Scrip is part of the Business Intelligence Division of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

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


Ultragenyx Pharmaceutical LLC

This article was originally published in Start Up

Executive Summary

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.

Related Content

Ultragenyx Takes On Rare Childhood Disease With In-licensing Deal
Ultragenyx Raises $75 Million Series B, Prepares For A 2014 IPO
The A-List: The Trend-Shaping Series A Financings Of 2011
Rare Disease Specialist Ultragenyx Takes $45MM Series A Round
Rare Disease Specialist Ultragenyx Takes $45MM Series A Round
Edimer Pharmaceuticals Inc.
Start-Up Previews (05/2011)
Afraxis Inc.
bluebird bio LLC
Rare Disease Research: Where Compassion Meets Commerce


Related Companies

Related Deals




Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

Your question has been successfully sent to the email address below and we will get back as soon as possible. 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