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A series of clinical trials on gene therapy for RPE65-associated LCA led to the approval of voretigene neparvovec-rzyl (Luxturna) by the Food and Drug Administration ... Gene therapy for glaucoma.
Adverum Biotechnologies, Inc., formerly known as Avalanche Biotechnologies, [2] is a publicly traded [3] clinical stage gene therapy company located in Redwood City, California. [4] The company is targeting unmet medical needs for serious ocular and rare diseases, including wet age-related macular degeneration (wet AMD). [5]
The California Institute for Regenerative Medicine (CIRM) is a state agency that supports research and education in the fields of stem cell and gene therapiesIt was created in 2004 after 59% of California voters approved California Proposition 71: the Research and Cures Initiative, [1] which allocated $3 billion to fund stem cell research in California.
The gene therapy is not a cure for the condition, but substantially improves vision in those treated. [8] It is given as a subretinal injection. Voretigene neparvovec was approved for medical use in the United States in December 2017, [9] in Australia in August 2020, [10] in Canada in October 2020, [11] and in Switzerland in February 2020. [12].
Morgan Micheletti, MD, FACS, is an American ophthalmologist specializing in cataract surgery, refractive surgery, and glaucoma surgery. [1] [2] He is a partner, fellowship director, [3] and research director at Berkeley Eye Center. [4] [5]
"Gene Therapy Arrives". Scientific American This page was last edited on 10 November 2024, at 06:28 (UTC). Text is available under the Creative Commons Attribution ...
Ultragenyx is based in Novato, CA and Brisbane, CA [2] and has a presence in the Boston area, including a gene therapy plant under construction as of 2021. [3] The company’s Latin American headquarters is located in Miami. [4]
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