Targeting 18 to 25 yo with a drug that could last just 3 to 10 years at $2m to $3m a pop is a big ask.
If the drug was successful, Dr Storey said it could galvanise CSL's investment into gene therapy and spur more acquisitions by it in the emerging field.
He said there was no doubt CSL would invest more in gene therapy. "You're seeing so much competition on so many aspects of their business, they inevitably have to participate more and more in that.
"[In terms of acquisitions] it's more likely to be of the sorts developing alternatives to intravenous immunoglobulin.
"They need things that are fairly close to market. Looking at its R&D pipeline, the next big thing is CSL 112, which is still some years away, so they do have a gap in the pipeline."
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