CRISPR Therapeutics Presents Preclinical Data at AACR 2021 Supporting CD70 Knockout as a Novel Approach to Increasing CAR-T Cell Function

On April 10, 2021 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported preclinical data from the Company’s allogeneic chimeric antigen receptor T cell (CAR-T) program at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 (Press release, CRISPR Therapeutics, APR 10, 2021, View Source [SID1234577837]). The data, presented today in an e-poster session entitled, CD70 knockout: A novel approach to augment CAR-T cell function, found that the generation of CAR-T cells including knockout of the CD70 show improved properties including potency and persistence over CAR T cells where the CD70 gene remains intact.

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The Company applied CRISPR/Cas9 editing to examine the effects of knocking out the gene function of multiple checkpoint-related genes in CAR-T cells, including PD1 and LAG3 where antagonism with antibodies has shown anti-cancer properties in humans and mice, as well CD70. The data demonstrated that CD70 knockout performed better than other checkpoint genes and provided advantages for CAR-T cells targeting multiple antigens beyond CD70. In contrast, CAR-T cells with classical checkpoint genes knocked out showed no improved properties and often proved detrimental to CAR-T function.

CRISPR Therapeutics is currently studying CTX130TM, an investigational allogeneic CAR-T cell therapy, in patients with CD70-expressing tumors, including clear cell renal cell carcinoma and B and T cell malignancies. CRISPR Therapeutics’ two independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 are ongoing. The Company expects to report top-line data from these trials in 2021.