Most biotechs develop medicines to treat diseases, but what makes CRISPR Therapeutics (CRSP) stand out is its use of “molecular scissors” to fix problems on the genetic level, asserts Mike Cintolo, growth stock expert and editor of Cabot Top Ten Trader.
Specifically, the company uses a unique gene-editing technology (dubbed CRISPR/Cas9) for creating therapies to treat cancer, blood diseases, diabetes and more. The company’s platform identifies DNA anomalies, then employs the protein Cas9 to remove the gene sequence “problem” and replace it.
CRISPR’s pipeline includes several clinical-stage trials, including VCTX210 (for type 1 diabetes), CTX110 (for B-cell tumors), CTX120 (for multiple myeloma) and CTX130 (for tumors and blood cancers), as well as research-stage in vivo approaches for treating Friedreich’s Ataxia, hemophilia and Amyotrophic Lateral Sclerosis.
Along with its partner Vertex Pharmaceuticals (VRTX), CRISPR is developing Exa-Cel, which edits a patient’s own stem cells. Both companies released Phase III clinical study data that showed Exa-Cel has the potential to be a one-time “functional cure” for both transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD) patients.
CRISPR expects to submit regulatory filings for Exa-Cel for both of those indications later this year. The numbers at this point are basically irrelevant, as the stock trades on clinical data and the potential of its pipeline and technology.
At its recent Innovation Day presentation, the company said its long-term focus is to be a leader in gene editing, with a major Wall Street bank observing that CRISPR’s pipeline is now among the “richest and most diverse in the gene-editing space.” Granted, it’s very early stage, though we’re intrigued that nearly 400 funds have stakes already.
After a rocket ride in 2020, CRSP hit a major peak at $220 early last year and spent the rest of 2021 in retreat, and this year has followed suit, with the stock surrendering most of its pandemic-year gains (down 80%) before bottoming near $42.
But CRSP has turned around impressively since then — there was no bottom building here, just a sharp rally off the low. Volatility here is very, very large (it’s been moving an average of five points per day from high to low), but we’re OK nibbling on dips if you want in.