Advancing treatments for rare diseases Together

Our focus

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

EspeRare concentrates its efforts on where the unmet medical need is the highest and where scientific knowledge is sound. Based on collaboration with pharmaceutical companies, biotechs and academia, the foundation builds a pipeline of pharmacotherapies (molecule and biologics) to be (re)positioned for rare diseases. We work with established 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, compounds can go back to the originator or be transferred to biomedical partners for later stage clinical trials, registration and commercialisation.

  • Our first focus is on Rimeporide, a Merck Serono molecule, with the potential to slow muscle degeneration in children with Duchenne Muscular Dystrophy, a genetic and fatal rare disease.
  • Our near-term goal is to complete the proof of concept for Rimeporide while working on in-licensing additional programmes to be incorporated into our R&D portfolio.
  • Mid-term we aim to build a diversified portfolio of programmes with strong therapeutic potential to treat a broader population of underserved patients.

 

  • Fostering access to health for patients that are the most in need is what this foundation is about, and is what I am about.
    Beatrice Greco
    EspeRare's founder and member of the Board

About drug repositioning

Existing or abandoned pharmacotherapies can be developed for new use in other diseases through drug repositioning approaches. Read more