Prime Medicine, a Cambridge-based biotech, is canceling its sole clinical-stage genetic medicine after a patient showed promising results in a phase 1/2 trial for chronic granulomatous disease. The company laid off 25% of its staff and CEO Keith Gottesdiener, M.D., is stepping down.
The lead candidate, PM359, uses autologous hematopoietic stem cells modified with “prime editors” to treat the rare inherited disease. No serious adverse events were reported in the preliminary results, and restoration of NADPH oxidase activity was seen in 66% of neutrophils by Day 30.
Despite this milestone, Prime Medicine is ending further development of PM359. The company is seeking external options for clinical development. Instead, it will focus on its preclinical in vivo liver franchise, with an anticipated IND submission in 2026.
The company’s stock has dropped 19% since market open, and CEO Keith Gottesdiener resigned after a layoff announcement that impacted 25% of the staff. Prime Medicine will continue working on CAR-T products for hematology and oncology with Bristol Myers Squibb. The biotech had previously partnered with Bristol Myers Squibb to develop these products.
The move comes amid a challenging industry environment, particularly in gene therapy, where numerous large players are shifting their focus away from the area.
Source: https://www.fiercebiotech.com/biotech/prime-medicines-ceo-exits-biotech-shelves-sole-clinical-gene-therapy-lays-staff