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Copy Number Variation clinical trials at UCSF

1 research study open to eligible people

Copy number variation refers to changes in the number of certain genes in our DNA. UCSF is recruiting participants for a clinical trial about a gene therapy called RGX-314, which aims to treat wet age-related macular degeneration. The therapy involves a one-time treatment to replace a faulty gene with a healthy one.

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  • Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD

    open to eligible people ages 50-89

    RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. RGX-314 is being developed as a potential one-time treatment for wet AMD.

    San Francisco, California and other locations

Our lead scientists for Copy Number Variation research studies include .

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