Genetic Medicine Approaches to Treat Rare Diseases

A biotech rare disease company focused on genetic medicine is looking for novel research and technologies to address rare metabolic diseases, in particular amino acid metabolism diseases and lysosomal storage disorders. Specifically, the team is looking for assets utilising genetic medicine approaches which have potential for development into novel treatments, with the ultimate aim of finding long-term solutions for these conditions.

The company is interested in amino acid metabolism and lysosomal storage disorders, with a particular interest in Phenylketonuria...