Overview
Karyopharm Therapeutics is a biopharmaceutical company focusing on the development of novel drugs for the treatment of cancer, inflammation and diseases related to cell proliferation. Karyopharm Therapeutics is developing small molecule drugs that will modulate the activity of critical pathways in cancer, inflammation, and cell proliferation (e.g. FOXO, NFкB, P53, pRB) by targeting the nuclear pore complex machinery, which controls the import and export of proteins between the nucleus and cytoplasm. To successfully control transport through the nuclear pore complex, the company will use its unique technology platform, developed by Sharon Shacham, to identify novel small molecules, optimize them into clinical candidates, and to evaluate them in subsequent human clinical studies.
Karyopharm Therapeutic’s first target, Crm1 is the main exporter of key tumor suppressor proteins and was found to be correlated with poor prognosis in cervical, ovarian, liver cancers, osteosarcoma, and gliomas. Karyopharm’s has undertaken genomic analysis that has identified genetic alterations of Crm1 in specific cancers. This in-depth validation provides a clear path to clinical targets. Using our technology platform we identified 4 new scaffolds resulting in Provisional patents for novel small molecule inhibitors of Crm1.
Karyopharm believes that specific and selective modulators of nuclear transport can provide new therapies for cancer, preneoplastic conditions, and other diseases of pathologic proliferation including inflammation and viral diseases.