T

he stock prices of companies developing gene therapies are surging on optimism that one-shot treatments will cure a host of rare, inherited diseases, so let’s take a moment to remember gene therapy is risky and prone to failure.

At an FDA advisory panel last week, kids with a rare and debilitating form of severe night blindness told emotional, joyous stories of seeing rainbows and playing outside with their friends at dusk for the first time because of a groundbreaking gene therapy called Luxturna developed by Spark Therapeutics (ONCE). As I noted on Twitter, the patient testimonies at the Luxturna advisory panel ranked among the most compelling I’ve ever heard, and underscore the incredible potential for gene therapy to change lives for the better.

This is a STAT Plus article and you can unlock it by subscribing to STAT Plus today. It's easy! Your first 30 days are free and if you don't enjoy your subscription you can cancel any time.
Already a subscriber? Log in here.

Leave a Comment

Please enter your name.
Please enter a comment.

Sign up for our Morning Rounds newsletter

Your daily dose of news in health and medicine.