New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...

Long-term follow-up shows 95% success rate, no serious complications in largest ADA-SCID gene therapy study to date ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...

In February, the biotech reported improvements in 10 of 11 children who received DB-OTO. The AAV gene therapy, which ...

Researchers have understood for decades that some forms of deafness are inherited, yet until now, few of those genetic clues ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

In May, a historic moment in science and medicine was captured in a single photo that circulated across news outlets ...

A new gene therapy approach aimed at protecting people with type 1 diabetes from developing diabetic kidney disease—a serious ...

Bayer agreed to acquire unlisted U.S. biotech firm Asklepios BioPharmaceutical Inc for as much as $4 billion in a bet on gene therapy with the help of modified viruses.