The FDA will issue a decision on the BLA for RGX-121 by November, which if positive, could make it the first gene therapy for Hunter syndrome.

India already has a homegrown CAR T-cell therapy, and other regions are exploring their own strategies, including developing them at the point of care.

In his first term, pharmaceutical companies took the "most favored nation" policy to court over the White House's effort to slash costs for patients.

The firms will screen compounds from natural sources for activity against gene variants linked to chronic diseases.

After seeing promising results in preclinical studies, the firm hopes to begin Phase I trials shortly after getting IND clearance.

From decoy DNA sequences to plasmapheresis, researchers are testing strategies to get around anti-AAV antibodies that hinder ...