Karyopharm Therapeutics Inc. is a clinical-stage pharmaceutical company focused on discovery and development and subsequent commercialization of novel first-in-class drugs directed against nuclear transport and related targets for the treatment of cancer and other major diseases.Our scientific expertise is focused on understanding the regulation of intracellular communication between the nucleus and the cytoplasm. We have discovered and are developing novel, small molecule Selective Inhibitor of Nuclear Export, or SINE, compounds that inhibit the nuclear export protein XPO1. These SINE compounds represent a new class of drug candidates with a novel mechanism of action that have the potential to treat a variety of diseases in areas of unmet medical need. Our SINE compounds were the first oral XPO1 inhibitors in clinical development.

Dedicated to changing the future for people with neurological diseases, but we also understand that people want to function better today. Our goal is to provide therapies that can restore function and improve the lives of people with neurological disorders. That means we’re focused every day on developing new medications that work differently than existing ones.We’re working on treatments that we hope will:Provide relief for people with Parkinson’s disease who experience OFF periodsAddress dementia associated with Parkinson’s diseaseRepair damage to myelin, the insulating layer of cells that surround nerve fibers in people with multiple sclerosisWe realize you may have questions about neurological conditions, treatment and research. There are many resources and Web sites that can provide in-depth information so you can better understand how different diseases affect the body, learn more about available treatment options, and find organizations that support people and their families who are impacted by neurological illnesses.

Alnylam is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of patients who have limited or inadequate treatment options. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of debilitating diseases with high unmet medical need. Alnylam was founded in 2002 on a bold vision to turn scientific possibility into reality, which is now marked by its robust discovery platform and deep pipeline of investigational medicines, including 4 programs in late-stage clinical development.