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Edimer Pharmaceuticals Inc.

This article was originally published in Start Up

Executive Summary

Edimer Pharmaceuticals Inc. is poised to begin the first human trials of a protein-replacement therapy for XLHED, a disorder that impacts the development of teeth, hair and skin. Although the start-up is planning to test its therapy first in adults, Edimer's ultimate goal is to permanently correct the disorder by treating developing fetuses in utero. The company's compound is designed to cross the placental barrier, so that it can bind an endogenous receptor and trigger normal fetal development with just a single treatment.

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Ultragenyx Pharmaceutical LLC

Biotech veteran Emil Kakkis founded Ultragenyx Pharmaceutical LLC to develop treatments for rare disorders. First up: a potential therapy for hereditary inclusion body myopathies. There are several variants of this neuromuscular disorder, which causes progressive muscle weakness. By the end of the disease course, patients can be almost quadriplegic in terms of motor function. Ultragenyx is betting it can help restore muscle function in people diagnosed with HIBM by providing a continuous and steady source of sialic acid, likely through thrice-daily oral administration.

Rare Disease Research: Where Compassion Meets Commerce

The advent of a new set of potential acquirers has heightened venture capital's interest in rare diseases, resulting in the creation of a new cohort of drug developers that are using cutting edge technologies to treat and in some cases cure grave illnesses. In this issue, we profile Afraxis, bluebird bio, Edimer Pharmaceuticals and Ultragenyx Pharmaceutical.

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