Presentation
Background
Our business model is to discover first-in-class, innovative therapeutics for the treatment of Rare and Ultra-Rare Diseases and license them to larger partners who will deliver them to market for the patients that need them.
Vision and Mission
Patient’s with Rare and Ultra-Rare diseases, and their families who care for and support them, face significant and numerous challenges in their daily lives.
Our focus is to use our innovative approach and expertise to discover new treatments, which these patients not only need but also clearly deserve, in order to improve their quality of life.
Approach
By designing and discovering pharmacological chaperones (drugs) which associate to these enzymes and stabilise them, we will be able to resolve the corresponding pathology of the disease and have a significant positive impact on patient’s lives.
Research and Development

Before joining Industry, he held different positions in Academia, initially in the Department of Medical Pathology at the University Hospital of Madrid (Spain), and later in the Department of Biochemistry at the University of Massachusetts Medical School (US). During these years, he focused on the role of G-protein coupled receptors in the activation of signal transduction pathways with special interest on the regulation of intracellular phospholipases and their role in disease.
Emilio has been very active in the development of the Society for Laboratory Automation and Screening (SLAS) where he served as Director on the SBS Board and as Chairman of the SLAS European Council. He has been very active in the creation and development of the Spanish Drug Discovery Network (SDDN).
Management and Advisory Team







Administration
Board of Directors

