The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...

In gene therapy, a person's faulty genes are either replaced or modified to treat or prevent disease. With roughly two dozen products on the market and hundreds of clinical trials in progress or ...

For cell therapy, they help engineer immune cells like CAR-T cells to fight cancer. Gene editing tools like CRISPR/Cas-9 molecular scissors are delivered to help correct genetic defects. It's also ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Christmas has come a few days early for Spark Therapeutics after the FDA approved its ground-breaking gene therapy for a rare eye disease. It looked likely that the FDA would approve the US ...

Researchers have identified a method to enhance the effectiveness of a promising cancer treatment. They found that modifying a specific gene improves the ability of immune cells to combat cancer for ...

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first-of-its-kind genetic therapy for glioblastoma, a severe form of brain ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia. The annualized bleeding rate went from 10 to 0. No worrisome gene integrations were seen. The content of this ...