Biosplice is developing first-in-class, small-molecule therapeutics based on pioneering science of alternative pre-mRNA splicing. Stemming from foundational discoveries in WNT pathway modulation, Biosplice has elucidated novel biology linking CLK/DYRK kinases to the therapeutic regulation of alternative splicing. Alternative splicing is an essential biological mechanism that regulates the diversification of proteins in a cell, which, in turn, determines cell type and function. Biosplice’s target class governs the selection of tissue-specific mRNA splice sites, making them attractive, druggable targets within the cellular “command and control” center. Biosplice’s drugs in clinical development include Lorecivivint for osteoarthritis (in Phase 3), Cirtuvivint for numerous cancers, and a broad pipeline that ranges from Alzheimer’s disease to other degenerative conditions.
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