CRISPR technology has the potential to revolutionize the way we approach medicine. This technology has already been used in a variety of applications, including gene editing, gene therapy, and disease diagnosis. As a result, companies developing CRISPR-based products and services have seen significant valuation growth since inception, with many CRISPR stocks having excellent long-term upside potential in this regard.
Several companies at the forefront of CRISPR technology have seen their stocks rise dramatically since going public. This can be attributed to the potential for CRISPR-based therapies to treat a wide range of genetic disorders and diseases, including cancer, sickle cell anemia, and cystic fibrosis. This technology also has potential applications for editing genes in animals and plants, and therefore has significant implications for the agriculture and food industries.
The CRISPR stocks below are among those I believe have the best long-term potential.
CRISPR Therapeutics (CRSP)
CRISPR Therapeutics (NASDAQ:CRSP) may be the first gene editing stock that comes to mind when discussing the sector. The company was not the first to successfully create gene editing technology and medicine. However, it is among the first to be commercially successful using the technology.
That success has led to dramatic price increases for CRSP stock since going public in 2016. Shares flirted with the $200 level in 2021 during the most recent market peak. They currently trade for around $50. That may either excite investors, or scare them away. However, the company’s pipeline of therapies addresses sickle cell anemia, beta-thalassemia, and other blood disorders. These diseases affect the function of the hemoglobin that carries oxygen in the blood.
CRISPR Therapeutics is also developing gene-editing therapies for solid tumors.
Investors must remain aware that while the stock has significant potential, the company continues to lose money. It reported a mere $94,000 in revenue in the most recent quarter, leading to a net loss of more than $175 million. Yet, at the same time, it maintained around $1.973 billion in liquidity to end the September quarter.
Editas Medicine (EDIT)
Editas Medicine (NASDAQ:EDIT) is another leading name among CRISPR stocks, although it trades below $10, squarely in the realm of penny stock territory. Like CRISPR Therapeutics, Editas Medicine is using gene editing technology to develop therapeutics for beta-thalassemia and sickle cell disease. The firm’s lead candidate products also include therapies against diseases that affect vision and can lead to blindness.
Like CRISPR Therapeutics, Editas Medicine’s financial picture includes substantial liquidity, but also low revenues and heavy losses. This is due to the intensive research & development investment required of biotech companies. Editas Medicine reported $478.5 million in liquidity to end the third quarter. It also recorded a net loss of $55.7 million, $41.3 million of which was attributable to R&D expenses.
But if the company is successful in commercializing its pipeline, then those losses will no longer matter. For now, the company remains in the early clinical stages of development with its key therapies, meaning this is still one of the CRISPR stocks that will have to move through late clinical stages before commercializing its technology.
Intellia Therapeutics (NTLA)
Intellia Therapeutics (NASDAQ:NTLA) stock represents a company utilizing CRISPR/Cas9, a powerful tool for precisely and efficiently editing genetic sequences. The company is using this technology to develop therapies for a variety of genetic diseases, including those that affect metabolism, liver diseases, and blood disorders.
Intellia Therapeutics’ commercial platform focuses on both in-vivo (taking place in a living body), and ex-vivo (those taking place outside of the body) therapies. Its in-vivo therapies focus on knocking out defective malfunctioning genes that cause disease, inserting functional genes to achieve specific outcomes, or both. Another way to say this is that CRISPR is the therapy for its in-vivo platform, whereas CRISPR creates the therapies for its ex-vivo platform.
The company has partnered with leading pharmaceutical firms including Novartis (NYSE:NVS) and Regeneron (NASDAQ:REGN). Intellia’s leading candidate therapy targets the treatment of transthyretin amyloidosis (ATTR), a rare genetic disease that leads to the buildup of amyloid protein in the heart and other organs.
Verve Therapeutics (VERV)
Verve Therapeutics (NASDAQ:VERV) is a biotech stock focused on developing gene therapies for cardiovascular disease. The company went public via a SPAC merger in late 2020, near the peak of SPAC-funded startups going public. The company is still in the clinical stage, and any news related to clinical trial results and FDA approvals will likely have a major impact on its stock price.
Verve Therapeutics is focused on editing genes related to low-density lipoprotein, also known as LDL, or bad cholesterol. The company is targeting its application in reducing dangerously high levels of cholesterol in a heriditary disease called familial hypercholesterolemia. The disease causes a genetic mutation leading to a buildup of cholesterol that causes advanced cardiovascular disease in a wide range of patients, even youth. Verve Therpeutics’ lead candidate, VERVE-101, is an in-vivo liver gene editing treatment. It targets the PCSK9 gene, interrupting PCSK9 protein production, thereby lowering LDL production.
The company maintained a cash position of $550.7 million to end Q3 while reporting $929,000 in revenues in the period. Clinical data from VERVE-101 1b trials are expected in the middle of this year.
Cellectis (NASDAQ:CLLS) is a biotech company based in France that specializes in gene editing technologies. The company has partnered with other biotech companies and pharmaceutical firms including Pfizer (NYSE:PFE). The company’s main commercial focus is on developing therapies using its gene editing technology, TALEN, for cancer and immune-related disorders.
Cellectis has been utilizing gene editing technology for 23 years, and specifically focuses TALEN on for electroporation. Electroporation is a process by which DNA is introduced into cells using a brief pulse of electricity.
That said, Cellectis has still done relatively little in terms of advancing commercial products through clinical trials. On Sept. 30, 2022, the company was undertaking 4 clinical trials, through which a relatively modest 40+ patients had been dosed with its products.
The company’s UCART platform is arguably its greatest asset. It utilizes CAR-T cells derived from healthy donors and can be used to treat multiple patients. Those cells can be used to create off-the-shelf therapies which eliminate the need for the collection of the patient’s own cells, thus potentially lowering overall costs significantly.
Sangamo Therapeutics (SGMO)
Sangamo Therapeutics (NASDAQ:SGMO) is a biotechnology company specializing in gene editing and gene therapy. It uses its proprietary zinc finger nuclease technology to edit the DNA of cells to treat genetic diseases and cancer. The company’s lead product candidate, SB-525, is relatively far along in the clinical trial process for Hemophilia A.
Sangamo Therapeutics’ Hemophilia A program is being co-developed with Pfizer, and is currently in Phase 3 clinical trials. The company also boasts significant programs in renal transplants, sickle cell disease, and Fabry disease in Phase 1/2 clinical trials.
The company’s pre-clinical pipeline has attracted significant partners including Takeda (NYSE:TAK), Novartis, Pfizer, and Biogen (NASDAQ:BIIB).
In the first 9 months of 2022, Sangamo Therapeutics recorded $84.07 million in revenues leading to a loss of $140.3 million. That said, the company had $350.26 million in cash at that time, and is rapidly nearing commercialization of its hemophilia A program and other therapeutics.
Beam Therapeutics (BEAM)
Last on this list of CRISPR stocks to buy is Beam Therapeutics (NASDAQ:BEAM), a company providing cutting-edge solutions that have caught investors’ attention. The company is utilizing base editing technology to correct genetic mutations in a precise and efficient manner, which has the potential to revolutionize the way genetic diseases are treated.
Beam’s team includes experts in the field of genome editing, and the company has notable partnerships with leading research institutions.
In addition, the company’s base editing technology allows for a more targeted approach than traditional gene editing methods, potentially reducing unintended side effects. This could be a game-changer in the field of gene therapy.
Like so many other companies on this list, Beam Therapeutics is developing therapies for the treatment of sickle cell disease, enrolling its first patient in a trial for patients with severe sickle cell disease in November.
The company posted a net loss that approached $223 million through the first 9 months of 2022. However, it maintained more than $1.1 billion in cash equivalents at that time. Additionally, its sickle cell program, which also utilizes electroporation, has a chance for commercialization in the near future. Like Cellectis, Beam Therapeutics is developing off-the-shelf cancer therapeutics that could eliminate the need to collect the patients’ own cells, lowering costs.
On the date of publication, Alex Sirois did not have (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.