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Gene Therapy

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November 30, 2021
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Opus Genetics announces licensing of third program for inherited retinal disease

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.

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November 29, 2021
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Topical gene therapy shows positive results in epidermolysis bullosa

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

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November 22, 2021
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FDA grants priority review to betibeglogene autotemcel for beta-thalassemia

FDA grants priority review to betibeglogene autotemcel for beta-thalassemia

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

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November 17, 2021
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Investigational gene therapy leads to sustained factor VIII expression in hemophilia A

Investigational gene therapy leads to sustained factor VIII expression in hemophilia A

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.

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November 14, 2021
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Suprachoroidal RGX-314 well tolerated, improves visual acuity in initial results

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.

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November 12, 2021
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VIDEO: Opus Genetics plans to bring therapies for rare retinal diseases to clinical trials

VIDEO: Opus Genetics plans to bring therapies for rare retinal diseases to clinical trials

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.

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November 10, 2021
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VIDEO: Suprachoroidal delivery ‘brings gene therapy into the office setting’ for wet AMD

VIDEO: Suprachoroidal delivery ‘brings gene therapy into the office setting’ for wet AMD

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.

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October 28, 2021
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VIDEO: Highlights in gene therapy for wet AMD from ASRS meeting

VIDEO: Highlights in gene therapy for wet AMD from ASRS meeting

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.

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October 27, 2021
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VIDEO: New therapies in diabetic macular edema

VIDEO: New therapies in diabetic macular edema

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

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October 02, 2021
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Retinal changes after COVID-19, gene therapy top Euretina congress coverage

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.

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