The company plans to file investigational new drug (IND) and clinical trial applications (CTA) for Wilson's Disease in the first half of 2026 and for AATD in mid-2026, with initial clinical data expected in 2027.

Allan Reine, M.D., has been appointed as the new CEO, taking over from Keith Gottesdiener, M.D., who stepped down immediately.

On May 19, 2025, Prime Medicine, a biotech company recognized for its CRISPR-based gene editing technology, announced a significant leadership change and workforce reductions.

As part of a strategic restructuring, the company will lay off approximately 25% of its workforce to cut costs and lower cash needs.

This reorganization aims to reduce anticipated cash requirements by nearly half through 2027, reflecting the company's response to a downturn in the biotech market.

Many companies in the gene editing field have faced similar challenges, leading to job cuts and program discontinuations.

Prime Medicine will now focus on developing treatments for large genetic liver diseases, specifically Wilson's Disease and Alpha-1 Antitrypsin Deficiency (AATD), while deprioritizing its Chronic Granulomatous Disease (CGD) programs.

Wilson's Disease affects over 20,000 individuals in the U.S. and EU, with no approved disease-modifying therapies available, while AATD impacts about 200,000 people, many of whom face severe health risks.

Although Prime Medicine's PM359 program shows promise in treating CGD, the company is exploring external development options following initial positive data.

Founded in 2019 based on technology developed by Broad Institute biochemist David Liu, Prime Medicine aims to enable precise genome edits.

Prime Medicine will continue its Cystic Fibrosis program with support from the Cystic Fibrosis Foundation and will also develop Prime Edited CAR-T products in partnership with Bristol Myers Squibb.

Dr. Reine emphasized the company's commitment to navigating market challenges while delivering on the potential of Prime Editing technology.