Genetic Therapies Course

Date & Time 22-24 June 2023
Location Heidelberg, Germany

Rare diseases have long been considered the “orphans” of Medicine: of insufficient importance to merit the investment needed for scientific and therapeutic advancement. But this situation has been rapidly changing. Orphan drug legislation has been successful in stimulating therapeutic innovation and patients and pharmaceutical companies have both benefited from the development of new drugs. Improved diagnostic strategies, such as new-born screening and modern high throughput genetic testing, have highlighted the enormous impact which the early initiation of disease-modifying therapiescan have on the prognosis of these conditions. The advent of genetic therapies is now set to make rare diseases a major area of innovation in Medicine, with single gene defects being the ideal targets for the development of advanced medicinal therapy products (ATMPs).

However, with new opportunities come new challenges, such as safety, long-term efficacy, sustainability, pricing, and the provision of access to these new innovative medications. This course organized by Recordati Rare Diseases sets out to explore how these technologies are being applied, using inherited metabolic diseases as examples, and how this might change our practice in the future.


  • 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 perspectives, expectations and concerns about genetic therapies.


Healthcare professionals and scientists with an interest in genetic therapies, those from the pharmaceutical industry and from regulators involved in the development and licensing of these novel therapies.


Learn more and register here.