Posted by Michael Wonder on 16 Oct 2019
ProQR receives rare paediatric disease designation from FDA for sepofarsen for the treatment of LCA10
15 October 2019 - ProQR Therapeutics today announced that it received rare paediatric disease designation from the U.S. FDA for sepofarsen for the treatment of Leber’s congenital amaurosis 10 (LCA10), the most common cause of blindness due to genetic disease in children.
Sepofarsen (QR-110) is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of LCA10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene.
