“Genetic therapies” course in Heidelberg – 22-24 June

Registration is now open for the “Genetic therapies” course promoted by Recordati Rare Disease Fondation d’Entreprise, which will be held in person from 22 to 24 June 2023, in Heidelberg (Germany).

The course will provide valuable insights into the rapidly evolving field of genetic therapies and their application in rare diseases, mainly inherited metabolic diseases. It will explore how new technologies are being used and how they can change medical practice in the future. In addition, some challenges associated with the development of genetic therapies, such as safety, long-term efficacy, sustainability, pricing, and access to innovative medications will be discussed.

Specifically, the learning objectives of the course will be:

• • to understand the principles of the techniques used for replacing, modulating and altering genetic information in order to cure disease or attenuate the clinical phenotype
  • to learn about current strategies used to deliver nucleic acids to different organs and compartments within the body
  • to explore the clinical development of genetic therapies, particularly the identification of meaningful endpoints, and the determination of dosing regimens
  • to define the challenges in providing access to high-priced medications for rare diseases and to look at alternative pricing models
  • to learn about the patient’s perspectives, expectations and concerns about genetic therapies.

This can also represent an opportunity for healthcare professionals and stakeholders to stay up-to-date with the latest advances in the field, network with other professionals, and better understand the opportunities and challenges in developing and implementing genetic therapies in rare diseases.

The course is open to healthcare professionals and scientists with interest in genetic therapies, as well as professionals from the pharmaceutical industry and regulatory bodies involved in the development and licensing of these therapies.

Online registration is required by the 5th of May 2023.

To see the detailed programme of the course and to have any further information about registration, fees and logistics, please visit the course website.