Barry J Byrne

Barry J Byrne, M.D., Ph.D.

Professor And Associate Chair

Department: MD-CHILD HEALTH RES INSTITUTE
Business Phone: (352) 294-5757
Business Email: barry.byrne@ufl.edu

About Barry J Byrne

Dr. Barry Byrne is the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. After obtaining a B.S. degree in Chemistry from Denison University, he pursued his medical education, as well as a Ph.D. in Microbiology and Immunology, at the University of Illinois. He completed his pediatric residency, cardiology fellowship training and post-doctoral training in Biological Chemistry at Johns Hopkins University. Joining the University of Florida in 1997, he has served in a variety of clinical, research and educational roles, and is now the Earl and Christy Powell University Chair in Genetics.

Dr. Byrne is currently conducting numerous clinical research studies for a variety of rare diseases, with specific attention to developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness, cardiac dysfunction and respiratory dysfunction. His research team has made significant contributions to the understanding and treatment of Pompe disease, a type of muscular dystrophy resulting from abnormal glycogen accumulation in the muscle. His current research has focused on developing new therapies using the missing cellular protein or the corrective gene to restore muscle function in Pompe, Duchenne muscular dystrophy and other inherited myopathies.

Accomplishments

UFRF Professor Award
2017-2020 · University of Florida
Faculty Research Prize in Clinical Research
2007 · University of Florida
Research Professor Award
2003-2006 · University of Florida
Clinician Scientist Award
1996 · Johns Hopkins University
Clinician Scientist Award
1994 · Johns Hopkins University
Grant Awardee
1992 · Johns Hopkins University
James Scholar
1983 · University of Illinois

Board Certifications

  • Pediatric Cardiology
    American Board of Pediatrics

Clinical Profile

Specialties
  • Pediatrics
Subspecialties
  • Cardiovascular Disease
  • Pediatric Cardiology
Areas of Interest
  • Autosomal recessive
  • Becker muscular dystrophy
  • Canavan disease
  • Duchenne muscular dystrophy
  • Echocardiogram
  • Friedreich ataxia
  • Gaucher disease
  • Heart CT scan
  • Heart MRI
  • Muscular dystrophy
  • Myasthenia gravis
  • Pediatric Cardiology
  • Pompe Disease
  • Spinal muscular atrophy

Research Profile

Areas of Interest
  • Canavan disease
  • Duchenne muscular dystrophy
  • Friedreich’s ataxia
  • Gene therapy
  • Myotubular myopathy
  • Pompe Disease
  • Rare neuromuscular disorders
  • Spinal muscular atrophy

Publications

2018 Molecular Therapy : The Journal Of The American Society Of Gene Therapy
2017 American Journal of Physiology-Lung Cellular and Molecular Physiology
2015 Molecular Therapy : The Journal Of The American Society Of Gene Therapy
2013 Respiratory Physiology & Neurobiology
2011 Molecular Genetics and Metabolism
2011 Human Molecular Genetics
2011 Molecular Genetics and Metabolism
2009 Acta Crystallographica. Section F, Structural Biology and Crystallization Communications
2009 Proceedings of the National Academy of Sciences
2008 Journal of Inherited Metabolic Disease
2006 Pediatrics American Academy of Pediatrics

Grants

Jun 2020 ACTIVE
Development of a novel gene therapy for lupus
SAREPTA THERAPEUTICS ·
Jun 2020 ACTIVE
Immune response to AAV Vector Capsid and Assessment of Eligibility for AAV-mediated Gene Therapy for Duchenne Muscular Dystrophy
DUCHENNE UK ·
May 2020 ACTIVE
Control of Breathing and Pompe Disease
NATL INST OF HLTH NICHD · Principal Investigator
Dec 2019 ACTIVE
Non-GLP Expression Study in C57BL/6 Mice
APPLIED GENETICS TECH CORP · Principal Investigator
Dec 2019 ACTIVE
Sarepta TO #1: Support of PGTC Cores
SAREPTA THERAPEUTICS · Principal Investigator
Nov 2019 ACTIVE
GLP Tissue and Shedding Biodistribution Study in Nonhuman Primates
APIC BIO · Principal Investigator
Aug 2019 ACTIVE
Development of a Novel AAV Vector Capsid Optimized for OA Gene Therapy
US ARMY MED RES ACQUISITION · Project Manager
Jul 2019 – May 2020
Sarepta Task Order #7 – Pompe Canine Colony Housing Program
SAREPTA THERAPEUTICS · Principal Investigator
Mar 2019 – Dec 2019
GLP Tissue-only Biodistribution Study in Nonhuman Primates
APIC BIO · Principal Investigator
Dec 2018 ACTIVE
MDA MOVR Data Hub Participation Agreement
MUSCULAR DYSTROPHY ASSO · Principal Investigator
Oct 2018 – Dec 2018
Analysis of Cincinnati Zoo Feline Shedding
MASS GENERAL BRIGHAM · Principal Investigator
Jul 2018 – Dec 2018
Limelight TO #1 – Optimizing Hydrodynamic Transduction Injection Procedure
LIMELIGHT BIO · Principal Investigator
Jun 2018 – Jun 2020
Solid TO #15 Evaluation of Immune Management in AAV-?Dys Gene Therapy
SOLID BIOSCIENCES INC · Principal Investigator
Apr 2018 – Apr 2020
Antisense oligonucleotide therapy for a novel humanized GAA rat model of Pompe Disease
SAREPTA THERAPEUTICS · Principal Investigator
Apr 2018 – Dec 2018
Lacerta Task Order #3 – NHP TAb Screening
LACERTA THERAPEUTICS · Project Manager
Dec 2017 ACTIVE
Analysis of Human Samples for Biodistribution and Circulating Antibodies
SOLID BIOSCIENCES INC · Principal Investigator
Nov 2017 – Jun 2018
Hex-003-004
UNIV OF MASSACHUSETTS MEDICAL SCHOOL · Principal Investigator
Oct 2017 – Mar 2019
Toxicology Study of BS01 in Rd1 mice
APPLIED GENETICS TECH CORP · Principal Investigator
Jun 2017 ACTIVE
Cure FA Research Foundation
UF FOU · Principal Investigator
May 2017 – Nov 2019
Process Development for AAV Manufacturing of AAV-CB-hFXN
CURE FA FOUNDATION · Principal Investigator
Apr 2017 ACTIVE
Breathing Research and Therapeutics (BREATHE)
NATL INST OF HLTH NHLBI ·
Mar 2017 ACTIVE
Expanded access trial of systemic delivery of aspartoacylase (rAAV0-CB6-AspA) gene vector in a single patient with canavan disease
THE LAWRENCE FAMILY · Principal Investigator
Jan 2017 – Jun 2020
MDA Care Center
MUSCULAR DYSTROPHY ASSO · Principal Investigator
Nov 2016 ACTIVE
TO #1 – Access and Transfer of rAAV9-CMV-hAADC Production and Testing Methods
PTC THERAPEUTICS ·
Sep 2016 – Jan 2019
Diaphragm pacing and rehabilitation in Pompe disease
NATL INST OF HLTH NICHD ·
Aug 2016 – Dec 2019
Solid TO#12 – Study ID:UFGTC15-007/ UFGTC16-007/UFGTC16-008
SOLID GT LLC · Principal Investigator
Aug 2016 – Dec 2019
Task Order #13 – Evaluation of SGT-01 in Neonatal NHP using Immune Modulation
SOLID GT LLC · Principal Investigator
Jul 2016 ACTIVE
CMS Newborn Screening – COQWX
FL DEPT OF HLTH CHILDRENS MED SERVS ·
Jul 2016 – Jun 2017
Solid Fellow
SOLID GT LLC · Principal Investigator
Mar 2016 – Mar 2019
POM002 LAB SCERVICES AGREEMENT – GAA ACTIVITY ASSAY AND
BIOMARIN PHARMACEUTICAL · Principal Investigator
Mar 2016 – Jul 2017
Production and Testing of rAAV9-DES-hGAA Clinical Produc
AUDENTES THERAPEUTICS · Principal Investigator
Jan 2016 – Dec 2016
Preclinical Study of Adeno-Associated Virus Mediated Gen
AUDENTES THERAPEUTICS · Principal Investigator
Oct 2015 – Mar 2019
OneFlorida Clinical Research Consortium Phase II
PATIENT-CENTERED OUTCOMES RES INST · Project Manager
Sep 2015 ACTIVE
Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease
NATL INST OF HLTH NHLBI · Principal Investigator
Sep 2015 – Mar 2017
Development therapeutic gene therapy vector for N-glycan
GRACE WILSEY FOUNDATION · Principal Investigator
Sep 2015 ACTIVE
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
NATL INST OF HLTH NIAMS · Project Manager
Sep 2015 – Aug 2018
SOLID TO #5 ASSESSMENT OF SAFETY/EFFICACY OF IV
SOLID GT LLC · Principal Investigator
Aug 2015 – Aug 2021
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy
INVENTIV HEALTH CLINICAL LLC · Principal Investigator
Aug 2015 ACTIVE
Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis and Fat
NATL INST OF HLTH NIAMS ·
Jul 2015 – Jul 2018
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
BIOMARIN PHARMACEUTICAL · Project Manager
Jul 2015 – Jun 2019
Respiratory Muscle Function in Untreated X-linked Myotub
AUDENTES THERAPEUTICS · Project Manager
May 2015 – May 2016
Request for Support to Plan Clinical Gene Therapy Progra
BARTH SYNDROME FOU · Principal Investigator
Apr 2015 – Apr 2018
A Study of Respiratory Muscle Strength
COVANCE INC · Project Manager
Apr 2015 – Dec 2016
Project Development Planning
SOLID GT LLC · Principal Investigator
Feb 2015 – Feb 2016
Pompe Gene Therapy
AUDENTES THERAPEUTICS · Principal Investigator
Dec 2014 – Dec 2016
AAV9 Pompe Rodent Study
AUDENTES THERAPEUTICS · Principal Investigator
Dec 2014 – Dec 2017
Recensus
AUDENTES THERAPEUTICS · Principal Investigator
Dec 2014 ACTIVE
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreichs Ataxia
TRIAL RUNNERS LLC · Project Manager
Oct 2014 – Jun 2023
An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
SAREPTA THERAPEUTICS · Principal Investigator
Sep 2014 – Jun 2018
Development of a Strength Training Protocol in Duchenne
NATL INST OF HLTH NIAMS · Project Manager
Sep 2014 – Jun 2017
A Pilot Study of the Effects of Oral Administration of Zavesca on Anti-rhGAA Immune Response in Subjects with Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
AMICUS THERAPEUTICS · Principal Investigator
May 2014 – May 2022
NEOGAA Extension Study / LTS13769
SANOFI US SERVICES · Principal Investigator
Mar 2014 – Feb 2017
Response to Diaphragmatic Pacing in Subjects with Pompe
ACID MALTASE DEFICIENCY ASSO · Project Manager
Jan 2014 – Dec 2018
BSF Human Material Storage
BARTH SYNDROME FOU · Principal Investigator
Nov 2013 – Nov 2016
A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) in rhGAA Exposed Subjects with Late-onset Pompe Disease
BIOMARIN PHARMACEUTICAL · Principal Investigator
Sep 2013 – Aug 2018
The relationship between genomic variants and MRI/MRS…
NATL INST OF HLTH NIAMS · Project Manager
Aug 2013 – Aug 2016
Tadalafil
ELI LILLY AND CO · Principal Investigator
Aug 2013 – Feb 2018
Advanced Gene Therapy for Treatment of Cardiomyopathy and Respiratory Insufficiency in Duchenne Muscular Dystrophy
US ARMY MED RES ACQUISITION · Principal Investigator
Aug 2013 – Mar 2016
Phase I/II Trial of Diaphragm Delivery of Recombinant Ad
SOCIAL AND SCIENTIFIC SYSTEMS · Principal Investigator
Feb 2013 – Jun 2016
Development of Clinical Translational Studies
AUDENTES THERAPEUTICS · Principal Investigator
Sep 2012 – Jun 2018
Control of Breathing and Pompe Disease
NATL INST OF HLTH NICHD · Principal Investigator
Sep 2012 – Sep 2017
MD110050 Optical Imaging of Dystrophic and Damaged Muscle
US DEPT OF DEFENSE · Project Manager
Sep 2012 – Aug 2018
Becker Muscular Dystrophy – A Natural History Study to Predict Efficacy of Exon Skipping
CHILDRENS RESEARCH INSTITUTE · Principal Investigator
Jul 2012 ACTIVE
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research – F017885/6
UF FOU · Principal Investigator
Jul 2012 ACTIVE
Fox Research Fund
UF FOU · Principal Investigator
Jul 2012 ACTIVE
Powell Gene Therapy Fund
UF FOU · Principal Investigator
Jul 2012 ACTIVE
Sunshine Children's Fund
UF FOU · Principal Investigator
Jun 2012 – May 2017
Heart and Skeletal Muscle Metabolism, Energetics and Function in Barth Syndrome
WASHINGTON UNIV · Principal Investigator
May 2012 – May 2018
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-US-201
SAREPTA THERAPEUTICS · Principal Investigator
Mar 2012 ACTIVE
UFRCC for Cardiovascular Cell Therapy Research Network
NATL INST OF HLTH NHLBI ·
Feb 2012 – Feb 2018
A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale
GENZYME CORP · Principal Investigator
Dec 2011 – Dec 2019
Neuromuscular Disease/Clinical Trial Support Fund
UF FOU · Principal Investigator
Aug 2011 – Apr 2017
A Long Term Study for Extended BMN 701 Treatement of Pat
BIOMARIN PHARMACEUTICAL · Principal Investigator
Apr 2011 – Apr 2022
A Long-term Study to Evaluate Growth and Development Outcomes in Patients with Infantile-Onset Pompe Disease Who Are Receiving Myzoyme (alglucosidase alfa)
GENZYME CORP · Principal Investigator
Nov 2010 – Oct 2018
McGollie – Longitudinal, Observations Study of Subjects
UF FOU · Principal Investigator
Dec 2009 – May 2021
Data Registry Services
GENZYME CORP · Principal Investigator
Jul 2007 – Aug 2016
CTSI
UF RESEARCH FOU · Project Manager

Patents

Issued November 2000
Method of Preparing Recombinant AAV Compositions
#6,146,874
Issued October 2002
Materials and Methods for Gene Therapy
#6,461,606
Published October 2002
rAAV Compositions for Gene Therapy
#US-2003-0082162-A1
Published January 2003
Adeno-Associated Viral Vectors for the Treatment and Prevention of Diabetes
#US-2003-0095949-A1
Published April 2003
Treatment for Pompe Disease
#US-2003-0223966-A1
Published June 2003
Production of Pseudotyped Recombinant AAV Virions
#2007-0015238-A1
Issued December 2003
Method of Preparing Recombinant Adeno-Associated Virus Compositions
#6,660,514
Published December 2003
Methods of Preparing Recombinant Adeno-Associated Virus Compositions
#2004/0121444-A1
Issued August 2004
Methods for Large-Scale Production of Recombinant AAV Vectors
#6,783,972
Published February 2005
Gel-based Delivery of Recombinant Adeno-Associated Virus Vectors
#US2006/0078542 A1
Published August 2006
High Titer Recombinant AAV Production
#US-2007-0202587
Issued August 2006
High Titer Recombinant AAV Production
#7,091,029
Issued August 2006
Production of Pseudotyped Recombinant AAV Virions
#7,094,604
Published October 2007
Adeno-Associated Viral Vectors for the Treatment and Prevention of Diabetes (CON)
#US-2009-0186002-A1
Published April 2008
Gel-Based Delivery of Recombinant Adeno-Associated Virus Vectors
#US2008/0279945-A1
Published April 2010
Compositions and Methods for Treating Glycogen Storage Diseases
#US2010/0221225 A1
Published June 2012
Recombinant Adeno-Associated Virus Vector Therapy for Treatment of the Neuromuscular Junction
#US-2012-0322861-A1
Issued October 2012
A Self-Complementary Adeno-Associated Virus Having a Truncated CMV-Chicken B-Actin Promoter
#8,298,818
Published November 2012
High Titer Recombinant AAV Production
#US-2013-0171719
Published December 2014
Recombinant Adeno-Associated Virus Vector Therapy for Treatment of the Neuromuscular Junction (CIP)
#US-2015-0196671-A1
Published April 2015
Methods of Permitting a Subject to Receive Multiple Doses of Recombinant Adeno-Associated Vrus
#US2017/0049887
Published July 2015
Novel Method for Purification of Raav9 by Ion Exchange Chromatography and Sodium Citrate and Citric Acid Buffers
#US2017/0130208
Published October 2015
High Titer Recombinant AAV Production
#US 2016-0024480 A1
Published January 2016
Compositions and Methods for Treating Diseases (CON)
#US2018/005120-A9
Published April 2016
Recombinant Adeno-Associated Virus Vector Therapy for Treatment of the Neuromuscular Junction (CON)
#US2017/0028002
Published December 2016
Methods of Packaging Multiple Aden-Associated Virus Vectors
#US2017/0218395
Published February 2017
Recombinant AAV Production in Mammalian Cells
#US17/0362577
Issued May 2017
Adeno-Associated Virus Vectors for Treatment of Glycogen Storage Disease
#9,644,216

Education

Postdoctoral Fellowship – Biological Chemistry
1987-1991 · Johns Hopkins University
Clinical Fellowship – Pediatric Cardiology
1987-1991 · Johns Hopkins University
Residency – Pediatrics
1984-1987 · The Johns Hopkins Hospital
Medical Degree
1978-1984 · University of Illinois
Doctor of Philosophy – Microbiology & Immunology
1978-1984 · University of Illinois

Contact Details

Phones:
Business:
(352) 294-5757
Emails:
ADMINISTRATIVE SPECIALIST III:
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