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Duchenne Muscular Dystrophy: A Patient-Funded Startup

December 18, 2013

This is the third Blog Post in a series over the next week that will examine Duchenne Muscular Dystrophy (DMD) in the rare disease and orphan drug space.

DART Therapeutics, a Cambridge-based company in Massachusetts, is an example of a rare disease startup company that uses the power of patient foundations and other stakeholders to drive the orphan drug development process. DART Therapeutics is a company financed and owned by patient foundations. It is an example of a successful model for research and drug development centered on the needs of families affected by a specific rare disease, DMD. While the company is patient-funded and founded, it has a professional staff and business processes like any other biotechnology company. DART stands for “Disease Action Research Therapy”.

DART Therapeutics is created in 2010 by two DMD foundations and DART Therapeutics’ CEO Gene Williams, a former Genzyme executive. The two foundations are started by parents of boys with DMD :

•    Charley’s Fund is a 501(c)(3) tax-exempt public charity in Great Barrington, Massachusetts, with the mission to “direct money into the hands of researchers who have the best shot at developing a treatment or cure for DMD”

•    Nash Avery Foundation in Minnesota.

In March 2011, the company sets up an affiliate, Halo Therapeutics, to license in and develop the IND-stage DMD drug candidate HT-100, Halofuginone Hydrobromide. HT-100 has orphan drug designation in both the US and the EU.  In October 2012, the company announces the completion of a study to evaluate a new non-invasive biomarker for DMD that measures muscle wasting in patients.

Recent 2013 DART Therapeutics News

•   March 2013 announcement that the company is developing a SARM (Selective Androgen Receptor Modulator) DMD drug candidate DT-200, obtained from Galapagos NV in Belgium

•   March 2013 announcement that the company has entered into a drug repositioning collaboration with Biovista targeting DMD

•   July 2013 announcement that a Phase Ib/IIa study is initiated to determine the safety and tolerability of lead drug candidate HT-100 (delayed-release Halofuginone)

•   November 2013 announcement that Halo Therapeutics and the Muscular Dystrophy Association (MDA) awards US $500,000 for the development of HT-100 for DMD.

DART Therapeutics’ mission is to move forward with its DMD drug candidates, invest in clinical trial designs, biomarkers, and to share all of this discovery with the DMD community. This is a great example of rare disease foundations working together to launch and co-manage a start-up company in a non-competitive way for the purpose of the larger DMD community. The DART Therapeutics model “joins patient foundation assets and pharmaceutical industry expertise … to speed development of new therapies.”

Please Note: “Technician Transfer Liquid To Test Tube” courtesy of US National Institute of Mental Health.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.


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