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FDA Advisers Endorse Gene Therapy To Treat Form of Blindness (cbsnews.com)
An anonymous reader quotes a report from CBS News: A panel of U.S. health advisers has endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine. A panel of experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics' injectable therapy, which aims to improve vision in patients with a rare mutation that gradually destroys normal vision. The vote amounts to a recommendation to approve the therapy. According to Spark Therapeutics' website, inherited retinal diseases are a group of rare blinding conditions caused by one of more than 220 genes. Some living with these diseases experience a gradual loss of vision, while others may be born without the ability to see or lose their vision in infancy or early childhood. Genetic testing is the only way to verify the exact gene mutation that is the underlying cause of the disease.
The FDA Advisory Committee materials and presentations are here, for those interested.
Note that this is for a very specific genetic cause of blindness, where a mutation in a gene for an enzyme results in effective loss of that specific enzyme. The drug is a retroviral vector encoding only the missing gene. Other causes of blindness, genetic or otherwise, wouldn't benefit from this treatment.
Pretty amazing, and a long time coming for gene therapy, since Jesse Gelsinger's death prompted a long close look at using adenoviral vectors for gene therapy.