The Latest Breaking News from Gene Therapy

Teddi Shaw, from Northumberland, first recipient on health service of Libmeldy, world’s most expensive drug

Scientists say delivering gene therapy directly to the brain holds great promise

Social media users shared a range of false claims this week

A novel gene therapy developed at UC San Francisco is on track to improve the health of ten young children who were born without functioning immune systems and without the capacity to fight infections. Read on for details:

(This Dec.13 story has been corrected to remove reference to Sanofi in the fifth paragraph)

UK teenager is first person in the world to receive ‘base-edited’ cells that could transform cancer medicine

U.S. regulators approved CSL Behring’s hemophilia B gene therapy, a one-off infusion that frees patients from regular treatments but costs $3.5 million a dose, making it the most expensive medicine in the world.

Hemgenix frees patients from repeated treatments but initial cost makes it most expensive medicine in the world

The FDA just approved its most expensive drug ever — a hemophilia gene therapy from CSL and UniQure — and QURE stock popped Wednesday.

U.S. health regulators have approved the first gene therapy for hemophilia, a blood-clotting disorder with few treatment options

Something like 50 million people worldwide have epilepsy. While the majority of these people are able to use medications to manage and prevent their seizures, around one-third don’t respond well to these treatments. In such cases, the only option available to bring seizures under control is to remove the part…