The firm will assess irpagratinib plus best supportive care in patients with FGF19-overexpressing advanced liver cancer.

The firm will stop shipping the gene therapy for these patients until a new immunosuppressive regimen is approved for managing acute liver failure.

The firm is developing the PHOX2B-targeted cell therapy, which has shown promise in preclinical xenograft models, with CHOP researchers.

Studies doing genome-wide testing can easily ID patients with naturally low neutrophil counts, but nobody's looking at it, ...

Medical ethicists are bringing attention to an early chapter of precision medicine history, an often overlooked aspect of an ...

The drug is already approved for KMT2A-rearranged AML, and Syndax is hoping for approval in NPM1-mutated disease by the end of the year.

NEW YORK – Ionis Pharmaceuticals has treated the first patient in a Phase III clinical trial evaluating the efficacy and safety of its investigational antisense oligonucleotide (ASO) therapy for ...

The firm will use the funds to further develop its lead genetic medicine in Stargardt disease and advance its gene therapy pipeline in other indications.

The multimodal ArteraAI Prostate Test combines digital images of patients' biopsies and clinical data to predict prognoses and treatment outcomes.

The agency approved a perioperative Keytruda regimen based on a 30 percent reduction in the risk of recurrence, progression, or death versus the control arm.

The company plans to use some of the funds to support development of a kinase inhibitor in Phase I testing for BCR-ABL fusion-positive chronic myeloid leukemia.