Unlike enzyme replacement therapies that are taken chronically for life, Edimer Pharmaceuticals Inc. hopes a single course of its EDI200 given either before or soon after birth will permanently correct a rare developmental disorder. The fusion protein entered human testing this year and the company hopes to achieve clinical proof of concept in mid-2014.

X-linked hypohidrotic ectodermal dysplasia (XLHED) is a developmental disorder caused by the absence of ectodysplasin A, a protein required for proper formation of glandular structures in the skin.