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We've funded more than 2700 projects and 1600 researchers to give people affected by a rare genetic condition a new hope.
What we do
We were made to give people affected by a rare genetic condition an answer – treatment for their condition. Rare genetic conditions are not statistically relevant when considered individually, but the same can’t be said about a person’s life.
We support the best projects chosen using a rigorous and transparent selection process, following consultation with an internationally-renowned committee of qualified scientists.
Orchard Therapeutics Receives Positive CHMP Opinion for LibmeldyTM for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)
First therapy recommended for EU approval for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants.
Osteopetrosis: new opportunity of treatment thanks to a licensing agreement
SiSaf announces a research collaboration and licensing agreement with the University of L’Aquila for the treatment of the debilitating bone disease, Autosomal Dominant Osteopetrosis Type 2.
Discovery of a novel drug candidate to develop effective treatments for brain disorders
Researchers at IIT-Istituto Italiano di Tecnologia discovered a novel chemical compound, which has the potential to became a new drug for the treatment of core symptoms of brain disorders like Down syndrome and autism.
How we fund research?
We select the best research projects using an internationally-recognised selection method i.e. the peer review process.
Millions of euros invested