A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...

An experimental MeiraGTx gene therapy that has clinical data showing reversal of blindness in children born with a rare, inherited eye disorder is heading to Eli Lilly. According to the terms of the ...

The two main approaches for discovering disease genes reveal distinct aspects of biology, a new study shows. While both methods are widely used, the research found that they identify different genes ...

The FDA is investigating the deaths of two boys with Duchenne muscular dystrophy who died soon after being treated with delandistrogene moxeparvovec (Elevidys) gene therapy, the agency said. The ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...

Opal Sandy underwent a gene therapy injection to her right ear in 2023. A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...

The first participant in an investigational gene therapy trial of STXBP1 encephalopathy died, Capsida Biotherapeutics said. The death occurred days after dosing, according to information posted on the ...

Biogen has discontinued all gene therapy programs using adeno-associated virus (AAV) capsids, instead shifting resources to modalities that have the highest likelihood of achieving better treatment ...

(CNN) — There may be some hope for those living with Huntington’s disease. A new experimental gene therapy was found to slow the progression of the disease. Drugmaker Uniqure announced the results of ...

The first study to evaluate both eyes treated with a gene therapy targeting neovascular, or wet, age-related macular degeneration (AMD) has found the treatment as safe and effective in the second eye ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...