"I'm feeling great," said 19-year-old Caden Major.

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative ...

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 ...

Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...

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

Aradigm Health, a new benefits platform aimed at easing the financial sting of coverage for cell and gene therapies, has ...

The treatment uses a modified, harmless virus to deliver healthy genes to a patient’s cells to treat genetic diseases. Read ...

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

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

Xmoor Pharma has established a strategic collaboration with the Translational Research Office (TRO) and University College ...

Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s ...

Determining which patients should receive the treatment will still require individual risk-benefit assessments, experts say.