Ocugen has reported preliminary phase 2 data on its eye disease gene therapy, providing more evidence that the candidate may outperform existing treatments from Apellis Pharmaceuticals and Astellas ...

This image shows three brain cells with the faulty protein that causes Huntington’s disease. The bright yellow cell in the middle has built up a clump of this protein inside it. The blue spots in 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 ...

Analysts say second fatality heightens safety concerns, raises doubts about future demand Both patients suffered from acute liver failure Sarepta suspends forecast, stops shipments of therapy to some ...

Encoded Therapeutics has linked its Dravet syndrome gene therapy ETX101 to a 78% reduction in seizures, keeping the biotech on track to start a pivotal study next year. The Californian company’s ...

For years, cell and gene therapies were mainly associated with rare and orphan diseases, those that impact small patient groups and lack effective treatment options. However, recent developments in ...

Retiree Chip Hailey, who has a severe form of hemophilia B, participated in a groundbreaking gene therapy trial. Hailey experienced a life-threatening delay in treatment after a car accident, ...