Advancing treatments for rare diseases Together

Discover the Undiscovered

 

 

Using a knowledge analytics platform, the EspeRare foundation discovers the potential of existing pharmacotherapies to address rare diseases. 
These repositioning opportunities are identified using a novel methodology that screens drugs mode of action against pathophysiological processes of selected rare diseases that have high unmet needs.

The methodology to identify repositioning candidates 

Through the combined efforts of selecting rare diseases and pharmacotherapies for repositioning, we discover attractive candidates with high therapeutic potential

Prioritise Rare Diseases

Working with patient groups, we prioritise rare diseases according to:

  • Chronicity and unmet medical needs
  • Paediatric condition
  • Cost of healthcare
  • Patient engagement
  • Availability of disease understanding
  • Availability of disease and animal models
  • Competitive landscape
  • Fundraising resources
  • Options for full development

Search & select pharmacotherapies

Existing drugs already developed by pharma, biotech and academia are selected for repositioning based on:

  • Toxicology and safety profiles
  • Understanding modes of action
  • Chemistry, manufacturing and control packages
  • Clinical data packages
  • Possible paths towards for full development and commercialisation

Discover Candidates with High Potential

Using our knowledge management approach we discover the potential of existing drugs to treat the selected rare diseases through:

  • Pathophysiological pathway analysis
  • Patient information integration
  • Mapping drug mode of action