FBX-101

FBX-101 is an investigational gene therapy developed for the treatment of Krabbe disease (globoid cell leukodystrophy), a rare and often fatal neurodegenerative disorder caused by mutations in the GALC gene leading to deficiency of the enzyme galactocerebrosidase. FBX-101 utilizes an adeno-associated viral vector serotype rh10 (AAVrh10) to deliver a functional copy of the human GALC gene intravenously, typically after hematopoietic stem cell transplantation (HSCT). This approach aims to restore GALC enzyme activity in both central and peripheral nervous systems, addressing demyelination and improving motor function. The therapy has shown promising results in preclinical models and early clinical trials, including improved myelination, gross motor function, and prolonged lifespan. It is currently being evaluated in Phase 1/2 clinical trials for infantile and late infantile Krabbe disease[1][2][3][4][5][6][7].

Other names

Adeno-associated virus serotype rh10 containing the human galactosylceramidase gene