The firm will test the drug with immune checkpoint inhibitors in NSCLC patients with low PD-L1 expression and KEAP1 and/or STK11 mutations.

Researchers will use Cellply's VivaCyte platform to conduct single-cell analyses of NK cells and evaluate their ability to treat the bone marrow cancer.

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.

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.

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

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

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.

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

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.

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