ABXC-29

ABXC-29 is an investigational **CD71-targeted Centyrin-small interfering RNA conjugate** developed by Aro Biotherapeutics for Pompe disease. The engineered Centyrin protein binds transferrin receptor 1 on muscle cells to facilitate intracellular delivery of an siRNA directed against **GYS1**, which encodes muscle glycogen synthase. By silencing GYS1, ABXC-29 is designed as a substrate-reduction therapy that decreases glycogen synthesis and thereby reduces pathological glycogen accumulation in skeletal and cardiac muscle. In a 6neo/6neo Pompe mouse model, it reduced Gys1 messenger RNA, GYS1 protein, glycogen synthase activity, and muscle glycogen, with improved treadmill performance in male mice. ([pmc.ncbi.nlm.nih.gov](https://pmc.ncbi.nlm.nih.gov/articles/PMC11764773/))

Other names

CD71 Centyrin:Gys1 siRNA conjugate