Gene Therapy

Opus Genetics announced an agreement to license its third preclinical program focused on an inherited retinal disease from Massachusetts Eye and Ear, Harvard Medical School’s ophthalmic teaching hospital, according to a press release.

Vyjuvek was effective in healing the majority of dystrophic epidermolysis bullosa wounds in a phase 3 trial, Krystal Biotech announced in a press release.

The FDA granted priority review to betibeglogene autotemcel, a gene therapy in development to treat patients with transfusion-dependent beta-thalassemia.

The novel gene therapy SPK-8011 induced sustained factor VIII expression in patients with hemophilia A, according to results of a nonrandomized phase 1/phase 2 dose-escalation study published in The New England Journal of Medicine.

NEW ORLEANS — Initial results from the ALTITUDE study suggest that suprachoroidal RGX-314 is well tolerated in the treatment of diabetic retinopathy, according to a speaker here.

NEW ORLEANS — In this Healio Video Perspective from the Eyecelerator meeting, Ben Yerxa, PhD, acting CEO of Opus Genetics, discusses the company’s plans, including the beginning of trials of OPGx-001, designed to target the LCA5 gene.

In a video interview, Nancy M. Holekamp, MD, director of retina services at Pepose Vision Institute in St. Louis, talked about suprachoroidal delivery of Regenxbio’s gene therapy RGX-314 for neovascular age-related macular degeneration.

Rishi P. Singh, MD, talked about gene therapy studies for neovascular age-related macular degeneration presented at the ASRS meeting in a video interview with Healio.

Rishi P. Singh, MD, of Cleveland Clinic, discussed new therapies for diabetic macular edema presented at the ASRS meeting in a video interview.

Coverage from the virtual Euretina congress included items investigating retinal changes in children who recovered from COVID-19 and vascular changes in patients with multiple sclerosis.