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

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

  • PLoS One. 2020 Dec 28;15(12):e0244368. doi: 10.1371/journal.pone.0244368.
Diogo Gonçalves 1,  Guylène Rignol 1,  Pierre Dellugat 1,  Guido Hartmann 1 2,  Stephanie Sarrazy Garcia 3,  Jeffrey Stavenhagen 4,  Luca Santarelli 5,  Elvire Gouze 6,  Christian Czech 1
Affiliations

Affiliations

  • 1 Research and Development, Rare Disease Unit, Pfizer, Nice, France.
  • 2 TOLREMO Therapeutics AG, Muttenz, Switzerland.
  • 3 Bionea, Biot, France.
  • 4 Therini Bio, South San Francisco, CA, United States of America.
  • 5 VectivBio, Basel, Switzerland.
  • 6 Université Côte d'Azur, CNRS, Inserm, iBV, Nice, France.
PMID: 33370388 DOI: 10.1371/journal.pone.0244368
Abstract

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.

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