Yes. The gene therapy approved last year that is relevant to me has
been in trial for....12 years? I knew about it, vaguely. I wasn't
eligible for the trials because I'd had a traumatic retinal
detachment, but I'd hear something about it every year or two and
think oh yeah. They're still at that? But never actually
expecting anything to come of it.
And then last winter there I was, minding my own business taking a
bath and listening to an economics pocast. And in passing one of the
hosts mentioned this brand new, first of its kind gene therapy for a
rare eye condition. And I thought what are the odds? But yeah,
there it was. Only applicable to a very specific mutation that 8% of
the diagnosed population has, but. It's real and it works. Apparently
they picked this one rare thing because it was, for some reason beyond
me, a simplified method to work out how to do this, thus clearing the
way for their bigger target, hemophilia. Which they have also cured, I
believe.
But yeah. It's uncomfortable knowing we are on the brink of a huge
explosion in this field and not knowing how to scope what that could
be. And how long it will take. And what it's limitations will be. But
it's definitely coming now, that's not science fiction anymore.
no subject
Yes. The gene therapy approved last year that is relevant to me has been in trial for....12 years? I knew about it, vaguely. I wasn't eligible for the trials because I'd had a traumatic retinal detachment, but I'd hear something about it every year or two and think oh yeah. They're still at that? But never actually expecting anything to come of it.
And then last winter there I was, minding my own business taking a bath and listening to an economics pocast. And in passing one of the hosts mentioned this brand new, first of its kind gene therapy for a rare eye condition. And I thought what are the odds? But yeah, there it was. Only applicable to a very specific mutation that 8% of the diagnosed population has, but. It's real and it works. Apparently they picked this one rare thing because it was, for some reason beyond me, a simplified method to work out how to do this, thus clearing the way for their bigger target, hemophilia. Which they have also cured, I believe.
But yeah. It's uncomfortable knowing we are on the brink of a huge explosion in this field and not knowing how to scope what that could be. And how long it will take. And what it's limitations will be. But it's definitely coming now, that's not science fiction anymore.