gene therapy

Scientist pipetting DNA (deoxyribonucleic acid) samples for testing during a clinical trial

Experimental gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions

By Laura Ungar, Associated Press

Sickle cell disease is complex on its own, but black men with the illness battle its stigmas and stereotypes too

Gene therapies for sickle cell disease approved in U.S.

Regulators on Friday approved two new gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the United States.

By Laura Ungar, Associated Press

Sickle Cell Disease, Human Blood Smear with Sickled cells, 500X. This blood smear shows numerous bizarre, abnormally shaped, and sickled erythrocytes (red blood cells). This single DNA base change causes an abnormal hemoglobin which causes the erythrocytes to bend into a sickle shape that can block circulation in small blood vessels.

The world’s first gene therapy for sickle cell disease has been approved in the UK

In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it had approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020.

By Maria Cheng, Associated Press

FILE PHOTO: Signage is seen outside of FDA headquarters in White Oak, Maryland

First gene therapy for deadly form of muscular dystrophy gets FDA approval for young children

The treatment from Sarepta Therapeutics was approved Thursday for children ages 4 and 5 with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes early death.

By Matthew Perrone, Associated Press

Human brain, computer illustration. Photo by Sebastian Kaulitzki/via Getty Images

New gene therapy delivers treatment directly to brain

About 30 U.S. studies testing gene therapy to the brain for various disorders are ongoing, according to the National Institutes of Health.

By Laura Ungar, Associated Press

FDA blocks much-anticipated BioMarin hemophilia gene therapy

By Linda A. Johnson, Associated PRess

Investors fled drug developer BioMarin in droves on Wednesday, driving shares down by a third after U.S. regulators rejected the company's potentially game-changing hemophilia A gene therapy.

Gene therapy was a boy’s last chance to stop leukemia. And it worked.

By Lesley McClurg, KQED Science

At least 50 children are in remission because of a breakthrough treatment that engineers the body’s own immune system to fight cancer.

At $850,000, price for new childhood blindness gene therapy four times too high, analysis says

By Andrew Joseph, STAT

Luxturna, a gene therapy with a list price $850,000, is the latest flashpoint in the debate over how to afford innovative medicines.

We may soon have our first $1 million drug. Who will pay for it? And how?

By Meghana Keshavan, STAT

As the first gene therapies approach the market, drug makers, insurers, and patients are debating complex questions about cost, value, and payment plans.

U.S. clears first ‘gene therapy’ for tough childhood leukemia

By Lauran Neergaard, Associated Press

Opening a new era in cancer care, the Food and Drug Administration on Wednesday approved the first treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.