Gene-editing tech like CRISPR is the key to unlocking medical innovations.
In the last few years, cutting-edge science has advanced the field of genetics further than many ever thought possible. Mapping the human genome was the first step in unraveling the secrets of genetics, and today the pinnacle of that research is what's known as CRISPR technology. CRISPR is short for clustered regularly interspaced short palindromic repeats, a fancy way of describing very precise genetic engineering that may be the key to curing a variety of genetic disorders. As biotech companies have come to recognize its potential, funding for CRISPR tech has increased exponentially since it was first developed, and the industry has grown considerably more crowded. This gives investors plenty of companies to choose from, but which CRISPR stocks are the best investments today? Here are six top gene-editing stocks leading the pack.
Crispr Therapeutics (ticker: CRSP)
If investors want to put their money into a company focused on the CRISPR industry, then the first company they should look at is Crispr Therapeutics. Founded by Emmanuelle Charpentier, one of the co-discoverers of CRISPR technology and co-recipient of the 2020 Nobel Prize in Chemistry, Crispr Therapeutics has a slew of therapies in the works. The company's main focus at the moment is CTX001, which treats patients with sickle cell disease and transfusion-dependent beta-thalassemia. Another candidate in its pipeline is CTX110, a treatment for patients with relapsed or refractory non-Hodgkin lymphoma, which just enjoyed positive early results in its Phase 1 trial in late October. These are just two candidates among many that Crispr is working on, and any one of them could be incredibly profitable for the biotech company -- and its shareholders.
Editas Medicine (EDIT)
Gene editing can take two forms. The first is in vivo, in which genes are edited while still inside of a patient's body. The second is ex vivo, in which genes are edited and then implanted within a patient's body. Editas Medicine does both, giving the company a wide range of treatments in its pipeline that are focused on three areas: blood diseases, cancers and ocular diseases. In addition, one treatment -- EDIT-101 -- has entered its Phase 1/2 trial for the treatment of Leber congenital amaurosis 10 (LCA10). This is the first time an in vivo CRISPR treatment has entered a clinical trial, and positive results could instantly make Editas a leader in this new field. In the meantime, Editas completed its equity offering in the second quarter and strengthened its cash position, providing the company with enough funding for operations through 2023. Strong financials and a deep pipeline make Editas one of the best CRISPR companies on the market right now.
Intellia Therapeutics (NTLA)
CRISPR is such new technology that there are still very few experts in the field -- but Intellia Therapeutics co-founder Jennifer Doudna is certainly among them. Along with Emmanuelle Charpentier, Doudna received the 2020 Nobel Prize in Chemistry for the discovery of CRISPR technology back in 2012 -- today, Intellia is utilizing that same technology to create therapies for a number of genetic diseases. At the top of the company's pipeline is NTLA-2001, an in vivo treatment for the genetic disease transthyretin amyloidosis, or ATTR, which began its Phase 1 clinical study in early November. Intellia has two other strong candidates, NTLA-2002 (for the treatment of hereditary angioedema) and NTLA-5001 (for acute myeloid leukemia), on deck for regulatory submission in 2021. An impeccable pedigree and a broad range of therapies make Intellia an excellent CRISPR investment.
Beam Therapeutics (BEAM)
Beam Therapeutics made its public debut in February, and shares quickly sank along with the rest of the market in the following month. You can't keep a good idea down, though, as the team at Beam has some very good ideas for how to utilize CRISPR technology. The company focuses on base editing, a technique in which it changes the very chemical compounds, or letters -- G, A, T, and C -- that make up a strand of DNA. According to the company, "If existing gene editing approaches are 'scissors' for the genome, our base editors are 'pencils,' erasing and rewriting one letter in the gene." Investors have good reason to be excited about this technology, and Beam has a whopping 12 programs in various preclinical stages that have a lot of potential. Shares are up more than 190% since its debut, and with the company's first investigational new drug heading to the U.S. Food and Drug Administration in the second half of next year, the hype is sure to climb even higher.
bluebird bio (BLUE)
Unfortunately for bluebird bio and its investors, while the science behind its products may be sound, the manufacturing capacity is not. In early November, the FDA asked bluebird to prove its commercial manufacturing of a new treatment for sickle cell disease will match the manufacturing process from clinical trials. This was a huge setback for the company, as pandemic-related delays mean it likely won't be able to submit for FDA approval until 2022 -- but investors shouldn't be distracted by short-term problems. In the long run, bluebird bio is positioned for success. The company received priority review from the FDA for its multiple myeloma drug ide-cel, and bluebird will still be able to market its sickle cell disease treatment in Europe under the name Zynteglo as a treatment for transfusion-dependent beta-thalassemia. Bluebird bio shares are down nearly 50% year to date, but all is not lost for this gene therapy company, and investors looking for a value pick in this space may want to take a closer look.
Regeneron Pharmaceuticals (REGN)
While most of these companies are focused solely on gene therapies, Regeneron has its fingers in several different pies. Its dermatitis and asthma drug Dupixent enjoyed an excellent year, while sales of Eylea, which treats a variety of eye diseases, remained steady. Meanwhile, the FDA granted a priority review for Libtayo, which could treat cutaneous squamous cell carcinoma, with a target action date in February of next year. And of course, Regeneron's treatment for the virus, REGN-COV2, enjoyed excellent clinical trial results in the third quarter. All of this amounted to a great year for Regeneron, but the future of the company may lie elsewhere: genetics. Regeneron heads up a massive genomics research effort in which it collaborates with a variety of research institutes and companies, including Intellia. CRISPR technologies fall under this umbrella, and while they aren't as important to Regeneron's bottom line as other treatments at the moment, their potential is staggering.
Six of the top gene-editing stocks to buy now:
-- Crispr Therapeutics (CRSP)
-- Editas Medicine (EDIT)
-- Intellia Therapeutics (NTLA)
-- Beam Therapeutics (BEAM)
-- bluebird bio (BLUE)
-- Regeneron Pharmaceuticals (REGN)