Posted by Michael Wonder on 16 Mar 2018
Abeona Therapeutics receives FDA rare paediatric disease designation for ABO-202 gene therapy program in CLN1 disease
15 March 2018 - Company’s fourth gene therapy program to receive rare paediatric disease designation, enabling priority review voucher.
Abeona Therapeutics announced today that the FDA has granted rare paediatric disease designation for the ABO-202 program (AAV-CLN1), an AAV-based gene therapy for the treatment of CLN1 disease (infantile and late infantile onset Batten disease). A fatal lysosomal storage disease of the nervous system caused by autosomal-recessive mutations in the CLN1 gene, also known as infantile neuronal ceroid lipofuscinosis, CLN1 disease is an inherited genetic disease that primarily affects the nervous system in newborns and progresses rapidly.
In February 2018, the ABO-202 program was granted orphan drug designation by the FDA.
