A company focused on the genetics of rare diseases to develop novel medicines. We are a clinical-stage biopharmaceutical company discovering and developing a pipeline of differentiated therapeutics for orphan indications that we intend to commercialize on our own, and for larger market indications that we intend to partner with global pharmaceutical industry leaders. We have built a core platform enabling the discovery of validated drug targets by studying rare human diseases with extreme traits, including diseases caused by mutations in ion channels, known as channelopathies. Our integrated platform has a full complement of in-house capabilities for human genetics and small molecule drug discovery, and for preclinical and clinical development.


PHARMACEUTICAL;BIOTECHNOLOGY

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