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.

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

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.

Makers of organoids and cell models cheered the decision and said it will bolster the development of precision medicines, ...

A "productive" meeting with the FDA has given the firm confidence in its regulatory filing and commercialization plans for ST-920.

NEW YORK – UTR Therapeutics is planning to start a Phase I clinical trial of UTRxM1-18, its investigational mRNA destabilizer for treating c-MYC-driven cancers, in 2026, if the US Food and Drug ...

The agency approved the treatment based on data from 57 patients with previously treated KRAS-mutant recurrent low-grade serous ovarian cancer.

The firm will test LP-184 as a monotherapy and in combination with a PARP inhibitor in patients who have DNA damage repair gene mutations.

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