Regenxbio (United States)

Anifrolumab, an Anti–Interferon‐α Receptor Monoclonal Antibody, in Moderate‐to‐Severe Systemic Lupus Erythematosus

Richard Furie, Munther A. Khamashta, Joan T. Merrill et al. · Arthritis & Rheumatology · 2016 · 829 citations · Full text

Preclinical and clinical evaluation of the LRRK2 inhibitor DNL201 for Parkinson’s disease

Danna Jennings, Sarah Huntwork‐Rodriguez, Anastasia G. Henry et al. · Science Translational Medicine · 2022 · 230 citations

AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression

Kun Ding, Jikui Shen, Zibran Hafiz et al. · Journal of Clinical Investigation · 2019 · 131 citations · Full text

Ocular Disease, Engineering, Gene Delivery +19

2019 White Paper on Recent Issues in Bioanalysis: FDA Immunogenicity Guidance, Gene Therapy, Critical Reagents, Biomarkers and Flow Cytometry Validation (Part 3 – Recommendations on 2019 FDA Immunogenicity Guidance, Gene Therapy Bioanalytical Challenges, Strategies for Critical Reagent Management, Biomarker Assay Validation, Flow Cytometry Validation & CLSI H62)

Steven P. Piccoli, Devangi Mehta, Alessandra Vitaliti et al. · Bioanalysis · 2019 · 92 citations · Full text

Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study

Peter A. Campochiaro, Robert L. Avery, David M. Brown et al. · The Lancet · 2024 · 91 citations

Regenerative Medicine, Dose-escalation Study, Gene Therapies +9