Biostrophin - (2010 - product launch) ?

[Andre]

Hi David,

according to Website of the company Asklepios the products Launch takes place 2010.
What do you think?

Link: www.askbio.com then "Patient Info"

Andre

[David Feder]

Biostrophin is a gene therapy. They start in USA in 2006 and I think that they need more 5 years to conclude the study.
But FDA control can delay this studies.

www.parentprojectmd.org/site/DocServer/biostrophin_abst.pdf?docID=1141

[Glen]

Dr. Feder,

In gene therapy the replacement gene is attenuated to only the important parts. In the studies with mice was the gene fully functional after gene therapy?

Is the goal of gene therapy to turn DMD into Beckers or to have a fully functional gene?

Thanks, Glen

[David Feder]

It's possible to cure with gene therapy. But I don't know the effect of therapy in muscles with some degeneration. How much muscle the therapy can cure? If only few muscles replace the gene (less than 40%) probably the functional results will be low and the disease will be more similar to BMD.

[Andre]

Hi David,

in the efficiency in the gene therapy from Dr. Cossu better then 40% muscles ?
It gives other realistic research for 80% ?
To live „normal“ how much muscles we need (replace 80%) ?

Thanks Andre

[David Feder]

See this article:
www.distrofiamuscular.net/genetherapy.pdf

An old review of gene therapy. In the table 3 you can see the results of each experimental study in mdx mice

[Andre]

Hi David,

Pat Furlong wrote to Biostrophin 2010:
---
The goal is 2010 though they are now conducting studies in the dogs to insure that there are able to deliver the aav systemically without adverse effects.
---
If they test now with animals, how do they want to arrive their goal 2010?
Is that possible?

André

[David Feder]

André:
Probably they will start human trial in 2010. Results in 2015 or more.

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