New Prospects for Gene Therapy in IRDs

Aaron Nagiel, MD, PhD, reflects on advances in retinal gene therapy since Luxturna's approval and the prospect of additional treatments.

By: Patricio G. Schlottmann, MD, Aaron Nagiel, MD, PhD

Retinal Physician November 11, 2026

5 Key Takeaways

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Aaron Nagiel, MD, PhD, described inherited retinal disease gene therapy as an exciting area of ongoing development.
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Luxturna, approved by the FDA in 2017, was the first gene therapy approved for an inherited retinal disease.
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Nagiel said there had been a dry spell in approvals since Luxturna, but many gene therapy trials are now ongoing.
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Beacon Therapeutics' laru-zova for X-linked retinitis pigmentosa caused by RPGR mutations was described as a potential approval.
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Nagiel said the ongoing trials bring energy and momentum toward developing additional inherited retinal disease therapies.

Patricio G. Schlottmann, MD

Affiliations: Charles Centro Oftalmológico

Specialties: Ophthalmology

Bio: Patricio G. Schlottmann, MD, is an ophthalmologist associated with Charles Centro Oftalmológico in Argentina. He received his medical degree from the University of Buenos Aires in 1998 and completed formative clinical training at Santa Lucía Hospital in Buenos Aires, Argentina’s largest public eye hospital. Dr. Schlottmann’s work has centered on ophthalmic care, diagnostic technology, and clinical research. His interest in ocular imaging began during specialist training with exposure to Stratus OCT technology, which led to his first publication in Investigative Ophthalmology and Visual Science and helped shape a career combining clinical practice with research.

Aaron Nagiel, MD, PhD

Specialties: Ophthalmology

Bio: Aaron Nagiel, MD, PhD, is an expert in retinal gene therapy with a focus on inherited retinal diseases (IRDs). He has contributed to advancing the field since the initial FDA approval of Luxturna in 2017 and actively participates in ongoing clinical trials for new gene therapies targeting conditions such as X-linked retinitis pigmentosa. Dr. Nagiel is recognized for his insights into emerging gene therapies and their clinical implications, reflecting a commitment to improving treatment options for patients with diverse genetic backgrounds.

New Prospects for Gene Therapy in IRDs

5 Key Takeaways

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