By ASHLEY WELCH, CBS NEWS October 06, 2015.
An experimental gene therapy has improved eyesight in patients with a rare, inherited eye disease that can cause blindness, according to its developer, Spark Therapeutics Inc.
The company said it plans to apply to the Food and Drug Administration next year for approval to market the treatment. If accepted, this would be the first gene therapy to win approval in the United States.
“We saw substantial restoration of vision in patients who were progressing toward complete blindness,” Dr. Albert M. Maguire, principal investigator in the trial and professor of ophthalmology at the Perelman School of Medicine of the University of Pennsylvania, said in a statement.
There were no serious adverse effects associated with the treatment, the company reported.The news comes about a week after a hospital in the U.K. announced it had performed a pioneering human embryonic stem cell operation on a patient with the aim of curing a different, more common cause of blindness known as macular degeneration.
Gene therapy involves injecting genetic material into a person’s cells to treat or prevent a disease. While research in this area has been going on since the late 1990’s, no such treatment has been approved in the U.S. The European Commission approved the Western world’s first gene therapy in 2012 for an extremely rare disease in patients with a particular enzyme deficiency.