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 ...

If the agency approves its application, the firm will begin a Phase I clinical trial in c-MYC-driven cancers in 2026.

Two family members with a germline ALK-mutant neuroblastoma both experienced complete response to ALK inhibitors in a case study.

The funds will support a Phase I/II trial of its gene therapy candidate in an aggressive form of dilated cardiomyopathy due to LMNA gene mutations.