Achondroplasia: Current Disease-Modifying Therapies and Genome Editing Strategies. (PubMed, Int J Mol Sci) - Oct 17, 2026 - "These include the activation of the NPR2/CNP axis with vosoritide and navepegritide and the pharmacologic attenuation of FGFR signaling with infigratinib, while extracellular biologics designed to modulate ligand-receptor interactions remain investigational. In parallel, gene-based approaches are increasingly discussed as routes toward the etiology-directed control of the causal driver, ranging from the cartilage-biased tuning of FGFR3 expression to variant-level genome editing concepts. Here, we synthesize the current therapeutic landscape, explicitly distinguish pathway-targeted disease modification from etiology-directed genetic strategies, and highlight the key translational barriers for gene-based approaches, including target-tissue delivery to the growth-plate, dose control and durability across development, redosing and immunogenicity constraints, and long-term safety."
Journal • Review • Gene Therapies • Genetic Disorders • Orthopedics • FGFR3
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Ekaterina Kondrateva; Svetlana Smirnikhina
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