P014: First-in-human phase 1/2 trial of intravenous FBX-101 with cord blood transplantation increases GALC, brain and motor development in infantile Krabbe

Infantile Krabbe disease (IKD) is a severe neurodegenerative disorder caused by galactocerebrosidase (GALC) deficiency which leads to death by 2 years. Umbilical cord blood transplantation (UCBT) is the accepted standard of care that has been used for 20 years in asymptomatic newborns demonstrating that survival is extended into their late teen years, but motor function progressively declines due to peripheral neuropathy. Since 2006, newborn screening has been available in New York State and currently 10 states screen for infantile disease.