Clinical trial download: Data on a Gene therapy for dry and wet AMD. Phase 1 study of intravitreal gene therapy with ADVM-022 for neovascular AMD (OPTIC trial). AREDS 2 supplements for age-related macular degeneration (AMD).Advm-022 intravitreal Gene therapy for wet AMD (Optic).You can learn more about how we ensure our content is accurate and current by reading our editorial policy. We link primary sources - including studies, scientific references, and statistics - within each article and also list them in the resources section at the bottom of our articles. Medical News Today has strict sourcing guidelines and draws only from peer-reviewed studies, academic research institutions, and medical journals and associations. Gyroscope Therapeutics also announced positive results from their ongoing Phase 1/2 clinical trial, stating that people tolerated the therapy well and demonstrated increased CFI levels compared with the patients’ baselines. This therapy is intended to slow the progression of geographic atrophy, which is an advanced form of dry macular degeneration.Ī 2020 study found that GT005 is safe and well-tolerated in mice, leading to a long-term secretion of human CFI in the animals. This helps counteract the inflammation caused by the complement system and prevents the body from attacking its retinal cells. Gene therapy delivers a copy of genes that correctly encode this protein and increase its production. Experts found that complement factor I (CFI) - proteins that prevent the immune system from attacking retinal cells - are low or not functioning in people with dry macular degeneration. GT005 clinical trialĪnother clinical trial, called GT005 by Gyroscope Therapeutics, also targets MACs. Phase 1 of the therapy has shown efficacy by slowing the rate of progression without producing adverse reactions. This gene therapy permanently alters the retinal cells to increase their expression of CD59. ![]() For the procedure, researchers administer an injection of the product into the vitreous cavity of the eye in an office setting.Ī protein found on the cell surface called CD59 prevents and blocks MACs from forming. There is a clinical trial underway, conducted by Hemera Biosciences, for a therapy called AAVCAGsCD59 (HMR59) for dry and wet macular degeneration. These proteins become overactive and attack the retinal cells, causing membrane attack complex (MAC) or holes in the eye. ![]() In dry macular degeneration, there is an imbalance in the complement system - a system of proteins in the immune system that fights bacteria and other pathogens. Gene therapy for dry macular degeneration
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