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-: CMC has been an ongoing challenge,
particularly around the consistency
and scaling of these processes.
I'd be interested to hear, in addition
to any general observations you have
about how companies address these issues,
is there anything that you'd like to see them do differently
about how they engage with FDA in this area?
-: Yeah, I'm not sure whether it's how they engage.
It's how we as a field might engage,
which is that if we could leverage
a given vector and reuse it
so that we weren't reinventing the manufacturing process
every time a new therapy came along,
we might be a lot better off.
Because right now,
I can say
from the cheap seats,
it looks like there's a lot of time spent
and a lot of effort on the secret sauce of manufacturing.
Whereas to me, it seems like
the real brainpower, the innovation here, should be
in the actual constructs of what we're actually doing
to address the disease on the business end:
so what's addressing the disease,
rather than how you're going to make it.
Something is more valuable if the probability
of success is higher.
And if you stay within these four walls
of how you do your development program
for your academic gene therapy,
you will allow us to know that we have a higher probability
of success in transferring your process into ours.
And therefore, the asset inherently has more value.
So I think if industry came together and thought about that,
it could really help this move forward.
Because
I think then a lot of the academic investigators
would have the incentive that they need to come
into using a certain set of processes.
Whereas right now, it's kind of like "Hey, I'm going
to do it the way I want to, and tough luck, industry.
You want a gene therapy for this enzyme deficiency.
You're just going to have to adapt the way I've come to it."
No, maybe the way to come at this is,
"Look, if you want to have an asset
as an academic investigator that's really valuable to us,
you do it in a certain way so that when we pick it up,
we can actually run with it."
And we'll compensate you for that,
the tech transfer piece of it, which I'm not the expert in.
There's got to be some way to incentivize it on that end.
That comes, by the way,
from experience. When I was in cross-academic medicine,
and when I was in industry,
as much as we would get really excited
about clinical results,
the things that usually were
some of the biggest speed bumps were not clinical results.
They were that you couldn't make the thing.
If you had it, you couldn't make it.
And that's a big problem.