The company's proprietary endosomal escape technology enables targeted delivery of large molecules such as PMOs, ASOs and siRNAs to intracellular targets in extrahepatic tissues as well as the liver.
With successful PoCs in rodents and non-human primates, Sapreme is building a pipeline of therapeutic applications to address high unmet needs in neuromuscular, renal, cardiometabolic and diseases.
Sapreme's lead program is designed as a best-in-class therapy for Duchenne muscular dystrophy, an incurable and fatal pediatric monogenic disease.
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze