This technology identifies a collection of compounds designed to treat myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML).
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.
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.
This technology has been validated in human cancer cell lines.
Patent Pending(WO/2025/171395)
IR CU24132, CU24133
Licensing Contact: Jerry Kokoshka
