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:6/24/2016)... ... June 24, 2016 , ... Researchers at the Universita Politecnica delle Marche in ... peritoneal or pleural mesothelioma. Their findings are the subject of a new article on ... biomarkers are signposts in the blood, lung fluid or tissue of mesothelioma patients that ...
(Date:6/23/2016)... , June 23, 2016 /PRNewswire/ - FACIT ... Ontario biotechnology company, Propellon Therapeutics ... development and commercialization of a portfolio of first-in-class ... Epigenetic targets such as WDR5 represent an exciting ... significantly in precision medicine for cancer patients. Substantial ...
(Date:6/23/2016)... MONICA, Calif. , June 23, 2016  The Prostate Cancer Foundation ... pioneer increasingly precise treatments and faster cures for prostate cancer. Members of the ... institutions across 15 countries. Read More About the Class ... ... ...
(Date:6/23/2016)... ... ... STACS DNA Inc., the sample tracking software company, today announced that Dr. Hays ... DNA as a Field Application Specialist. , “I am thrilled that Dr. Young ... DNA. “In further expanding our capacity as a scientific integrator, Hays brings a wealth ...
Breaking Biology Technology:
(Date:6/15/2016)... , June 15, 2016 ... report titled "Gesture Recognition Market by Application Market - Global Industry ... - 2024". According to the report, the  global gesture ... in 2015 and is estimated to grow at ... billion by 2024.  Increasing application of ...
(Date:6/7/2016)... 7, 2016  Syngrafii Inc. and San Antonio ... that includes integrating Syngrafii,s patented LongPen™ eSignature "Wet" ... collaboration will result in greater convenience for SACU ... while maintaining existing document workflow and compliance requirements. ... Highlights: ...
(Date:6/2/2016)... , June 2, 2016   The Weather Company ... announcing Watson Ads, an industry-first capability in which consumers will ... being able to ask questions via voice or text and ... Marketers have long sought an ... consumer, that can be personal, relevant and valuable; and can ...
Breaking Biology News(10 mins):