Navigation Links
MDA Awards Valerion Therapeutics $1.2 Million to Continue Myotubular Myopathy Research

Tucson, Ariz. (PRWEB) May 30, 2013

The Muscular Dystrophy Association has awarded Valerion Therapeutics $1,195,762 to continue development of an experimental cell-penetrating treatment that may represent a unique approach to treating myotubular myopathy (MTM).

The experimental treatment, 3E10Fv-MTM1, uses a cell-penetrating antibody to deliver myotubularin to muscle fibers. Myotubularin is the protein that is deficient in MTM1.

Researchers at the Concord, Mass., biotech have taken a different approach to creating an effective enzyme-replacement therapy by utilizing an antibody-protein fusion that can penetrate the muscle cell. After receiving injections of 3E10Fv-MTM1 for only two weeks, mice showed improved muscle strength, function and structure.

“We are interested in this technology because it may provide a novel method of delivering proteins to muscle,” said MDA Vice President of Research Jane Larkindale. “In January, the company published data that showed that the technology allowed the protein to get into the muscles of a mouse model of the disease, where it had a positive effect on the disease course. It also may be able to be applied to other proteins for other diseases in MDA's program."

MTM is an inherited muscle disease that can cause severe, general muscle weakness complicating basic activities such as breathing and swallowing.

The award was made through MDA Venture Philanthropy (MVP), the drug development arm of MDA. It will allow the company to continue testing 3E10Fv-MTM1 in mice and develop a pharmaceutical grade treatment that eventually may be tested in individuals with MTM.

"We are extremely grateful for the continued support of MDA,” said Dustin Armstrong, Ph.D., vice president, research at Valerion Therapeutics. “This preclinical funding will advance testing of our myotubular myopathy product candidate and further validate the potential of our therapeutic platform. Valerion is committed to utilizing our antibody-based delivery system to target genetic diseases, and our partnership with MDA provides additional encouragement for addressing these neuromuscular disorders with high unmet need."

MDA began supporting development of this therapy in 2010 through a grant to Armstrong, who was chief scientific officer at 4s3 Bioscience at the time. 4s3 is now Valerion Therapeutics, taking its name from the Latin term valere meaning “to flourish, be strong and healthy.”

To learn more, read "MTM: MDA Funds Development of Myotubularin-Based Treatment."

About Valerion
Valerion Therapeutics (formerly 4s3 Bioscience) is a biotechnology company focused on developing therapies for orphan genetic diseases using a novel antibody-based targeting system.

Valerion takes its name from the Latin word valere meaning “to flourish, to be strong and healthy.”
Valerion’s platform technology allows for enhanced intracellular delivery of active biological therapeutics for neuromuscular diseases, including enzyme-deficiency disorders and muscular dystrophies. Currently, the company’s lead preclinical programs focus on the unmet medical needs of myoytubular myopathy and myotonic dystrophy. Valerion completed a $20 Million Series A investment with Alopexx Enterprises and Genzyme Ventures in 2012. In addition to this venture funding, the company has obtained competitive grant support from the Massachusetts Life Sciences Center (MLSC), the NIH and the Muscular Dystrophy Association.
For more information please go to

About MDA

MDA is the nonprofit health agency dedicated to finding treatments and cures for muscular dystrophy, ALS and related diseases today affecting more than 1 million Americans. It does so by funding research, providing comprehensive health care and support services, and through advocacy and education.

In addition to funding more than 250 research projects worldwide, MDA maintains a national network of 200 medical clinics; facilitates hundreds of support groups for families affected by neuromuscular diseases; and provides local summer camp opportunities for thousands of youngsters living with progressive muscle diseases.

For more information, visit and follow us on Facebook ( and Twitter @MDAnews.

  • MDA-

Read the full story at

Source: PRWeb
Copyright©2012 Vocus, Inc.
All rights reserved

Related medicine news :

1. CSL® (Creative Systems Lighting) Wins Two 2013 Green GOOD DESIGN™ Awards
2. T.E.N. Announces Nominees for The 2013 ISE® West Awards
3. HMHP Wound Care Earns ‘Center of Distinction’ Awards
4. Connecticut Community Foundation Awards More Than $373,000 in Grants & Sponsorships to Area Nonprofits
5. USA Book News Announces Simon Says, By William Poe; Award-Winner In 2013 International Book Awards
6. New York Law Journal Releases Top Medical Malpractice Case for 2012: NY Supreme Court Awards $8.6 Million to Plaintiff after Doctor's Misdiagnosis Led to Personal Injury
7. Tim Smucker and Danny Wegman Honored With GS1 Lifetime Achievement Awards
8. ISM Announces Winners of 2013 Top 15 CRM Software Awards for both ‘Enterprise’ and ‘Small & Medium Business’ Solutions
9. Mystic Ink Publishing's "The Infinity Zone" Wins Top Honor - International Book Awards
10. Dorland Health Announces the 2013 Case In Point Platinum Awards Winners
11. Wolters Kluwer Health receives 13 awards from the ASHPE
Post Your Comments:
(Date:6/27/2016)... ... June 27, 2016 , ... A revolution is underway. ... transport experience for the millions of people who require these medical transport services ... industry through the use of technology. Now, SmartEMS has put forth an industry-changing ...
(Date:6/26/2016)... ... June 26, 2016 , ... PawPaws brand pet supplements owned ... developed to enhance the health of felines. The formula is all-natural and is made ... in the PawPaws Cat Kidney Support Supplement Soft Chews are Astragalus Root ...
(Date:6/25/2016)... FL (PRWEB) , ... June 25, 2016 , ... The temporary closing of Bruton Memorial ... Plant City Observer , brings up a new, often overlooked aspect of head lice: the ... closing for fumigation is not a common occurrence, but a necessary one in the event ...
(Date:6/25/2016)... ... ... On Friday, June 10, Van Mitchell, Secretary of the Maryland Department of Health and ... their exemplary accomplishments in worksite health promotion. , The Wellness at Work Awards took ... the BWI Marriott in Linthicum Heights. iHire was one of 42 businesses to receive ...
(Date:6/24/2016)... CA (PRWEB) , ... June 24, 2016 , ... Marcy was in a crisis. Her ... would lash out at his family verbally and physically. , “When something upset him, he ... he would use it. He would throw rocks at my other children and say he ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)...   Pulmatrix, Inc ., (NASDAQ: PULM ... announced today that it was added to the Russell ... comprehensive set of U.S. and global equity indexes on ... milestone for Pulmatrix," said Chief Executive Officer Robert ... progress in developing drugs for crucial unmet medical needs, ...
(Date:6/23/2016)... June 23, 2016 Any dentist who has made ... the current process. Many of them do not even offer ... difficulties and high laboratory costs involved. And those who ARE ... at such a high cost that the majority of today,s ... Dr. Parsa Zadeh , founder of Dental Evolutions Inc. ...
(Date:6/23/2016)... June 23, 2016 Capricor ... ), a biotechnology company focused on the discovery, ... that patient enrollment in its ongoing randomized HOPE-Duchenne ... exceeded 50% of its 24-patient target. Capricor expects ... third quarter of 2016, and to report top ...
Breaking Medicine Technology: