Who We Fund

Vanderbilt University Medical Center

Dr. Savona is the Head of Hematology, Cellular Therapy and Stem Cell Transplant, the Beverly and George Rawlings Director of Hematologic Malignancies Research, and Professor of Internal Medicine and Cancer Biology at Vanderbilt University.

He is a physician scientist specializing in the development of experimental therapies for myeloid malignancies. He has led development and approval of several novel treatments targeting the proteosome, BCL2 family proteins, PI3 kinase delta, the JAK/STAT pathway, and various epigenetic agents. He studies clonal hematopoiesis (CH) and experimental opportunities to alter the progression of CH to myeloid neoplasia and/or vascular disease. He has been involved medical research for over 20 years and has published over 100 manuscripts in major academic journals including: Cancer Discovery, Journal of Clinical Oncology, Nature Medicine, Blood, Cell Stem Cell, Lancet Oncology, Lancet Haematology, JAMA, and Nature Reviews.

He is a veteran of Operation Iraqi Freedom/Operation Enduring Freedom for the USAF.

Program Name(s)

Career Development Program

IMPACT

Project Title

Manipulation of cell fate in myeloid disease

Reaching out to underserved & minority patients with hematological diseases in the southeastern US

TAP Partner

Ryvu Therapeutics is a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology using a proprietary discovery engine platform.

Program Name(s)

Therapy Acceleration Program

Project Title

A phase 2 study of RVU120, a novel CDK8 inhibitor, in combination with venetoclax in patients with AML

TAP Partner

Constellation Pharmaceuticals was a clinical-stage biopharmaceutical company developing novel therapeutics that selectively modulate gene expression to address serious unmet medical needs in patients with cancer. MorphoSys acquired Constellation in 2021 and then Novartis acquired MorphoSys in 2024. Novartis continues to develop pelabresib in patients with myeloproliferative neoplasms.

Program Name(s)

Therapy Acceleration Program

Project Title

Development of BET protein bromodomain inhibitors for the treatment of patients with hematologic malignancies

Dana-Farber Cancer Institute

Program Name(s)

Career Development Program

Project Title

Mechanisms of oncogenic transcription in NPM1-mutant myeloid leukemia

TAP Partner

BioInvent International AB is a clinical-stage biotech company that discovers and develops novel and first-in-class immuno-modulatory antibodies for cancer therapy, with currently four drug candidates in five ongoing clinical programs in Phase 1/2 trials for the treatment of hematological cancer and solid tumors, respectively. The Company's validated, proprietary F.I.R.S.T™ technology platform identifies both targets and the antibodies that bind to them, generating many promising new drug candidates to fuel the Company's own clinical development pipeline and providing licensing and partnering opportunities.

Program Name(s)

Therapy Acceleration Program

Project Title

A phase 1/2 study of BI-1206, a monoclonal antibody to CD32b (FcyRIIB), in combination with rituximab and acalabrutinib in patients with indolent NHL that has relapsed or is refractory to rituximab

A phase 2 study of BI-1808, a monoclonal antibody to TNFR2, as a single agent and in combination with pembrolizumab in patients with solid tumors and CTCL

TAP Partner

Crossbow Therapeutics is a biotechnology company determined to improve the lives of people with cancer by unlocking the therapeutic potential of T-cell receptor (TCR)-mimetic antibodies. The company’s T-Bolt™ therapies are next-generation, easily assembled immunotherapies directed with high precision at previously unreachable cancer cell targets.

Program Name(s)

Therapy Acceleration Program

Project Title

A phase 1 study of CBX-250, a TCR-mimetic antibody, in patients with AML, MDS or CMML

University of California, San Francisco

Dr. Arun Wiita is a physician-scientist and Associate Professor in the Dept. of Laboratory Medicine and Dept. of Bioengineering & Therapeutic Sciences at the University of California, San Francisco. Dr. Wiita’s research focuses on mass spectrometry-based proteomics, target discovery, protein engineering, and cellular engineering toward the development of novel therapies for blood cancers. Dr. Wiita also directs the UCSF Stephen and Nancy Grand Multiple Myeloma Translational Initiative (MMTI) Laboratory. Dr. Wiita is a prior recipient of the NIH Director’s New Innovator Award, a Clinical Scientist Development Award from the Doris Duke Charitable Foundation, and a Chan Zuckerberg Biohub Investigator Award, among others, and is an elected member of the American Society for Clinical Investigation. He completed his residency in Clinical Pathology at UCSF, his MD and PhD at Columbia, with graduate training in single molecule biophysics, and undergraduate studies in Chemistry at Princeton.

Program Name(s)

Translational Research Program

Project Title

Optimized, computationally engineered CD70-targeting CAR-T cells for high-risk multiple myeloma

TAP Partner

Caribou is a clinical-stage biotechnology company, co-founded by CRISPR pioneer and Nobel Prize winner Jennifer Doudna, Ph.D., using next-generation CRISPR genome-editing technology to develop “off-the-shelf” (allogeneic) CAR therapies for hard-to-treat blood cancers.

Program Name(s)

Therapy Acceleration Program

Project Title

A phase 1 study of CB-010, a CRISPR-edited allogeneic CAR-T targeting CD19, in patients with B-cell NHL

A phase 1 study of CB-011, a CRISPR-edited allogeneic CAR-T targeting BCMA, in patients with multiple myeloma

Stanford University

Dr. Kathleen Sakamoto is Professor of Pediatrics at Stanford University School of Medicine. She has been studying the causes of AML and developing new therapies for the past 30 years. Her research funded by the LLS currently focuses on repurposing a drug used to treat tapeworms, niclosamide, for children with relapsed/refractory AML. Niclosamide is an FDA approved drug and is well tolerated in children. Dr. Sakamoto’s research has resulted in a Phase I clinical trial that will study toxicity, response in AML cells, and drug levels. She is also studying mechanisms of resistance of AML cells to niclosamide to look for drugs that will act synergistically for future clinical trials. Her goal is to take discoveries in the laboratory and translate them to the clinics to improve the overall survival and quality of life in children with AML.

Program Name(s)

Translational Research Program

Project Title

Niclosamide for the treatment of relapsed pediatric acute myeloid leukemia

Niclosamide for the Treatment of Relapsed/Refractory Pediatric Acute Myeloid Leukemia

Houston Methodist Research Institute

My research focuses on the translation of bench works into new therapeutic approaches/strategies for multiple myeloma and other blood cancers. One of my research goals is to study the mechanism underlying the pathogenesis of myeloma and its associated tumor microenvironment. I have been investigating the interaction of myeloma cells with bone marrow adipocytes, bone cells, and the signaling pathways associated with obesity, inflammation, chemo-, immune-therapeutic resistance, and bone disease, which are the major hurdles in treating myeloma. As a PI, I have managed many projects funded by extramural grants, private funds, and industry alliances. Some highlights include the NIH/NCI R01s and funding received from ASH and ACS. I have an excellent publication track record with peer-review articles that are in top-tier journals with high impact factors (e.g. Cell Metabolism, Science Translational Medicine, Cancer Cell). My research experience shows my capacity to be a PI for this Project.

Program Name(s)

Translational Research Program

Project Title

Targeting acetyl-CoA synthetase 2 to remodel obesity-evoked inflammatory microenvironment in myeloma

Weill Cornell Medicine

I completed my Ph.D. in pharmacology at Zhejiang University, China, in June 2020, specializing in cancer immunology. My doctoral research provided in-depth training in mouse models, particularly the construction of humanized immune recombinants such as PBMC and CD34-engrafted xenografts for pharmacodynamic studies. I developed skills in in vitro drug screening, immune cell sorting, and animal models, solidifying my expertise in experimental design. Currently, I am conducting research in Dr. Wendy Béguelin's lab, focusing on the role of the tumor microenvironment in B lymphoma progression. I pioneered a multiphoton microscopy approach for real-time imaging of cellular interactions within lymphoid tissues, enabling a detailed view of cellular interactions in lymphoma. Supported by experts from the Béguelin, Chris Xu and Melnick groups, our team leverages advanced murine lymphoma models and deep-tissue imaging techniques.

Program Name(s)

Career Development Program

Washington University in St. Louis

Dr. Fehniger is a Professor of Medicine at WashU Medicine in St. Louis. As a physician-scientist he leads a research program focused on developing novel immunotherapies for patients with blood cancer. His group pioneered studies discovering memory-like natural killer (NK) cell immunobiology and activity against acute myeloid leukemia and other hematologic malignancies. His team developed the platform for production of GMP-grade memory-like NK cells for use in academic clinical trials and translated these findings into early phase clinical trials advancing memory-like NK cellular therapy for both adult and pediatric patients. Correlative immunology from these trials identified multiple mechanisms whereby memory-like programs enhance NK cell response to blood cancers. Current research focuses on engineering memory-like NK cells with synthetic proteins to enhance targeting and function, identifying immunosuppressive mechanisms within blood cancer microenvironments, and translating these new findings to the clinic. He has mentored over 30 graduate, medical, and post-doctoral trainees as well as junior faculty. Dr. Fehniger serves as Co-leader of the Siteman Cancer Center Hematopoietic Development and Malignancies Program, Laboratory Director of the Center for Gene and Cellular Immunotherapy, and Director of the Siteman Cancer Center Biologic Therapies Core Facility. 

Program Name(s)

Academic Clinical Trials Program (ACT)

Project Title

NK cell immunotherapy to reduce relapse after haploidentical transplant for high-risk pediatric AML