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NCT04794101Phase 3ACTIVE NOT RECRUITING

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

Study Summary

A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)

Conditions
X-Linked Retinitis Pigmentosa
Interventions / Drugs
Genetic: AAV5-hRKp.RPGR Intermediate DoseGenetic: AAV5-hRKp.RPGR Low Dose
View on ClinicalTrials.gov ↗
Sponsor
JNJ
Johnson & Johnson
Diversified Pharma
C51/100
Study Details
Enrollment97 participants
Primary Completion—
Lead SponsorJanssen Research & Development, LLC
Data Retrieved2026-04-11 02:55:31.549853+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 97 participants
Sponsor Catalyst Profile

Johnson & Johnson (JNJ) is the sponsoring company for this trial. BiotechSign currently grades this company C (51/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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