Biosplice’s mission is to restore health by delivering first-in-class therapies that harness alternative splicing.
Our scientific platform is based on biological discoveries that govern tissue specialization and enable us to selectively eliminate harmful proteins using small molecules. Our foundational discoveries in Wnt pathway modulation unlocked the broad therapeutic potential of alternative splicing by targeting the CLK/DYRK family kinases. These kinases govern the selection of tissue-speciﬁc and disease-selective RNA splice-sites, thus deﬁning them as targets for drug therapy. Selectively reprogramming cellular behavior could lead to the discovery of new, potentially curative therapies for certain diseases.
Biosplice’s clinical pipeline is focused on osteoarthritis and oncology, with early-stage programs in neurology and other areas of significant unmet medical need.