On May 17, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, and its research collaborator, Ospedale San Raffaele (OSR), presented at the 21st Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) the first update on their joint discovery and development efforts of Wilms’ Tumor 1 (WT1)-specific transgenic T cells (Press release, Intellia Therapeutics, MAY 17, 2018, View Source [SID1234526747]). In conjunction with this presentation, Intellia reported that its first cell therapy target is WT1 for the treatment of acute myeloid leukemia and other potential hematological malignancies, as well as for solid tumors.
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"As Intellia expands its wholly owned T cell therapy ex vivo efforts, we are pleased to present our first set of data in this cell therapy area," said Intellia President and Chief Executive Officer John Leonard, M.D. "WT1 is over-expressed in many tumor types, including both leukemias and solid tumors, and is an ideal target for immuno-oncological therapies seeking to treat these malignancies. This early data being presented is part of Intellia’s broader strategy to develop next-generation solutions for immuno-oncology and autoimmune disorders, with novel, modular platforms based on genome editing."
Intellia and OSR are collaborating to develop best-in-class CRISPR-edited T cells directed to WT1, a tumor-associated antigen expressed across a wide range of different tumor types and a known driver of leukocyte blasts in hematological cancers. At this year’s ASGCT (Free ASGCT Whitepaper) Annual Meeting, OSR researchers, led by Chiara Bonini, M.D., Ph.D., shared findings showing the generation, characterization and advancement of WT1-specific, transgenic T cells against multiple WT1 epitopes presented on HLA-A*02:01 and other Class I alleles. Initial data demonstrating both recognition and killing of acute myeloid leukemia cells also was presented.
OSR researchers and Intellia presented the poster, entitled "Hunting WT1-Specific T Cell Receptors for TCR Gene Editing for Acute Myeloid Leukemia." The abstract is available on the ASGCT (Free ASGCT Whitepaper) website here.
"Our collaboration with Intellia represents our shared belief that the characteristics of T cell receptors as a targeting moiety may be an excellent approach to broadening the field of oncology cell therapy, opening the door to many more potential antigen targets in liquid and solid tumors," added Bonini, full professor at Università Vita-Salute San Raffaele; deputy director, Division of Immunology, Transplantation and Infectious Diseases; and head, Experimental Hematology Unit, Ospedale San Raffaele, Italy.
This ex vivo research effort is part of an integrated strategy to develop CRISPR-edited T cell therapies to address hard-to-treat cancers and overcome certain limitations of current-generation cell therapies, includin