Posted by Michael Wonder on 28 Sep 2020
Orchard Therapeutics receives EMA PRIME designation for OTL-203 for the treatment of MPS-I
28 September 2020 - Orchard Therapeutics today announced that the EMA has granted Priority Medicines (PRIME) designation to OTL-203, an investigational ex vivo autologous haematopoietic stem cell gene therapy in development for the treatment of mucopolysaccharidosis type I at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
Additional interim data was recently presented from the ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203 in the severe Hurler subtype of mucopolysaccharidosis type I.
