
The first commercial gene therapy to provide an outright cure - jerryhuang100
https://www.technologyreview.com/s/601390/gene-therapys-first-out-and-out-cure-is-here/
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Apfel
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 -
[http://thelancet.com/journals/langlo/article/PIIS2214-109X(1...](http://thelancet.com/journals/langlo/article/PIIS2214-109X\(15\)00156-4/fulltext))

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synapticfire
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. [https://avexis.com](https://avexis.com)

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. [http://www.curesma.org/sma/about-
sma/](http://www.curesma.org/sma/about-sma/)

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Frozenlock
As one of the unlucky ones with this disease, I'm very happy to see some
advances made in the field. (Tho I've been hearing about promising advances
for decades, without ever seeing any concrete result...)

Did you find other organizations working on gene therapy for SMA?

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synapticfire
I know of no other gene therapies, but other treatments are very real, and
very close to being approved. My son is in a trial of nusinersen with Ionis
Pharmaceuticals. The results of this drug are astounding.

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Frozenlock
Thank you very much!

I contacted the company and am now waiting for a response.

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baldfat
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

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)

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.
[https://en.wikipedia.org/wiki/Gattaca](https://en.wikipedia.org/wiki/Gattaca)

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.

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greggman
I know Gattaca is a favorite of many people I had a fairly negative reaction
to it.

Vincent has bad genes and he's trying to get in to the Space Program. That's
not Sci-Fi, that's the actual Space Program, at least NASA's and always has
been. As one example, bad eyes genes? No space travel for you!

And why shouldn't it be that way? It costs billions of dollars to put people
in space. Seems like it would be irresponsible not to stack the deck toward
success from every possible dimension. Sure some guy with a genetically
diagnosed bad heart has some chance his heart will hold out but it seems
practically criminal for him to risk the mission and all his fellow astronauts
lives on a known diagnosis of high risk.

Even today AFAIK US fighter pilots also effectively have gene based selection.

Although hey, to keep it relevant it sure would be awesome if they could
correct the genes as an adult. Need 20/20 vision get this gene therapy. Need a
good heart get that gene therapy...

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dragonwriter
Actually, the real current space program doesn't exclude people with bad
genotype, it excludes people with bad phenotype or inadequated demonstrated
abilities. (Which are more or less loosely correlated with genetics, but not
the same thing as genetics.)

> Even today AFAIK US fighter pilots also effectively have gene based
> selection.

No, again, its phenotype and abilities, not genotype, on which selection is
based.

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ianai
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.

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fapjacks
And I will happily dump most of my income into whatever they're hawking.
Nobody _chooses_ to get old. It's forced upon us in the most awkward way. I
will happily pay someone to prevent (or cure) aging in myself, and shift the
time of my death to a time of my choosing.

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isoprophlex
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?

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Natanael_L
Depends on the disease. Disclaimer, I'm not a doctor or researcher! YMMV

Those diseases with _active_ bad genes controlling the behavior of cells that
are replaced at a fast rate would be more likely to effectively disappear
after just the treatment, such as some blood or skin diseases.

Those who changes how the body grows (like say skeleton growth or other cells
with slow replacement) would still leave the body in the condition it was in
just before the treatment. They would effectively be chronic unless you can
simultaneously fix the existing faults using for example surgery or a
secondary therapy.

But they wouldn't continue to deteriorate in the cases where the continued
activity of the bad gene drives the deterioration.

And syndromes caused by genes that only had to be active once (like in
childhood) and now is dormant won't get fixed by gene therapy (but it MIGHT
prevent it from being hereditary, if properly propagated in the body)

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amelius
Question: can this type of gene therapy be reversed?

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maxerickson
The procedure in the article could not be repeated to reverse the effect. Once
the altered stem cells take hold in the body it would be necessary to destroy
them to reverse the effect.

It could later become possible to undo the procedure using some method that
was able to alter the cells in the body.

