Exploring FBX-101 For Krabbe Disease

Forge is developing innovative gene therapies that aim to help patients suffering from devastating rare diseases. This article explainsa and explores Krabbe disease and FBX-101.

Krabbe disease is a devastating neurodegenerative disease affecting about 1 -2.5 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner.

FBX-101 utilizes adeno-associated viral (AAV) gene therapy after hematopoietic stem cell transplant (HSCT) to deliver a functioning copy of the GALC gene, which encodes an enzyme needed to prevent the buildup of psychosine in myelinated cells of both the central and peripheral nervous system.