Shares of Crispr Therapeutics (NASDAQ:CRSP) jumped nearly 23% last month, according to data provided by S&P Global Market Intelligence. The biopharmaceutical company announced a quarterly update demonstrating multiple areas of progress. The lead drug candidate, CTX001, is enrolling patients at six global sites for a phase 1/2 trial in transfusion-dependent beta thalassemia (TDT) and at 10 global sites for a phase 1/2 trial in sickle cell disease. Preliminary results are expected to be released before the end of 2019, which could have a profound effect on the pharma stock.
The gene-editing pioneer ended September with nearly $630 million in cash, which will come in handy next year when the company expects to have up to five clinical trials ongoing simultaneously. That includes plans to initiate the first clinical trial of CTX120 as a treatment for multiple myeloma in the first half of 2020, which should be followed by multiple trials involving CTX130 in solid tumors and white blood cell cancers.
It's certainly difficult for investors to argue against the continuous execution of Crispr Therapeutics. The company was the first using CRISPR gene-editing technology to enter clinical trials, continues to advance multiple assets through the rigors of preclinical work and closer to the clinic, and partners with outside companies to augment its own capabilities. There's the highly visible partnership with Vertex Pharmaceuticals for CTX001 and other pipeline assets, but that's far from the only collaboration.
Crispr Therapeutics previously created a joint venture with Bayer, called Casebia Therapeutics, although control of the start-up will revert to Crispr before the end of 2019. Casebia will focus on programs in hemophilia, eye disorders, and autoimmune diseases. Bayer will have opt-in rights for multiple drug candidates.
Crispr is also collaborating with ViaCyte to develop a cellular medicine for treating type 1 diabetes, and with KSQ Therapeutics to develop CAR-T drug candidates with enhanced allogeneicity (read: grown from a single cell line and able to be used in any individual, in contrast to the strict donor matching required for current CAR-T medicines).
There's a long way to go before the company proves CRISPR gene editing can live up to the hype in human therapeutics, but the pioneer is the best positioned among its peer group. While there are major flaws with CRISPR gene editing that could keep the initial tools from ever being commercialized, Crispr Therapeutics is taking a "softer" approach with ex vivo engineering of white blood cells in blood disorders and for immuno-oncology. Whether the approach yields success remains to be seen, but investors will get their first glimpse of the potential when preliminary results from CTX001 trials are announced in the coming months.