LANSING, Mich. (AP) — Michigan's Republican-led Legislature has blocked proposed regulations of a breakthrough gene therapy that combats some blood cancers, citing concerns that patients could not...
SANFORD, N.C. (AP) — Drug giant Pfizer is expanding its North Carolina manufacturing plant for gene-therapy medicines to treat rare diseases, adding about 300 jobs.
WASHINGTON (AP) — Expanding access to a promising but costly treatment, Medicare said Wednesday it will cover for some blood cancers a breakthrough gene therapy that revs up a patient's own immune...
TRENTON, N.J. (AP) — U.S. regulators want to know why Novartis didn't disclose a problem with testing data until after the Swiss drugmaker's $2.1 million gene therapy was approved.
U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease...
They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with "bubble boy disease" have had it fixed by a gene therapy made from one of the...
LISBON, Portugal (AP) — Researchers in the United States and Britain are sharing a 1 million-euro ($1.16 million) prize from a Portuguese scientific foundation for developing revolutionary gene therapy that has restored the sight of children.
The Lisbon, Portugal-based Champalimaud Foundation's annual Vision Award was given Tuesday for work on the treatment of children blinded from birth by genetic disease.
U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly becoming an established form of medical care with no extraordinary risks.
LAWRENCEVILLE, Ga. (AP) — Easton Walker is a 17-month-old charmer. With a flop of red hair and pale blue eyes, the toddler smiles at just about everyone he meets.
But behind the playful smile is a debilitating disease. A feeding tube, as well as a port catheter (or port for short), for weekly infusions, are hidden underneath Easton's sky blue T-shirt.
MADISON, Wis. (AP) — As a doctor injected cloudy fluid into Makena Eighmy's intravenous line, it looked like the 13-year-old animal lover from near Watertown was getting a standard procedure for her cancer.
But the infusion this month at UW Health's American Family Children's Hospital was far from routine.
DURHAM, N.C. (AP) — A gene therapy company says it will create 200 jobs in North Carolina.
AveXis on Tuesday agreed to set up operations in Durham County, after getting a state incentive package worth up to $3 million.
AveXis makes treatments for spinal muscular atrophy.
GENEVA (AP) — Swiss pharmaceuticals giant Novartis says it has agreed to buy the U.S.-based gene therapy company AveXis Inc. for $8.7 billion, part of its goal to become a leader in the treatment of neurodegenerative diseases.
The two companies say their respective boards have voted unanimously to approve the deal, to be paid for through cash and short-term borrowing.
For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that made this possible.
Scientists removed some of his blood cells, disabled a gene to help them resist HIV, and returned these "edited" cells to him in 2014. So far, it has given the San Francisco man the next best thing to a cure.
Scientists Use Gene Therapy to Fight HIV
A second patient has been treated in a historic gene editing study in California, and no major side effects or safety issues have emerged from the first man's treatment nearly three months ago, doctors revealed Tuesday.
Gene editing is a more precise way to do gene therapy, and aims to permanently change someone's DNA to try to cure a disease.
WASHINGTON (AP) — A first-of-its kind genetic treatment for blindness will cost $850,000 per patient, making it one of the most expensive medicines in the world and raising questions about the affordability of a coming wave of similar gene-targeting therapies.
After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.
WASHINGTON (AP) — U.S. health officials on Tuesday approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major advance for the emerging field of genetic medicine.
(The Conversation is an independent and nonprofit source of news, analysis and commentary from academic experts.)
Ian Haydon, University of Washington
(THE CONVERSATION) If you’ve ever bought a new iPhone, you’ve experienced good packaging.
Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can give long-lasting help and perhaps even cure the blood disease.
Hemophilia almost always strikes males and is caused by lack of a gene that makes a protein needed for blood to clot. Small cuts or bruises can be life-threatening, and many people need treatments once or more a week to prevent serious bleeding.
LONDON (AP) — Doctors treating a critically ill boy with a devastating skin disease used experimental gene therapy to create an entirely new skin for most of his body in a desperate attempt to save his life.
Two years later, the doctors report the boy is doing so well that he doesn't need any medication, is back in school and even playing soccer.
TRENTON, N.J. (AP) — U.S. regulators on Wednesday approved a second gene therapy for a blood cancer, a one-time, custom-made treatment for aggressive lymphoma in adults.
The Food and Drug Administration allowed sales of the treatment from Kite Pharma. It uses the same technology, called CAR-T, as the first gene therapy approved in the U.S. in August, a treatment for childhood leukemia from Novartis Pharmaceuticals.
SILVER SPRING, Md. (AP) — A potentially groundbreaking treatment for a rare form of blindness moved one step closer to U.S. approval Thursday, as federal health advisers endorsed the experimental gene therapy for patients with an inherited condition that gradually destroys eyesight.
The panel experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics' injectable therapy, which aims to improve vision by replacing a defective gene needed to process light.
SILVER SPRING, Md. (AP) — The Latest on an FDA panel's discussion on a gene therapy treatment for blindness (all times local):
U.S. health advisers have endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine.
In a story Oct. 9 about an experimental gene therapy for blindness, The Associated Press erroneously reported the deadline for a decision by the U.S. Food and Drug Administration. The date is Jan. 12, not Jan. 18.
A corrected version of the story is below:
Seeing hope: FDA panel considers gene therapy for blindness
FDA Panel Considers Gene Therapy For Blindness
- Business WireRocket Pharmaceuticals Reports Third Quarter 2019 Financial Results and Operational HighlightsNovember 7, 2019
- Globe NewswireClearside Biomedical Suprachoroidal Injection Platform Featured in Multiple Presentations at the American Academy of Ophthalmology (AAO) 2019 Annual MeetingOctober 17, 2019
- Globe NewswirePrevail Therapeutics Reports Second Quarter 2019 Financial Results and Recent Business HighlightsAugust 14, 2019
- Business WireGene and Cell Therapy Solutions Provider, SIRION Biotech Announces Record First Half 2019 Financial ResultsJuly 31, 2019
- Globe NewswireAdverum Biotechnologies Appoints Senior LeadersJuly 15, 2019
WASHINGTON (AP) — Opening a new era in cancer care, U.S. health officials have approved a breakthrough treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.
WASHINGTON (AP) — Opening a new era in cancer care, U.S. health officials on Wednesday approved a breakthrough treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.
The Food and Drug Administration called the approval historic, the first gene therapy to hit the U.S. market. Made from scratch for every patient, it's one of a wave of "living drugs" under development to fight additional blood cancers and other tumors, too.
SANFORD, N.C. (AP) — Biopharmaceutical giant Pfizer Inc. plans to invest $100 million in a North Carolina plant to focus on a process by which genetic material is introduced to counter defective or missing genes.
The company confirmed Gov. Roy Cooper's news on Monday that it will expand its Sanford operations in preparation for producing gene therapy medicines based on technology developed at UNC-Chapel Hill. Cooper's news release says the investment will lead to 40 new jobs.
A treatment for a common childhood blood cancer could become the first gene therapy available in the U.S.
A Food and Drug Administration advisory panel voted 10-0 on Wednesday in favor of the leukemia treatment developed by the University of Pennsylvania and Novartis Corp. The FDA usually follows recommendations from its expert panels, but isn't obligated to do so.
CHICAGO (AP) — Doctors are reporting unprecedented success from a new cell and gene therapy for multiple myeloma, a blood cancer that's on the rise. Although it's early and the study is small — 35 people — every patient responded and all but two were in some level of remission within two months.
In a second study of nearly two dozen patients, everyone above a certain dose responded.
A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.
The first-in-the-world case is detailed in Thursday's New England Journal of Medicine.
An experimental gene therapy that turns a patient's own blood cells into cancer killers worked in a major study, with more than one-third of very sick lymphoma patients showing no sign of disease six months after a single treatment, its maker said Tuesday.
In all, 82 percent of patients had their cancer shrink at least by half at some point in the study.