Alnylam has partnered with the Foundation For Rare Diseases to reward two innovative projects in the field of rare diseases with a grant of €20,000 each.
The two winners are :
After obtaining my PhD in 2009, I continued my training with a postdoctoral fellowship at the University of California at Los Angeles (UCLA) in the laboratory of Prof. Tomas Ganz. My project led to the identification of a new hormone that plays a major role in the regulation of iron metabolism and anemia repair mechanisms: erythroferrone (ERFE). I was recruited as an Inserm research fellow in 2016 and since 2017 I lead the team "Erythroferrone and martial homeostasis" at the Institute for Research in Digestive Health (IRSD) in Toulouse with the support of an ERC Starting grant.
Beta-thalassemia is a rare genetic disease affecting 1-9 people out of 1 million and caused by a defect in hemoglobin synthesis. This pathology is characterized by a profound anemia and iron overload, which causes severe clinical complications that can be life threatening. Our project aims to develop new therapeutic strategies based on the inhibition of a new target identified in the laboratory using antisense oligonucleotides for the treatment of iron overload associated with beta-thalassemia and for which effective treatments are lacking.