Scrip is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By


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.

You may also be interested in...

With $18M In Series B Funds, Edimer To Test Protein-Replacement Therapy

Building on Swiss research, the biotech hopes to develop the first therapy for X-linked hypohidrotic ectodermal dysplasia, a genetic mutation that can impact development of teeth, hair and skin. After Phase I testing in adults, Edimer now plans to test EDI200 in six to 10 infants in Phase II.

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.

Related Content


Related Companies

Related Deals

Latest Headlines
See All



Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Thank you for submitting your question. We will respond to you within 2 business days. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts