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Intellia Therapeutics Announces First Patient Dosed in Phase 1/2a Clinical Trial of NTLA-5001 for the Treatment of Acute Myeloid Leukemia
NTLA-5001 is a novel investigational T cell receptor (TCR)-T cell therapy which leverages Intellia’s proprietary cell engineering platform NTLA-5001 targets
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"NTLA-5001 is a novel investigational T cell receptor (TCR)-T cell therapy which leverages Intellia’s proprietary cell engineering platform NTLA-5001 targets Wilms’ Tumor 1 (WT1), an overexpressed intracellular antigen frequently found in acute myeloid leukemia (AML) and other hematologic and solid tumors CAMBRIDGE, Mass., March 01, 2022 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced that the first patient has been dosed with NTLA-5001, the company’s ex vivo CRISPR/Cas9 genome editing candidate for the treatment of acute myeloid leukemia (AML). NTLA-5001 is an autologous T cell receptor (TCR)-T cell therapy designed to target the Wilms’ Tumor (WT1) antigen, which is found in AML and many other hematologic and solid tumors. It is the company’s first ex vivo candidate developed using Intellia’s advanced lipid nanoparticle cell engineering platform, designed to improve cell performance as compared to traditional ex vivo delivery technologies. “As Intellia’s first wholly-owned ex vivo candidate to be dosed in a patient, this NTLA-5001 milestone represents a significant step forward in our full-spectrum approach to genome editing,” said Intellia President and Chief Executive Officer John Leonard, M.D. “AML is the most common type of acute leukemia in adults, where despite recent advancements, a significant therapeutic need still exists. We look forward to advancing this investigational engineered cell therapy as a treatment for people living with this aggressive cancer of the blood and bone marrow.” About the NTLA-5001 Clinical Program The Phase 1/2a study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy. The study includes a dose escalation and expansion phase, with up to 54 total participants. The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5%...