Rare diseases research

There are more than 7,000 rare diseases. These are rare pathologies for which there is no cure or effective treatment. They are generally chronic and degenerative, and can be severe and disabling.

Most of them are genetic in origin and more than half affect children. 

In the European Union they affect between 27 and 36 million people (6-8% of the population). In Spain, more than 3 million people are affected.

At the Cima Universidad de Navarra we do research to understand the origin and development of these diseases and to design novel strategies for their treatment. Our innovations in gene therapy have already reached clinical trials.

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Rare diseases we investigate

Icono blanco microscopio
The patient, at the center of our research

To understand the cellular and molecular mechanisms that cause the different rare diseases we investigate.

To identify new genetic mutations or specific therapeutic targets for these pathologies.

To develop efficient and safe gene therapy vectors for the treatment of severe diseases.

To design new advanced gene and cell therapies.

Innovate in biotechnology products that can be transferred to clinical trials.


Our research programs and platforms

Research at Cima is multidisciplinary, carried out by teams of professionals from various fields who collaborate with the clinical professionals of the Clínica Universidad de Navarra.