DrBarry Byrne

CMS Newborn Screening - COQHX

Pre-IND Activities, IND Preparation, and FDA Submission Support for SPG56 (CYP2U1) Gene Therapy SinglePatient Protocol

Management of pre-existing immunity against AAV9 in humanized mice using Rozanolixizumab-noli (RYS GGO)

A phase 2, multicenter, randomized, placebo-controlled, double-blind study to investigate the safety, pharmacodynamics, and preliminary efficacy of S-606001 as an add-on to enzyme replacement therapy in patients with late-onset Pompe disease

Vector Biodistribution in Feline Tissue

A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (DMD)

PC8.1-135: Integrating High-Throughput Chromatography and Data Science in a Platform for the Rapid and Accurate Quantification of Full, Partial, and Empty AAV Capsids

MR Assessment of the effect of nNOS on Bioenergetics and Microvascular Function in Becker and Duchenne Muscular Dystrophy

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

UF Health Center for Advanced Therapeutics

Evaluation of High-throughput Extracellular Vesicle Loading Platform for Therapeutic Genome Editing INDs

Early Cardiac Biomarkers of Inflammation and Remodeling in DMD

Addressing Shortcomings of AAV GeneTherapies for the Muscular Dystrophies

A Phase 1-2, open-label, dose escalation study to evaluate the safety of 2 doses of intravenous ATA-200, an adeno-associated viral vector carrying the human gamma-sarcoglycan gene, in patients with gamma-sarcoglycanopathy (limb-girdle muscular dystrophy LGMDR5, formerly LGMD2C).

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of Delandistrogene moxeparvovec following plasmapheresis in Subjects with Duchenne Muscular Dystrophy and pre-existing Antibodies to AAVrh74 (HORIZON)

Development and optimization of a gene therapy vector for MEPAN Syndrome

MRI/MRS Markers of Skeletal Muscle Disease Progression and Vascular Function in Non-Ambulatory Individuals with DMD

Procession to IND of a capsid mutated AAV8 codon optimized NAGLU vector for treatment of Sanfilippo Syndrome type B

MDA Care Center Network Designation Award

Novel Gene Therapy for Critical Limb Ischemia and Limb Salvage in Vascular Occlusive Disease

Task Order#1 for UF #AGR00019321- Rocket Pharmaceuticals

Frataxin Potency assay in support of an AAV-mediated gene therapy treatment for Friedreich’s ataxia

Clinical Study of Anti-AAV Antibody Depletion using Efgartigimod alfa- fcab in Boys with DMD Prior to and Following AAV Gene Therapy (MDA 1020019)

A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

Clinical Study of Anti-AAV Antibody Depletion using Efgartigimod alfa- fcab in Boys with DMD Prior to and Following AAV Gene Therapy (MDA 1020019)