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

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ZUG, Switzerland and CAMBRIDGE, Mass., April 16, 2018 (GLOBE NEWSWIRE) -- 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.

“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 and eradicated MM cells in and models.

To address the need for effective and durable therapies for both hematologic and solid tumors, allogeneic CAR-T cells targeting the CD70 antigen were generated by CRISPR/Cas9 genome editing. CD70 is expressed in both hematologic malignancies as well as in solid tumors such as renal cell carcinoma (RCC), while its expression in normal tissues is restricted. In Poster 2551, the B2M and TRAC genes from healthy donor cells were disrupted and the CAR was introduced into the TRAC locus, similar to other allogeneic CAR-T cell therapies in CRISPR’s portfolio. Data showed that over 60% of the cells contained all three of the targeted edits. The study found that the CD70 CAR-T cells displayed potent cell killing function against CD70 expressing lymphoid and renal cancer derived cell lines across a broad range of antigen expression levels. These cells also secreted IFNg, released granzyme B and proliferated in a CD70 specific manner, all indications of potent anti-tumor activity. The CD70 targeting CAR-T cells eradicated RCC cells in an murine model.

“The future of cell therapies lies in the ability to create more sophisticated versions of CAR-Ts by incorporating multiple modifications with precision and consistency. We believe that CRISPR-based gene editing will be the engine to enable this next generation of CAR-Ts,” said Samarth Kulkarni, Chief Executive Officer of CRISPR Therapeutics. “We continue to make rapid progress on our first CAR-T program, CTX101, directed towards CD19+ malignancies, and aim to file an IND for this program by the end of this year.”

These results were presented at the American Association for Cancer Research Annual Meeting on April 162018 in two poster sessions entitled and

Investors:Chris Erdman 617-307-7227 orChris Brinzey Westwicke Partners339-970-2843

Media:Jennifer Paganelli WCG for CRISPR 347-658-8290

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Globe Newswire: 21:01 GMT Monday 16th April 2018

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