Branaplam

Branaplam (CAS No.: 1562338-42-4) is a synthetic, orally bioavailable, and selective SMN2 pre-mRNA splicing modulator, suitable for mechanistic studies and early discovery research workflows centered on RNA processing and the survival motor neuron pathway; it promotes exon 7 inclusion by stabilizing the interaction between U1 small nuclear ribonucleoprotein (U1 snRNP) and SMN2 pre-mRNA, thereby increasing full-length SMN2 transcripts and correspondingly upregulating SMN protein levels in cellular and animal research systems; in terms of in vitro activity, its effect on the SMN pathway is typically in the nanomolar range (for example, EC50 approximately 20 nM), while in electrophysiology-related evaluations it may show low micromolar inhibition of the hERG potassium channel (for example, IC50 approximately 6.3 μM), suggesting that appropriate concentration selection and corresponding counterscreening are needed in experimental design; in established mouse models of spinal muscular atrophy, dose-related increases in SMN protein in the brain and spinal cord have been observed, along with prolonged survival compared with the control group, supporting central nervous system target engagement at the research level; typical applications include modulating SMN2 splicing in disease-relevant cellular models, validating biomarkers related to splicing correction, and serving as a reference tool compound in splicing modulator screening cascades, with the specific concentration or dose to be used determined according to the respective experimental design and research objectives; this product is for laboratory research use only and is not intended for medical, clinical, diagnostic, or any form of ingestion or consumption in humans or animals.