We recently compiled a list of the 10 Best Gene Therapy Stocks to Buy Right Now. In this article, we are going to take a look at where Prime Medicine, Inc. (NASDAQ:PRME) stands against the other gene therapy stocks.
Gene therapy is referred to as a medical technique that involves modifying or manipulating genes to treat or prevent disease. The therapy focuses on addressing the underlying genetic cause of a condition by repairing, replacing, or altering genes within an individual’s cells. Industry experts opine that this innovative therapy has the potential to treat a variety of genetic disorders, cancers, and other diseases.
As per IMARC, healthcare infrastructure in the US adapted to accommodate gene therapies. As per IQVIA’s tally, 114 gene therapy trials were initiated in the year 2023. Out of this, a large majority (~77%) were sponsored by the healthcare industry. As per Kella Kapnisi, Head of Cell and Gene Therapy at Team Consulting (a medical technology design and development consultancy), as of now, there are 38 cell and gene therapies approved by the FDA. Notably, many of these have reached commercialization through mostly manual laboratory manufacturing processes.
Growth Drivers of The Gene Therapy Industry
As per IMARC, the US gene therapy market size has been pegged at US$1,312.2 million in 2024. The market should reach US$3,697.8 million by 2033. This phenomenal growth is expected to stem from the increasing prevalence of genetic disorders, genetic engineering advancements, a strong focus on strategic collaborations, supportive regulatory frameworks, and heightened investments in R&D.
Ever since the initiation of the Orphan Drug Designation program, the FDA designated over 1,000 medicines for uncommon diseases as orphan drugs, many of which are gene therapies. Furthermore, optimism around the gene therapy industry grew after the regulatory body showcased its dedication to promoting the development of these medicines in 2023. IMARC added that the FDA approved 3 novel gene therapies for conditions such as spinal muscular atrophy and hemophilia.
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Trends to Drive Growth in 2025
As per Kella Kapnisi, as the cell and gene therapy industry has been continuously evolving, the need and requirement for purpose-built technology is of utmost importance. Small batch manufacturing systems that are customized for personalized therapies and decentralized manufacturing, real-time process control with the help of advanced inline and online analytics, and sophisticated data analysis and management systems using ML technology remain important trends that might drive the growth of cell and gene therapy manufacturing.
As per IMARC, the collaborations between biotech companies, pharmaceutical firms, and university research institutes can pool the funds needed to address challenges related to creating and promoting gene treatments.
IMARC further added that the increased prevalence of genetic disorders and chronic diseases, together with increasing strategic collaborations and partnerships are important trends in the US gene therapy market. The growing cases of cancer and genetic disorders remain one of the key factors in the gene therapy market. Gene therapy is a key component of cancer treatment as it can precisely research and target malignant cells to prevent further damage.
Our Methodology
To list the 10 Best Gene Therapy Stocks to Buy Right Now, we conducted extensive research and scanned through several online rankings. After getting the initial list of 15-20 stocks, we shortlisted the ones having high hedge fund holdings. Finally, the stocks are arranged in ascending order of their hedge fund sentiments, as of Q3 2024.
At Insider Monkey we are obsessed with the stocks that hedge funds pile into. The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter’s strategy selects 14 small-cap and large-cap stocks every quarter and has returned 275% since May 2014, beating its benchmark by 150 percentage points (see more details here).
A scientist examining a microchip and circuit board in a hi-tech lab.
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.
Overall PRME ranks 7th on our list of the best gene therapy stocks to buy right now. While we acknowledge the potential of PRME as an investment, our conviction lies in the belief that some deeply undervalued AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for a deeply undervalued AI stock that is more promising than PRME but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
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Disclosure: None. This article is originally published at Insider Monkey.