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Explain this story: Prime Medicine Gets FDA Green Light for Gene Editing Study
Prime Medicine (NASDAQ: PRME) has received FDA clearance for its Investigational New Drug (IND) application for PM647, a gene-editing therapy for Alpha-1 Antitrypsin Deficiency (AATD). The company plans to initiate a global Phase 1/2 clinical trial to assess the safety and efficacy of PM647, which aims to correct the E342K (Pi*Z) mutation in the SERPINA1 gene. PM647 is designed to restore functional M-AAT protein levels to address both liver and lung complications of AATD.
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Prime Medicine (NASDAQ: PRME) has received FDA clearance for its Investigational New Drug (IND) application for PM647, a gene-editing therapy for Alpha-1 Antitrypsin Deficiency (AATD). The company plans to initiate a global Phase 1/2 clinical trial to assess the safety and efficacy of PM647, which aims to correct the E342K (Pi*Z) mutation in the SERPINA1 gene. PM647 is designed to restore functional M-AAT protein levels to address both liver and lung complications of AATD.
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Published Sep 25, 2026, 14:22 ET · on this site +1d 11h