"With more than a decade of experience in ex vivo genome editing, we have developed a deep understanding of T cell immunology that enables us to optimize the T cell editing process," said Dr. Gary Lee, Senior Director of Genome Editing at Sangamo. "The improved ZFN platform provides an extremely potent editing platform that allows us to use significantly reduced doses of mRNA and AAV. As a result, the gene editing process is highly efficient and well tolerated during ex vivo cell expansion, and, we believe, is the 'manufacturing ready' profile needed for clinical product development."At the Keystone Symposium, Jain presented work leading to a T cell with four edits achieved in a single step:
- Elimination of endogenous T cell receptor (TCR) expression by knock-out of the TCR alpha constant locus (TRAC) with greater than 97% efficiency
- Elimination of (human leukocyte antigen) HLA Class I proteins by knock-out of b 2-microglobulin (B2M) with greater than 91% efficiency
- Targeted integration into either the TRAC or B2M locus with double knock-out of TCR and HLA Class I:
- 91% efficiency with green fluorescent protein (GFP)
- 77% efficiency with CD19 chimeric antigen receptor (CAR)
- Four simultaneous edits including triple knockout of TCR (93%), B2M (96%), CISH, a checkpoint gene (93%), and targeted insertion of GFP (91%) resulting in 76% of all cells with all four edits
About Sangamo TherapeuticsSangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the Company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. For more information about Sangamo, visit www.sangamo.com.Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to Sangamo's expectations for cellular immuno-oncology treatments, the clinical and therapeutic potential of Sangamo's ZFN gene editing platform, Sangamo's strategy to advance its T cell editing platform in collaboration with partners, and other statements that are not historical facts. These forward-looking statements are based on Sangamo's current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with: gene therapy product candidate development and the inherent uncertainty of clinical success, including the risks that Sangamo and/or its collaborators may encounter unanticipated toxicity or adverse events or fail to demonstrate efficacy in clinical development; the initiation, enrollment and completion of the stages of its clinical trials; technological challenges; technological developments by its competitors and others in the gene therapy and/or cellular immuno-oncology treatment fields; Sangamo's dependence on collaborations to further the development of its technology platforms, including Sangamo's potential inability to successfully enter into new collaborations with third parties on acceptable terms, or at all, in order to advance its T cell editing platform. A more detailed discussion of these and other risks and uncertainties may be found under the caption "Risk Factors" and elsewhere in Sangamo's SEC filings and reports, including Sangamo's Quarterly Report on Form 10-Q for the quarter ended September 30, 2017 and future filings and reports by Sangamo. Sangamo assumes no obligation to update the forward-looking information contained in this press release. View original content with multimedia: http://www.prnewswire.com/news-releases/sangamo-presents-oncology-genome-editing-capabilities-at-keystone-symposium-on-emerging-cellular-therapies-300597725.html SOURCE Sangamo Therapeutics, Inc.