University of Colorado Anschutz Gates Institute's CD64 CAR T-cell therapy gets IND for AML

The University of Colorado Anschutz Gates Institute announced FDA clearance for an Investigational New Drug (IND) application for a CD64-directed CAR T-cell therapy in adults with relapsed or refractory acute myeloid leukemia, as per an announcement. The clearance marks the first CAR T-cell therapy developed entirely on a US academic campus to receive FDA authorization for clinical testing, according to the Gates Institute. Enrollment in the Phase I first-in-human study is anticipated to begin in June 2026.

The Phase I study will evaluate safety, tolerability, and optimal dosing of CD64 CAR T cells, with treatment to be administered at UCHealth University of Colorado Hospital. Manufacturing will be conducted at the Gates Biomanufacturing Facility on the CU Anschutz campus.

As per the trial's registry listing (NCT07347418), the study will enroll adults with relapsed/refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (HR-MDS), with documented CD64 expression. A separate pediatric trial, to be led by Sanam Shahid, MD, at Children's Hospital Colorado, is expected to open later in 2026.

CD64 (FcγRI) is a high-affinity receptor for immunoglobulin G expressed on monocytic AML cells, and was identified as a marker of disease that persists following venetoclax-based therapy in work from Craig Jordan's laboratory at CU Anschutz. The CAR T-cell construct was engineered to direct cytotoxic T cells against CD64-expressing leukemia cells. No approved CAR T-cell therapy currently exists for AML; the field has faced persistent challenges with target selection given antigen overlap between leukemic blasts and normal hematopoietic progenitors.

---

Spot something wrong? Report an issue with this article