Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote

Krabbe Disease

https://www.forgebiologics.com/forge-biologics-announces-positive-fbx-101-clinical-trial-update-in-patients-with-krabbe-disease-identified-by-newborn-screening-ahead-of-rusp-vote/

Five patients with Krabbe Disease who were identified by newborn screening (NBS) in the US have been treated with hematopoietic stem cell transplantation (HSCT) and FBX-101 gene therapy in clinical trials with positive safety and efficacy results. FBX-101 is an adeno-associated viral (AAV) gene therapy that is supposed to treat the peripheral nerve disease that is not treated by HSCT. In Krabbe, the GALC gene is mutated resulting in lowered enzyme activity and leads to toxic levels of built-up psychosine in myelin producing cells leading to demyelination. Patients who have had FBX-101 after HSCT have been shown to have increased galactocerebrosidase (GALC) expression, decreased psychosine levels, improved motor function and correct brain white matter growth.