Natali_Mis/iStock via Getty Images CRISPR Therapeutics (NASDAQ: CRSP ) is a company at the forefront of developing cutting-edge solutions for very difficult to treat diseases. If CRISPR Therapeutics is successful in developing therapies currently in the company’s pipeline, the stock would have enormous upside from the stock’s currently low prices.
CRISPR Therapeutics currently has six named programs in clinical trials and an additional four named programs at the research stage. What is starting to excite investors at this point is that CRISPR Therapeutics has shown huge progress in the first diseases that it decided to target, which are β-thalassemia and Sickle Cell Anemia. CRISPR Therapeutics now has trial data for twenty-two patients consisting of fifteen β-thalassemia and seven Sickle Cell patients, that shows all of these patients were functionally cured after one treatment with CTX001. CRISPR Therapeutics is expected to launch CTX001 in 2023.
The revenues that are expected to be generated from CTX001 will be used to develop solutions to treat and perhaps one day cure several forms of blood cancer including Lymphoma, Multiple Myeloma, and Myeloid Leukemia. If CRISPR Therapeutics is able to create an effective solution to cure difficult to treat blood cancers like Myeloid Leukemia, then the upside in this stock would be enormous. Investors that buy into CRISPR Therapeutics are speculating that the company’s CRISPR gene editing platform can produce revolutionary therapies that can possibly do things that people once thought impossible like curing cancer or making replacement organs for the pancreas, liver or kidneys. CRISPR Therapeutics Roadmap Moving Forward
CRISPR Therapeutics roadmap β-thalassemia and Sickle Cell Anemia
The CTX001 program started as a 50/50 collaboration with Vertex Pharmaceuticals (NASDAQ: VRTX ) providing the experience in regulatory, clinical operations, and commercialization, while CRISPR Therapeutics brought the gene editing IP. In April 2021, Vertex purchased an additional ten percent of the rights to CTX001 globally for a total of $1.1 billion. CRISPR Therapeutics did this for several reasons which included adding to the balance sheet. CRISPR Therapeutics now has a large war chest to invest in other new large opportunities on the platform. CRISPR Therapeutics CTX001 Vertex and CRISPR Therapeutics have planned regulatory submissions for CTX001 in late 2022. CRISPR Therapeutics CEO Sam Kulkarni believes the company’s approach will result in a functional cure for sickle cell disease and beta thalassemia, two inherited blood diseases. Next Generation Immuno-Oncology Platform
One of the areas where CRISPR Therapeutics will invest money derived from the CTX001 program is into several allogeneic CAR-T programs, with the first one being CTX110, which is an allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapy for the treatment of CD19+ malignancies.
Typically, B cell lymphomas , acute lymphoblastic leukemia ( ALL ), and chronic lymphocytic leukemia ( CLL ) will often express the CD19+ marker. The fundamental bet that CRISPR Therapeutics is making here is that smart engineered cells are the best way to kill cancers. What CRISPR’s Immuno-Oncology platform essentially does is engineer immune cells using a CRISPR gene editing tool so those immune cells will be able to recognize specific cancers and kill them.
CTX110 is in phase I CARBON trial , which is an open-label, multicenter clinical trial evaluating the safety and efficacy of CTX110 in adult patients with relapsed or refractory B-cell CD19+ malignancies who have received at least two prior lines of therapy. CRISPR Therapeutics recently reported data from this trial and investors were disappointed by question marks over the durability of CTX110 . and dropped the stock 6% by the end of the next day. The disappointment with CTX110 when combined with disappointments from other companies using allogeneic CAR-T approach was likely a contributing factor for the stock declining 34% since October 12, when the data was released. I think in the moment of comparing data with between ten different drugs people lose sight of the fact that it’s a historic moment. We’ve opened up the era of smart cells and cancer and we now believe that we can tweak the regimen, do consolidation dosing where we get two doses to one, improve the lymphodepletion and all that to further enhance the data. Source: CRISPR Therapeutics CEO Sam Kulkarni – Piper Sandler 33rd Annual Virtual Healthcare Conference CRISPR Therapeutics frames the results as not being a disappointment because the data showed that a single dose of CTX110 injected into a cancer patient made some patients cancer free for what they call an extended period of time, with an extended period of time being defined as over a year of being cancer free. It […]
source Crispr Therapeutics: Tons Of Upside From Current Prices
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