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12:00 AM
 | 
Aug 27, 2012
 |  BioCentury  |  Emerging Company Profile

Edimer: Gland idea

Edimer aims to test fusion protein in newborns with rare X-linked disorder

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.

The condition is characterized by an inability to sweat, sparse hair formation, few and malformed teeth, as well as sequelae that result from an absence of mucous glands, such as dry eye, respiratory infections and asthma.

Six to 10 males per 100,000 births are affected by XLHED, which is associated with a three times higher infant mortality rate, according to President and CEO Neil Kirby.

There is no available therapy, nor is there anything in development aside from EDI200, he noted. Treatment is consigned to symptom management, including cosmetic work, childhood dentures and avoidance of physical...

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