X-linked retinitis pigmentosa
Clinical trials for X-linked retinitis pigmentosa explained in plain language.
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Gene therapy targets second eye in rare blindness disorder
Disease control OngoingThis study tests a gene therapy called botaretigene sparoparvovec in the second eye of people with X-linked retinitis pigmentosa, a genetic condition that causes vision loss. The 24 participants previously received the therapy in one eye and are now being followed for safety and …
Matched conditions: X-LINKED RETINITIS PIGMENTOSA
Phase 2 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
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Gene therapy shows promise for rare blindness disease
Disease control OngoingThis study is a follow-up to a Phase 3 trial testing a gene therapy called AAV5-hRKp.RPGR for people with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. The treatment aims to slow or stop vision decline by delivering a working copy of the RPGR gene …
Matched conditions: X-LINKED RETINITIS PIGMENTOSA
Phase 3 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
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Experimental gene therapy aims to halt vision loss in rare eye disease
Disease control OngoingThis Phase 3 study tests a gene therapy called AAV5-hRKp.RPGR in 4 Japanese people with X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The therapy is injected into the eye to deliver a working copy of the RPGR gene. The main goal is to che…
Matched conditions: X-LINKED RETINITIS PIGMENTOSA
Phase 3 • Sponsor: Janssen Pharmaceutical K.K. • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
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Gene therapy offers hope for rare Blindness-Causing eye disease
Disease control OngoingThis study tests a gene therapy called AGTC-501 in males aged 12 to 50 with X-linked retinitis pigmentosa, a genetic eye disease that causes vision loss. Participants receive one of two doses of the therapy or no treatment, and researchers measure changes in vision under low ligh…
Matched conditions: X-LINKED RETINITIS PIGMENTOSA
Phase 2/3 • Sponsor: Beacon Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC