Sapreme is a preclinical-stage biotech company developing next-generation RNA therapeutics for patients with genetically driven diseases. Our proprietary endosomal escape technology enables targeted delivery of large molecules such as ASOs and siRNAs to intracellular targets in liver and extrahepatic tissues, such as muscle, heart, kidney and the brain.
With successful PoCs in rodents and non-human primates, Sapreme is now building a pipeline of therapeutic applications to address high unmet needs in neuromuscular, renal, cardiometabolic diseases and beyond.
Our lead program is designed as a best-in-class therapy for Duchenne muscular dystrophy, an incurable and fatal pediatric monogenic disease. In addition, we are pursuing strategic partnerships to co-develop products with select pharma/biotech companies.


The problem: Less than 1% of next-generation therapeutics cross cellular barriers efficiently

Large molecules do not cross cell- or endosomal membranes efficiently. Therefore, intracellular target engagement is inefficient. This leads to limited new treatment options for patients, despite validated and promising disease targets. There is a high need for improved intracellular delivery of next-generation oligonucleotide therapeutics (siRNAs and ASOs).


Our solution: Harnessing the power of endosomal escape enhancers evolved in plant biology

Sapreme has selected a unique class of plant secondary metabolites (glycosylated triterpenoids) that specifically enhance delivery of large molecules to the cellular cytoplasm by mediating endosomal escape. Through advanced chemical modification of these natural compounds, Sapreme has developed a proprietary technology platform (SPT) with optimized medicinal properties. Our SPT platform selectively enhances receptor-targeted delivery to endosomes and enables pH-driven endosomal release of the therapeutic. Endosomal targeting and proprietary linker technology means that SPT remains inactive in circulation and has unprecedented endosomal escape potency and tolerability.