Gene Therapy Is Nearing a Breakthrough

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.

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...

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 :-)

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