Human Gene Therapy - April 2023 - 254

254
INTERVIEW
temic dosing is manufacturing capacity.
Although we have a somewhat
feasible level of manufacturing, these
are relatively small patient populations
and the cost is still extremely
high. As we improve manufacturing
technology and purity of the products,
we hope that there will be an enhanced
safety profile. For some conditions
such as Duchenne with
potential for early diagnosis, there is a
consideration for the need to readminister
products. There are two parts
to immunogenicity issues: one is the
acute toxicities that have been observed
with systemic dosing of AAV
and the second is for long-term access
and maintenance of the expression
level that is needed to get a clinically
meaningful benefit. That will require
re-exposure, and so we are working
very hard to establish a mechanism
for redosing and maintaining patients,
for example, who are in current clinical
trials, to be eligible for future
clinical products.
I do think that the field has helped
move us along in terms of understanding
the third generation of
capsid biology through molecular
evolution. These techniques have
yielded really exciting new opportunities
for improving efficacy and
possibly lowering manufacturing
needs. We know that many of these
variants of AAV that are engineered
through directed evolution have less
manufacturability than their wildtype
counterparts. There is an urgent
need to improve manufacturing and
lower costs to improve access around
the world. Many of the products that
we see coming to market now will
only be accessible in the United
States because of their costs.
Human Gene Therapy: In the
context of this issue being dedicated
to the life and career of
Nick Muzyczka, people who are
new to gene therapy only know
someone like Nick through the
articles that they have published.
Could you tell me about how you
knew Nick and your experiences
with him?
Dr. Byrne: Nicholas Muzyczka was
the founding director of the Gene
Therapy Center at the University of
Florida. Then he served as the interim
director of the Genetics Center.
He was a great mentor to the
many people who came through this
program, and now have established
programs of their own. He was at
the nidus for learning and discovery;
he trained individuals such as Jude
Samulski, Terry Flotte, myself, and
we have now another generation
coming along to move the field to
the next level.

Human Gene Therapy - April 2023

Table of Contents for the Digital Edition of Human Gene Therapy - April 2023

Contents
Human Gene Therapy - April 2023 - CT1
Human Gene Therapy - April 2023 - CT2
Human Gene Therapy - April 2023 - Cover1
Human Gene Therapy - April 2023 - Cover2
Human Gene Therapy - April 2023 - 239
Human Gene Therapy - April 2023 - 240
Human Gene Therapy - April 2023 - 241
Human Gene Therapy - April 2023 - 242
Human Gene Therapy - April 2023 - 243
Human Gene Therapy - April 2023 - 244
Human Gene Therapy - April 2023 - Contents
Human Gene Therapy - April 2023 - 246
Human Gene Therapy - April 2023 - 247
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Human Gene Therapy - April 2023 - Cover3
Human Gene Therapy - April 2023 - Cover4
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