Looking for a way to invest in the "biggest biotech discovery of the century" -- CRISPR gene editing? Two of the top candidates are CRISPR Therapeutics AG (NASDAQ:CRSP) and Intellia Therapeutics, Inc. (NASDAQ:NTLA). Both biotechs are focused on harnessing CRISPR gene editing to fight disease.
CRISPR Therapeutics is the hands-down winner in terms of stock performance so far in 2018. But which of these two up-and-coming biotechs is the better pick for long-term investors? Here's how CRISPR Therapeutics and Intellia stack up against each other.
The case for CRISPR Therapeutics
CRISPR Therapeutics was co-founded by Emmanuelle Charpentier, one of the pioneers of CRISPR gene-editing research. It quickly attracted a lot of attention -- and money -- from venture capital firms and one big biotech: Celgene.
As CRISPR Therapeutics' preclinical research programs advanced, the company also caught the eye of big drugmakers interested in the promise of gene editing. Both Vertex Pharmaceuticals and Bayer announced collaborations with CRISPR Therapeutics in 2015.
The Vertex partnership has gained the most momentum. CRISPR Therapeutics and Vertex have already started enrolling patients in a phase 1/2 clinical study being conducted in Europe for the gene-editing therapy CTX001 in treating the rare blood disorder beta-thalassemia. And although the U.S. Food and Drug Administration (FDA) placed another phase 1/2 study of CTX001 in treating sickle cell disease on clinical hold a few months ago, that hold was recently lifted. CRISPR Therapeutics and Vertex expect to begin the study by the end of 2018.
CRISPR Therapeutics also plans to submit an application by the end of the year to request FDA approval to begin an early-stage clinical study of another CRISPR therapy. CTX101 is the biotech's lead off-the-shelf chimeric antigen receptor T cell (CAR-T) therapy targeting cancers with high levels of the CD19 protein.
The company should be in great shape to go for a long time without having to raise additional capital. CRISPR Therapeutics recently conducted a stock offering that generated around $200 million in gross proceeds.
The case for Intellia Therapeutics
Intellia Therapeutics also has a close connection with one of the key scientists who paved the way for the use of CRISPR in gene editing. The biotech was formed by venture capital firm Atlas Venture and Caribou Biosciences, which in turn was co-founded by noted CRISPR pioneer Jennifer Doudna.
Like CRISPR Therapeutics, Intellia caught the eyes of big drugmakers. Novartis invested in the small biotech and formed a partnership focusing on the use of CRISPR gene editing in CAR-T and hematopoietic stem cell (HSC) therapies. In 2016, Regeneron teamed up with Intellia, gaining exclusive rights to CRISPR therapies that target up to 10 indications.
Intellia and Regeneron hope to submit an application by the end of 2019 to begin clinical testing of a CRISPR gene-editing therapy targeting treatment of transthyretin (TTR) amyloidosis. This rare genetic disease is caused by mutations in the TTR gene, which lead to buildup of abnormal deposits of the amyloid protein.
The company also expects to identify the first hematopoietic stem cell target from its collaboration with Novartis by the end of this year. Intellia has already identified Wilms' tumor 1 (WT1) as the first cell therapy target from research with Italian university and research hospital Ospedale San Raffaele.
Intellia's cash position should carry the company well into the future. As of June 30, 2018, the biotech's cash and cash equivalents totaled $305.5 million.
CRISPR Therapeutics appears to be the better pick for investors right now. It has a significant head start over Intellia, with one clinical study already enrolling and others soon to start. It will be well over a year before Intellia will be able to start clinical testing of its lead CRISPR candidate -- and that's if all goes well.
Keep in mind, though, that there's still a long way to go for CRISPR Therapeutics. Several potential risks have been identified with CRISPR gene editing. More issues could arise. CRISPR Therapeutics could be a huge winner eventually, but it's a stock suitable only for aggressive investors.