FBT101

FBT101 is a modified antisense oligonucleotide (ASO) gapmer designed to target and suppress the expression of the double homeobox 4 (DUX4) gene. DUX4 is a transcription factor that, when aberrantly expressed in skeletal muscle, activates myotoxic gene programs leading to muscle damage and the progressive muscle-weakening pathology characteristic of facioscapulohumeral muscular dystrophy (FSHD). FBT101 was originally developed through a collaboration between the University of Alberta (Yokota Lab) and Children's National Research Institute, and was subsequently licensed globally to Facio Biotherapies in December 2024. The therapeutic candidate is currently in preclinical development for the treatment of both genetic subtypes of FSHD (FSHD1 and FSHD2).