In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia.

Achondroplasia is a rare genetic disorder caused by mutations in the Fibroblast Growth Factor receptor 3 (FGFR3). These mutations lead to aberrant increase of inhibitory signaling in proliferating chondrocytes at the growth plate. Recifercept is a potential treatment for this disease using a decoy approach to sequester FGFR3 ligands subsequently normalizing activation of the mutated FGFR3 receptor. Recifercept binds to FGF isoforms in vitro and in cellular model systems and reduces FGFR3 signaling. In addition, in a transgenic mouse model of achondroplasia, Recifercept restores reduced body weight and long bone growth in these mice. These data suggest that Recifercept treatment could lead to clinical benefits in children treated with this molecule.
Competing Interests: I have read the journal's policy and the authors of this manuscript have the following competing interests: Diogo Gonçalves, Guylène Rignol, Pierre Dellugat, Christian Czech are employees of Pfizer. Guido Hartmann, Stephanie Garcia, Jeffrey Stavenhagen, Luca Santarelli, are former employees of Therachon. Elvire Gouze is a former consultant of Therachon. Guido Hartmann is currently employed by TOLREMO therapeutics AG, Stephanie Garcia is currently employed by Bionea Lab, Jeffrey Stavenhagen is currently employed by Therini Bio, and Luca Santarelli is currently employed by VectivBio. None of these authors were employed by these companies at the time of the study, and they had no influence on the work presented here. There are no patents, products in development or marketed products to declare. This does not alter our adherence to PLOS ONE policies on sharing data and materials.