Charles O'Neill

Accreditations

Dr. O'Neill serves as a member of the Executive Board and provides consulting on many aspects of vision science with a specific focus on study design and regulatory requirement considerations for IND submission.  Dr. O'Neill is an Assistant Adjunct Professor in the Pharmacology and Toxicology Graduate Group at University of California-Davis. He has contributed to 26 regulatory submissions in the US, EU, Australia and Japan, coauthored over 40 peer-reviewed publications, and been a Diplomate of the American Board of Toxicology since 1995. With 14 years of biotechnology and pharmaceutical development experience, Dr. O'Neill has led non-clinical development efforts regarding anti-angiogenic molecules including the targeted oncology monoclonal antibody AvastinTM, and the intra-vitreally delivered monoclonal antibody fragment LucentisTM, a treatment for the neovascular form of age-related macular degeneration.

Recent Publications

2023

Safety Findings of Dosing Gene Therapy Vectors in NHP With Pre-existing or Treatment-Emergent Anti-capsid Antibodies
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Safety, pharmacokinetics and CNS distribution of tralesinidase alfa administered via intracerebroventricular infusion to juvenile cynomolgus monkeys
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2022

Brain Alterations in Aged OVT73 Sheep Model of Huntington's Disease: An MRI Based Approach
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Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB
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Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy
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Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers
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2021

Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease
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2020

Intravitreal enzyme replacement inhibits progression of retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis
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Intravitreal enzyme replacement preserves retinal structure and function in canine CLN2 neuronal ceroid lipofuscinosis
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Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B
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2019

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy
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<em>Ex Vivo</em> Gene Therapy: Graft-versus-host Disease (GVHD) in NSG™ (NOD.Cg-Prkdc<sup>scid</sup> Il2rg<sup>tm1Wjl</sup>/SzJ) Mice Transplanted with CD34<sup>+</sup> Human Hematopoietic Stem Cells
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2018

Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study
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Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice
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2017

Maternal phenylketonuria syndrome: studies in mice suggest a potential approach to a continuing problem
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Partial rescue of neuropathology in the murine model of PKU following administration of recombinant phenylalanine ammonia lyase (pegvaliase)
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2016

Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study
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Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease
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Antibodies that neutralize cellular uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndrome
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Workshop Proceedings: Streamlined Development of Safety Assessment Programs Supporting Orphan/Rare Diseases-Are We There Yet?
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Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6months of therapy in cats using different IV infusion durations
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2015

Effects of Food Intake on the Relative Bioavailability of Amifampridine Phosphate Salt in Healthy Adults
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Genetic variation in aryl N-acetyltransferase results in significant differences in the pharmacokinetic and safety profiles of amifampridine (3,4-diaminopyridine) phosphate
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Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism
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2014

Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial
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Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis
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Pharmacokinetic and pharmacodynamic evaluation of elosulfase alfa, an enzyme replacement therapy in patients with Morquio A syndrome
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Enzyme replacement therapy delays pupillary light reflex deficits in a canine model of late infantile neuronal ceroid lipofuscinosis
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Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease)
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Recombinant human tripeptidyl peptidase-1 infusion to the monkey CNS: safety, pharmacokinetics, and distribution
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2012

Corneal inflammation is inhibited by the LFA-1 antagonist, lifitegrast (SAR 1118)
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Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia
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Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats
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2011

Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis
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Bi-modal dose-dependent cardiac response to tetrahydrobiopterin in pressure-overload induced hypertrophy and heart failure
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Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations
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The pharmacologic assessment of a novel lymphocyte function-associated antigen-1 antagonist (SAR 1118) for the treatment of keratoconjunctivitis sicca in dogs
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2010

Delivery of SAR 1118 to the retina via ophthalmic drops and its effectiveness in a rat streptozotocin (STZ) model of diabetic retinopathy (DR)
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Safety and pharmacokinetics of a novel lymphocyte function-associated antigen-1 antagonist ophthalmic solution (SAR 1118) in healthy adults
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Tetrahydrobiopterin supplementation reduces atherosclerosis and vascular inflammation in apolipoprotein E-knockout mice
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2009

Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen
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2003

Characterization of a Monkey Model of Laser-Induced Choroidal Neovascularization (CNV)
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26-Week Intravitreal Injection Toxicity Study with rhuFab VEGF in Cynomolgus Monkeys with an 8-Week Recovery
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2001

Safety Evaluation of Differing Schedules of Intravitreal Administration of rhuFab VEGF in Cynomolgus Monkeys for 2 Months
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2000

Safety Evaluation of Intravitreal Administration of rhuFab VEGF in Cynomolgus Monkeys for 3 Months
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