CRISPR Therapeutics Presents Positive Data on Allogeneic CRISPR-based CAR-T Cell Therapies at AACR 2018

CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced the presentation of new data from the company’s allogeneic chimeric antigen receptor T cell (CAR-T) program at the American Association for Cancer Research (AACR) Annual Meeting 2018. The data presented today demonstrate the generation of CAR-T cells targeted to BCMA and CD70 through CRISPR/Cas9 gene editing that have high editing rates, consistent expression, and selective and potent cell killing. These data confirm and expand on the work already completed on CTX101, CRISPR’s lead allogeneic CAR-T cell therapy in development for CD19+ malignancies.

As quoted in the press release:

“In the studies presented today, we used multiplexed CRISPR gene editing to modify healthy donor T cells to make CAR-T cells that selectively and potently target the tumor antigen of choice,” said Tony Ho, MD, Head of R&D, CRISPR Therapeutics. “These data provide further evidence that CRISPR/Cas9 can play a major role in enabling the creation of next-generation CAR-T cell therapies that may work for a broader population of patients including those with solid tumors.”

In Poster 1540, allogeneic CAR-T cells targeting B cell maturation antigen (BCMA) were evaluated as a potential approach for the treatment of multiple myeloma (MM). Using the CRISPR/Cas9 system, allogeneic CAR-T cells targeting the BCMA receptor were generated by disrupting the beta-2-microglobulin (B2M) and TCR alpha constant region (TRAC) genes and inserting an anti-BCMA CAR into the TRAC locus. Over 60% of the cells contained all three of the targeted edits. The study found that the CAR-T cells selectively and potently killed BCMA+ cells in vitro and eradicated MM cells in in vitro and in vivo models.

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