Gene Therapy for IRDs: Real-World Lessons and New Directions

Aaron Nagiel, MD, PhD, discusses long-term experience with Luxturna and early clinical findings from Opus Genetics’s OPGx-BEST1 program at SARyV 2026.

By: Aaron Nagiel, MD, PhD

Retinal Physician November 11, 2026

Clinical Scorecard: Gene Therapy for IRDs: Real-World Lessons and New Directions

At a Glance

Category	Detail
Condition	Inherited retinal diseases, including RPE65-associated disease and BEST1-related retinal diseases
Key Mechanisms	The speaker discussed Luxturna (voretigene neparvovec-rzyl) and Opus Genetics’ OPGx-BEST1 gene therapy. Chorioretinal atrophy was reported in post-authorization experience with Luxturna.
Target Population	Patients treated with Luxturna and patients with autosomal recessive or dominant BEST1-related retinal disease in the OPGx-BEST1 study
Care Setting	The report describes post-authorization safety experience with Luxturna and findings from five patients in the OPGx-BEST1 study presented at SARyV 2026.

Key Highlights

The speaker said real-world post-authorization safety data for Luxturna behaved as expected based on clinical-trial experience.
The speaker said chorioretinal atrophy was not really found in trials and was subsequently described in post-authorization experience.
The speaker said certain vectors, in the right setting, could cause photoreceptor and retinal pigment epithelium degeneration.
The speaker described OPGx-BEST1 as well tolerated in five patients, with no major safety issues.
The speaker described potential signs of efficacy and said cohort two would include more patients and a higher dose.

Guideline-Based Recommendations

Diagnosis

Management

Monitoring & Follow-up

Risks

Patient & Prescribing Data

The OPGx-BEST1 findings described involved five patients with autosomal recessive or dominant BEST1-related retinal disease.

The speaker described OPGx-BEST1 as well tolerated in five patients, with no major safety issues and potential signs of efficacy. The speaker said Luxturna’s real-world post-authorization safety experience behaved as expected based on clinical trials and noted chorioretinal atrophy.