Rio Biofarma

Rio Biopharma is a Brazilian biotechnology company dedicated to developing innovative small molecule therapies for rare genetic conditions and neurodegenerative disorders. Founded as a spin-off from the Federal University of Rio de Janeiro (UFRJ) and the D'Or Institute for Research and Education (IDOR), the company utilizes a proprietary drug discovery platform based on patient-derived induced pluripotent stem cells (iPSCs). Their lead research programs focus on the activation of the glucocerebrosidase (GBA1) enzyme, aiming to provide disease-modifying treatments for Gaucher disease and GBA-linked Parkinson's disease. By combining advanced cellular modeling with medicinal chemistry, Rio Biopharma seeks to address significant unmet medical needs in the lysosomal storage disorder space. The company represents a significant step in the Brazilian life sciences ecosystem toward original drug discovery and development.