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7.2¢ percheron therapeutics limited

Very interesting, thanks for this. Having CD49+ as the...

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dalts66

4,336 Posts.

2023

23/07/23

15:04

Post #: 68932753
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Originally posted by waynesworld: ↑

You could sense the disappointment and almost a resetting of expectations @George1972

Thanks George,
There was a very sobering discussion about regulatory and treatment implications arising from the advent of gene therapy re: the session on Combination Therapies: How do we get there. The issue arises from the fact that gene therapy fundamentally changes the genotype of that individual and creates a heterogeneity that divides the patient population between respective treatment and non-treatment groups and within the treatment groups, a further classification according to which gene therapy the patient has received. Instead of hope there are question marks and red flags. It would be less of an issue if the gene therapy worked but what if the confirmatory trials end up in a cul-de-sac.

Gene therapy may limit a patient's option for future treatment in ways which have not been previously imagined. The genie may be out of the bottle and the path forward may be how to put it back in if all the promises are unfulfilled.

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Very interesting, thanks for this. Having CD49+ as the biomarker, and not dystrophin will probably aid in any combination trial, or treatment post gene therapy.

ANP Price at posting: 5.5¢ Sentiment: Buy Disclosure: Held
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