AVI Enters Into Collaborations for the Development of Two Additional Exon-Skipping Products for Duchenne Muscular Dystrophy (DMD)

 Collaborations Represent Key Inflection Point in AVI's Progress in Developing Treatments for Broader DMD Population BOTHELL, WA, Nov 15, 2011 (MARKETWIRE via COMTEX) --

 

AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based therapeutics, today announced collaborations for the development of two additional exon-skipping drugs, one for exon 45 and one for exon 50, to support AVI's broad-based development program for the treatment of DMD. AVI's collaboration with Children's National Medical Center in Washington, D.C. and the Carolinas Medical Center (CMC) will support certain IND-enabling activities for an exon 45-skipping therapeutic. AVI's collaboration with the National Institutes of Health (NIH) will support IND-enabling activities for an exon 50-skipping therapeutic and will be supported through in-kind research conducted either by the Therapeutics for Rare and Neglected Diseases (TRND) program or by contract research organizations. AVI is currently conducting a Phase IIb trial of eteplirsen, its exon 51-skipping therapeutic candidate for the treatment of DMD.

 

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you beat me

 

its still worth posting twice

 

thanks for catching it so quick and i hope this brings some good news to many families

 Yes, worth posting many times. Really good piece of news. I PRAY TO GOD that it help all our duchenne community.

 

Raktim

Yet again those outside the hotzone are left out. "Rare and Neglected" indeed.

Can someone answer a question for me? If a boy, who is missing exons 38 thru 43, benefit from exon
skipping?

Karen, I'm no M.D., but as I read the chart, it looks like the skipping of exon 44 would help.  (As it happens, that's what my boy, Carl, needs.  He has a deletion of 40 through 43.)  And the good news is that the drug is in the pipeline.  Here's a link to Prosensa's page.  And here's the chart.  Hope that's helpful!

check out this web page from prosensa's web site. It makes it very easy to understand which deletions would benefit from skipping of particular exons. It looks like he could benefit from 51 or 44.
http://www.prosensa.eu/patients-family/duchenne-muscular-dystrophy/...
Karen Jones said:

Can someone answer a question for me? If a boy, who is missing exons 38 thru 43, benefit from exon
skipping?
Is there any information on the status of exon 45 at Children's National/Carolina's Medical? How close to trials?

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