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Apc001 (Hong Kong Baptist University)
- Development stage
- Preclinical
- Lead developer
- Hong Kong Baptist University
- Modality
- RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Apc001 (also known as aptscl56, Apc001PE, or Apc001OA) is a next-generation, cardiovascular-safe bone anabolic aptamer that specifically targets the loop3 domain of sclerostin (SOST). Developed by Hong Kong Baptist University in collaboration with Aptacure Therapeutics, Apc001 blocks the interaction between osteoblastic sclerostin loop3 and LRP4, which is required for sclerostin to bind to LRP6 and inhibit the Wnt/β-catenin signaling pathway. Unlike traditional sclerostin-targeting monoclonal antibodies (such as romosozumab) that target loop2 and carry a black box warning for cardiovascular risks, Apc001 preserves the macrophagic sclerostin loop2-ApoER2 interaction that protects the cardiovascular system. Apc001 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the US FDA for the treatment of osteogenesis imperfecta (OI) and X-linked hypophosphatemia (XLH), and is also in development for osteoporosis, sarcopenia, fracture healing, and breast cancer.
- Other names
- PEG40k-aptscl56PEG-40k-aptscl56PEG 40k-aptscl56anti-sclerostin loop3 aptamersclerostin loop3-specific aptamerstapled Aptscl56
02
Targets
SOST (Sclerostin)
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