Prime Medicine, Inc. (NASDAQ:PRME), a biotechnology firm developing one-time curative genetic therapies, announced that the U.S. Food and Drug Administration (FDA) cleared its Investigational New Drug (IND) application for PM647.

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Prime Medicine’s Investigational AATD Therapy Gets FDA Clearance

The clearance paves the way for PM647, an investigational in vivo Prime Editor for Alpha-1 Antitrypsin Deficiency (AATD), to enter clinical studies in the U.S.

AATD is an inherited genetic disorder resulting from variants in the SERPINA1 gene that cause low levels or an abnormal form of the alpha-1 antitrypsin (AAT) protein, leading to an increased risk of lung and liver damage.

The PiZZ genotype, which PM647 aims to correct, is present in roughly 100,000 individuals in the U.S. and an estimated 200,000 across the US and Europe.

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Details on the Phase 1/2 Clinical Trial

The Phase 1/2 clinical trial for PM647 will be a global, single-arm, open-label, first-in-human study.

It aims to assess the safety, tolerability, and initial clinical efficacy of ascending doses of PM647 administered as a one-time intravenous infusion in adult patients.

Initially, the trial will enroll adult participants who only exhibit lung manifestations of AATD.

Once tolerability is established in this group, the study will expand to include a separate cohort of adults with significant liver disease, whether or not they also have lung manifestations of AATD.

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What You Need to Know About PM647

PM647 is designed to correct the E342K (Pi*Z) mutation in the SERPINA1 gene, the primary cause of AATD.

By directly targeting the mutation, PM647 aims to restore the production of functional M-AAT, addressing both the liver and lung complications associated with the disease.

In fully humanized mouse models, PM647 demonstrated significant editing efficiency, successfully restoring corrected M-AAT protein levels to a healthy human range with a single infusion at clinically relevant doses.

PM647 utilizes the same liver-directed lipid nanoparticle (LNP) as PM577a, Prime Medicine’s investigational program for Wilson disease.

PRME Price Action: Prime Medicine shares were down 6.19% to $2.88 at the time of publication on Friday, according to Benzinga Pro data.

Photo by Panchenko Vladimir via Shutterstock

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