CRISPR Therapeutics to expand into autoimmune disease

Dec. 5, 2023

CRISPR Therapeutics has shared updates to its immuno-oncology pipeline, as well as an expansion into autoimmune disease.

The biotech shared preliminary data from trials of its next-gen CAR T-cell product candidates, CTX112 targeting CD19, and CTX131 targeting CD70, suggesting potential improvements in efficacy compared to first-generation candidates.

According to CRISPR, these next-gen candidates demonstrate increased manufacturing robustness and scalability, with higher and more consistent CAR T-cell production per batch. The biotech is now looking to focus on CTX112 and CTX131, with plans to transition patients treated with CTX110 and CTX130 to long-term follow-up.

In addition to the ongoing trials, CTX112 will be evaluated in autoimmune diseases, with a clinical trial in systemic lupus erythematosus set to begin in the first half of 2024. Simultaneously, trials of CTX131 will broaden to include hematologic malignancies, encompassing T- and B-cell malignancies.

The announcement comes days before the FDA is scheduled to share its landmark regulatory decision on CRISPR and Vertex's gene-therapy, exa-cel. The therapy, branded Casgevy, addresses sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) and recently became the world's first CRISPR-based gene-editing therapy when it received authorization by the U.K.'s MHRA a few weeks ago.

MHRA granted conditional marketing authorization for eligible SCD and TDT patients aged 12 and above, lacking a suitable donor. Approximately 2,000 patients in the U.K. qualify for Casgevy, according to Vertex.

The drug, a result of the 2015 collaboration between CRISPR Therapeutics and Vertex, is also under review by the European Medicines Agency, the Saudi Food and Drug Authority, and the U.S. FDA. Despite reservations about off-target risks, the FDA has granted Priority and Standard Reviews for SCD and TDT, with action dates on December 8, 2023 and March 2024.