Posted by Michael Wonder on 11 Oct 2020
Axovant Gene Therapies receives rare paediatric disease designation for AXO-AAV-GM1 for GM1 gangliosidosis
9 October 2020 - Company on-track to report data from on-going Phase 1/2 clinical study in Q4 2020.
Axovant Gene Therapies today announced that it has received rare paediatric disease designation from the U.S. FDA for AXO-AAV-GM1, an AAV9 based gene therapy delivered via a single intravenous administration that is in Phase 1/2 development for GM1 gangliosidosis.
In addition to the rare paediatric disease designation, AXO-AAV-GM1 has orphan drug designation and is the only gene therapy in clinical development for both infantile (Type I) and juvenile (Type II) GM1 gangliosidosis.
