Following the success of mRNA-based vaccines during the COVID-19 pandemic, which prompted the development of antisense drugs targeting a broader range of diseases, the European Commission is kicking off a doctoral training network called EFFecT to address treatments for rare and ultra-rare diseases with new drugs.
EFFecT is a doctoral training network, funded by the European Commission, dedicated to preparing highly specialized male and female researchers in antisense therapies.
EFFecT is a collaborative training initiative that brings together universities, research institutes, industry, and noncommercial partners from across Europe to provide doctoral and postdoctoral students with multidisciplinary and international training and the skills needed to develop innovative and personalized therapies for rare and neurodegenerative diseases.
Antisense therapy makes it possible to treat diseases such as Sma (spinal muscular atrophy) and Sla (amyotrophic lateral sclerosis) in a personalized way, slowing their course. In the future, these therapies could also open new vistas for complex diseases such as prion diseases, Huntington's disease, retinal diseases, and other ultra-rare diseases.
Antisense drugs are composed of artificial RNA fragments that precisely target the mutated RNA that causes the disease. The 'mutated RNA is thus degraded, and the disease does not occur.
Correcting the molecular mechanism of the disease opens up new possibilities and numerous challenges. Investment is needed in training specialized researchers who can follow the drug development process in all its steps.
Professor Michela Denti, who has been working on RNA for years and its applications in therapeutics for rare diseases, is among the experts involved in the EFFEcT project.
Initiatives such as EFFecT are essential for training specialized researchers capable of responding to scientific and medical challenges of great complexity.