Prime Medicine Focuses on Genetic Liver Diseases, Partnered Programs After Positive CGD Data

Prime Medicine Inc. (NASDAQ:PRME) is one of the best hot stocks to buy according to Wall Street analysts. In May, Prime Medicine announced a strategic restructuring, which included a reprioritization of its pipeline, cost-cutting measures, and a leadership transition. The company will now focus its internal efforts on in vivo programs for large genetic liver diseases, specifically Wilson’s Disease and Alpha-1 Antitrypsin Deficiency/AATD, and continue its in vivo Cystic Fibrosis program with external support, as well as partnered programs for Prime Edited CAR-T products.

This shift follows the announcement of initial positive data from the Phase 1/2 clinical trial of PM359 in Chronic Granulomatous Disease/CGD, which provided clinical proof-of-concept for Prime Editing as a transformative gene editing technology.

Prime Medicine Focuses on Genetic Liver Diseases, Partnered Programs After Positive CGD Data

A scientist examining a microchip and circuit board in a hi-tech lab.

However, Prime Medicine is now exploring options for the continued clinical development of PM359 external to the company and ceasing further internal efforts in X-linked CGD. For its internal programs, Prime Medicine expects to file an Investigational New Drug/IND application and/or Clinical Trial Application/CTA for its Wilson’s Disease program in H1 2026, and for its AATD program in mid-2026. Initial clinical data from both programs are anticipated in 2027.

Prime Medicine Inc. (NASDAQ:PRME) is a biotechnology company that delivers genetic therapies to address the spectrum of diseases by deploying gene editing technology in the US.

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Disclosure: None. This article is originally published at Insider Monkey.