Regenxbio Inc.
Clinical trials sponsored by Regenxbio Inc., explained in plain language.
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Gene therapy trial halted: hope for kids with rare brain disease
Disease control TerminatedThis study tests a gene therapy called RGX-121 for children with Hunter syndrome, a rare genetic disease that affects the brain and body. The therapy aims to deliver a working gene to the brain to help improve development. The trial is currently suspended and enrolled only 2 chil…
Phase: PHASE3 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 26, 2026 08:54 UTC
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Gene therapy trial aims to treat rare brain disease
Disease control TerminatedThis study tests a gene therapy called RGX-111 for people with MPS I, a rare genetic disease 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 tolerable…
Phase: PHASE1, PHASE2 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 26, 2026 08:28 UTC
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Gene therapy watch: researchers track Long-Term safety of RGX-111 in MPS i patients
Disease control ENROLLING_BY_INVITATIONThis study follows 21 people with MPS I who previously received RGX-111 gene therapy in an earlier trial. Researchers will monitor side effects and measure changes in thinking and behavior over time to see if the treatment remains safe and continues to help.
Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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Gene therapy for duchenne MD: Long-Term safety check begins
Disease control ENROLLING_BY_INVITATIONThis study follows 66 boys with Duchenne muscular dystrophy who already received RGX-202 gene therapy in an earlier trial. Researchers will monitor safety and track how well the therapy helps with muscle function over time, such as standing, walking, and climbing. The goal is to …
Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated May 22, 2026 13:53 UTC