Gene Therapy

RGX-314 gene therapy administered via in-office suprachoroidal injection could provide long-lasting therapy for diabetic retinopathy.

AUSTIN, Texas — In this Healio Video Perspective from the North American Neuro-Ophthalmology Society meeting, Jonathan C. Horton, MD, PhD, describes an alternative method of gene therapy delivery.

In this Healio Video Perspective, Jay S. Duker, MD, chief operating officer of EyePoint Pharmaceuticals, discusses EYP-1901 for the treatment of wet age-related macular degeneration.

Nanoscope Therapeutics has completed enrollment in a phase 2b clinical trial investigating the efficacy and safety of MCO-010 in patients with retinitis pigmentosa, according to a press release.

LentiGlobin led to a reduction in hemolysis and complete resolution of severe vaso-occlusive events among a small cohort of patients with sickle cell disease, according to study results published in The New England Journal of Medicine.

WAIKOLOA, Hawaii — A gene-based therapy, specifically designed to neutralize VEGF activity, may be the next approach to the treatment of diabetic retinopathy, with the advantage of a single suprachoroidal administration.

NR082, a gene therapy drug candidate for the treatment of Leber hereditary optic neuropathy, was granted orphan drug designation by the European Medicines Agency, according to a press release from Neurophth Therapeutics.

The FDA has granted investigational new drug clearance to Nanoscope Therapeutics for a phase 2 trial investigating MCO-010 in patients with Stargardt macular degeneration, according to a press release.

Lumevoq demonstrated continued vision improvement in Leber hereditary optic neuropathy 4 years after a single gene therapy injection, according to a press release from GenSight Biologics.

ADVM-062, a novel gene therapy candidate, received orphan drug designation from the FDA for the treatment of blue cone monochromacy, according to a press release from Adverum Biotechnologies.