Forge Biologics, A Columbus, Ohio-based gene therapy manufacturing and development company, is set to begin Phas I and 2 clinical trials of a gene therapy to treat Krabbe Disease, a rare neurodegenerative disorder that is frequently fatal before age 5.
The technology was licensed last year from Pitt. It comes from the work of Maria Escolar, professor of pediatrics and neurodevelopmental disabilities at the School of Medicine.
Krabbe disease is a rare and fatal pediatric leukodystrophy affecting about 1-2.6 in 100,000 people in the United States.There is currently no approved treatment.