Gene-therapy pioneer Jean Bennett, M.D., Ph.D., may have been the keynote speaker for the closing session at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), but Yannick Duwe, a 15-year-old patient with Leber congenital amaurosis (LCA) who was treated in her clinical trial six years ago, stole the show.
Children's Hospital of Philadelphia has invested $50 million in a new biotech start-up that seeks to be the nation's first commercial provider of gene therapy, company officials announced Tuesday.
Spark Therapeutics will assume control over two clinical trials that originated at the prominent teaching hospital - one in which patients with a rare form of blindness already have regained some vision, the other an early-stage effort to treat hemophilia B.
In 2009, 12 people participated in a clinical trial through which they received an experimental gene therapy for an inherited form of blindness. The correct form of the gene, meant to replace their faulty ones, was injected into the retinal cells of one eye. Limiting the injection to one eye was precautionary, since the researchers didn’t know if there would be any adverse affects. When the trial began all of the participants were legally blind, but two weeks after the injection their vision began to improve. Although their vision didn’t recover enough to be considered normal vision, half of the participants improved enough to lose their legally blind status.
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