Biosplice Therapeutics

Biosplice Therapeutics is a private, clinical-stage biotech based in San Diego, founded in 2012, with a novel platform targeting alternative splicing via CLK/DYRK kinases. The company is advancing a pipeline led by lorecivivint for osteoarthritis, which has progressed to Phase 3 trials, representing a potential disease-modifying therapy in a large, underserved market. Its platform also holds promise in oncology and other degenerative diseases, though it faces significant scientific, clinical, and competitive risks inherent in pioneering a new drug class.

Small molecule modulation of alternative pre-mRNA splicing via inhibition of the CLK/DYRK family of kinases.

In osteoarthritis, Biosplice competes with other companies developing disease-modifying agents, including biologics and other small molecules. In splicing modulation, it faces competition from RNA-targeting companies (e.g., using antisense or siRNA) in specific diseases, but its small-molecule approach to the core splicing machinery is relatively unique, though other kinase inhibitors may have overlapping effects.