Gene Therapy Long-Term Data for Retinal Dystrophy

Stephen Russell, MD, Professor of Ophthalmology and Visual Sciences and Dina J. Schrage Professor of Macular Degeneration Research at the University of Iowa Carver College of Medicine, discusses long-term data on Luxturna (voretigene neparvovec-rzyl) for retinal dystrophy treatment.

Retinal dystrophies are a rare group of degenerative disorders. Over 270 genes are known to be associated with retinal dystrophies.

Voretigene neparvovec is an adeno-associated virus that is approved for the treatment of biallelic RPE65 mutation-associated retinal dystrophy. The treatment delivers a normal copy of the RPE65 gene directly to retinal cells, which then produce the normal protein to convert light into an electrical signal in the retina. This works to restore vision loss.

Long-term safety and durability data for voretigene neparvovec was presented at the 2024 American Society of Retina Specialists conference. The presentation included up to nine years of data. It was observed that at the 8 and 9 year follow-ups, both mobility and visual function were improved and maintained for at least five years following treatment. These improvements were also maintained for up to 8 and 9 years, respectively. This was observed in both original intervention and delayed intervention patients. Additionally, the safety profile was consistent with that in vitrectomies and AAV subretinal injections.

Chapters:
Luxturna Overview and Clinical Trials 00:00
After Approval 10:58