GA-003

GA-003 (also known as LBMD10) is an investigational bicistronic adeno-associated virus (AAV) gene therapy being developed by Genosera for the treatment of Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). Utilizing a proprietary bicistronic technology platform originating from research at the Center for Gene Therapy at Nationwide Children's Hospital, GA-003 is designed to deliver two therapeutic genes within a single vector. This approach aims not only to halt the progression of the disease but also to potentially reverse pre-existing muscle damage by addressing the underlying genetic deficiency of fukutin-related protein (FKRP). GA-003 is currently in the IND-enabling stage of development.