WEBVTT

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- And if we can define

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how the regulators will approve
the manufacturing of

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these drugs, it sort of becomes a platform

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and it makes it a lot
easier for the next company.

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- The Bespoke Gene Therapy

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Consortium is one that you're leading.

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It's a public-private partnership
in order to provide access to
treatment for rare diseases.

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Tell us a little bit
about that consortium.

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- Well, you know, rare
diseases in isolation are rare--

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by definition, of course.

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But in aggregate they're common

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because there are so many rare diseases,

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and an increasing number of
them appear to be amenable

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to gene therapies. When you're
only studying it in a few

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people and you can't really do
a, a gold standard, you know,

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phase three randomized clinical trial.

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And it creates a lot of uncertainty

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and worry among investors as to whether

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or not the company

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that's working on a specific
therapy is ever going

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to get to the finish line.

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So the Gene Therapy Consortium

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or the the Bespoke Gene
Therapy Consortium was really

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designed to create a kind

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of standard approach to the
manufacturing of vectors.

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And if we can define

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how the regulators will
approve the manufacturing

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of these drugs, it
sort of becomes a platform

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and it makes it a lot
easier for the next company.

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We're not inventing each time a new wheel.

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Same thing at the other end.

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Once we kind of have a
regulatory path toward

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how we do approve drugs for rare diseases,

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then everyone benefits.

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And in fact, just a couple of weeks ago,

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we published the first playbook
on gene therapy, working

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with the whole consortium
of the 33 partners

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that are involved in this
effort, including the FDA,

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and really worked out some

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of the early lessons
learned about manufacturing

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and regulatory approvals.