Regenxbio Inc.
Clinical trials sponsored by Regenxbio Inc., explained in plain language.
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Brain-Targeting gene therapy tested for devastating rare disease
Disease control TerminatedThis early-stage study is testing a one-time gene therapy called RGX-111 for people with MPS I, a rare genetic disorder that affects the brain. The therapy aims to deliver a working copy of a missing gene directly to the central nervous system to help the brain produce a needed e…
Phase: PHASE1, PHASE2 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC
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Tracking a potential lifeline: 5-Year study monitors boys after groundbreaking gene therapy
Disease control ENROLLING_BY_INVITATIONThis study is tracking boys for up to five years after they received an experimental gene therapy called RGX-202 for Duchenne muscular dystrophy (DMD). The main goal is to understand the long-term safety of the treatment and see if its benefits on muscle strength and movement las…
Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC
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One-Shot gene therapy aims to halt devastating brain disease in toddlers
Disease control TerminatedThis study is testing a one-time gene therapy called RGX-121 for young boys with a severe form of Hunter syndrome that affects the brain. The goal is to see if delivering a working gene directly to the brain can slow or stop the loss of thinking and developmental skills. The stud…
Phase: PHASE3 • Sponsor: REGENXBIO Inc. • Aim: Disease control
Last updated Mar 05, 2026 14:00 UTC