This Gene Therapy Can Restore Vision

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FOLLOW subscribe SEND TIP December 19, 2017 Hello, readers! This is Sy. The Food and Drug Administration (FDA) on Tuesday approved the first true gene therapy in the U.S.—Luxturna from Spark Therapeutics, which treats a form of inherited vision loss which can lead to blindness. The landmark approval continues a string of significant medical advances to be blessed by regulators this year. But Spark is staying mum on Luxturna’s price so far amid speculation that the treatment’s list price could be as high as $1 million. Spark Therapeutics stock stayed relatively flat in early Tuesday trading. Luxturna is a gene therapy that’s directly administered to patients who have a condition called biallelic RPE65 mutation-associated retinal dystrophy. The disorder is caused by defects in a gene which helps produce a protein critical to normal vision. With Luxturna, a healthy copy of the gene is inserted into retinal cells (with the help of engineered viruses) in order to produce the necessary protein and restore vision. The science is futuristic, and the fact the idea that gene therapies like Luxturna may be able to rectify lifelong genetic defects over the long term is tantalizing. “I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb in a statement. But groundbreaking science can come at a backbreaking price. There has been speculation, for instance, that Luxturna could cost as much as $1 million (Spark will not be revealing the list price until January). While patients wouldn’t have to pay such a price out-of-pocket, the sticker shock could prove a problem to insurers and benefits managers, and discourage widespread prescriptions by doctors. Cutting-edge but high cost drugs that target a relatively small portion of the population have been on the rise recently and spurred debates about how the health care system should balance patient access and market incentives for innovation. Earlier this year the FDA approved two groundbreaking new types of cancer treatments—Novartis’ Kymriah and Gilead/Kite’s Yescarta—which reengineer immune cells to fight cancer but also come with list prices in the hundreds of thousands of dollars. Read on for the day’s news. Sy Mukherjee @the_sy_guy sayak.mukherjee@fortune.com . DIGITAL HEALTH The search for the meatless meat. My colleague Beth Kowitt has a must-read feature on Silicon Valley's quest for a meat without the actual meat—a project that could, theoretically, improve both human diet and planetary health. "It's the type of world-saving vision that has oft captured the imagination of Silicon Valley—the kind of entrenched problem that technologists believe only technology can solve: feeding a fast-growing, protein-hungry global population in a way that doesn't blow up the planet," writes Beth. "Conjuring up meat without livestock—whose emissions are responsible for 14.5% of global greenhouse gases—is core to that effort." ( Fortune) . INDICATIONS Shire faces setback with Hunter syndrome trial failure. Rare disease drug maker Shire's experimental SHP609, being tested on child patients with Hunter syndrome, missed the main goals of a late-stage clinical trial in a setback to the European pharma company. Hunter syndrome is a very rare genetic metabolic disease which can cause physical symptoms like enlarged head and abnormal bone size as well as aggressive behavior, among other symptoms. There is no cure for the condition. (Reuters) People with egg allergies can get the flu vaccine. New research suggests that even Americans with egg allergies can safely receive flu vaccines. Previously, these people were encouraged to explore alternative vaccine options because traditional inoculations are manufactured using chicken eggs, raising concerns of potential allergic reactions. "People with egg allergy of any severity can receive the influenza vaccine without any special precautions," said Dr. Matthew Greenhawt, lead study author and chairman of the American College of Allergy, Asthma and Immunology Food Allergy Committee. (CNN) . THE BIG PICTURE Humana, private equity firms team up to buy Kindred Healthcare. Kindred Healthcare, home health services giant valued at about $4.1 billion including debt, is being purchased by health insurer Humana and a pair of private equity firms for $800 million. This is yet another example of the cross-sector consolidation wave (including CVS-Aetna) in the medical industry; Humana is a large operator of Medicare plans, and the marriage with a firm that focuses on seniors' care makes could make Humana's access to the elderly customer market that much easier. (Wall Street Journal) The tax plan expected to pass Congress today would still hit Obamacare. Congress is expected to pass its tax reform bill today. But the legislation would also affect U.S. health care by nixing Obamacare's mandate to carry health insurance—a move that nonpartisan budget scorekeepers like the CBO have estimated will lead to 13 million fewer insured Americans over the next decade relative to current law and higher premiums in individual insurance markets. A pair of bills to shore up the ACA's marketplaces that may be included in a government spending vehicle would do almost nothing to counteract the coverage losses projected by CBO. . . REQUIRED READING Tesla Secures Its Largest Order of Semi Trucks Yet With UPS, by David Z. Morris Can This Man Build a Better Bitcoin? by Robert Hackett Airbnb Lands $200 Million Investment for Branded Apartment Complexes, by Casey Quackenbush Warren Buffett's Berkshire Hathaway Hit a Record $300,000 Per Share, by Reuters Produced by Sy Mukherjee @the_sy_guy sayak.mukherjee@fortune.com Find past coverage. Sign up for other Fortune newsletters. . Email Clifton Leaf subscribe share: . This message has been sent to you because you are currently subscribed to Brainstorm Health Unsubscribe here Please read our Privacy Policy, or copy and paste this link into your browser: http://www.fortune.com/privacy FORTUNE may receive compensation for some links to products and services on this website. Offers may be subject to change without notice. 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