You've probably heard about the tremendous potential for gene editing. The technology could change the world of medicine by enabling cures to be developed for a wide range of genetic diseases. But you've probably also heard about the significant risks associated with gene editing.
Because of these risks, investing in gene-editing stocks like CRISPR Therapeutics (NASDAQ:CRSP) and Editas Medicine (NASDAQ:EDIT) isn't for the faint of heart. There is a way, though, for investors to potentially profit from gene editing without taking on nearly as much risk. How? By buying the stocks of the small biotechs' major partners. In particular, investors might want to consider Allergan (NYSE:AGN), Celgene (NASDAQ:CELG), and Vertex Pharmaceuticals (NASDAQ:VRTX).
Allergan entered into a strategic alliance with Editas Medicine in 2017 to develop CRISPR-based gene-editing therapies targeting eye diseases. Earlier this month, Allergan exercised its option to develop and market Editas' lead candidate EDIT-101 for the treatment of Leber congenital amaurosis type 10 (LCA10), the most common cause of hereditary childhood blindness.
Editas plans to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for EDIT-101 in October. The biotech recently announced that the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC) has completed the NIH protocol registration process for EDIT-101. A public meeting of the committee won't be required for approval of clinical testing of EDIT-101 in humans.
Focusing on gene-editing therapies for eye diseases is a good fit for Allergan. The big drugmaker currently markets Restasis for treating chronic dry eye disease and Lumigan for treating glaucoma. Allergan also has several late-stage eye-disease drugs in its pipeline, including abicipar.
With more than $16 billion in revenue generated over the last 12 months, Allergan is a much safer pick than its partner, Editas. My colleague Todd Campbell recently picked Allergan as his top stock to buy because of its attractive valuation and potential for a turnaround. I agree with Todd and view Allergan as a pretty good long-term investment.
Celgene offers a way for investors to benefit from gene-editing advances made by both CRISPR Therapeutics and Editas Medicine. The big biotech was one of the earliest investors in CRISPR Therapeutics and still owns 10% of the company. But while Celgene hasn't teamed up on development with CRISPR Therapeutics, it is working closely with Editas.
Editas and Juno Therapeutics began collaborating in 2015 on using CRISPR-based gene editing to engineer T cells to fight cancer. The two biotechs hope to improve the efficacy and safety of chimeric antigen receptor T cell (CAR-T) and T cell receptors (TCR) in treating several types of tumors. Celgene acquired Juno earlier this year and has continued and even expanded Juno's close relationship with Editas.
Celgene could see a quicker return from its stake in CRISPR Therapeutics, though. All of the research with Editas is still in preclinical studies with no timeline for advancing to clinical trials in humans. CRISPR Therapeutics, on the other hand, hopes to initiate phase 1 clinical studies by early next year for gene-editing therapies targeting rare blood disorders beta-thalassemia and sickle cell disease.
Some might question whether or not Celgene is actually a safer pick than CRISPR Therapeutics or Editas since its stock has underperformed both smaller biotechs so far in 2018. Celgene remains highly dependent on one drug, Revlimid, for its revenue. I think, though, that Celgene is in great shape to defend Revlimid and generate solid earnings growth for years to come with its strong pipeline, which market research firm EvaluatePharma ranks as the third-best in the biopharmaceutical industry.
Another way to profit from CRISPR Therapeutics' gene-editing advances is to buy shares of Vertex. The two biotechs teamed up to develop and commercialize CRISPR Therapeutics' lead candidate CTX001 in treating beta thalassemia and sickle cell disease.
There is one catch -- for now, at least. On May 30, 2018, the FDA placed a clinical hold on initiation of a phase 1 study evaluating CTX001 in treating sickle cell disease. CRISPR Therapeutics and Vertex are working with the FDA to answer all questions. Samarth Kulkarni, CEO of CRISPR Therapeutics, recently stated that the companies "have a clear path" to move past the clinical hold.
I think that the U.S. study of CTX001 in sickle cell disease and the European study of the therapy in beta thalassemia will soon move forward. While there's a long way to go, I'm also cautiously optimistic that Vertex and CRISPR Therapeutics could have a big winner on their hands with CTX001.
In the meantime, Vertex already has multiple winners in its lineup. The biotech essentially enjoys a monopoly in treating cystic fibrosis (CF) with three approved drugs on the market. Vertex also has late-stage studies underway for triple-drug CF combos that should turbocharge its revenue growth.
An important adverb
There's no such thing as a completely safe stock. That's especially true with biotech and pharma stocks. Even big drugmakers can experience huge share drops due to regulatory and pipeline setbacks.
My use of the adverb "relatively" in describing Allergan, Celgene, and Vertex as safer investment choices is important to note. They are relatively safer than Editas Medicine and CRISPR Therapeutics because they have approved products generating solid revenue. But they're certainly not completely safe picks.
Investing is all about taking on risk in the hopes of making an acceptable return on investment. I think that many moderately aggressive investors will find the risk-reward propositions of Allergan, Celgene, and Vertex to their liking. And really aggressive investors might like these big drugmakers' partners, CRISPR Therapeutics and Editas, as well.