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Sarepta gene therapy study missed key target in Duchennes, page-117

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Sander51

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15/01/21

14:01

Post #: 50197508
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Originally posted by waynesworld ↑

A line in the sand appears to have been drawn. Gene therapy has been discredited as a foundational treatment in DMD. I don't think we can go back to the prevailing consensus that existed prior to the release of the Sarepta clinical trial data i.e. a transformative therapeutic benefit in DMD will require a significant increase in dystrophin production. Fortunately, for the boys and families in the DMD community we can go forward.

What will be the new consensus in the treatment of DMD? Although the genesis of the disease is widely understood, there is an altogether reasonable thesis that the progression of the disease is dependent upon a biological pathway independent of dystrophin - chronic inflammation. Enter ATL1102 and the remarkable data points from the recent Phase II trial.

It will take Pharma a little while to digest the implications arising from the failure of the Sarepta gene therapy trial. It may take even a little longer to discover that CD49d is a novel target in the control of inflammation. Who knows when they will focus upon the results of ATL1102 in a small clinical trial in DMD in Australia.

Rest assured, however, that when the stars align and the dots are connected a new paradigm will come into view that will be transformative not only for DMD sufferers but multiple indications where chronic inflammation is the major or secondary causative biological pathway in disease progression.

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@waynesworld, care to shed any light on where this trial fits into the Gene Therapy picture as it appeared in the latest MD news - seems they are excited about the news?

Dosing Begins in Phase 3 Trial of Pfizer's Gene Therapy for Duchenne (musculardystrophynews.com)

Thanking you in anticipation of your reply
Cheers
.

ANP Price at posting: 21.0¢ Sentiment: Hold Disclosure: Held
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Sarepta gene therapy study missed key target in Duchennes, page-117

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Sarepta gene therapy study missed key target in Duchennes, page-117

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