A novel Centyrin:siRNA targeting and delivery platform inhibits glycogen synthesis and reduces glycogen levels in skeletal and cardiac muscle in a mouse model of Pompe disease

A novel Centyrin:siRNA targeting and delivery platform inhibits glycogen synthesis and reduces glycogen levels in skeletal and cardiac muscle in a mouse model of Pompe disease

Discovery of ABX1100, a CD71 Centyrin-Gys1 siRNA Conjugate Clinical Candidate for the Treatment of Pompe Disease

Nonclinical Studies in Non-Human Primates on ABX-1100:
A Centyrin:Gys1 siRNA Conjugate for the Treatment of Pompe Disease