In this article, we will be taking a look at the 8 gene-editing stocks to buy.
Gene editing is an advanced medical technique within gene therapy that involves precisely modifying an individual’s DNA to treat or prevent diseases. This approach directly alters genetic material to correct mutations, enhance cellular functions, or eliminate disease-causing genes. Industry experts believe that gene editing has the potential to revolutionize the treatment of genetic disorders, cancers, and various other conditions.
According to IMARC, the U.S. healthcare infrastructure has adapted to support gene-editing therapies. IQVIA reported that 114 gene therapy trials were initiated in 2023, with approximately 77% sponsored by the healthcare industry. Kella Kapnisi, Head of Cell and Gene Therapy at Team Consulting, noted that the FDA has approved 38 cell and gene therapies, many of which have reached commercialization through predominantly manual laboratory manufacturing processes.
U.S. Gene Editing Market Growth, Breakthrough Therapies, and Challenges Ahead
The U.S. gene editing market has experienced significant growth, valued at $3.19 billion in 2024, while genome editing stood at $3.55 billion in 2023. Looking ahead, projections indicate a substantial surge, with gene editing expected to reach $13.99 billion and genome editing forecasted at $16.49 billion by 2034. These growth trends reflect compound annual growth rates (CAGRs) of 15.93% and 16.6%, respectively, underscoring the increasing adoption and investment in gene-editing technologies.
Several breakthrough therapies are paving the way for advancements in gene editing. Precision BioSciences’ PBGENE-HBV represents the first FDA-cleared in vivo gene-editing trial for chronic hepatitis B. Early data from the trial indicate a 70% reduction in the hepatitis B surface antigen (HBsAg) in two out of three patients at the lowest dose (0.2 mg/kg), targeting covalently closed circular DNA (cccDNA) to address the root cause of HBV persistence. YolTech Therapeutics has also made strides with its hyperoxaluria treatment, demonstrating a 70% reduction in harmful oxalate levels in patients with primary hyperoxaluria type 1 through lipid nanoparticle-delivered gene editing. Additionally, AccurEdit’s cholesterol therapy has shown promising results, achieving a 70% reduction in LDL cholesterol with a single-dose treatment by silencing PCSK9.
The number of patients receiving gene therapies is expected to fluctuate over the next decade. In 2020, 16,244 patients were treated, with this figure projected to rise to 94,696 by 2025 before gradually declining to 65,612 by 2034 as existing patient stocks deplete. Over the next decade, an estimated 1.09 million patients could benefit from gene therapies, with cancer patients making up approximately 48% of recipients.
From a financial and economic standpoint, annual spending on gene editing therapies is anticipated to peak at $25.3 billion in 2026 before stabilizing at $21.0 billion by 2034. These therapies are projected to yield an additional 5.12 quality-adjusted life years (QALYs) per patient, with each QALY valued at $43,110. Insurance coverage is expected to play a crucial role in facilitating access, with Medicare, Medicaid, and private insurers projected to cover $8.1 billion, $5.44 billion, and $12.2 billion annually, respectively.
Despite these advancements, the gene-editing industry faces key challenges. Delivery risks remain a concern, as adverse events associated with viral vectors and conditioning regimens pose safety hurdles. Regulatory scrutiny is also increasing, with the FDA’s pending decision on Abeona Therapeutics’ recessive dystrophic epidermolysis bullosa (RDEB) therapy, expected by April 29, 2025, highlighting the evolving standards for gene-editing approvals.
A scientist in a laboratory working on a gene editing tool, to create treatments for rare genetic diseases.
Our Methodology
For this list, we scanned Insider Monkey’s database of over 1,000 hedge funds as of Q4 2024 and selected companies involved in the development and commercialization of gene-editing technologies. From there, we picked eight companies with the highest number of hedge fund investors and ranked them in ascending order.
Why are we interested in 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 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here).
Here is our list of the 8 best gene-editing stocks to buy.
8. Verve Therapeutics, Inc. (NASDAQ:VERV)
Number of Hedge Fund Holders: 27
Verve Therapeutics, Inc. (NASDAQ:VERV) is a biotechnology company focused on developing gene-editing therapies to treat cardiovascular diseases, particularly for patients with genetic risks like familial hypercholesterolemia. Their innovative treatments aim to permanently lower cholesterol levels with a single-shot approach, potentially reducing heart attack risks.
Verve Therapeutics, Inc. (NASDAQ:VERV) is advancing its gene-editing programs targeting key cholesterol drivers of atherosclerosis. Its lead programs include VERVE-102 (PCSK9 program), which is currently in the Heart-2 Phase 1b clinical trial, with initial data expected in Q2 2025 and final dose escalation data anticipated in H2 2025. The VERVE-201 (ANGPTL3 program) is also in Phase 1b trial, with updates expected in H2 2025, while VERVE-301 (LPA program) has recently been nominated as a development candidate and is being advanced in collaboration with Eli Lilly. As one of the best gene editing stocks, Verve is positioned to make significant strides in its innovative gene-editing efforts.
In its Q4 2024 financial report, the company reported a cash position of $524.3 million and a revenue of $13.1 million for the quarter, totaling $32.3 million for the full year. R&D expenses were $55.0 million for Q4 and $204.3 million for the year, reflecting the company’s heavy investment in its pipeline. Despite the revenue growth from increased collaboration agreements, Verve Therapeutics, Inc. (NASDAQ:VERV) posted a net loss of $50.0 million for Q4 and $198.7 million for the full year as it continues to focus on research and development.
The corporation is targeting a large market opportunity, particularly in cardiovascular diseases. For example, over 1.4 billion people worldwide have lipoprotein(a) concentrations above the risk threshold for ASCVD, making VERVE-301 a potentially game-changing treatment. In recent developments, Verve Therapeutics, Inc. (NASDAQ:VERV) announced that Frederick T. Fiedorek, M.D., will retire as Chief Medical Officer, with Scott Vafai, M.D., taking over clinical development. Additionally, the business regained rights to a novel gene-editing program for liver disease, previously part of its collaboration with Vertex Pharmaceuticals.
7. Ginkgo Bioworks Holdings, Inc. (NYSE:DNA)
Number of Hedge Fund Holders: 27
Ginkgo Bioworks Holdings, Inc. (NYSE:DNA) stands seventh on our list of the 8 best gene-editing stocks to buy. It is a biotechnology company that uses genetic engineering to create bacteria for industrial applications. Its cell programming platform produces a variety of products, such as food ingredients, therapeutics, and chemicals.
Ginkgo Bioworks Holdings, Inc. (NYSE:DNA) has formed strategic partnerships with major biopharma firms and government agencies, which allows it to scale operations and diversify its revenue streams. The company also achieved significant cost savings, reducing its operating expenses by $190 million annually by Q4 2024, which improves its path to profitability.
In Q4 2024, the corporation saw strong growth in Cell Engineering revenue, up 29% year-over-year to $35 million, thanks to expanding relationships with large biopharma customers. The company ended 2024 with $562 million in cash, no bank debt and reduced its cash burn to $55 million, providing a solid financial foundation. Total revenue for Q4 was $44 million, up from $35 million in Q4 2023, while the adjusted EBITDA loss narrowed to $57 million.
However, Ginkgo Bioworks Holdings, Inc. (NYSE:DNA) faced challenges, including a 51% decline in Biosecurity revenue due to the end of K-12 COVID testing contracts. For 2025, the company projects revenue between $160 million and $180 million, with Cell Engineering expected to contribute $110 million to $130 million and Biosecurity at least $50 million. The company aims to achieve adjusted EBITDA breakeven by the end of 2026 through cost reductions and strategic growth initiatives.
6. Intellia Therapeutics, Inc. (NASDAQ:NTLA)
Number of Hedge Fund Holders: 31
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a clinical-stage biotechnology company focused on developing gene-editing therapies using CRISPR/Cas9 technology to treat severe genetic diseases and cancers. The company specializes in both in vivo (inside the body) and ex vivo (outside the body) therapies, with a strong emphasis on in vivo approaches.
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is focused on developing gene-editing therapies for diseases with high unmet needs, such as hereditary angioedema (HAE), transthyretin amyloidosis (ATTR), and alpha-1 antitrypsin deficiency (AATD). Their lead therapies, like NTLA-2002 for HAE and NTLA-2001 for ATTR, aim to offer curative, single-dose treatments, potentially disrupting multi-billion dollar markets. The company is also exploring applications in immuno-oncology and autoimmune diseases.
In recent developments, Intellia Therapeutics, Inc. (NASDAQ:NTLA) is progressing with Phase 3 studies for HAE and ATTR, with plans to complete enrollment in the HAELO study by 2025. Positive results from its Phase 1 trial of Nex-z for ATTR show promising reductions in transthyretin protein, which could slow disease progression.
Financially, the company reported $861.7 million in cash as of December 31, 2024, and is expected to fund operations through 2027. Collaboration revenue surged to $12.9 million, signaling strong progress in its partnerships. However, the corporation continues to invest heavily in R&D, with R&D expenses rising to $116.9 million. Despite these investments, Intellia Therapeutics, Inc. (NASDAQ:NTLA) anticipates a 5% to 10% reduction in operating expenses in 2025 due to a restructuring plan.
5. Sanofi (NASDAQ:SNY)
Number of Hedge Fund Holders: 33
Sanofi (NASDAQ:SNY) is a global healthcare company that develops and markets a wide range of pharmaceutical products, vaccines, and consumer healthcare items. Its portfolio includes treatments for diseases like cancer, multiple sclerosis, diabetes, and rare diseases, as well as vaccines for bacterial and viral infections. The company generates revenue through sales to healthcare providers, pharmacies, and consumers. The company is also expanding its gene-editing and gene therapy capabilities by partnering with innovative biotech firms, positioning itself as a key player in the gene-editing market.
Sanofi (NASDAQ:SNY) strengthened its gene-editing capabilities through an expanded partnership with Scribe Therapeutics in January 2025, focusing on in vivo genetic medicines for sickle cell disease (SCD) and other genomic disorders. The collaboration includes the company’s exclusive use of Scribe’s CRISPR X-Editing (XE) technology for in vivo therapies. The initial focus is on developing a streamlined SCD treatment using CRISPR XE technology combined with the company’s non-viral delivery systems, which could improve patient access and reduce complications compared to ex vivo treatments. Success in SCD could lead to broader applications for other genomic diseases, making Sanofi one of the best gene editing stocks in the field.
Sanofi (NASDAQ:SNY) reported €41.1 billion in total revenues for FY 2024, a rise of 11.3% at constant exchange rates (CER). In its first full year, Dupixent made almost €13 billion, while Beyfortus, a new RSV vaccine for infants, made €1.7 billion and became a blockbuster. Despite a 1.8% drop in reported terms, SNY’s EPS increased 4.1% at CER to €7.12, beyond expectations. The company also suggested paying out €3.92 per share in dividends in 2024, which it has grown for 30 consecutive years.
4. Beam Therapeutics Inc. (NASDAQ:BEAM)
Number of Hedge Fund Holders: 35
Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology company focused on precision genetic medicines using base editing, a technology that allows for precise, single-letter changes to DNA without cutting the strands. The company aims to develop treatments for genetic disorders, hematological diseases, and cancers. Its unique base editing technology offers a more accurate, predictable approach compared to traditional CRISPR, potentially reducing risks associated with DNA breaks.
Beam Therapeutics Inc. (NASDAQ:BEAM) recently reached a key milestone by achieving its adult enrollment target in the BEACON Phase 1/2 clinical trial of BEAM-101 for sickle cell disease. The company also presented promising early results from this trial at the 66th American Society of Hematology Annual Meeting. Additionally, the corporation plans to release initial data from its Phase 1/2 study of BEAM-302 for Alpha-1 Antitrypsin Deficiency (AATD) in the first half of 2025, solidifying its position among the best gene editing stocks in the biotech sector.
Beam Therapeutics Inc. (NASDAQ:BEAM) ended 2024 with a solid cash position of $850.7 million, supporting ongoing research and trials. R&D expenses were $101.4 million for the fourth quarter, reflecting a decrease from the previous year, which may indicate greater efficiency in its operations. General and administrative expenses were $28.7 million for Q4. However, the business reported a net loss of $90.4 million for the quarter and $376.7 million for the year, which is typical for biotech companies in the clinical stage.
The company’s revenue from licenses and collaborations significantly dropped from $316.2 million in Q4 2023 to $30.1 million in Q4 2024. This decrease warrants further investigation, as it may affect the company’s future financial stability. Despite this, Beam Therapeutics Inc. (NASDAQ:BEAM) expects its current cash reserves will fund operations into 2027, giving the company ample time to advance its key programs.
3. Moderna, Inc. (NASDAQ:MRNA)
Number of Hedge Fund Holders: 44
The third stock on our list of the best gene-editing stocks to buy is Moderna, Inc. (NASDAQ:MRNA). It is a biotechnology company focused on developing mRNA-based therapeutics and vaccines for a variety of diseases, including COVID-19, cancer, and cardiovascular conditions. What sets the company apart in the gene-editing field is its innovative use of mRNA technology combined with gene-editing tools for in vivo therapies, which directly alter genes within a patient’s body. This approach aims to overcome challenges faced by traditional gene therapies. The corporation’s rapid development of the COVID-19 vaccine highlights its ability to quickly create mRNA-based treatments, positioning the company as a leader in addressing emerging health threats and unmet medical needs.
Moderna, Inc. (NASDAQ:MRNA)’s target market spans a wide range of areas, including infectious diseases, cancer, and rare genetic disorders. After the success of its COVID-19 vaccine, Spikevax, the company is expanding its pipeline to address other critical areas, such as respiratory vaccines for COVID-19, influenza, and RSV, as well as vaccines for rare diseases like CMV and EBV. The corporation is also working on cancer immunotherapies, particularly for melanoma.
For 2024, Moderna, Inc. (NASDAQ:MRNA) recorded a revenue of $3.2 billion, a 53% decrease from the previous year, mainly due to lower sales of Spikevax. The company reported a net loss of $3.6 billion for the year, an improvement from the $4.7 billion loss in 2023. This drop in revenue was expected, as the demand for COVID-19 vaccines has slowed. However, the business’s efforts to cut costs—by reducing operating expenses and optimizing operations—helped mitigate the impact on its profitability.
Moderna, Inc. (NASDAQ:MRNA) ended the year with $9.5 billion in cash, up slightly from the previous quarter. Despite the revenue decline, the company’s cost-saving measures, reduced R&D expenses, and a focus on strategic investments in its mRNA-based pipeline position it for continued growth beyond the pandemic era.
2. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)
Number of Hedge Fund Holders: 68
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company that develops transformative medicines for serious diseases, with a strong focus on gene editing, particularly in collaboration with CRISPR Therapeutics. The company specializes in treatments for genetic diseases, especially cystic fibrosis, and generates revenue by selling medications to patients with rare genetic conditions. Recently, the company has expanded its focus to include sickle cell disease and beta-thalassemia.
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has partnered with CRISPR Therapeutics to develop CASGEVY (exagamglogene autotemcel), a gene-edited cell therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). This treatment involves editing the BCL11A gene in a patient’s stem cells to increase the production of fetal hemoglobin, which helps manage these inherited blood disorders.
The company’s Q4 2024 financial results highlight continued growth, with a 16% increase in revenue from the same period in 2023, reaching $2.91 billion. Full-year 2024 revenue reached $11 billion, exceeding expectations. The growth was driven by strong sales of CF treatments and the launch of CASGEVY. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is also expanding its reach with new therapies like ALYFTREK for CF and JOURNAVX for acute pain, solidifying its place among the best gene editing stocks.
With a broad total addressable market (TAM) covering diseases like CF, SCD, TDT, acute pain, type 1 diabetes, and renal diseases, the company aims to treat millions of patients worldwide. The business is focused on growing its market presence and advancing its innovative therapies.
1. Eli Lilly and Company (NYSE:LLY)
Number of Hedge Fund Holders: 115
Eli Lilly and Company (NYSE:LLY) tops the list of the best gene editing stocks. It is a global pharmaceutical leader focused on developing and selling medications for various conditions, including diabetes, obesity, oncology, and neuroscience. With a strong emphasis on scientific innovation, the company is at the forefront of gene editing and biotechnology, positioning itself as a key player in these fields.
Eli Lilly and Company (NYSE:LLY) is making significant advancements in genetic medicine through initiatives like the Lilly Seaport Innovation Center in Boston, which fosters collaboration on gene-based therapies. The company is also expanding its capabilities through acquisitions, such as the purchase of Morphic Therapeutics, and partnerships, like the one with OpenAI to develop novel antimicrobials.
In its Q4 2024 earnings call, the company reported impressive growth. Revenue increased by 45%, which was driven by strong demand for new products like Mounjaro and Zepbound, contributing over $3.1 billion to the growth. The company also saw solid performance across the oncology, immunology, and neuroscience segments. Key regulatory approvals were achieved, and positive Phase 3 trial results for several drugs were announced.
Eli Lilly and Company (NYSE:LLY) continued investing in manufacturing, committing over $23 billion since 2020 to expand its capacity. It also returned $3 billion to shareholders through dividends and repurchases and introduced a $15 billion share repurchase program. The company’s successful international expansion, particularly in Europe, Japan, and China, contributed to its growth.
Overall, Eli Lilly and Company (NYSE:LLY) ranks first among the 8 gene-editing stocks to buy. While we acknowledge the potential of gene-editing companies, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than LLY but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
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