Advanced Therapies Week 2022 | Treating Krabbe disease with FBX-101

Krabbe disease is a rare genetic disorder caused by a deficiency of the enzyme galactocerebrosidase (GALC) and can result in mortality in infants. John Maslowski, CCO, Forge Biologics, Columbus, OH, provides a brief overview of FBX-101, an adeno-associated virus (AAV)-based gene therapy for patients with Krabbe disease. The therapy is currently being assessed in the Phase I/II RESKUE trial (NCT04693598), where patients also receive a bone marrow transplant. FBX-101 has demonstrated preliminary efficacy and the trial will additionally assess safety as well as mobility in patients. This interview took place at Advanced Therapies Week 2022.