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Karyopharm Therapeutics  
Karyopharm Therapeutic是一家美國(guó)抗癌藥物研發(fā)公司

Founded in 2008, Karyopharm Therapeutics is a clinical-stage pharmaceutical company focused on discovery and development of novel drugs for the treatment of cancer, inflammation and other diseases related to cell proliferation.
Our lead products are first in class, oral Selective Inhibitors of Nuclear Export (SINE), which irreversibly block nuclear-cytoplasmic transport mediated by Exportin-1 (XPO1, also called CRM1).
XPO1 is the only exporter of the tumor suppressor proteins p53, p21, FOXO, pRB, BRACA1, and PP2A, as well as the anti-inflammatory protein IkB, the inhibitor of NFκB. XPO1 over expression leads to the transport of tumor suppressor proteins (TSPs) out of the nucleus and into the cytoplasm where they are unable to function. Elevated levels of XPO1 correlate with poor prognosis in ovarian, cervical, pancreatic, and liver cancers, acute myeloid leukemia, as well as in osteosarcoma and gliomas. Natural product inhibitors of XPO1 have shown potent anti-cancer activity in the laboratory, but are unsuitable as therapeutic agents.
Recognizing its central role in cancer, we have used our unique in silico drug design platform to discover and develop novel irreversible drug like inhibitors of XPO1. Our most advanced compounds, KPT-330 and KPT-335, are currently undergoing Phase 1 testing: KPT-330 is being tested in patients with advanced tumors, both solid and hematologic, and KPT-335 is being tested in dogs with non-Hodgkin’s lymphoma.
We believe that our SINE compounds can provide novel, oral, well-tolerated therapies for various cancers. In addition, evidence suggests that SINE will also show activity in inflammatory, dermatologic and other proliferative diseases as well as infectious diseases.
In addition, Karyopharm has a growing pipeline addressing novel oncology targets which complement our XPO1 SINE program.