Gene therapies permanently alter a person’s DNA to produce a therapeutic benefit, often with breathtaking results. As a result, biotech companies, financiers, and the Food and Drug Administration are working extra hard to bring as many of these drugs to market as quickly as possible.
Even a general public raised on science fiction can appreciate recently approved Luxturna from Spark Therapeutics (NASDAQ: ONCE). This is the first directly administered gene therapy in the U.S. that targets an inherited disease caused by a mutation in a specific gene. It uses an engineered virus to deliver a normal copy of a gene that allows retinal cells to produce a protein necessary for sight.
Right now nobody really knows how gene therapies like Luxturna are going to shake out from a financial perspective. The treatment only needs to be injected once, yet it costs $425,000 per eye. With perhaps just 2,000 potential U.S. patients, end payers might not put up a major fight when it comes time to provide reimbursement, but that attitude could change.
There are at least 10,000 known disorders caused by problems with a single gene. That means there are a lot of potential targets just waiting for a group of scientists with enough funding to attack them. Venture capital funding of privately held biotech companies broke through the $10 billion barrier last year led by gene therapy start ups. Publicly traded companies, such as Crispr Therapeutics, have amassed multi-billion valuations before giving a single dose of their experimental gene therapies to anyone. They can’t all be zingers, but all that money is going to push a lot of new therapies onto the market, for better or for worse.
John Mackey, CEO of Whole Foods Market, an Amazon subsidiary, is a member of The Motley Fool’s board of directors. Cory Renauer has no position in any of the stocks mentioned. The Motley Fool owns shares of and recommends Amazon, Athenahealth, Berkshire Hathaway (B shares), and Salesforce.com. The Motley Fool owns shares of CRISPR Therapeutics. The Motley Fool recommends CVS Health. The Motley Fool has a disclosure policy.