Prime Medicine Inc. (NASDAQ:PRME) is one of the best biotech penny stocks to buy according to analysts. On December 23, LifeSci Capital initiated coverage of Prime Medicine with an Outperform rating and $6 price target. The firm highlighted that Prime Medicine is advancing one-and-done gene editing treatments for conditions with high unmet medical needs, specifically Wilson’s disease, alpha-1 antitrypsin deficiency/AATD, and cystic fibrosis. LifeSci Capital expressed particular optimism regarding the commercial and clinical opportunities Prime Medicine is pursuing within the Wilson’s disease and AATD landscapes
In other news, on December 7, Prime Medicine Inc. (NASDAQ:PRME) announced the publication of Phase 1/2 clinical data for its investigational therapy, PM359, in the New England Journal of Medicine/NEJM. PM359 is an autologous hematopoietic stem cell product designed to treat p47phox chronic granulomatous disease/CGD. These findings represent the first-in-human demonstration of Prime Editing’s safety and efficacy.
The trial results focused on two patients, one adult and one pediatric, both of whom had a history of CGD-defining complications, such as skin infections and CGD-associated colitis. Following treatment with PM359, both patients achieved rapid neutrophil and platelet engraftment. By Day 30, they reached 69% and 83% dihydrorhodamine-positive/DHR+ neutrophils, respectively. These figures significantly exceed the 20% threshold typically required for clinical benefit. Because the DHR activity remained stable over time, researchers believe the gene correction successfully occurred in the long-term repopulating hematopoietic stem cells within the bone marrow. Clinically, both participants have remained free of new CGD-related complications.
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.