CRSP
I would like to summarize some of the developments at CRSP over the last year and pose a question at the end.
CTX001 is a drug made from the patient’s own cells which are modified to secrete fetal hemoglobin in the treatment of sickle cell disease and beta thalasemia. There is an ongoing phase I/II study
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease - Full Text View - ClinicalTrials.gov
One year followup of first patients was published in the New England Journal of Medicine In January (N Engl J Med 2021; 384:252-260). One year after treatment fetal hemoglobin is still being made and patients are not needing transfusions or having veno-occlusive events.
June 2021 presentation from European Hematomolgy Association meeting shows similar results at 3 months follow-up for 15 thalassemia patients and 7 sickle cell patients (CLIMB THAL-111 and CLIMB SCD121 trials). Press release has more patients than the abstract so I am including both below.
CTX001 FOR SICKLE CELL DISEASE: SAFETY AND EFFICACY RESULTS FROM... by Stephan Grupp
Vertex and CRISPR Therapeutics Present New Data in 22 Patients With Greater Than 3 Months Follow-Up Post-Treatment With Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at European Hematology Association Annual Meeting | Vertex Pharmaceuticals
There are three CAR T-cell therapies being developed and in clinical trials. Other targets are possible in the future but lack of immune rejection for an off-the-shelf treatment is really what is being tested initially.
CTX 110 - allogeneic CAR T-cells that targets CD19
CTX 120 - allogeneic CAR T-cell therapy that targets BCMA for multiple myeloma
CTX 130 - allogeneic CAR T-cell therapy that targets CD70 (variety of tumor types)
Webcast held Oct 2021 released interim results of CARBON trial of CTX 110 in relapse or refractory CD19+ B-cell malignancies.
CRISPR Therapeutics Reports Positive Results from its Phase 1 CARBON Trial of CTX110™ in Relapsed or Refractory CD19+ B-cell malignancies | CRISPR Therapeutics
Single dose CTX 110 was given and there was a 58% overall response rate and 38% complete response (remission). Most important was the favorable safety profile .
Despite giving the patient cells from someone else, the gene editing prevented rejection. This trial shows that the treatment worked and there is really a low concern regarding this approach to allogeneic T-CAR cell therapy. Basically we should now be able to develop off the shelf CAR T-cell therapies that do not have require removal of cells from a specific patient and processing. This will decrease delay in starting therapies and reduce cost.
CTX 110 given RMAT designation in November 2021 by FDA
VCTX210 is being codeveloped by Veritas and CRSP for the treatment of type 1 diabetes. Vertex Pharmaceuticals has recently been in the news for curing the first patient with type 1 diabetes using allogeneic pancreatic islet cells derived from stem cells in a lab.
A Cure for Type 1 Diabetes? For One Man, It Seems to Have Worked.
This is great breakthrough but requires patients to remain on immunosuppressive drugs or their body will reject the foreign cells. Veritas is their competitor and has partnered with CRSP to implement a similar treatment that is off the shelf and should not require immunosuppressive drugs. Remember CRSP has already shown this works clinically with CTX 110.
This is a company that has likely cured sickle cell disease and beta thalassemia and has developed a platform for off the shelf CAR T-cell cancer treatments. Time will tell regarding type 1 diabetes. I see the current stock level of 60% off highs for the last year as a potential opportunity. I would like insight into what factors have caused this change in stock price because I do not see bad news in the products they are developing. I recognize the trials are still early.
