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COMING UP:Share Opener Variant 3



>> The concept of gene therapy, using a virus to deliver a gene into a cell in order to fix faulty DNA, has been around for decades. But the science is now delivering on its potential to cure hereditary diseases, such as hemophilia. The latest development, in the coming months we may well see the first gene therapy approvals in the US.
As Reuters' Ben Hirschler explains.>> We have a gene therapy coming up that could get approved in the United States, and would be the first pure gene therapy to be approved there, in January. And that's for an inherited rare form of blindness. And then there's another related area, cell therapy, which involves gene modified cells that are injected into the body.
And we could get an approval in the United States for the first kind of treatment along those lines as early as next month.>> But the progress comes with a catch, US pharmaceutical companies just need to look across the Atlantic to see the problems these treatments can run into.
The first two gene therapies approved in Europe are Uniqure's Glybera, approved in 2012 for a rare blood condition. And GlaxoSmithKline's Strimvelis, approved last year for a rare immunodeficiency condition. These very expensive one-off treatments, $1 million and $700,000 respectively, have only been used commercially since Is then to treat three people.
>> I think the problem that we've seen with these two gene therapies shows that it's really challenging for the reimbursement system, particularly in Europe perhaps, to get to grips with drugs that cost such huge amounts of money upfront. And that's why the industry and also payers are beginning to think about new payment systems.
>> Glybera is now being taken off the market due to a lack of demand, and the future for Strimvelis is uncertain after GSK decided to review its rare diseases unit. But if the US and the rest of the world can get the payments right, one-off treatments like this could actually save money compared to the costs of long-term care.