pp-056 Injection

For achondroplasia, the most common form of disproportionate short stature, caused by dysregulated FGFR3 signaling leading to impaired endochondral bone growth. Phase 2 clinical development. Anti-FGF2 RNA aptamer (chemically modified oligonucleotide). Mechanism / Target: Selective inhibition of fibroblast growth factor 2 (FGF2), a key ligand that overstimulates FGFR3 signaling in achondroplasia. By blocking FGF2–FGFR3 interaction, RBM-007 aims to normalize cartilage growth plate activity and promote physiologic bone elongation. Route / form: Subcutaneous injection. Differentiation: Provides a ligand-targeting approach distinct from FGFR3-blocking biologics; may reduce excessive FGFR3 activation without full receptor blockade. Designed to improve bone growth while potentially lowering safety concerns associated with systemic FGFR3 inhibition.