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 ...

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

JABSOM Cell and Molecular Biology researcher Dr. Jesse Owens has spent the better part of two decades chasing a vision that ...

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

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

Gene therapy is now being used for certain cancers and sickle cell anemia.A South Florida teen was among the first to get ...

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

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

A long-term, trust-based CDMO partnership can support cell and gene therapy developers from early process development through ...

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

Qalsody is one step closer to being covered by public Canadian healthcare systems when used to treat adults with ALS with ...

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...