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REGENXBIO

9 clinical trials

Clinical trial
A Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)
Status: Recruiting, Estimated PCD: 2025-12-01
Clinical trial
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
Status: Active (not recruiting), Estimated PCD: 2024-09-29
Clinical trial
A Phase I/IIa (Phase 1/Phase 2a), Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX-314 in Subjects With Neovascular AMD (nAMD)
Status: Completed, Estimated PCD: 2019-11-24
Clinical trial
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-111
Status: , Estimated PCD: 2027-09-01
Clinical trial
A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With RGX 381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
Status: Active (not recruiting), Estimated PCD: 2025-05-20
Clinical trial
A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
Status: Active (not recruiting), Estimated PCD: 2024-05-01
Clinical trial
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 in Subjects With Mucopolysaccharidosis Type I
Status: Active (not recruiting), Estimated PCD: 2023-03-01
Clinical trial
AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)
Status: Terminated, Estimated PCD: 2020-11-27
Clinical trial
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Children 5 Years of Age and Older With MPS II (Hunter Syndrome)
Status: Active (not recruiting), Estimated PCD: 2024-05-01