first Prime Editing therapy administered to humans, demonstrated rapid restoration of NADPH oxidase activity in a patient with Chronic Granulomatous Disease, exceeding therapeutic thresholds.
Prime Medicine expects to release initial clinical data from its Phase 1/2 trial of PM359 for p47phox chronic granulomatous disease in 2025, potentially demonstrating the curative potential of its Prime Editing technology.
Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors.
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