ABX1100

ABX1100 is an investigational, tissue-targeted therapy for Pompe disease, specifically late-onset Pompe disease (LOPD). It consists of a CD71 receptor-binding Centyrin protein conjugated to a small interfering RNA (siRNA) that targets and knocks down GYS1 messenger RNA in muscle tissue. By inhibiting the expression of glycogen synthase 1 (GYS1), the enzyme responsible for synthesizing glycogen in muscle, ABX1100 aims to reduce pathological glycogen accumulation characteristic of Pompe disease. This substrate reduction approach offers an alternative to enzyme replacement therapy and has shown durable knockdown of GYS1 mRNA and protein in preclinical models and early clinical trials. The drug is being developed by Aro Biotherapeutics, has received Orphan Drug Designation and Rare Pediatric Disease status from the FDA, and is currently in Phase 1b clinical trials[1][3][4][5][6][7][8].