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.
- Forums
- ASX - By Stock
- PER
- Sarepta gene therapy study missed key target in Duchennes
Sarepta gene therapy study missed key target in Duchennes, page-115
-
-
- There are more pages in this discussion • 299 more messages in this thread...
You’re viewing a single post only. To view the entire thread just sign in or Join Now (FREE)
Featured News
Add PER (ASX) to my watchlist
(20min delay)
|
|||||
Last
8.0¢ |
Change
0.000(0.00%) |
Mkt cap ! $72.12M |
Open | High | Low | Value | Volume |
0.0¢ | 0.0¢ | 0.0¢ | $0 | 0 |
Buyers (Bids)
No. | Vol. | Price($) |
---|---|---|
1 | 246 | 8.0¢ |
Sellers (Offers)
Price($) | Vol. | No. |
---|---|---|
8.2¢ | 10000 | 1 |
View Market Depth
No. | Vol. | Price($) |
---|---|---|
1 | 246 | 0.080 |
1 | 24999 | 0.079 |
3 | 78158 | 0.078 |
1 | 13844 | 0.077 |
1 | 8000 | 0.076 |
Price($) | Vol. | No. |
---|---|---|
0.082 | 10000 | 1 |
0.083 | 281992 | 3 |
0.084 | 100000 | 1 |
0.085 | 136669 | 3 |
0.086 | 135412 | 3 |
Last trade - 07.02am 10/09/2024 (20 minute delay) ? |
Featured News
PER (ASX) Chart |
Day chart unavailable