23 May 2023 - CAMPSIITE trial is enrolling mucopolysaccharidosis type II patients as part of a pivotal program that incorporates material from the NAVXpress platform process manufactured at the RegenxBio manufacturing innovation centre and continues to support plan to file biologics license application in 2024 using the accelerated approval pathway
RegenxBio today announced that the US FDA has granted regenerative medicine advanced therapy designation for RGX-121, an investigational one time AAV therapeutic for the treatment of mucopolysaccharidosis type II, also known as Hunter syndrome.