{"id":"CU24132","slug":"small-molecule-therapeutics--CU24132","source":{"id":"CU24132","dataset":"techtransfer","title":"Small-molecule therapeutics for the treatment of myeloid cancers","description_":"<p>This technology identifies a collection of compounds designed to treat myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML).</p>\r\r<h2>Unmet Need: Targeted treatments for splicing-driven myeloid cancers</h2>\r\r<p>Myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) are a heterogeneous group of blood cancers that arise from mutations in hematopoietic stem cells. Most MDS cases involve mutations in genes coding for splicing factors which result in ineffective blood cell production, marrow dysplasia, low blood cell counts, and an increased risk for AML. Current therapeutic attempts to target splicing factor proteins have been limited because they also impact splicing in healthy tissue, resulting in adverse effects and reduced clinical effectiveness. As a result, there is a need for targeted, functional treatments for MDS and AML.</p>\r\r<h2>The Technology: Small molecules selectively targeting splicing factor-mutant myeloid cancers</h2>\r\r<p>This technology describes a collection of small molecules with the capacity to selectively kill cells harboring mutations in splicing factor genes. Identified through a phenotypic high-throughput screen of genetically engineered leukemia cells, these compounds are capable of selectively targeting cells harboring hotspot splicing factor mutations while sparing healthy, wild-type cells. This technology offers a promising solution for the treatment of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) via the targeting of splicing factor mutations.</p>\r\r<p>This technology has been validated in human cancer cell lines. </p>\r\r<h2>Applications:</h2>\r\r<ul>\r<li>Small-molecule cancer therapeutics</li>\r<li>Treatment for myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML)</li>\r<li>Research tool for splicing factors and splicing modulators</li>\r<li>Research tool for studying hematopoietic stem cell mutations</li>\r</ul>\r\r<h2>Advantages:</h2>\r\r<ul>\r<li>Targeted treatment for MDS and AML</li>\r<li>Specifically targets cells with splicing factor mutations</li>\r<li>Reduced harmful side effects in healthy wide-type cells</li>\r</ul>\r\r<h2>Lead Inventor:</h2>\r\r<p><a href=\"https://biology.columbia.edu/content/james-manley\">James Manley, Ph.D.</a></p>\r\r<h2>Patent Information:</h2>\r\r<p>Patent Pending(<a href=\"https://patents.google.com/patent/WO2025171395A1\">WO/2025/171395</a>)</p>\r\r<h2>Related Publications:</h2>\r\r<h2>Tech Ventures Reference:</h2>\r\r<ul>\r<li><p>IR CU24132, CU24133</p></li>\r<li><p>Licensing Contact: <a href=\"mailto:techtransfer@columbia.edu\">Jerry Kokoshka</a> </p></li>\r</ul>\r","tags":["Acute myeloid leukemia","Dysplasia","Haematopoiesis","Hematopoietic stem cell","High-throughput screening","Myelodysplastic syndrome","Myeloid leukemia","Phenotype","Tumors of the hematopoietic and lymphoid tissues"],"file_number":"CU24132","collections":[],"meta_description":"Targeted small molecules kill splicing-factor–mutant myeloid cancers (MDS/AML) while sparing healthy cells; potential therapy and research tool.","apriori_judge_output":"{\"scores\":{\"novelty\":3.0,\"potential_impact\":3.0,\"readiness\":3.0,\"scalability\":2.0,\"timeliness\":2.0},\"weighted_score\":2.8,\"risks\":[\"Timeline: preclinical with in vivo safety profiling not yet performed\"],\"one_sentence_take\":\"The approach shows mutation-selective cytotoxicity and potential for targeted therapy, but novelty and readiness are modest and near-term risks limit impact and scalability.\"}","inventors":["James Manley","Pedro Galhardo Bak-Gordon","Siddhartha Mukherjee M.D., D.Phil."],"manager":"Jerry Kokoshka","depts":["Biological Sciences","Medicine"],"divs":["Columbia University Medical Center (CUMC)","Faculty of the Arts & Sciences"],"date_released":"2026-06-12"},"highlight":{},"matched_queries":null,"score":0.0}