Presentations and Publications

Media

Presentations & Publications

October 11, 2019

Key Takeaways From The RGX-314 Phase I/IIa Clinical Trial For Wet AMD (Cohorts 1-5)

Jeffrey Heier, MD – Ophthalmic Consultants of Boston

October 26, 2018

Six Month Results of the Phase I Study to Evaluate Safety & Tolerability of RGX-314 Gene Therapy in nAMD Subjects

Jeffrey Heier, MD – Ophthalmic Consultants of Boston

May 15, 2018

AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related MacularDegeneration

Yuanyuan Liu5, Seth D. Fortmann, Jikui Shen, Erik Wielechowski, Anna Tretiakova, Stephen Yoo, Karen Kozarsky, Jiangxia Wang, James M. Wilson, Peter A. Campochiaro'

January 12, 2018

Adeno-Associated Virus Serotype 8 Gene Therapy Leads to Significant Lowering of Plasma Cholesterol Levels in Humanized Mouse Models of Homozygous and Heterozygous Familial Hypercholesterolemia

Kassim et al. Human Gene Therapy

March 12, 2017

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques.

Greig et al. Human Gene Therapy

March 12, 2017

Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia

Greig et al. Human Gene Therapy

August 10, 2016

Delivery of an adeno-associated virus vector into CSF attenuates central nervous system disease in mucopolysaccharidosis type II mice

Human Gene Therapy

July 4, 2016

Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model

Molecular Genetics and Metabolism

July 16, 2014

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

Molecular Therapy

May 5, 2014

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial

Human Gene Therapy

Media

Presentations & Publications

Key Takeaways From The RGX-314 Phase I/IIa Clinical Trial For Wet AMD (Cohorts 1-5)

Six Month Results of the Phase I Study to Evaluate Safety & Tolerability of RGX-314 Gene Therapy in nAMD Subjects

AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related MacularDegeneration

Adeno-Associated Virus Serotype 8 Gene Therapy Leads to Significant Lowering of Plasma Cholesterol Levels in Humanized Mouse Models of Homozygous and Heterozygous Familial Hypercholesterolemia

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques.

Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia

Delivery of an adeno-associated virus vector into CSF attenuates central nervous system disease in mucopolysaccharidosis type II mice

Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial

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