To date, rare and neglected diseases have not attracted the attention they deserve. Poor understanding of the underlying biology, small patient populations and limited returns for the required investment have meant that what small progress is achieved, has usually been supported by those immediately affected: patients, self-organized into Patient Advocacy Groups (PAGs) that fund research and promote the interests of their members.

This situation however is changing, as new technologies and knowhow promise to deliver results more cost effectively than in the past, and as new collaboration models are explored by interested stakeholders.

At Biovista, we believe that drug repositioning is a highly relevant strategy for therapy development in the case of rare and orphan diseases. It promises to identify new therapies or improve existing ones at a significantly lower cost than before. It thereby makes it possible for PAGs to fund the initial research and development stages that are necessary to de-risk an opportunity to a sufficient degree so that it can then be picked up for further development by biotech and pharma companies.

Patient Responses to Drug Repositioning

Justin Reilly

Some of these grants look like very good ideas to this non-science person, especially the drug search project…


I’m so excited to see these approaches! There are so many new big-picture technologies available to start interrogating the bodies of PWCs, like genomics, proteomics, metabolomics, etc. I’m especially excited to see the drug repurposing…

Reproduced with the kind permission of the CFIDS Association of America

How we can help

Biovista is already in collaboration with PAGs in areas such as Chronic Fatigue Syndrome (CFS) and beta-Thalassaemia working to advance the understanding of these diseases, find new therapies and improve existing ones. We work to:

  • Identify existing drugs to reposition to your disease
  • Identify drugs to combine with the existing standard of care therapy
  • Develop the repositioned drug to the PoC in man stage
  • Identify biomarkers and clinical trial endpoints in cases where these do not exist
  • Define inclusion/exclusion criteria for patient recruitment purposes
  • Mine anonymized patient record data to help understand the disease mechanism

How we work

  • We use our COSS platform to identify repositioning opportunities to be used either in isolation or in combination with existing therapies
  • We combine our own resources with your in-house data, if available, to explore patient sub-population therapy optimization and disease understanding. This latter knowledge can be made available to any other organizations (e.g. academic groups) you may be collaborating with
  • We generate and protect any relevant intellectual property (IP) and assign it to dedicated structures (e.g. a new company) for its further exploitation.

Case Study 1

See how Biovista’s repositioning work is used in the discovery of treatments for Chronic Fatigue Syndrome.

Case Study 2 

See how Biovista’s J-Anaemia EHR technology is supporting healthcare provision to patients with hemoglobinopathies.

For more information

To find out more information on our work, visit our pages on Drug Repositioning, Combination Therapies, and the CFIDS collaboration, or send an email directly to ps{at}