Our functional genomics discovery engine screens for novel therapeutic targets at whole genome scale using sh/sgRNA libraries and relevant CMTX cancer cell line models. Through this approach, we identify novel genetic perturbations capable of reversing or inactivating molecular or cellular phenotypes associated with cancer malignancy. We discovered a novel druggable target gene that regulates cell surface expression of PD-L1, potentially providing opportunities for developing small molecule immuno-oncology drugs that can effectively treat solid tumors with limited drug penetration or located behind the blood-brain barrier, where current antibody therapies are often in effect. Building on this success, we are expanding the application of our tool to uncover novel therapeutic targets that hold the potential for the development of first-in-class drugs that can cure cancers or neuro-diseases.

Cancer cell lines serve as primary models in cancer research, enabling the study of cancer mechanisms, validation of therapeutic targets, and assessment of drug effectiveness. Our extensive efforts involve characterizing and subtyping more than 200 human cancer cell lines, encompassing 14 distinct tumor types, by analyzing their genetic mutations, mRNA expression patterns, protein expression levels, and metabolomic profiles. CMTX is particularly dedicated to uncovering novel therapeutic opportunities for recurrent, metastatic, and treatment-refractory cancer subtypes. To achieve this, we systematically evaluate the therapeutic potential of small molecule compounds and genetic perturbagens against these challenging cancer subtypes that lack effective treatments. Through this comprehensive approach, we unveil groundbreaking therapeutic targets and small molecule compounds that demonstrate promise for these previously deemed incurable cancer subtypes, illuminating promising avenues for potential treatment and offering new hope to patients in need.