Gene therapy offers hope for rare blindness

NCT ID NCT04671433

Completed Disease control Sponsor: Janssen Research & Development, LLC Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 3 trial tests a gene therapy called botaretigene sparoparvovec for X-linked retinitis pigmentosa, a genetic condition that causes progressive vision loss. The study involves 105 participants who receive the therapy via injection under the retina. The main goal is to see if it improves their ability to navigate a maze after one year.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

botaretigene sparoparvovec (AAV5-RPGR gene therapy)

What this could lead to

If successful, this gene therapy could slow or partially reverse vision loss in people with X-linked retinitis pigmentosa, potentially preserving independence.

What could go wrong

This is an early-stage Phase 3 trial with only 105 participants. The treatment involves eye surgery and may not work for everyone. Long-term safety and effectiveness are still unknown.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

retinitis pigmentosa retinitis pigmentosa 3

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

X-LINKED RETINITIS PIGMENTOSA

Contacts and locations

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Locations

Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli

Naples, 80131, Italy
Azienda Ospedaliero Universitaria Careggi

Florence, 50134, Italy
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts

Paris, 75012, France
Childrens Hospital

Los Angeles, California, 90027, United States
Duke Eye Center

Durham, North Carolina, 27705, United States
Emory University

Atlanta, Georgia, 30322, United States
Gartnavel General Hospital

Glasgow, G12 0YN, United Kingdom
Hadassah Medical Center

Jerusalem, 91120, Israel
Hosp Univ Fund Jimenez Diaz

Madrid, 28040, Spain
Hospital For Sick Children

Toronto, Ontario, M5G 1X8, Canada
IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS

Roma, 00198, Italy
Massachusetts General Hospital - Center for Celiac Research and Treatment

Boston, Massachusetts, 02114, United States
Moorfields Eye Hospital

London, EC1V 2PD, United Kingdom
NHS Lothian

Edinburgh, EH3 9HA, United Kingdom
Ospedale San Paolo

Milan, 20142, Italy
Radboudumc

Nijmegen, 6525EX, Netherlands
Retina Consultants of Houston

Bellaire, Texas, 77401, United States
Rigshospitalet Glostrup

Glostrup Municipality, 2600, Denmark
Shiley Eye Institute Jacobs Retina Center

La Jolla, California, 92093 0946, United States
St James University Hospital

Leeds, LS9 7TF, United Kingdom
Stanford Health Care

Palo Alto, California, 94303, United States
UZ Gent

Ghent, 9000, Belgium
Univ of Michigan Medical Center

Ann Arbor, Michigan, 48105, United States
Universite de Lausanne, Hopital ophtalmique Jules-Gonin

Lausanne, 1004, Switzerland
University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical

Basel, 4031, Switzerland
University of Pittsburgh Medical Center (UPMC)

Pittsburgh, Pennsylvania, 15213, United States
VUMC Amsterdam

Amsterdam, 1105AZ, Netherlands
VitreoRetinal Associates, PA

Gainesville, Florida, 32607, United States