Searchable abstracts of presentations at key conferences on calcified tissues

ba0007oc24 | (1) | ICCBH2019

TA-46, a recombinant soluble FGFR3 receptor for the treatment of achondroplasia, is safe and well-tolerated in healthy volunteers

Collins Samuel , Greig Gerard , Porter Richard , Stavenhagen Jeff , Santarelli Luca , Meyer Christian

Objectives: To assess the safety, tolerability and pharmacokinetic (PK) profile of single and multiple doses of TA-46 administered subcutaneously to healthy volunteers.Methods: This was a double-blind, randomized, placebo-controlled trial in a total of 72 subjects. Cohorts of 8 subjects were randomised to receive either TA-46 or placebo in a 3:1 ratio in single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. SAD doses were 0.3, 1, 3, 10 a...

ba0007p15 | (1) | ICCBH2019

TA-46 prevents premature synchondrosis and restores foramen magnum size in a mouse model of achondroplasia

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...