PACT – When Personalized Medicine Brings New Hope to Patients with Rare Diseases
Context
In recent years, a new therapeutic approach has emerged: antisense oligonucleotides (ASOs). These personalized treatments make it possible to design medicines tailored to a patient’s genetic profile and pave the way for truly individualized medicine.
However, turning this scientific progress into clinical reality remains a major challenge and requires close collaboration between researchers, physicians, regulatory authorities, and specialized organizations.
Project
In this context, HUG aims to establish a Center for Innovative Therapies, in partnership with the n-Lorem Foundation and the espeRare Foundation, to facilitate access to these treatments for rare diseases.
The PACT project will be the first initiative of this kind in Switzerland. It will identify patients who could benefit from personalized ASO therapies, develop these treatments, and ensure their medical follow-up within a secure clinical framework.
Beyond treatment itself, PACT will rely on an ecosystem bringing together physicians, geneticists, and researchers to advance knowledge and improve care in the field of rare diseases.
This project carries a clear ambition: to open a new chapter in the management of rare diseases and to offer hope to patients and their families.
Project Lead
Klara Posfay-Barbe, Medical Director, Medical and Quality Directorate, Hôpitaux universitaires de Genève & Full Professor, Department of Pediatrics, Gynecology and Obstetrics, Faculty of Medicine, Université de Genève
