Gene Therapy

One subretinal injection of medium-dose OCU410 gene therapy reduced geographic atrophy growth by up to 32%.

The FDA has accepted Nanoscope's BLA for sonpiretigene isteparvovec in retinitis pigmentosa, backed by RESTORE and REMAIN trial data.

Benjamin Bakall, MD, PhD, weighs the RESTORE efficacy and safety data, the practical realities of in-office delivery, and how genotype-independent treatment reshapes candidacy and patient counseling.

The authors of a review of 16 gene therapy trials for inherited retinal diseases explain why the pediatric evidence base remains thin and argue that individual-level, age-stratified reporting is the change most needed before CRISPR-based editing reaches children.

The FDA accepted Nanoscope's BLA for MOGENRY (sonpiretigene isteparvovec, MC-010), a gene therapy for retinitis pigmentosa with severe vision loss, supported by RESTORE trial data showing sustained BCVA improvement.

Jay Chhablani, MD, on how MacTel eyes turn neovascular, when to refer, and what Encelto means for treatment.

Noah G. Banoub discusses a study finding MacTel2 misdiagnosed in 25.5% of cases, common mimics, misleading imaging, and Encelto's approval.

The FDA has cleared an IND for RAG-1C, a first-in-class saRNA therapy targeting proliferative vitreoretinopathy.

Charles Wykoff, MD, PhD, of Retina Consultants of Texas, discusses 1 year of real-world revakinagene taroretcel experience, covering patient selection, surgical technique, early safety outcomes, and the platform’s potential beyond MacTel.