University of Colorado Advances First-In-Human CD64 CAR-T Therapy for AML Following FDA IND Clearance

April 12 2026-

The University of Colorado Anschutz Gates Institute has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration for a novel CAR-T cell therapy targeting CD64, marking the first cell therapy developed entirely on the CU Anschutz campus to enter clinical testing.

The investigational therapy is designed to target CD64, a protein identified on aggressive leukemia cells that can evade existing treatments. By engineering chimeric antigen receptor (CAR) T cells to recognize and eliminate CD64-expressing cells, the approach offers a new strategy for patients with relapsed or refractory acute myeloid leukemia (AML), a population with limited treatment options and poor prognosis.

The therapy originated from foundational work identifying CD64 as a marker of therapy-resistant leukemia cells and has undergone extensive preclinical development and optimization. Its advancement into the clinic reflects a coordinated effort across basic science, translational research, and clinical teams within the Gates Institute ecosystem.

A first-in-human Phase 1 clinical trial is expected to begin in summer 2026, enrolling adult patients with relapsed or refractory AML. The study will evaluate safety, tolerability, and dose optimization, with patients treated at UCHealth University of Colorado Hospital. A separate pediatric trial is also planned for later in the year, expanding the potential impact of this approach to younger patient populations.

CAR-T therapies have demonstrated transformative outcomes in certain hematologic malignancies, particularly B-cell cancers, but extending this success to myeloid malignancies such as AML has proven challenging due to target selection and safety considerations. The CD64-directed strategy represents a novel attempt to address these barriers by targeting a marker associated with disease persistence and resistance.

The IND clearance underscores the growing capability of academic institutions to translate early scientific discoveries into clinical-stage therapies. If successful, this program could contribute to a new generation of CAR-T approaches tailored for difficult-to-treat myeloid leukemias.