- And if we can define
how the regulators will approve the manufacturing of
these drugs, it sort of becomes a platform
and it makes it a lot easier for the next company.
- The Bespoke Gene Therapy
Consortium is one that you're leading.
It's a public-private partnership in order to provide access to treatment for rare diseases.
Tell us a little bit about that consortium.
- Well, you know, rare diseases in isolation are rare--
by definition, of course.
But in aggregate they're common
because there are so many rare diseases,
and an increasing number of them appear to be amenable
to gene therapies. When you're only studying it in a few
people and you can't really do a, a gold standard, you know,
phase three randomized clinical trial.
And it creates a lot of uncertainty
and worry among investors as to whether
or not the company
that's working on a specific therapy is ever going
to get to the finish line.
So the Gene Therapy Consortium
or the the Bespoke Gene Therapy Consortium was really
designed to create a kind
of standard approach to the manufacturing of vectors.
And if we can define
how the regulators will approve the manufacturing
of these drugs, it sort of becomes a platform
and it makes it a lot easier for the next company.
We're not inventing each time a new wheel.
Same thing at the other end.
Once we kind of have a regulatory path toward
how we do approve drugs for rare diseases,
then everyone benefits.
And in fact, just a couple of weeks ago,
we published the first playbook on gene therapy, working
with the whole consortium of the 33 partners
that are involved in this effort, including the FDA,
and really worked out some
of the early lessons learned about manufacturing
and regulatory approvals.