FDA Okays Initiation of Phase 1/2 Trial of Gene Therapy for Krabbe Disease

The Food and Drug Administration (FDA) gave clearance for the first-in-human adeno-associated viral (AAV) gene therapy (FBX-101; Forge Biologics, Columbus, OH) for potential treatment of Krabbe disease. In preclinical trials in animal models, this gene therapy corrected the central and peripheral myelination deficits and behavior impairments caused by Krabbe disease and lengthened life-span of the animals. 

This gene therapy is administered after hematopoietic stem cell transplant (HSCT) that delivers a operative model of the galactosylceramidase (GALC) gene to prevent the buildup of psychosine in myelinated nerve cells in the central and peripheral nervous system. The transplant and therapy infusion have the potential to overcome some of the immunologic safety setbacks of earlier AAV gene therapies.

"The ground-breaking treatment approach using HSCT and AAV gene therapy, initially developed by Dr. Escolar, has safely demonstrated superior benefits in preclinical animal studies of Krabbe disease than either treatment method alone," said Timothy J. Miller, PhD, Forge's chief executive officer, president, and cofounder. "We are grateful for the FDA's engaged review and allowance and look forward to enrolling patients very soon." 

The research resulting in this gene therapy was led by Maria Escolar, MD, MS, chief medical officer at Forge Biologics and a pioneer for evaluating the natural history and new treatment approaches for individuals with Krabbe disease. "This combination approach is extremely exciting because the preclinical data demonstrate significant correction of survival, behavior, and neuromuscular function in animal models compared to either transplant or AAV treatment alone. This is a significant milestone for Krabbe disease families suffering from this deadly disease," said Dr. Escolar.