Major is one of the first pediatric patients in Florida to receive gene therapy for sickle cell disease since the FDA ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

ORLANDO -- A gene therapy that could transform CAR T-cell treatment achieved rapid and potentially durable measurable ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...

ROME, Italy, Dec. 10, 2025 /PRNewswire/ -- Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex ...

A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

T therapy has helped previously untreatable for T-cell acute lymphoblastic leukemia patients achieve remission, with 82% ...

A breakthrough therapy is driving aggressive leukaemia into remission. Here’s what the headlines don’t tell you about life ...

Discover why Cryoport (CYRX) offers deep value for investors as the leader in temperature-controlled logistics for cell and gene therapies.

A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) ...