Gene therapy has been in the news a lot of late as a potential cure for a variety of genetic diseases. The biggest breakthrough involved a unanimous backing from U.S. Food and Drug Administration’s advisory panel for a new gene therapy from Spark Therapeutics, intended to treat rare eye diseases. But what exactly is gene therapy, and does it deserve all that hype?
CNBC sat down with Canaan Venture Partners‘ biotech investor Nina Kjellson for a simple explanation of how it all works, and why she’s excited about it. As Kjellson explains, in the most simple terms, gene therapy is a technique that uses genes to treat disease.
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A big obstacle remains — and that’s price. The cost for each patient could be as high as $1 million, which insurance companies might decline to cover. For Kjellson, these experimental therapies have a lot of promise as they could treat a wide variety of medical conditions, starting with genetic disorders caused by mutations in single genes. It’s still early days, but the hope is that gene therapy might someday replace drugs or surgery by fixing the problem at its root.
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