Regenxbio Inc.
Clinical trials sponsored by Regenxbio Inc., explained in plain language.
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Gene therapy watch: 21 patients tracked for years after single treatment
Disease control ENROLLING_BY_INVITATIONThis study follows 21 people with MPS I who already received RGX-111 gene therapy in an earlier trial. Researchers will monitor them for years to check for side effects and see if the treatment helps with thinking, learning, and daily skills. The goal is to understand the long-te…
Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 17, 2026 09:24 UTC
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Gene therapy trial aims to treat rare brain disease MPS i
Disease control TerminatedThis study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that affects the brain and body. The therapy delivers a working copy of the missing IDUA gene directly into the fluid around the brain. The main goal is to see if it is safe and tolerabl…
Phase: PHASE1, PHASE2 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 17, 2026 09:21 UTC
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Gene therapy trial offers hope for kids with rare brain disease
Disease control TerminatedThis study tests a gene therapy called RGX-121 in children with Hunter syndrome, a rare genetic disorder that affects the brain and body. The therapy delivers a working copy of the missing gene to help improve brain development and reduce harmful substance buildup. The trial is f…
Phase: PHASE3 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 14, 2026 12:06 UTC
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Gene therapy for duchenne MD: Long-Term safety and strength check
Disease control ENROLLING_BY_INVITATIONThis study follows 66 boys with Duchenne muscular dystrophy who previously received RGX-202 gene therapy in an earlier trial. Researchers will monitor safety and measure how well the therapy helps with standing, walking, and climbing over time. The goal is to see if the treatment…
Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 04, 2026 16:19 UTC