retinal disease in children by replacing a mutation with a corrective gene. In March, Mass Eye and Ear performed the first-ever procedure to administer the drug to a patient. "It opened up the avenue for gene-based treatments, and some of that might be applicable to AMD," said Miller. While macular degeneration is thought to be influenced not only by genetics but by environmental factors, such as smoking, "gene therapies have incredible potential" to treat the disease, said Luk Vandenberghe, cofounder of Odylia Therapeutics, a nonprofit working to commercialize retinal disease research. Decades of research to understand diseases is now helping to power the new approaches to treatments, he said. There's also hope that the success of gene therapies for maladies of the eye could help launch similar kinds of treatments for other diseases. Ben Shaberman, an official at the Foundation Fighting Blindness, a patient advocacy group, said the retina - a thin tissue lining the back of the eye - is emerging as an ideal proving ground for the young gene therapy field. "The retina is accessible and a really good target," he said. "If you get things to work in the retina, a good chance you could apply them to neurodegenerative disorders of the brain or the central nervous system." Gemini, in , is trying to bring the precision medicine model being deployed in targeted cancer treatments to AMD. Unlike drug developers that try to make one-size-fits-all treatments for macular degeneration, it's focusing on treatments tailored to subsets of patients with distinct genetic variations that put them at risk. "We believe that genetics plays a key role, and we're spending a lot of time trying to understand these subpopulations," said Gemini chief executive James McLaughlin. Sometimes patients themselves aren't sure what role genetics has played in their disease. Teare, who was diagnosed with the disease in 2016, doesn't
retinal disease in children by replacing a mutation with a corrective gene. In March, Mass Eye and Ear performed the first-ever procedure to administer the drug to a patient. "It opened up the avenue for gene-based treatments, and some of that might be applicable to AMD," said Miller. While macular degeneration is thought to be influenced not only by genetics but by environmental factors, such as smoking, "gene therapies have incredible potential" to treat the disease, said Luk Vandenberghe, cofounder of Odylia Therapeutics, a nonprofit working to commercialize retinal disease research. Decades of research to understand diseases is now helping to power the new approaches to treatments, he said. There's also hope that the success of gene therapies for maladies of the eye could help launch similar kinds of treatments for other diseases. Ben Shaberman, an official at the Foundation Fighting Blindness, a patient advocacy group, said the retina - a thin tissue lining the back of the eye - is emerging as an ideal proving ground for the young gene therapy field. "The retina is accessible and a really good target," he said. "If you get things to work in the retina, a good chance you could apply them to neurodegenerative disorders of the brain or the central nervous system." Gemini, in , is trying to bring the precision medicine model being deployed in targeted cancer treatments to AMD. Unlike drug developers that try to make one-size-fits-all treatments for macular degeneration, it's focusing on treatments tailored to subsets of patients with distinct genetic variations that put them at risk. "We believe that genetics plays a key role, and we're spending a lot of time trying to understand these subpopulations," said Gemini chief executive James McLaughlin. Sometimes patients themselves aren't sure what role genetics has played in their disease. Teare, who was diagnosed with the disease in 2016, doesn't
Visions-October-2018-DIGITAL.docx