Based on market cap, CRISPR Therapeutics (NASDAQ:CRSP) ranks as the top biotech focused on developing CRISPR gene-editing therapies. It's more than 2 1/2 times the size of Editas Medicine (NASDAQ:EDIT) and nearly four times larger than Intellia Therapeutics (NASDAQ:NTLA).
But based on stock performance so far in 2020, Intellia wins the prize as the hottest CRISPR biotech stock. Its shares have soared more than 40%, thanks in large part to the expansion of its partnership with Regeneron.
While CRISPR Therapeutics and Intellia have captured investors' attention lately, Editas Medicine could now be the CRISPR stock to really watch. There are both near-term and long-term reasons why investors should keep their eyes on this company.
In March, Editas and its partner Allergan announced the dosing of the first patient in a phase 1/2 clinical study evaluating EDIT-101 in treating Leber congenital amaurosis type 10 (LCA10), an inherited form of blindness. Editas CEO Cynthia Collins called it "a truly historic event," as it was the world's first human study of an in vivo (inside the body) CRISPR gene-editing therapy.
Editas' Chief Scientific Officer Charlie Albright stated in the company's Q1 conference call last month that the study "has been cleared to continue based on a review of safety data on the first patient." That's great news, especially considering the pioneering nature of the LCA10 therapy.
I don't necessarily look for this clinical trial to provide a big catalyst for Editas over the next few months, at least not directly. But it could give the biotech an indirect catalyst.
Editas Medicine's experience with EDIT-101 in targeting LCA10 has enabled it to move forward with EDIT-102, a CRISPR therapy targeting another genetic eye disease, Usher syndrome 2A. Allergan is currently reviewing a preclinical data package for the potential licensing of EDIT-102. Editas expects a decision from Allergan on exercising its option for EDIT-102 by the third quarter of 2020.
My hunch is that Allergan will decide to license EDIT-102 unless some safety issue emerges in the phase 1/2 study for EDIT-101. A positive decision would likely cause Editas' shares to jump.
CRISPR Therapeutics is the leader in developing a CRISPR therapy for treating rare blood diseases sickle cell disease and beta-thalassemia. The company and its partner, Vertex Pharmaceuticals, expect to report additional data from two phase 1/2 studies in progress evaluating CRISPR/Cas9 gene-editing therapy CTX001 later this year.
Editas is behind CRISPR Therapeutics right now. But I won't be surprised if Editas emerges as a winner in sickle cell disease and beta-thalassemia over the long term.
The company plans to file for FDA approval by the end of 2020 to begin clinical testing of EDIT-301 in treating sickle cell disease. EDIT-301 uses its proprietary enzyme Cas12a (also known as Cpf1) instead of Cas9, the enzyme most commonly used in CRISPR gene-editing therapies.
Editas thinks that EDIT-301 could be the best-in-class CRISPR therapy for treating both sickle cell disease and beta-thalassemia. One reason behind the biotech's confidence is that the therapy edits the HBG1 and HBG2 genes rather than the BCL11Ae gene targeted by CRISPR Therapeutics' CTX001. Editas believes that this difference will give EDIT-301 a better safety profile than CTX001 will have. The company also thinks that using Cas12a will lead to sustained higher fetal hemoglobin levels than using the Cas9 enzyme will.
There's another intriguing possibility for Editas Medicine. Its partner on EDIT-101, Allergan, was recently acquired by AbbVie (NYSE:ABBV). The primary reason for this deal was for AbbVie to reduce its dependence on Humira, which faces biosimilar competition in the U.S. beginning in 2023.
AbbVie has other arrows in its quiver for offsetting the inevitable loss of revenue from Humira -- notably including its new immunology drugs Rinvoq and Skyrizi. However, the closer the date approaches for Humira's U.S. sales decline, the more I suspect that AbbVie will be interested in making additional smaller deals to boost its top line.
If EDIT-101 is successful in phase 1 testing and advances to phase 2, Editas Medicine could very well be on AbbVie's acquisition radar. The biotech wouldn't be so expensive that it would require AbbVie to take on a lot of additional debt. Buying Editas could also boost AbbVie's oncology program since Editas has several preclinical programs that use CRISPR gene editing in cancer cell therapies.
A speculative play
To be sure, Editas Medicine is a speculative play. For that matter, so are CRISPR Therapeutics and Intellia Therapeutics. All of these biotech stocks face significant risks that their gene-editing therapies won't work or won't be safe. But the possibility of near-term catalysts and the tremendous long-term potential for Editas make this CRISPR biotech one for investors to closely watch.