ba0007p15 | (1) | ICCBH2019
Garcia Stephanie
, Rignol Guylene
, Goncalves Diogo
, Dellugat Pierre
, Tosello Lionel
, Marsault Raphael
, Stavenhagen Jeff
, Santarelli Luca
, Gouze Elvire
Objectives: Achondroplasia, the most common form of short limb dwarfism, is a rare genetic disorder caused by a gain-of-function mutation of the FGFR3 receptor (FGFR3-G380R) and there are currently no effective treatments available. We have developed TA-46, a recombinant human soluble FGFR3(sFGFR3) form of the FGFR3 cell surface receptor containing an extra-cellular ligand-binding domain, including three IgG domains, which confer binding specificity. TA-46 is designed to block...