FDA Marks Potential Gene Therapy for Juvenile Batten Disease as Orphan Drug to Speed Development
Abeona Therapeutics’ ABO-201 program was designed at orphan drug by the U.S. Food and Drug Administration (FDA) as a potential gene therapy treatment for juvenile Batten disease. A clinical study of the therapy in patients is in the planning stages. ABO-201 therapy uses a virus to deliver a normal copy of…