Certain disorders of the skin and ectodermal structures start in-utero and are caused by a key protein being missing. These proteins act like a light switch by turning on a set of embryological steps that result in the formation of normal organs and tissues. Our lead drug candidate, DMX-101, is an in-utero protein replacement therapy for the orphan disease XLHED. DMX-101 is delivered into the amniotic fluid in the late second, and early third trimesters of pregnancy to mothers carrying affected children.
DMX 101 mechanism of action
DMX-101 is a first-in-class protein therapy designed to replace the function of endogenous ectodysplasin A1 (EDA) during late fetal development through a single-course of treatment delivered into the amniotic fluid during the later stages of pregnancy. This approach has already demonstrated significant potential in humans by normalization of sweat gland function and associated thermoregulation, and improvements in dentition and respiratory function observed in a case series of three patients treated in utero with DMX-101 during the third trimester of pregnancy. These results were recently published in the New England Journal of Medicine (N Engl J Med 2018; 378; 1604-1610) and featured in Nature Medicine’s 2018 Research Highlights (Nature Medicine 2018; 24: 702).
History of DMX101 for XLHED
For more information, please refer to Orphan: The Quest to Save Children with Rare Genetic Disorders by Philip R. Reilly, MD, JD