Human Gene Therapy - April 2023 - 253

PATHWAYS/INTERVIEW
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Interview with Barry Byrne, MD/PhD
Editor's note: Dr. Byrne is the Associate Chair of Pediatrics, the Earl and Christy Powell
University Chair in Genetics, and Director of the Powell Gene Therapy Center at the
University of Florida. This interview has been lightly edited for clarity and length.
Barry Byrne, MD/PhD
Human Gene Therapy: You have
been in the adeno-associated virus
(AAV) gene therapy field since
its inception, can you tell me
about your career path and how
you became interested in this
approach?
Dr. Byrne: I began my laboratory
with an interest in skeletomuscular
diseases and one ofmy coresidents in
Baltimore at Johns Hopkins, a scientist
well known in the field, was
Terry Flotte. Terry helped introduce
me to the AAV field when he was
working with Barrie Carter, who was
one of the pioneers in the field of
AAV gene therapy. Barrie was a
contemporary of Nick Muzyczka
and their labs together helped launch
this field that we now know as genetic
medicine. It is with great tribute
to Nick and to Barrie that the generations
that followed can be working
in this area to be helping patients
with genetically defined diseases.
Human Gene Therapy: You have
continued to work in neuromuscular
diseases, are there any ongoing
projects or clinical trials that
you are most excited about?
Dr. Byrne: We have been very involved
in Duchenne muscular dystrophy,
Pompe disease, and
Friedreich's ataxia, and those programs
have reached a stage where
this summer there might be a product
for Duchenne muscular dystrophy.
There are also a number of trials in
the Pompe space, including ones we
have initiated here at the University
of Florida and by industry sponsors.
HUMAN GENE THERAPY, VOLUME 34, NUMBERS 7 and 8
ª 2023 by Mary Ann Liebert, Inc.
There is a really robust pipeline for
the neuromuscular area. We are
working on critical problems related
to durability and issues of immunogenicity
and safety, but I think that
those problems are solvable. I really
am optimistic that we will continue
to make progress for patients and
advance the science.
Human Gene Therapy: What do
you think is the most important
challenge to address? Does the
field need better molecular tools,
or do we need to see how the clinically
approved products work in
humans?
Dr. Byrne: I think that there are two
key areas. As I mentioned, immunogenicity
is one. But key to providing
body-wide exposure to AAV in sysDOI:
10.1089/hum.2023.29239.int j 253

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
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Human Gene Therapy - April 2023 - 244
Human Gene Therapy - April 2023 - Contents
Human Gene Therapy - April 2023 - 246
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Human Gene Therapy - April 2023 - Cover3
Human Gene Therapy - April 2023 - Cover4
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