10 Best Gene Therapy Stocks to Buy Right Now

7) Prime Medicine, Inc. (NASDAQ:PRME)

Number of Hedge Fund Holders: 21

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

One promising program in Prime Medicine, Inc. (NASDAQ:PRME)’s pipeline focuses on Wilson’s Disease, which is a genetic disorder affecting copper metabolism. Wall Street believes that this program offers significant opportunities because of the large patient population it can potentially serve.

In October, Prime Medicine, Inc. (NASDAQ:PRME) presented the first in vivo preclinical data from its Wilson’s Disease program, demonstrating that Prime Editors can efficiently correct pathogenic mutations, without introducing safety concerns or detectable off-target edits. Notably, Prime Editors are advanced gene-editing tools developed by Prime Medicine, Inc. (NASDAQ:PRME).

These data are meaningful both for the company’s efforts in Wilson’s Disease (a devasting condition that can affect over 20,000 people in the US and Europe and for which there are currently no approved disease-modifying therapies) and in liver disease programs more broadly.

Prime Medicine, Inc. (NASDAQ:PRME)’s Wilson’s Disease program leverages its proprietary, universal LNP platform, and these interim results suggest that its LNP is expected to successfully deliver increased potency, and an improved safety profile and biodistribution as compared to other commonly used LNPs in development. Prime Medicine, Inc. (NASDAQ:PRME) is looking forward to advancing its Wilson’s Disease program toward an investigational new drug (IND) and/or clinical trial application (CTA) filing in H1 2026.

By focusing on Wilson’s Disease, Prime Medicine, Inc. (NASDAQ:PRME) targets to address a significant unmet medical need, potentially capturing a niche market with limited competition.