Science & Innovation

Presentations & Publications

March 06, 2024

RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy:
Interim Clinical Data

Aravindhan Veerapandiyan, MD, Arkansas Children's Hospital
February 07, 2024

RGX 121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II (MPS II): CAMPSIITE ® Phase I/II/III: A Clinical Study Update

Paul Harmatz, M.D., UCSF Benioff Children's Hospital
January 16, 2024

Suprachoroidal Delivery of Investigational ABBV-RGX-314 for Neovascular AMD: Results from the Phase II AAVIATE® Study

John D. Pitcher, III, M.D. Eye Associates of New Mexico
November 05, 2023

Suprachoroidal Delivery of Investigational ABBV-RGX-314 for Diabetic Retinopathy: The Phase II ALTITUDE® Study Dose Levels 1 and 2: One Year Results

Mark R. Barakat, M.D., Retina Consultants of Arizona
October 03, 2023

RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data

Aravindhan Veerapadiyan, M.D., Pediatric Neuromuscular Neurologist, Arkansas Children's Hospital
August 30, 2023

First-in-Human Intracisternal Dosing of RGX-181 (Adeno-Associated Virus 9 / Human Tripeptidyl Peptidase 1) for a 5-Year-Old Child With Late Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2): 6-Month Follow-up

Carolina Fischinger de Souza
August 30, 2023

RGX-121, An Investigational Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II (MPS II) - CAMPSIITE™ Phase I/II/III:  A Clinical Study Update

Paul Harmatz, MD
August 30, 2023

RGX-111, An Investigational Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type I (MPS I) - Interim Analysis of the First-in-Human Study and a Single IND Patient 

Raymond Y. Wang, MD
August 30, 2023

Daily Living Skills on the Vineland Adaptive Behavioral Scale Version 2 (VABS-II) Following Investigational RGX-121 Treatment in Neuronopathic Mucopolysaccharidosis Type II (MPS II)

Michelle Wood
August 01, 2023

Suprachoroidal Delivery of Investigational ABBV-RGX-314 for Diabetic Retinopathy: The Phase II ALTITUDE® Study

Dilsher S. Dhoot, MD
July 30, 2023

Subretinal Delivery of Investigational ABBV-RGX-314 for Neovascular AMD: A Phase II Pharmacodynamic Study

Ashkan Abbey, MD
July 30, 2023

Suprachoroidal Delivery of Investigational ABBV-RGX-314 for Neovascular AMD: Results from the Phase II AAVIATE® Study

David Boyer, MD
Adjunct Clinical Professor Ophthalmology Keck School of Medicine Partner Retina Vitreous Associates Medical Group
February 25, 2023

RGX-381: First-In-Human Clinical Trial Of An Investigational AAV9 Gene Therapy Encoding TPP1 For The Treatment Of Ocular Manifestations Of CLN2 Batten Disease

Christina Ohnsman, M.D., Senior Clinical Development Lead at REGENXBIO Inc.
 
February 24, 2023

RGX-111 Gene Therapy For The Treatment Of Severe Mucopolysaccharidosis Type I (MPS I): Interim Analysis Of Data From The First In Human Study

Raymond Wang, M.D., Division of Metabolic Disorders, CHOC Children's Hospital, Department of Pediatrics, University of California, Irvine, CA
February 22, 2023

RGX-121 Gene Therapy For The Treatment Of Neuronopathic Mucopolysaccharidosis Type II (MPS II): Interim Analysis Of Data From The First In Human Study (Abstract #LB-21)

Can Ficicioglu, M.D., Ph.D., Professor of Pediatrics at Perelman School of Medicine at the University of Pennsylvania, Director of the Newborn Metabolic Screening Program and the Lysosomal Storage Diseases Program, Clinical Director of the Metabolic Disease Program Children's Hospital of Philadelphia
February 11, 2023

Subretinal Delivery Of RGX-314 For Neovascular AMD: A Phase II Pharmacodynamic Study

Charles C. Wykoff MD, PhD, Director of Research at Retina Consultants of Texas; Chairman of Research, Retina Consultants of America; and Deputy Chair of Ophthalmology for the Blanton Eye Institute, Houston Methodist Hospital
November 02, 2022

Suprachoroidal Delivery Of RGX-314 For Diabetic Retinopathy Without CI-DME: The Phase II ALTITUDE® Study

Lejla Vajzovic, MD, FASRS, Associate Professor of Ophthalmology and Director of Duke Vitreoretinal Fellowship Program, Vitreoretinal Surgery and Disease, Department of Ophthalmology, Duke University School of Medicine
October 03, 2022

Suprachoroidal Delivery Of RGX-314 For NAMD: AAVIATE® Study

REGENXBIO
October 01, 2022

Gene Therapy For Neovascular AMD: Subretinal RGX-314: Phase I/IIa Long-Term Follow-Up Results Up To 4 Years

Peter Campochiaro M.D., Director, Retinal Cell and Molecular Laboratory, Professor of Ophthalmology, The Wilmer Eye Institute, Johns Hopkins University School of Medicine
August 31, 2022

RGX 121 Gene Therapy For The Treatment Of Severe Mucopolysaccharidosis Type II: CAMPSIITE™ Phase I/II/III: A Clinical Study Update

Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil
February 12, 2022

Suprachoroidal Delivery Of RGX-314 For Diabetic Retinopathy Without CI-DME: Results From The Phase II ALTITUDE™ Study

Michael Klufas, M.D., Retina Service, Wills Eye Hospital, Assistant Professor of Ophthalmology, Thomas Jefferson University
February 09, 2022

RGX-111 Gene Therapy For The Treatment Of Severe Mucopolysaccharidosis Type I (MPS I): Interim Analysis Of Data From The First In Human Study

Raymond Wang, M.D., Division of Metabolic Disorders, CHOC Children's Hospital, Department of Pediatrics, University of California, Irvine, CA
February 09, 2022

RGX-121 Gene Therapy For The Treatment Of Severe Mucopolysaccharidosis Type II (MPS II): Interim Analysis Of Data From The First In Human Study

Roberto Giugliani, M.D., Ph.D., Presenter title: Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil
November 12, 2021

Two Year Results From The Subretinal RGX-314 Gene Therapy Phase 1/2a Study For The Treatment Of NAMD, And An Update On Suprachoroidal Trials

Robert L. Avery, M.D., Founder of California Retina Consultants and Research Foundation
October 09, 2021

Suprachoroidal Delivery Of RGX-314 For Diabetic Retinopathy Without CI-DME: Early Results From The Phase II ALTITUDE™ Study

Dennis Marcus, M.D., F.A.S.R.S., Southeast Retina Center
October 01, 2021

Suprachoroidal Delivery Of RGX-314 For Neovascular AMD: Initial Results From The Phase II AAVIATE® Study

Nikolas London, M.D., M.S., M.S., F.A.C.S., Partner and Director of Research, Retina Consultants of San Diego, Chief of Ophthalmology, Scripps Memorial Hospital
October 01, 2021

Subretinal Delivery Of RGX-314 For Neovascular AMD: End Of Phase I/IIa Study Results

Allen Ho, M.D., Director of Retina Research at Wills Eye Hospital and Mid Atlantic Retina
February 08, 2021

RGX-121 Gene Therapy For Severe Mucopolysaccharidosis Type II (MPS II): Interim Results Of An Ongoing First In Human Trial

Marie-Laure Névoret, MD – Senior Clinical Development Lead, REGENXBIO
 
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 04, 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 05, 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
December 22, 2011

Adenovirus-Associated Virus Vector–Mediated Gene Transfer In Hemophilia B

The New England Journal of Medicine
October 12, 2010

Gene Therapy In A Humanized Mouse Model Of Familial Hypercholesterolemia Leads To Marked Regression Of Atherosclerosis.

Kassim et al. PLoS One
February 28, 2010

Rescue Of The Spinal Muscular Atrophy Phenotype In A Mouse Model By Early Postnatal Delivery Of SMN

Nature Biotechnology