Intellia Therapeutics Announces WT1 for treating Acute Myeloid Leukemia as Its First Cell Therapy Target

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) the first update on their joint discovery and development efforts of Wilms’ Tumor 1 (WT1)-specific transgenic T cells. In conjunction with this presentation, Intellia announced on 5/17/18 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.

“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 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.

Intellia is seeking to unlock the applications of the CRISPR/Cas9 technology and create a new class of therapeutic products for genome based diseases. Where previous treatments may have only addressed symptoms Intellia intends to create therapies that address the causes of disease.