When you buy through affiliate links in our content, we may earn a commission at no extra cost to you. Learn The best gene-editing stocks are having a moment this month with a Food and Drug Administration (FDA) advisory committee reviewing exa-cel, a gene-editing therapy by Vertex Pharmaceuticals and CRISPR Therapeutics. Exa-cel is being looked at as a potential cure for two genetic blood disorders – transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). The FDA is expected to issue a final ruling on the therapy to treat SCD by December.
With the advisory committee not raising any particular red flags, exa-cel could be the first CRISPR (clustered regularly interspaced short palindromic repeats) gene-editing therapy to be approved. In response to the meeting, gene-editing stocks saw double-digit increases.
Gene-editing is showing a great deal of promise to treat the root causes of disease with single-dose therapies, using genomic data, that could reduce the long-term costs for certain diseases. The Institute for Clinical and Economic Review (ICER) says the therapy could be worth as much as $1.35 million to $2 million per treatment because it would end the need for long-term therapies for SCD or TDT.
This guide presents the 10 best gene-editing stocks for solid long-term gains, looking at which companies stand to benefit the most from growth in the sector. Let’s take a deeper look at the case for investing in the above gene-editing stocks. Read on to find the best dividend stocks this month. The FDA advisory meeting regarding exa-cel spurred the recent push for Crispr Therapeutics shares as the comments regarding the therapy were generally positive. While SCD is rare, it affects 100,000 people in the U.S. each year, according to the Centers of Disease Control and Prevention, and each dose of exa-cel could be priced as high as $1 million. That’s still a pretty good-sized target group and Crispr is slated to get 40% of the profits from exa-cel, with Vertex receiving the rest. On top of that, exa-cel has a solid chance of being approved to treat another genetic blood disorder, TDT. Crispr’s pipeline includes 22 programs spread across four classifications: hemoglobinopathies, immuno-oncology, regenerative medicine, and in vivo approaches. The company, if exa-cel is approved by the FDA, gains a first-to-market advantage, both in profits to drive more development and in reputation among investors.
The clinical-stage gene-editing company’s lead therapy, NTLA-201, has huge potential because its target disease, transthyretin (ATTR) amyloidosis, affects between 200,000 and 500,000 people worldwide. The company sees the market for ATTR therapies to be worth $11 billion by 2026. The company recently got approval from the FDA to enter the therapy in a phase 3 trial. It’s the first such test of a gene-editing therapy that has the potential to cure or treat a hereditary condition by changing patients’ genomes. It is expected the trial will begin by the end of this year. Intellia has more than 10 programs in its pipeline to treat diseases with a high unmet need and it uses CRISPR editing to create therapies. In the first week of November, the stock rose more than 24% but it is down more than 17% so far this year. The company, as of the second quarter, had $1.1 billion in cash. Its only revenue is from collaborations, and it reported $13.6 million in revenue in the quarter, down from $14 million in the same period a year ago.
Vertex has a big advantage over most gene-editing companies because it is already profitable, thanks to its domination of cystic fibrosis therapies. In the third quarter, the company reported revenue of $2.48 billion, up 6%, year over year, thanks to strong sales from CF therapy Trikafta. Vertex also reported net income of $1.035 billion, up 22% over the same period last year. The company also has huge cash reserves of $13.8 billion, giving it plenty of money to develop exa-cel as well as the other 14 programs in its pipeline. The company raised its 2023 CF revenue prediction from an earlier range of between $9.7 billion to $9.8 billion to $9.85 billion.
Beam’s CRISPR base editing, which is considered to be more precise, with fewer off-target results, could set it apart in the industry. The company just extended its cash position into 2026 with a deal with Eli Lilly (NYSE: LLY) that gives Lilly the commercial rights to the base editing program to treat cardiovascular disease developed by Verve Therapeutics (NASDAQ: VERV). In return, Beam receives a $200 million upfront payment, a $50 million equity investment and is eligible for an additional $350 million in development-stage payments. The company has seven programs in its pipeline, led by BEAM-101 to treat SCD and TBD, as well as BEAM-201 to treat aggressive CD7+ leukemias and BEAM-302 as a one-time treatment to end AATD, with the potential for the cell therapy to treat lung and liver disease. The company’s base editing has attracted collaborations with Pfizer, Apellis Pharmaceuticals (NASDAQ: APLS), Sana Biotechnology (NASDAQ: SANA), Verve, Prime Medicine (NASDAQ: PRME) and Orbital Therapeutics.
The company’s shares are up more than 18% so far this month, but down more than 49% for the year. The impetus for the recent rise was the company’s third-quarter earnings, which showed increased sales for Zynteglo and Skysona, with 22 patient starts across both programs. In the quarter, the company reported revenue of $12.4 million, compared with only $100,000 in the same period last year. The company also narrowed its net losses, albeit barely, losing $71.7 million in the quarter compared to a loss of $76.5 million in the third quarter of 2022. The biggest concern for Bluebird is its cash position. It reported it had $227 million in cash. Bluebird is looking to possibly sell a Rare Pediatric Disease Priority Review Voucher (PRV), if granted by the FDA, for $103 million to improve its cash situation. It works by placing a functional beta-globin gene into a patient’s own stem cells, allowing them to produce healthy hemoglobin. If approved, lovo-cel would change Bluebird’s financial situation significantly. Visible Alpha says there’s a good possibility the therapy could generate $520 million in revenue by 2030.
The company is poised to benefit regardless of which gene-editing biotechs fare well. Its connection to gene-editing is only part of its business, with the company having groups for Life Sciences & Applied Markets, Diagnostics and Genomics, and Agilent CrossLab. In the third quarter, Agilent, citing lower business in China, saw revenue fall, but its profitability improved. It reported revenue of $1.67 billion, down 2.7%, year over year. Operating margin was listed as 29.3%, up 180 basis points compared to the same period last year. Earnings per share grew 7%, year over year, to $1.43.
The company uses CRISPR editing to produce allogeneic “off-the-shelf” CAR-T cell therapies, using healthy donor cells to target cancer cells. The company focuses on blood-related cancers by armoring the body to potentially improve antitumor activity. Its lead program is CB-010, which is in a phase 1 trial to treat relapsed or refractory B cell non-Hodgkin lymphoma. Two other therapies in phase 1 trials in the pipeline include CB-011 to treat relapsed or refractory multiple myeloma and CB-012 to treat refractory acute myeloid leukemia. The company’s main collaboration agreement is with AbbVie (NYSE: ABBV), but Pfizer has also recently become one of its main backers, making a $25 million equity investment in Caribou. The stock trades for less than $5 and its low cash position is a concern. However, considering the early success of its therapies, there’s a huge potential upside for the stock.
Regeneron is dipping more than its toes into gene editing. The pharmaceutical company’s financial strength gives it the means to see potential gene-editing therapies through to the finish line, and the company already has a sizable marketing staff. In the third quarter, Regeneron reported revenue of $3.36 billion, up 15%, year over year and EPS of $8.89, down 24% over the same period last year, but that includes a one-time unfavorable impact of about $0.77 in EPS from an acquired in-process research and development charge. The drug is used to treat asthma, eczema, and some other inflammatory conditions. Overall sales from the drug were up 33% to $3.1 billion. The company’s fastest-growing drug, though, was immunotherapy treatment Libtayo, which saw revenue increase 62% to $232 million. The Regeneron Genetics Center is one of the largest genetics sequencing efforts in the world and the company just increased its CRISPR gene-editing collaboration with Intellia and the collaboration has already produced prospective therapy NTLA-2001 to treat ATTR. Regeneron, in September, acquired Decibel Therapeutics, gaining the promising gene therapy DB-OTO, which is designed to cure hearing loss caused by mutations of the otoferlin gene.
Like Regeneron, Gilead is a large-cap pharmaceutical company that is seeking collaborations to make sure it isn’t left out of the gene-editing therapies game. Last month, Gilead company Kite entered into a research collaboration with EpiccrispR Biotechnologies that will use Epic Bio’s gene regulation platform to find next-generation cancer cell therapies. Kite will use the platform as a way to change certain genes to enhance CAR T-cell functions. Gilead already has a huge portfolio, including 61 programs in clinical trials. Gilead has eight cell therapies in clinical trials. The possibility of better engineering the company’s growing oncology portfolio through gene-editing is what drove the Epic Bio deal. One advantage to Gilead is it offers a quarterly dividend that it has raised for eight consecutive years, which has a yield of around 3.86%, more than double that of the S&P 500 average yield.
The company doesn’t find gene-editing therapies but helps other gene-editing companies through its platform that synthesizes DNA on silicon chips instead of well-plastic plates, the traditional method of synthesizing DNA. This increases the production speed of high-quality DNA. The company also recently launched a RNA sequencing portfolio. Like Agilent, Twist is more of a picks-and-shovels gene-editing stock. The company isn’t profitable but seems to be headed in the right direction. In the third quarter, the company reported revenue of $63.7 million, up 14%, year over year, but it also had a net loss of $57.4 million, a slight improvement over the $60.5 million it lost in the same quarter a year ago. Since the company released its third-quarter report on Aug. 4, the company signed an antibody discovery and licensing option agreement with Bayer, a collaboration agreement to find antibodies to fight immune-mediated inflammatory diseases with privately held biotech company IMIDomics and a drug discovery agreement with Ono Pharmaceutical (OTC: OPHLY).
Gene-editing stocks are companies that are developing or using gene-editing technology, which has the potential to change medicine by correcting genetic defects, treat diseases that are caused by genetic disorders and create new therapies. These therapies work by modifying genes in living organisms. There are pure-play gene-editing companies that are directly making therapies to correct genetic diseases, and there are other companies who help make materials used by gene-editing companies. There are also larger pharmaceutical companies that are beginning to, particularly through collaborations, develop a connection to gene-editing technology. According to a report by Grand View Research, the gene-editing market, listed as a $6.39 billion market in 2022, is expected to have a compound annual growth rate (CAGR) of 17.8 from 2023 to 2030. The rise is expected to be aided by government funding and an increasing emphasis from companies on treating and potentially curing genetic disorders. The relatively new science has huge growth potential to not only treat diseases but cure them. That kind of revolutionary medicine has the potential to drive revenue and share growth. Investing in gene-editing stocks presents another opportunity for diversification, especially in portfolios that are heavily weighted toward traditional pharmaceutical companies. The gene-editing therapies will be more difficult to copy than small molecule drugs and as such, present a greater moat for gene-editing companies. These companies also share the support of several governments, which could help speed gene-editing therapies to market. CRISPR/Cas9 is the most widely used gene-editing technique today because it can efficiently make precise changes to DNA, rewriting the code of a DNA sequence. Essentially, CRISPR/Cas 9 is an enzyme that can be used like scissors to cut DNA, allowing the cell to repair the break by inserting, deleting, or replacing nucleotides. Other common gene-editing techniques include TALENs (transcription activator-like effector nucleases), base editing, prime editing, and zinc finger nucleases (ZFNs). Beyond the types of edits, some gene-editing processes are done in vivo and some are done ex vivo. The difference is ex vivo gene therapies involve removing cells from the body, modifying them, and placing them back into the body. With in vivo gene therapy, new genes are inserted directly into the body. It’s a very new field with very few companies that are profitable, so there’s plenty of risk, but there’s a big opportunity for growth as well. To choose the best gene-editing companies, look for those with large pipelines. Another strong sign is a number of collaborative agreements with larger companies, because that speaks well of a particular gene-editing company’s expertise. The barriers to approval for gene-editing therapies are high, so it’s also important to look at gene-editing companies with either a strong cash position, or those who are already growing revenue, thanks to increased sales or collaboration revenue growth. There are gene-editing ETFs that can take the guesswork out of investing in gene-editing companies and can add balanced gene-editing exposure to your portfolio. With most gene-editing stocks down this year, there are only a handful of gene-editing ETFs whose total returns year to date are down less than 12%. Some of the more successful this year include the ARK Genomic ETF (NYSEMKT: ARKG), the Goldman Sachs Future Health Care Equity ETF (NYSEMKT: GDOC) and Franklin Genomic Investments ETF (NYSEMKT: HELX). We’ve established that finding the best gene-editing stocks to invest in is a difficult task. The process requires significant research and analysis, considering the number of companies that operate in this market. To streamline the process, consider using AltIndex – which is one of the best alternative data providers. Read on to find out how AltIndex can help you become a successful gene-editing investor in 2023. Investors can use AltIndex, a subscription service that uses artificial intelligence (AI) and alternative data to help find gene-editing stocks capable of outperforming the market. AltIndex uses a variety of metrics that can be particularly useful in buying, or selling a gene-editing stock at the right time. Once you’ve found several gene-editing stocks you are interested in, the AltIndex Stock Screener feature can help monitor the performance of the stocks on a watchlist. The watchlist can help you follow the stocks’ performance, including alerts when AltIndex has new information regarding a stock, such as when a company announces positive clinical trial results or if it receives regulatory approval for a new therapy. Another use of AltIndex is the company’s price predictions tool to decide on a stock. The service uses AI-generation price predictions that could, say, predict when a gene-editing stock is overpriced, so that you may want to wait before buying the stock. Gene-editing stocks are quite volatile because they are dependent on a new class of therapies and gene-editing stocks can rise or fall for reasons that have little to do with their current earnings. This is where AltIndex’s various social-media metrics can prove helpful. The company tracks billions of data points across news sites, web traffic, job postings and social media channels to find good investment opportunities, then assigns each stock a score of 0 to 100 based on market sentiment and other factors. Stocks with higher scores are considered better buys. AltIndex’s starter plan costs $29 per month and that comes with 10 AI stock picks every month. To use the services’ more advanced features, the pro plan costs $99 a month and provides 25 AI stock picks every month. Investing in gene-editing stocks is best done with a long-term approach as the science is still in the early stages. There’s little doubt that the ceiling for these stocks is quite high because they are helping to revolutionize medicine, in some cases potentially curing genetic disorders. The growing knowledge about the role of genes in various diseases, the increased demand for personalized medicine and increased investment in gene-editing research and development will drive the growth of gene-editing medicines. However, there are plenty of risks because the science is so new and there are plenty of hurdles, both scientifically and in regards to regulatory agencies that could slow the progress of individual companies. As in any other investment, the companies that will survive have solid financial backing to go along with gene-editing expertise. This isn’t a zero-sum game, though, as there will be several winners as more gene-editing therapies are developed. The success that exa-cel is expected to have with the FDA is just the beginning for the industry. 10 Best gene-editing Stocks to Invest in 2024
A Closer Look at the Top Gene-Editing Stocks to Buy
1. Crispr Therapeutics: Best Gene-Editing Stock to Buy Now
2. Intellia Therapeutics: Best Underrated Gene-Editing Stock
3. Vertex Pharmaceuticals: Best Gene-Editing Stock for Security
4. Beam Therapeutics: Base-Editing Sets it Apart
5. Bluebird Bio: Lovo-Cel Could Change Company’s Future
6. Agilent Technologies: Solid Stock for Pick-and-Shovel Gene-Editing Investing
7. Caribou Biosciences: High-Risk, High-Reward Stock
8. Regeneron Pharmaceuticals: Balance Gives it Dependable Growth
9. Gilead: Company is Branching Out Beyond Antivirals
10. Twist Biosciences: Risky Stock with Long-Term Potential
What are Gene-Editing Stocks?
Why Invest in Gene-Editing Stocks?
What Are the Types of Gene-Editing?
How to Choose the Best Gene-Editing Stocks
Are There Gene-Editing ETFs?
Where to Get Gene-Editing Stock Tips
Conclusion
References
FAQs
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