George,
Re gene therapy, it's not just Sarepta who are struggling, it's other Big Pharma aiming at other indications. A couple of clinical trials have been stopped because of safety concerns...there was a death in one of the trials this year.
I keep saying it but, Sarepta have $400m to protect with Exondys....and they need to provide efficacy data to FDA in 2021.
I would be very surprised if Sarepta are not the partner in the US.
.
Funds, Sarepta GT, new technologies, page-2
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