Prime Medicine Clears FDA Hurdle For Gene-Editing Therapy Targeting Rare Genetic Disease; Stock Up
(RTTNews) – Prime Medicine, Inc. (Nasdaq: PRME) has received U.S. FDA clearance to begin clinical testing of PM647, a gene-editing therapy designed to treat Alpha-1 Antitrypsin Deficiency, marking another regulatory milestone for the company’s liver-focused genetic medicine pipeline.
Alpha-1 Antitrypsin Deficiency (AATD) is a rare inherited disorder caused by mutations in the SERPINA1 gene, which can lead to progressive lung and liver damage. Current treatment options primarily focus on managing symptoms or replacing the missing protein rather than correcting the underlying genetic defect.
The FDA’s clearance of the Investigational New Drug (IND) application allows Prime Medicine to advance PM647 into human clinical studies in the United States. According to the company, approximately 100,000 people in the U.S. carry the PiZZ genotype that PM647 is designed to address.
PM647 is being developed as a one-time in vivo Prime Editing therapy intended to correct the E342K (Pi*Z) mutation, the most common genetic cause of AATD. By repairing the mutation at its source, the therapy aims to restore production of functional alpha-1 antitrypsin protein and potentially address both the lung and liver complications associated with the disease.
The upcoming Phase 1/2 study will be a global, open-label, single-arm trial evaluating the safety, tolerability and preliminary efficacy of escalating doses of PM647 administered through a one-time intravenous infusion. The trial will initially enroll adults with lung manifestations of AATD. If the therapy demonstrates acceptable tolerability, enrolment will expand to include patients with significant liver disease, with or without accompanying lung complications.
Prime Medicine said PM647 builds on the same liver-directed lipid nanoparticle delivery system used in PM577a, its investigational program for Wilson disease. The company noted that the latest regulatory clearance highlights the potential versatility of its Prime Editing platform across multiple liver-focused genetic disorders.
Preclinical studies conducted in fully humanized mouse models showed that PM647 achieved high levels of gene editing and restored corrected alpha-1 antitrypsin protein into the healthy human range following a single infusion, according to the company.
Prime Medicine expects to report initial clinical data from the PM647 program in 2027.
PRME has traded between $2.67 and $6.94 over the past year. The stock closed Thursday’s trading at $3.07, down 1.92%. In pre-market trading the stock is at $3.42, up 11.62%.
