Crispr Therapeutics AG (NASDAQ:CRSP) announced in its latest business update that 2020 provided favorable data to validate its CTX001 clinical program with great potential in immune-oncology.
The company seeks to produce new types of therapeutics to target a variety of health conditions through gene therapy, a departure from traditional approaches. Crispr CEO Samarth Kulkarni noted in the business update that the positive results from the CTX001 program and other programs highlight the company’s strong momentum in 2021. Crispr reported favorable topline data from its Phase 1 CARBON trial in which it has been using CTX110 to combat CD19+ B-cell malignancies.
Crispr reported notable progress in the immune-oncology program in October 2020. The company plans to release updates on its ongoing allogeneic CAR-T programs. Dr. Kulkarni revealed that the gene therapeutics manufacturer is aggressively working towards the launch of its large-scale production facility, as well as its commercial network, in 2021. These announcements paint a better picture of what to expect from the company this year as far as production and commercialization are concerned.
“We hope to make meaningful progress in bringing our large-scale manufacturing facility online and in building our commercial infrastructure,” stated Dr. Kulkarni.
What to expect from Crispr in 2021
CTX001 is currently one of the company’s most promising products. It received various drug designations from the FDA, including Rare Pediatric Disease designation, Orphan Drug Designation, Medicine Advanced Therapy, and fast track designation for severe SCD and TDT. These designations put the company in a position to rapidly commercialize the product, which means that it can contribute to its revenue in 2021.
CTX001 also garnered Priority Medicines designation for SCD from the European Medicines Agency and Orphan Drug Designation in the EU. This means that commercialization activities will focus on the American and European markets, which are currently the largest commercial markets for the pharmaceutical industry. The designations expand Crispr’s potential commercial opportunities.
The pipeline CTX001 is designed to boost etal hemoglobin (HbF; hemoglobin F), and it can potentially eliminate the need for blood transfusion in TDT patients. The treatment also demonstrated the ability to reduce painful episodes in SCD patients significantly.