ABX1100 (Aro Biotherapeutics)

ABX1100 (Centyrin-GYS1 siRNA conjugate) is an investigational, first-in-class tissue-targeted genetic medicine being developed by Aro Biotherapeutics for the treatment of late-onset Pompe disease (LOPD). The therapeutic is a conjugate consisting of a CD71 (transferrin receptor 1)-binding Centyrin protein scaffold covalently linked to a small interfering RNA (siRNA) targeting glycogen synthase 1 (GYS1). By binding to CD71, which is highly expressed on muscle cells, the Centyrin facilitates muscle-selective delivery and internalization of the siRNA payload. Once inside the cell, the siRNA degrades GYS1 mRNA, thereby inhibiting the synthesis of glycogen (substrate reduction therapy) and preventing its toxic accumulation in skeletal and cardiac muscle tissues. ABX1100 has received Orphan Drug and Rare Pediatric Disease designations from the FDA and is currently in Phase 1b clinical development.

Other names

CD71 Centyrin-Gys1 siRNA conjugateCD-71 Centyrin-Gys1 siRNA conjugateCD 71 Centyrin-Gys1 siRNA conjugateCentyrin-GYS1 siRNA conjugateCentyrin-GYS-1 siRNA conjugateCentyrin-GYS 1 siRNA conjugateCentyrin-targeted Gys1 siRNA conjugate