Otter Pharms

Otter Pharmaceuticals is a UK-based biotechnology company dedicated to developing novel small molecule therapies for rare lysosomal storage disorders. As a portfolio company of the RxCelerate group, Otter focuses on the discovery and development of brain-penetrant inhibitors of glucosylceramide synthase (GCS). Its lead candidate, OTR-001, is being developed for the treatment of Gaucher disease, with potential applications in GBA-associated Parkinson's disease. The company aims to address the significant unmet need for oral treatments that can cross the blood-brain barrier to treat the neurological symptoms of these conditions. By leveraging the integrated drug discovery platform of its parent company, Otter Pharmaceuticals accelerates the translation of scientific insights into clinical candidates.