On October 8, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company using its ARCUS genome editing platform to develop allogeneic CAR T and in vivo gene editing therapies, reported that issued the following statement about the safety of its allogeneic CAR T cell therapies (Press release, Precision Biosciences, OCT 8, 2021, View Source [SID1234591036]).
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1) Precision BioSciences’ allogeneic CAR T cells are made using its proprietary ARCUS genome editing platform designed for precision, specificity, and safety.
2) Precision BioSciences’ CAR T cells are the only allogeneic CAR T cells in human clinical trials made with a single gene editing step to specifically avoid the potentially deleterious effects of making multiple edits to T cells.
It is known in the field that making multiple edits in T cells can result in chromosomal abnormalities. Specifically, it has been shown to be an issue in TCR/CD52 edited cells. As published in Cancer Research[1], "Translocation frequencies ranged from 10-4 to 2×10-2 with translocations resulting in acentromeric or dicentromeric chromosomes occurring the least frequently."
Precision’s lymphodepletion strategy does not include an anti-CD52 monoclonal antibody, and therefore does not require editing CD52 in the CAR T cells.
3) In addition, Precision BioSciences believes the oligo-capture method is the most sensitive method available for off-target detection allowing for superior product characterization with respect to gene editing safety. Importantly, this method is used to engineer out off-target editing of the ARCUS nucleases during the research phase of product development.
4) As part of product release testing, Precision BioSciences evaluates chromosomal abnormalities and confirms that the CAR T cells are not transformed.
5) Across four clinical programs in more than 100 patients treated with PBCAR T cells, Precision BioSciences has seen no evidence of chromosomal abnormalities.
Precision BioSciences is actively recruiting patients in ongoing clinical studies of PBCAR0191 (NCT03666000) for patients with relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) and R/R B-cell acute lymphoblastic leukemia, PBCAR19B (NCT04649112) for patients with R/R NHL, and PBCAR269 (NCT04171843) for patients with R/R multiple myeloma.