The FDA could soon approve gene therapy for blindness
Children whose vision improved following gene therapy, plus their parents, doctors, and scientists, will speak in front of a Food and Drug Administration panel on Thursday, as the committee decides whether it will recommend approving the therapy.
The FDA has until Jan. 18 to decide if it wants to approve Luxturna, which would be the first gene therapy available in the United States for an inherited disease and the first where a corrective gene is directly given to a patient, The Associated Press reports. Luxturna has been tested on people with Leber congenital amaurosis, who are unable to make a protein needed for the retina due to flaws in the RPE65 gene. They typically are only able to see blurred shapes and bright lights, until they lose their sight all together.
A study, funded by Luxturna's manufacturer, Spark Therapeutics, found that while it does not give patients 20/20 vision, it did improve the vision of nearly everyone who participated in the trials. The company hopes patients would only need one treatment, which involves injecting a modified virus with the corrective gene into the retina. It's not known yet how long the benefits last, but it usually only takes about a month for sight to start to improve. Thanks to the therapy, children have been moved from Braille classrooms to sighted classrooms, and adults who have never held jobs before due to limited sight can now work, Dr. Katherine High, president of Sparks Therapeutics, told AP.
Subscribe to The Week
Escape your echo chamber. Get the facts behind the news, plus analysis from multiple perspectives.
Sign up for The Week's Free Newsletters
From our morning news briefing to a weekly Good News Newsletter, get the best of The Week delivered directly to your inbox.
From our morning news briefing to a weekly Good News Newsletter, get the best of The Week delivered directly to your inbox.
Create an account with the same email registered to your subscription to unlock access.
Sign up for Today's Best Articles in your inbox
A free daily email with the biggest news stories of the day – and the best features from TheWeek.com
Catherine Garcia is night editor for TheWeek.com. Her writing and reporting has appeared in Entertainment Weekly and EW.com, The New York Times, The Book of Jezebel, and other publications. A Southern California native, Catherine is a graduate of the University of Redlands and the Columbia University Graduate School of Journalism.
-
The hunt for Planet Nine
Under The Radar Researchers seeking the elusive Earth-like planet beyond Neptune are narrowing down their search
By Chas Newkey-Burden, The Week UK Published
-
Magazine interactive crossword - April 26, 2024
Puzzles and Quizzes Issue - April 26, 2024
By The Week US Published
-
Magazine solutions - April 26, 2024
Puzzles and Quizzes Issue - April 26, 2024
By The Week US Published
-
Post Office's Capture software to be reviewed over 'glitches'
Speed Read Solicitor representing accused postmasters says flaws in the IT system follow 'very similar pattern' to Horizon
By Arion McNicoll, The Week UK Published
-
Empty-nest boomers aren't selling their big homes
Speed Read Most Americans 60 and older do not intend to move, according to a recent survey
By Peter Weber, The Week US Published
-
Brazil accuses Musk of 'disinformation campaign'
Speed Read A Brazilian Supreme Court judge has opened an inquiry into Elon Musk and X
By Rafi Schwartz, The Week US Published
-
Disney board fends off Peltz infiltration bid
Speed Read Disney CEO Bob Iger has defeated activist investor Nelson Peltz in a contentious proxy battle
By Rafi Schwartz, The Week US Published
-
Disney and DeSantis reach detente
Speed Read The Florida governor and Disney settle a yearslong litigation over control of the tourism district
By Peter Weber, The Week US Published
-
Visa and Mastercard agree to lower swipe fees
Speed Read The companies will cap the fees they charge businesses when customers use their credit cards
By Peter Weber, The Week US Published
-
Reddit IPO values social media site at $6.4 billion
Speed Read The company makes its public debut on the New York Stock Exchange
By Peter Weber, The Week US Published
-
Housing costs: the root of US economic malaise?
speed read Many voters are troubled by the housing affordability crisis
By Peter Weber, The Week US Published