This section is dedicate to all types of European Initiatives that are related to the research on rare diseases.



European Network for the advancement of Clinical Gene Transfer & Therapy

The role of the European Network for the Advancement of Clinical Gene Transfer and Therapy (CLINIGENE) is to mobilise efficiently all interested parties, mostly involving academic research and production centers together with companies, patients' groups and regulatory bodies. Its main goal is to integrate multidisciplinary research in order to decipher the key elements which can lead to improved safety and clinical efficacy of gene transfer / therapy medicinal products, i.e. for clinical applications. Control and test methods may be applied as platforms for particular gene transfer products. Besides quality control, safety is of germane concern since in the event where the treatment would be proven safe, it could be administered early enough in the course of the disease to achieve genuine cure, so that clinical gene transfer may be called therapy.

The general objectives of Clinigene-NoE thus are the following:

  • Foster interaction between all stakeholders: regulators, pre-clinical & clinical investigators, scientists, companies (otherwise competitors), patients’ groups, in order to streamline integration of multidisciplinary expertise.
  • Establish quality, safety, efficacy and morally acceptable standards for clinical gene transfer products.
  • Identify the “critical path” to accelerate the transit phase from preclinical to clinical phase by integrating expertise and generating new knowledge.
  • Improve European competitiveness by spreading of excellence and disseminating knowledge.
  • Obtain clinically significant improvement in the treatment of some human diseases by gene therapy.

E-Rare 2012 - Created by Toussaint Biger