REGN17235

REGN17235 is an investigational small interfering RNA (siRNA) developed by Regeneron Pharmaceuticals. It is designed to target and reduce the expression of the Splicing factor 3b subunit 1 (SF3B1) protein. SF3B1 is a critical component of the U2 snRNP complex in the spliceosome, and mutations in this gene are frequently observed in hematologic malignancies, including myelodysplastic syndromes (MDS) and clonal cytopenia of undetermined significance (CCUS). These mutations lead to aberrant splicing of multiple genes, contributing to the pathogenesis of the disease. REGN17235 is currently being evaluated in Phase 1 clinical trials for patients with SF3B1-mutated low-risk MDS and CCUS to assess its safety, tolerability, and potential to mitigate the effects of spliceosome dysfunction.