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:5/26/2016)... NEW YORK , May 26, 2016 ... industry, investors see value in this space. Today,s pre-market research ... Healthcare equities: Radius Health Inc. (NASDAQ: RDUS ), ... (NASDAQ: ARWR ), and Five Prime Therapeutics Inc. ... these stock technical briefings at: http://www.activewallst.com/ ...
(Date:5/25/2016)... ... May 25, 2016 , ... Lajollacooks4u has become a rising hotspot ... rated one of its top attractions. Fortune 500 companies, such as Illumina, Hewlett-Packard, ... unique and intimate team-building experience. , Each event kicks off with an olive oil ...
(Date:5/25/2016)... ... May 25, 2016 , ... The American Medical Informatics Association ... the National Coordinator for Health IT (ONC) outlining a measurement approach to interoperability ... available when and where it was needed. The organization of health informatics professionals ...
(Date:5/25/2016)... ... May 25, 2016 , ... Biohaven ... Administration (FDA) has granted the company’s orphan drug designation request covering BHV-4157 for ... designation granted by the FDA. , Spinocerebellar ataxia is a rare, debilitating ...
Breaking Biology Technology:
(Date:3/22/2016)... 22, 2016 According ... Market for Consumer Industry by Type (Image, Motion, ... (Communication & IT, Entertainment, Home Appliances, & ... to 2022", published by MarketsandMarkets, the market ... reach USD 26.76 Billion by 2022, at ...
(Date:3/15/2016)... -- --> --> According ... "Digital Door Lock Systems Market - Global Industry Analysis, Size, ... digital door lock systems market in terms of revenue was ... to grow at a CAGR of 31.8% during the period ... (MSMEs) across the world and high industrial activity driving inclusive ...
(Date:3/11/2016)... India , March 11, 2016 ... a new market research report "Image Recognition Market by ... Application (Marketing and Advertising), by Deployment Type (On-Premises and ... Forecast To 2022", published by MarketsandMarkets, the global market ... 2015 to USD 29.98 Billion by 2020, at a ...
Breaking Biology News(10 mins):