Navigation Links
Milo Biotechnology Announces FDA Orphan Drug Designation for AAV1-FS344 for Treatment of Duchenne and Becker Muscular Dystrophy
Date:12/12/2012

CLEVELAND, Dec. 12, 2012 /PRNewswire/ -- Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.

The program is currently in a Phase I/II trial at Nationwide Children's Hospital in adult patients with Becker muscular dystrophy and inclusion body myositis, a trial funded by the foundation Parent Project Muscular Dystrophy. "The Orphan Drug designation will provide incentives for Milo Biotechnology's continued development of the therapy in rare muscular dystrophies with the greatest medical need," said CEO Al Hawkins. "We hope that our potent muscle strengthening approach will someday augment emerging genetic strategies to transform care for these intractable diseases."

Duchenne muscular dystrophy is the most common inherited myopathy, caused by the absence of dystrophin protein. Becker muscular dystrophy, a less severe form of Duchenne muscular dystrophy, is caused by reduced levels of dystrophin protein. With both types, patients experience progressive muscle weakness, and cardiac and respiratory complications.

The Orphan Drug Act (ODA) grants special status for products to treat rare diseases. Orphan designation qualifies the sponsor of the product for a tax credit and seven years of marketing exclusivity via the ODA. A marketing application for a prescription drug product that has been designated as a drug for a rare disease or condition is not subject to a prescription drug user fee. The disease or condition for which the drug is intended must affect fewer than 200,000 people in the United States.   

About Milo Biotechnology Milo Biotechnology is an early clinical stage startup company developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases.  Its lead program, AAV1-FS344, leads to the local expression of follistatin, a myostatin inhibitor. The company is developing earlier stage programs to increase expression of follistatin regionally to muscle groups and systemically via intravascular injection.  Milo's core technology was developed at and is exclusively licensed from Nationwide Children's Hospital in Columbus, Ohio; the company is based in Cleveland, Ohio, and has received funding from JumpStart Inc. and The North Coast Opportunities Technology Fund.


'/>"/>
SOURCE Milo Biotechnology
Copyright©2012 PR Newswire.
All rights reserved

Related biology technology :

1. Puma Biotechnology Announces Completion of Private Placement
2. Animal Biotechnology - Technologies, Markets and Companies
3. The European Union Signs Grant Contract to Fund a 2 Million Euro Biotechnology Research Project Led by Hadassah College Jerusalem
4. Puma Biotechnology Announces Positive PB272 (Neratinib) Phase II Data at CTRC-AACR San Antonio Breast Cancer Symposium
5. JumpStart Invests $250,000 in Milo Biotechnology
6. Global Biotechnology Industry
7. Apexigen and Shanghai Duyiwei Biotechnology Co. Ltd Sign a License and Collaboration Agreement to Develop and Commercialize APX004
8. Lixte Biotechnology Receives US Patent for Its Proprietary Histone Deacetylase Inhibitor
9. New Study: Health Professionals Support Biotechnologys Use In Food Products And Sustainable Farming
10. Global Biotechnology Instrumentation Industry
11. Choose New Jersey, Inc. to Tout New Jerseys Life Sciences Hub At Worlds Largest Biotechnology Event
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/29/2016)... ... 2016 , ... Proove Biosciences, Inc ., the commercial ... of the Proove Health Foundation . The Foundation is a non-profit organization ... of personalized medicine for tackling the nation’s most-pressing healthcare epidemics. As part of ...
(Date:4/29/2016)... ... ... Intelligent Implant Systems announced today that the two-level components for the Revolution™ ... States. These components expand the capabilities of the system and allow Revolution™ to ... of 2015, the company has seen significant sales growth in 1Q 2016, and the ...
(Date:4/28/2016)... ... April 28, 2016 , ... Next week on May ... its first-in-class technologies for tissue stem cell counting and expansion to gene-editing scientists ... Reprogramming & CRISPR-based Genome Engineering in Burlington, Massachusetts. , The attention of most ...
(Date:4/27/2016)... ... ... The Pittcon Organizing Committee is pleased to announce that Charles “Chuck” Gardner was ... since 1987. Since then, he has served in a number of key leadership positions ... the program and exposition committees. In his professional career, Dr. Gardner is the director ...
Breaking Biology Technology:
(Date:4/13/2016)... 2016  IMPOWER physicians supporting Medicaid patients in ... clinical standard in telehealth thanks to a new partnership ... platform, IMPOWER patients can routinely track key health measurements, ... index, and, when they opt in, share them with ... a local retail location at no cost. By leveraging ...
(Date:3/23/2016)... March 23, 2016 ... Sicherheit Gesichts- und Stimmerkennung mit Passwörtern ... (NASDAQ: MESG ), ein führender Anbieter ... Unternehmen mit SpeechPro zusammenarbeitet, um erstmals dessen ... wird die Möglichkeit angeboten, im Rahmen mobiler ...
(Date:3/18/2016)... -- --> --> Competitive ... Unmanned Vehicles, Physical infrastructure and Perimeter Surveillance & Detection Systems ... security market and the continuing migration crisis in the ... has led visiongain to publish this unique report, which ... defence & security companies in the border security market ...
Breaking Biology News(10 mins):