Prime Medicine Stock Jumps After FDA Clears Gene Editing Therapy for Clinical Trial

Prime Medicine (NASDAQ:PRME) shares gained over 12% in after-hours trading Thursday after the company announced it received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for PM647. The regulatory clearance allows the gene editing company to proceed with human clinical trials for its therapy targeting Alpha-1 Antitrypsin Deficiency (AATD).

FDA Greenlights Clinical Trial

The FDA's decision gives Prime Medicine the go-ahead to initiate a Phase 1/2 clinical study for PM647 in the United States. This marks a significant milestone for the company, moving its second therapeutic candidate into clinical development.

PM647 is an *in vivo* gene editing therapy designed to correct the genetic mutation that causes AATD. According to the company, the therapy is intended to be a one-time intravenous infusion that addresses both the liver and lung manifestations of the disease.

Targeting the Root Cause of AATD

Alpha-1 Antitrypsin Deficiency is an inherited disorder caused by mutations in the SERPINA1 gene. PM647 is specifically designed to correct the E342K mutation, the most common cause of the disease. This condition affects an estimated 100,000 people in the U.S. and 200,000 across the U.S. and Europe who carry the severe PiZZ genotype, for which there is currently no approved curative treatment addressing the underlying genetic cause.

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The therapy aims to restore the body's production of functional M-AAT protein. The company noted that PM647 utilizes the same liver-directed lipid nanoparticle delivery system as its other investigational program, PM577a, which is being developed for Wilson disease.

What's Next for PM647

Prime Medicine plans to launch a global, single-arm, open-label Phase 1/2 trial to evaluate the safety, tolerability, and preliminary efficacy of PM647 in adults with AATD. The study will begin by enrolling patients with lung-only symptoms before expanding to a separate cohort of patients with significant liver disease.

Investors and the medical community will be watching for initial clinical data, which the company expects to be available in 2027. This clearance represents the company's second regulatory milestone in recent months, signaling progress in its pipeline of gene editing therapies.