EspeRare, Dermelix to develop in utero therapy for rare disease

Dermelix partnered with the EspeRare Foundation to develop DMX-101 for X-linked hypohydrotic ectodermal dysplasia, a rare pediatric genetic disease. The partners plan to begin a pivotal study of the protein replacement therapy next half in the U.S. and EU.

DMX-101 comprises the Fc portion of human IgG1 linked to ectodysplasin 1 (EDA). According to EspeRare, there is no approved therapy for the indication, which is caused by EDA deficiency, and the therapy has the potential to become the first drug administered in utero to correct a genetic disease before birth. ...