Jim Wilson‘s pioneering gene therapy explorations have helped launch eight companies focused on translating his 40 years of ...

Researchers from the Centenary Institute have uncovered a reason why some people with hemophilia A develop resistance to ...

Cell and gene therapies harbor great hopes for biomedicine. They are becoming increasingly important for diseases that are ...

Genentech’s short film draws on the experiences of multiple families caring for children with hemophilia A, seeking to ...

A man’s acquired hemophilia coincided with a rare liver disease, a connection that had not been reported before, per a case ...

FVIII, the blood clotting protein missing in hemophilia A, adopts multiple shapes, making it more likely to be targeted by ...

Intellia has leveraged its expertise in Crispr/Cas9 gene editing to advance a pipeline of in vivo and ex vivo therapies for diseases with high unmet medical needs. We believe Intellia's proprietary ...

Aug. 29, 2024 — For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of ...

Market Value and Growth In 2025, the Gene Therapy For Rare Disease Market Size is valued at approximately US$ 3.01 Billion in 2024, marking a substantial increase from previous years. This growth ...

The death of a patient in a trial of an experimental gene therapy casts new shadows over a technology once seen as the future ...

New research has found that men who carry a common genetic variant are twice as likely to develop dementia in their lifetime ...

Genetic testing for hereditary cancer unexpectedly uncovered a case of myelodysplastic syndrome (MDS), highlighting the need ...