Message from the Director

Michel Sadelain, MD, PhD

We are at a pivotal moment for advancing therapeutic cell engineering. The progress in T cell engineering achieved over the past two decades, underscored by the emergence of CD19 chimeric antigen receptor (CAR) T cell therapy in oncology, signals a promising era for inventing, developing, and applying a broad range of immune cell-based medical interventions–not only for cancer but for all fields of medicine where engineered cells may address unmet medical needs. A concerted institutional initiative to advance research in therapeutic cell engineering is both timely and essential.

CAR T cell therapy has already transformed the treatment of several hematological malignancies. CAR T cells are lymphocytes that have been repurposed through genetic engineering to identify and destroy tumor cells. As of 2025, seven CAR therapies are approved, providing a curative option for patients with refractory leukemia, lymphoma, or multiple myeloma. CAR T cells targeting CD19 are now being investigated for treating refractory autoimmune rheumatic and neurologic disorders such as systemic lupus erythematosus and multiple sclerosis. This expansion of CAR T cell indications illustrates the remarkable potential of engineered immunity.

Following in the footsteps of CAR T cell therapy, an expanding spectrum of immune cell types and genetic engineering tools is under intensive prospection to devise novel interventions. Researchers are not only investigating the therapeutic potential of αβ-T cells but also γδ-T cells, regulatory T cells, NK cells, macrophages, B cells, neutrophils, and their precursors, including pluripotent stem cells. Beyond the well-established γ-retroviral and lentiviral vectors, CRISPR Cas9, a variety of base editors, adeno-associated vectors, single-stranded DNA, targeted replication-defective viruses, and lipid nanoparticles are rapidly advancing in preclinical studies. Therapeutic cells may thus be produced from the patient’s own cells, or from “off-the-shelf” cell sources, or from within, in vivo.

The future holds promise for yet a broader range of cell and gene therapies. Hematopoietic stem cells, neurons and microglia, retinal pigment epithelium, epidermal cells, muscle cells, hepatocytes, and others may be repaired or enhanced for treating a range of pathologies. Starting from patient cells or alternative cell sources, their engineering will be undertaken in specialized laboratories following current Good Manufacturing Practices (GMP) or, in some instances, in vivo (in the patient).

To reach these goals requires assembling diverse and complementary expertise. The Vagelos College of Physicians and Surgeons (VP&S) at Columbia University Irving Medical Center is ideally positioned to spearhead the development of these future medicines. Our medical school encompasses a vast array of departments and specialized centers, including the Herbert Irving Comprehensive Cancer Center, that cover extensive swaths of physiology and disease-focused research, providing outstanding venues for conducting clinical trials and delivering cell and gene therapies. This wealth of medical knowledge and practice is complemented by expertise in genetic engineering, systems biology, biomedical engineering, and artificial intelligence, within VP&S as well as the School of Engineering and the Chan-Zuckerberg BioHub New York.

The Columbia University Initiative in Cell Engineering and Therapy (CICET) will provide the intellectual impetus, infrastructure, and regulatory guidance needed for this endeavor. CICET will recruit researchers, physician-scientists, and specialized manufacturing engineers across all medical specialties. Its three main purposes are: (1) to promote and strengthen the research continuum between the biological, translational, manufacturing, and clinical dimensions of cell engineering and therapy; (2) to establish comprehensive academic programs that formally train the next generation of leaders in cell engineering and advanced cell therapy manufacturing; and (3) to advocate for equitable and inclusive access to emerging cell and gene therapies for Columbia’s local patient population, ensuring broad societal benefits.

In addition to scientific recruitments, CICET will foster collaborations to support clinically oriented research, from early exploration and preclinical proof-of-concept to clinical evaluation in first-in-human studies. This effort will leverage the cell and vector GMP production facility in the Russ Berrie building, along with its annexes for Process Development (PD), Quality Control (QC), and Quality Assurance (QA). Regulatory support and integration with clinical operations under CICET will facilitate seamless transitions in the continuum of lab-to-clinic and clinic-to-lab studies. 

CICET is an intersectional, collaborative program that bridges basic, clinical, and cell manufacturing sciences across medical specialties and departments, as well as academia-industry partnerships. We encourage all members of our community to engage in discussion about advancing cell engineering technologies or innovative clinical investigation in cell and gene therapy. Together, we can shape the future of medicine.

Michel Sadelain, MD, PhD
Director of the Columbia Initiative in Cell Engineering and Therapy (CICET)
Herbert and Florence Irving Professor of Medicine