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Intellia Therapeutics Begins Phase I/IIa Trial of T-Cell Receptor Therapy in AML

NEW YORK – Intellia Therapeutics on Tuesday dosed the first acute myeloid leukemia patient in a Phase I/IIa study of its autologous T-cell receptor therapy NTLA-5001.

NTLA-5001 is an autologous TCR therapy that targets the Wilms' tumor (WT1) antigen, which is overexpressed in AML and other hematologic and solid tumors. The trial is enrolling up to 54 AML patients who have detectable disease after receiving standard first-line therapy. Patients also must carry the HLA-A*02:01 allele.

In the first phase of the study, Intellia will enroll AML patients with lower disease burden, or less than 5 percent blasts in bone marrow samples, in one arm; and patients with higher disease burden, or greater than or equal to 5 percent blasts in bone marrow samples, in another arm. The company will test three dose levels of NTLA-5001 in both arms to determine the recommended dose.

"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," Intellia CEO John Leonard said in a statement. "AML is the most common type of acute leukemia in adults, where despite recent advancements, a significant therapeutic need still exists."

NTLA-5001 is Intellia's lead ex vivo cancer drug candidate. The therapy uses CRISPR/Cas9 genome editing to engineer a patient's own T cells to target WT1 in cancer cells. Intellia, based in Cambridge, Massachusetts, is also using its ex vivo platform to develop a TCR therapy targeting WT1 in solid tumors, a CAR T-cell therapy for CD30-positive lymphomas, and other undisclosed targets.