Charles A. ONeill, PhD

Dr. O'Neill is 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


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Partial rescue of neuropathology in the murine model of PKU following administration of recombinant phenylalanine ammonia lyase (pegvaliase).

Mol Genet Metab. 2017 Apr 29;:

Authors: Goldfinger M, Zeile WL, Corado CR, O'Neill CA, Tsuruda LS, Laipis PJ, Cooper JD


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Long-Term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study.

Clin Ther. 2016 Dec 09;:

Authors: Long B, Tompkins T, Decker C, Jesaitis L, Khan S, Slasor P, Harmatz P, O'Neill CA, Schweighardt B

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Reveglucosidase alfa (BMN 701), an IGF 2 Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease.

J Pharmacol Exp Ther. 2016 Nov 16;:

Authors: Peng J, Dalton J, Butt M, Tracy K, Kennedy D, Haroldsen P, Cahayag R, Zoog S, O'Neill CA, Tsuruda L

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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.

J Immunol Methods. 2016 Oct 24;:

Authors: Melton A, Soon R, Tompkins T, Long B, Schweighardt B, Qi Y, Vitelli C, Bagri A, Decker C, O'Neill CA, Zoog SJ, Jesaitis L

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Workshop Proceedings: Streamlined Development of Safety Assessment Programs Supporting Orphan/Rare Diseases-Are We There Yet?

Int J Toxicol. 2016 Jul;35(4):393-409

Authors: Allamneni KP, Parker S, O'Neill CA, Wright TL, King S, Andrews L


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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.

Mol Genet Metab. 2015 Oct 21;

Authors: Ruane T, Haskins M, Cheng A, Wang P, Aguirre G, Knox VW, Qi Y, Tompkins T, O'Neill CA

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Effects of Food Intake on the Relative Bioavailability of Amifampridine Phosphate Salt in Healthy Adults.

Clin Ther. 2015 Jul 1;37(7):1555-63

Authors: Haroldsen PE, Musson DG, Hanson B, Quartel A, O'Neill CA

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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.

Pharmacol Res Perspect. 2015 Feb;3(1):e00099

Authors: Haroldsen PE, Garovoy MR, Musson DG, Zhou H, Tsuruda L, Hanson B

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Neutral endopeptidase resistant C-type natriuretic peptide (CNP) variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism.

J Pharmacol Exp Ther. 2015 Feb 3;

Authors: Wendt DJ, Dvorak-Ewell M, Bullens S, Lorget F, Bell SM, Peng J, Castillo S, Aoyagi-Scharber M, O'Neill CA, Krejci P, Wilcox WR, Rimoin DL, Bunting S


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Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial.

Clin Ther. 2014 Dec 5;

Authors: Schweighardt B, Tompkins T, Lau K, Jesaitis L, Qi Y, Musson DG, Farmer P, Haller C, Shaywitz AJ, Yang K, O'Neill CA

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Pharmacokinetic and Pharmacodynamic Evaluation of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients with Morquio A Syndrome.

Clin Pharmacokinet. 2014 Sep 19;

Authors: Qi Y, Musson DG, Schweighardt B, Tompkins T, Jesaitis L, Shaywitz AJ, Yang K, O'Neill CA

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Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis.

Mol Genet Metab. 2014 Sep 16;

Authors: Vuillemenot BR, Kennedy D, Cooper JD, Wong AM, Sri S, Doeleman T, Katz ML, Coates JR, Johnson GC, Reed RP, Adams EL, Butt MT, Musson DG, Henshaw J, Keve S, Cahayag R, Tsuruda LS, O'Neill CA

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Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease).

J Neurosci Res. 2014 Jun 17;

Authors: Katz ML, Coates JR, Sibigtroth CM, Taylor JD, Carpentier M, Young WM, Wininger FA, Kennedy D, Vuillemenot BR, O'Neill CA

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Enzyme replacement therapy delays pupillary light reflex deficits in a canine model of late infantile neuronal ceroid lipofuscinosis.

Exp Eye Res. 2014 Jun 19;125C:164-172

Authors: Whiting RE, Narfström K, Yao G, Pearce JW, Coates JR, Castaner LJ, Jensen CA, Dougherty BN, Vuillemenot BR, Kennedy D, O'Neill CA, Katz ML

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Recombinant human tripeptidyl peptidase-1 infusion to the monkey CNS: Safety, pharmacokinetics, and distribution.

Toxicol Appl Pharmacol. 2014 Mar 15;277(1):49-57

Authors: Vuillemenot BR, Kennedy D, Reed RP, Boyd RB, Butt MT, Musson DG, Keve S, Cahayag R, Tsuruda LS, O'Neill CA


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Evaluation of the Therapeutic Potential of a CNP Analog in a Fgfr3 Mouse Model Recapitulating Achondroplasia.

Am J Hum Genet. 2012 Dec 7;91(6):1108-14

Authors: Lorget F, Kaci N, Peng J, Benoist-Lasselin C, Mugniery E, Oppeneer T, Wendt DJ, Bell SM, Bullens S, Bunting S, Tsuruda LS, O'Neill CA, Di Rocco F, Munnich A, Legeai-Mallet L

Corneal Inflammation Is Inhibited by the LFA-1 Antagonist, Lifitegrast (SAR 1118).

J Ocul Pharmacol Ther. 2012 Dec 5;

Authors: Sun Y, Zhang R, Gadek TR, O'Neill CA, Pearlman E

Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats.

Pediatr Res. 2012 Jan;71(1):39-45

Authors: Auclair D, Finnie J, Walkley SU, White J, Nielsen T, Fuller M, Cheng A, O'Neill CA, Hopwood JJ


Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis.

Mol Genet Metab. 2011 Nov;104(3):325-37

Authors: Vuillemenot BR, Katz ML, Coates JR, Kennedy D, Tiger P, Kanazono S, Lobel P, Sohar I, Xu S, Cahayag R, Keve S, Koren E, Bunting S, Tsuruda LS, O'Neill CA

Bi-modal dose-dependent cardiac response to tetrahydrobiopterin in pressure-overload induced hypertrophy and heart failure.

J Mol Cell Cardiol. 2011 Oct;51(4):564-9

Authors: Moens AL, Ketner EA, Takimoto E, Schmidt TS, O'Neill CA, Wolin MS, Alp NJ, Channon KM, Kass DA

Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations.

Mol Genet Metab. 2011 Jul;103(3):268-74

Authors: Vite CH, Wang P, Patel RT, Walton RM, Walkley SU, Sellers RS, Ellinwood NM, Cheng AS, White JT, O'Neill CA, Haskins M

The pharmacologic assessment of a novel lymphocyte function-associated antigen-1 antagonist (SAR 1118) for the treatment of keratoconjunctivitis sicca in dogs.

Invest Ophthalmol Vis Sci. 2011 May;52(6):3174-80

Authors: Murphy CJ, Bentley E, Miller PE, McIntyre K, Leatherberry G, Dubielzig R, Giuliano E, Moore CP, Phillips TE, Smith PB, Prescott E, Miller JM, Thomas P, Scagliotti R, Esson D, Gadek T, O'Neill CA

Safety and pharmacokinetics of a novel lymphocyte function-associated antigen-1 antagonist ophthalmic solution (SAR 1118) in healthy adults.

J Ocul Pharmacol Ther. 2011 Feb;27(1):99-104

Authors: Semba CP, Swearingen D, Smith VL, Newman MS, O'Neill CA, Burnier JP, Haughey DB, Gadek TR


Delivery of SAR 1118 to the retina via ophthalmic drops and its effectiveness in a rat streptozotocin (STZ) model of diabetic retinopathy (DR).

Invest Ophthalmol Vis Sci. 2010 Oct;51(10):5198-204

Authors: Rao VR, Prescott E, Shelke NB, Trivedi R, Thomas P, Struble C, Gadek T, O'Neill CA, Kompella UB

Tetrahydrobiopterin supplementation reduces atherosclerosis and vascular inflammation in apolipoprotein E-knockout mice.

Clin Sci (Lond). 2010 Aug;119(3):131-42

Authors: Schmidt TS, McNeill E, Douglas G, Crabtree MJ, Hale AB, Khoo J, O'Neill CA, Cheng A, Channon KM, Alp NJ

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

Mol Genet Metab. 2010 Feb;99(2):132-41

Authors: Auclair D, Finnie J, White J, Nielsen T, Fuller M, Kakkis E, Cheng A, O'Neill CA, Hopwood JJ