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pp-056

Injection

Dossier type
CTD
Dossier status
Under development
Country of origin
Japan
GMP approvals
Japan
Comments
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.
Manufacturer #38604

A Japan-based biotech venture specializing in RNA aptamer drug discovery, with clinical-stage programs in ophthalmology and rare diseases. It leverages a proprietary aptamer-discovery platform and seeks global collaborations to bring next-generation nucleic-acid therapies to market.

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