As the article mentions, the pricing of this will be extremely interesting. The outright Hep C cure (Sofosbuvir/Sovaldi) was one of the big stories in pharmacoeconomics last year, which is very interestingly priced globally (Nice paywalled article discussing this - <a href="http://thelancet.com/journals/langlo/article/PIIS2214-109X(15)00156-4/fulltext" rel="nofollow">http://thelancet.com/journals/langlo/article/PIIS2214-109X(1...</a>)
Another promising gene therapy, close to being submitted to the FDA for approval, has been developed by Ohio State University, Nationwide Children's hospital, and Avexis for the treatment, and possibly cure, of Spinal Muscular Atrophy.
<a href="https://avexis.com" rel="nofollow">https://avexis.com</a><p>If you have not heard of SMA, you should look it up. I had not heard of it until my 2 month old son was diagnosed.
<a href="http://www.curesma.org/sma/about-sma/" rel="nofollow">http://www.curesma.org/sma/about-sma/</a>
The future is now - 1) Self-driving cars 2) Flights to Mars in the next 10 years 3) Gene Therapy and Crisper 4) Robots are actual real world discussions<p>Happily no flying cars (We would all die), No AI yet (In the future AI I love and respect you and welcome your presence in our world)<p>Gattaca - The film presents a biopunk vision of a future society driven by eugenics where potential children are conceived through genetic manipulation to ensure they possess the best hereditary traits of their parents. The film centers on Vincent Freeman, played by Hawke, who was conceived outside the eugenics program and struggles to overcome genetic discrimination to realize his dream of traveling into space. <a href="https://en.wikipedia.org/wiki/Gattaca" rel="nofollow">https://en.wikipedia.org/wiki/Gattaca</a><p>This is just the crack in the wall. Philosophy and Ethical questions will make this one of the biggest stories for the next decade or more.
Here's how you price & sell this. I figure the long term cost structure of all/most gene therapies diminish rapidly with each additional cure. i.e. The first two are prohibitively expensive to develop. The next two will be less, potentially a TON less. What a big pharma like GSK does is spread their cost of R&D across all future incomes of future genetic treatments. Then develop genetic treatments for anything and everything that can be treated genetically. So you start with the rare things, like here. Then you go for the cancers and things that have many more patients. Over time you wind up with, say, cures for things like male pattern baldness. Things where there are millions of highly willing to pay individuals. In the longer term they're suddenly selling a product multiple times to nearly everyone. Suddenly they're not selling the cure for a rare condition, but selling a gene 'vehicle' - possibly at used or new car prices.
I wonder how this scales to other single-defect diseases? After the initial R&D, do you just swap out one sequence of genetic material for another in order to repair other diseases?