
Gene Therapy Is Nearing a Breakthrough - JumpCrisscross
http://www.barrons.com/articles/gene-therapy-is-nearing-a-major-breakthrough-1506140340?shareToken=st6ea3eae0818340d09e66102bc654b51c&utm_source=newsletter&utm_medium=email&utm_campaign=newsletter_axiosam&stream=top-stories
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reasonattlm
The article focuses on inherited diseases, but that is a proving ground for
the later, larger market of enhancement. An order of magnitude more healthy
people will be willing to undergo gene therapy to gain greater muscle mass and
less fat tissue (myostatin / follistatin) or greater stem cell activity
(telomerase) than is the case for patients with serious inherited conditions
willing to undergo gene therapy.

The breakthrough waiting to take place is the development of a methodology of
reliable transfection of large enough number of cells to produce the desired
outcome in adults. That seems to be not that far away, but it isn't the case
today; the gene delivery mechanisms work, but that isn't the same thing as
succeeding in altering a sufficient number of cells every time.

This is one of the reasons why there are no clinics offering enhancement gene
therapies via medical tourism. That will happen soon, however, in a similar
pattern to that followed for stem cell therapies at the outset of their
clinical availability.

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blackbagboys
Wait, stem cell therapies are actually effective? For what? I'm only aware of
shady cosmetic clinics advertising in Mexico or Greece.

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csomar
I'm a bit suspicious of the hype + Ticker symbol. The Ticker Symbol for Spark
Therapeutics is "ONCE". It is a $3bn valued company although is doesn't look
like it has any revenue (around $1.5m) and has a huge deficit of -$74m
(probably will be higher for the next year).

Seems like I have seen this movie before...

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fgimenez
Most biotechs don't have any revenue when they go public. They are traded on
scientific progress through clinical trials. The idea being that if they do
get through a phase 3, they are immediately valuable since they have a
monopoly on a valuable therapeutic. Most likely, they get acquired by large
pharma before that point - see Kite & Gilead.

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lwhalen
When can I gene-therapy away my 'seasonal' allergies? As an otherwise-healthy
thirtysomething, that's the big one for me at this time in my life :-)

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refurb
This plus Alnylam's recent success with their interference RNA approval means
we're making amazing progress in new therapies! Exciting times.

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agumonkey
The next ethics wall is gonna be steep.

