Our focus

Our focus is to translate research findings into new therapies for rare diseases through drug (re)positioning

EspeRare concentrates its efforts on rare diseases that have the highest unmet medical needs and for which there is sufficient scientific understanding. Based on collaboration with pharmaceutical companies, biotechs and academia, the foundation builds a pipeline of therapies to be (re)positioned for rare diseases. We work with established patients networks and biomedical centres of excellence to validate the therapeutic opportunities from preclinical to early clinical stages.

Once proof of concept in humans is validated and a conclusive data package is generated, therapies can go back to the originator or be transferred and/or co-developed with biomedical partners for later stage clinical trials, registration and commercialisation.

The validity and viability of our model was achieved with our first programme in Duchenne Muscular Dystrophy and our most advanced therapy, in X-linked Hypohidrotic Ectodermal Dysplasia. We have driven therapeutic proof of concept for these therapeutic opportunities and obtained orphan drug designation from the European and US regulatory agencies and other multiple regulatory schemes such as the FDA Breakthrough designation and Rare Pediatric Disease designation. We continuously develop our portfolio of programmes to address life-threatening rare diseases and bring hope to these underserved patients and their families.