AURORA, Colo., April 2, 2026
The University of Colorado Anschutz Medical Campus Gates Institute announced it has received U.S. Food and Drug Administration (FDA) Investigational New Drug (IND) clearance for a novel CD64-targeted CAR T-cell therapy, marking the first campus-developed CAR T therapy in the United States to advance into clinical testing. The therapy is designed to treat relapsed or refractory acute myeloid leukemia (AML), a highly aggressive blood cancer with limited treatment options and poor patient outcomes, and represents a significant milestone in cell and gene therapy innovation.
FDA IND Clearance Enables First-in-Human Trial
The FDA IND clearance allows the Gates Institute to initiate a Phase 1 clinical trial, expected to begin in summer 2026, evaluating the safety, tolerability, and optimal dosing of CD64-directed CAR T cells in adult patients with relapsed or refractory AML. The trial will be conducted at UCHealth University of Colorado Hospital, with additional expansion into pediatric studies later in the year, highlighting a comprehensive clinical development strategy.
This regulatory milestone reflects years of translational research and demonstrates the successful transition from laboratory discovery to clinical application under strict GxP standards, ensuring compliance with Good Clinical Practice (GCP) and regulatory oversight.
Novel CAR T Approach Targets Resistant Leukemia Cells
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The investigational therapy is engineered to target CD64, a protein expressed on aggressive leukemia cells that often evade existing treatments, offering a new mechanism of action in AML therapy. Developed through extensive research and optimization, the CAR T cells are designed to recognize and eliminate malignant cells with high specificity, potentially improving outcomes in patients who have failed conventional therapies. Early preclinical studies demonstrated promising anti-leukemic activity, supporting advancement into human trials.
This innovative approach addresses a critical unmet need in oncology, where current treatment options for relapsed AML remain limited and survival rates are low.
Integrated Biomanufacturing Supports Clinical Translation
A key strength of this program is its end-to-end development within a single academic ecosystem, with manufacturing conducted at the Gates Biomanufacturing Facility, ensuring seamless integration of research, development, and clinical production. This model supports rapid translation of scientific discoveries into clinical-grade therapies, while maintaining compliance with Good Manufacturing Practice (GMP) standards for cell therapy production.
The ability to manufacture CAR T-cell therapies on campus represents a transformative advancement in academic biopharmaceutical development, enabling greater control over quality, scalability, and regulatory compliance.
Advancing Cell and Gene Therapy Innovation
This achievement underscores the growing importance of cell and gene therapies in modern oncology, particularly for diseases with limited treatment options such as AML. CAR T-cell therapies have already transformed treatment paradigms in certain blood cancers, and the development of next-generation targets like CD64 expands the potential for broader clinical impact.
The Gates Institute’s success highlights the power of collaborative, multidisciplinary research environments in accelerating innovation, bringing together expertise in immunology, oncology, and biomanufacturing. As the clinical trial progresses, the therapy has the potential to reshape treatment strategies for aggressive leukemias, offering new hope for patients facing limited therapeutic options.
Source: The University of Colorado Anschutz Medical Campus Gates Institute press release
