Research we fund

Leukemia stem cells (LSCs) are self-renewing cells that frequently drive relapse in AML. LSCs are typically not eradicated by conventional therapies, highlighting the urgent need for new therapies. Proteostasis pathways play a critical role in maintaining hematopoietic stem cell (HSC) self-renewal and emerging data suggest that LSCs co-opt HSC proteostasis pathways to support growth and resist treatment, suggesting that disrupting proteostasis in AML stem cells could impair their self-renewal.

This project leverages the Wiita lab’s “structural surfaceomics” to map protein conformations on AML cells, revealing cryptic, activation-dependent epitopes absent in normal hematopoietic cells. Using structure-guided design and protein engineering, I will develop conformation-specific antibody drug conjugates(ADCs) targeting ITGB2/ITGAL to enhance specificity, reduce toxicity, and guide selective immunotherapy design.

In October 2026, TAP made a strategic investment in AvenCell Therapeutics to support the clinical development of investigational, allogeneic, or “off-the-shelf,” CAR-T cell therapy for people with relapsed or refractory blood cancers. 

AvenCell Therapeutics is a clinical-stage biotechnology company developing next-generation allogeneic CAR-T cell therapies for blood cancers. The company’s technology combines healthy donor-derived, gene-edited T cells with a switchable targeting system designed to give physicians greater control over CAR-T activity. Unlike traditional CAR-T therapies that are manufactured individually from a patient's own cells, AvenCell's approach is designed to provide an off-the-shelf therapy that can be available when patients need treatment.

TAP support will help advance the Phase 1b expansion of AVC-201 and generate additional evidence about its safety, durability of response, blood-count recovery, and potential role for patients with AML.

AvenCell is also developing AVC-203, an investigational allogeneic CAR-T therapy targeting CD19 and CD20 for patients with B-cell malignancies, including B-cell lymphomas (NCT07284433). AVC-203 is earlier in clinical development and provides an opportunity to evaluate AvenCell's allogeneic CAR-T platform across another group of blood cancers.

In October 2026, TAP made a strategic investment in AvenCell Therapeutics to support the clinical development of investigational, allogeneic, or “off-the-shelf,” CAR-T cell therapy for people with relapsed or refractory blood cancers. 

AvenCell Therapeutics is a clinical-stage biotechnology company developing next-generation allogeneic CAR-T cell therapies for blood cancers. The company’s technology combines healthy donor-derived, gene-edited T cells with a switchable targeting system designed to give physicians greater control over CAR-T activity. Unlike traditional CAR-T therapies that are manufactured individually from a patient's own cells, AvenCell's approach is designed to provide an off-the-shelf therapy that can be available when patients need treatment.

TAP support will help advance the Phase 1b expansion of AVC-201 and generate additional evidence about its safety, durability of response, blood-count recovery, and potential role for patients with AML.

AVC-201 is AvenCell's lead program and is being developed for relapsed or refractory AML. The therapy targets CD123, a protein found on many AML cells, using a separately administered targeting module that connects the CAR-T cells to the cancer cells. This switchable approach allows treatment activity to be controlled by adjusting or stopping the targeting module, an important feature because CD123 can also be found on healthy blood-forming cells. Early clinical findings have shown encouraging activity in heavily pretreated patients, and the program is advancing into Phase 1b expansion. AVC-201 is being evaluated in the Phase 1/1b RevSTAR-123 clinical trial (NCT05949125).

Acute myeloid leukemia (AML) is the leading cause of leukemia-related deaths despite intensive chemotherapy and stem cell transplantation. New approaches are urgently needed to improve survival. My research focuses on optimizing chimeric antigen receptor (CAR) T cells that target CLEC2A, a newly identified AML-specific target. I aim to expand the therapeutic potential of CLEC2A CAR T cells to safely and effectively treat AML and explore how prior treatments influence CAR T cell response.

Our proposal aims to expand upon a novel, successful strategy to treat chronic myelomonocytic leukemia. It involves use of a novel oral medication (can be taken as a pill) called onvansertib to selectively kill cancer cells and spare normal ones. The goal is to add an arm to study combination of this effective medication with decitabine which is already an approved medication for this cancer.

Acute myeloid leukemia is a type of blood cancer that occurs in children and adults, requiring intensive treatment with chemotherapy and many times stem cell transplant. Despite aggressive therapy, nearly half of children with AML will relapse and a majority will die from this disease. In this project, I will test a treatment called anti-LILRB4 CAR-T cells, which we have shown to specifically and effectively target and kill leukemia cells with the protein, LILRB4, on its surface. In this project we will test anti-LILRB4 CAR-T cells in pediatric patients with relapsed or refractory AML who have no further treatment options in a "first in human" clinical trial which we believe has the potential to offer a novel, curative treatment for patients with LILRB4+ relapsed or refractory acute myeloid leukemia.

This five-year study will implement, evaluate, and scale a multilevel intervention to improve clinical trial enrollment among adolescents and young adults and older adults with blood cancers. The intervention combines real-time electronic health record (EHR)-based identification of potentially eligible patients with coordinated provider support, patient-centered trial matching, and navigation through Blood Cancer United’s Clinical Trial Support Center. It will be tested in a three-arm randomized trial at Columbia University Irving Medical Center and adapted for community oncology affiliates, generating real-world evidence to support broader dissemination.

Acute myeloid leukemia (AML) is subject to macrophage-mediated immune clearance, although the mechanisms remain poorly understood. Using genome-wide CRISPRi screens in AML cells co-cultured with macrophages, we identified new regulators of susceptibility to phagocytosis, including a therapeutically targetable link to RHOA signaling. I will dissect the underlying mechanisms and test combinatorial RHOA and PI3Kγ inhibition to nominate novel treatment strategies that overcome AML immune evasion.

MYST family acetyltransferases KAT6A and KAT7 are targetable dependencies in MLLr and NPM1-mutant AML. However, it is unclear how they regulate gene expression and if they have distinct or partially redundant roles. We will deploy systems where endogenous KAT6A or KAT7 can be rapidly degraded, to understand how these proteins regulate chromatin and transcription. By elucidating how KAT6A/KAT7 sustain oncogenic programs, we hope to define how best to use KAT6 and KAT7 inhibitors for AML therapy.