APC003OA

APC003OA is an AI-generated, chemically modified nucleic acid aptamer designed to target the C-terminal (CT) domain of connective tissue growth factor (CTGF/CCN2). Developed by Aptacure Therapeutics in collaboration with The Chinese University of Hong Kong and Hong Kong Baptist University, APC003OA was discovered using the generative AI platform AptGEN. Unlike traditional CTGF-targeting therapies (such as the antibody pamrevlumab/FG-3019) that target the VWC domain, APC003OA specifically inhibits the CT-domain, which is a key driver of fibrotic signaling, without inducing compensatory TGF-β1 elevation. To enhance in vivo stability and extend its half-life, the aptamer is modified with 2'-O-methyl groups on cytosine and guanine nucleotides and conjugated with octadecadienoic acid (OA). APC003OA is being developed for the treatment of progressive muscle fibrosis in Duchenne muscular dystrophy (DMD) and has received Orphan Drug Designation and Rare Pediatric Disease Designation from the US FDA. It is also being investigated for pulmonary fibrosis and pneumoconiosis.

Other names

OA-modified Apc003octadecadienoic acid-modified Apc003