Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available.

Scientists have reversed diabetic heart failure with a genetic therapy in mice and miniature human "hearts" grown from stem cells. The new gene therapy may be a promising treatment for diabetic heart ...

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 ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

"I am absolutely thrilled and excited that we now have a potential treatment for this truly devastating disorder and what the data means is that for a person who has Huntington's disease, they will be ...

Once a booming field, bustling with innovation and money driving that innovation, the cell and gene therapy sector is now struggling to fuel therapeutic growth to bring medicines to patients. It doesn ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

It can cure deadly diseases, save long-term healthcare costs and transform lives. But the U.S. insurance system still isn’t ready to pay for it. Imagine telling a child with sickle cell disease that a ...

Gene therapy biotech Kriya Therapeutics has secured $313 million in a new series fundraise. The private U.S. company disclosed the equity financing in a Securities and Exchange Commission document ...