Meeting on the Mesa 2021 | Treating Krabbe disease with AAV-based gene therapies

Timothy Miller, PhD, Forge Biologics, Columbus, OH, provides an overview of the current treatment landscape for Krabbe disease and the potential for gene therapy as a curative treatment. Krabbe disease is a lysosomal storage disorder that affects the nervous system, and the current standard of care is a bone marrow transplant. Dr Miller additionally describes an adeno-associated virus (AAV)-based gene therapy that when administered in conjunction with the bone marrow transplant, lacks the immunogenicity-related issues associated with AAV vectors. This interview took place at Meeting on the Mesa 2021.