We are developing a new class of therapeutics targeting the effects of sRNA mutations

Gatehouse Bio is developing oligonucleotide therapeutics that correct disease-driving mutations in small RNA genes. Our platform, based on decades of research in small RNA biogenesis and function, identifies mutations in patients that correlate to clinical phenotypes, pathological variables, and mortality. We use these mutations to stratify patients into novel endotypes and design corrective therapies for each group. This results in a portfolio of targeted therapeutics, each designed for a particular endotype. Our approach is to focus on therapeutic areas such as, inflammatory, fibrotic and neurodegenerative disease where patients are known to have variable clinical courses.