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

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

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 ...

NEW YORK – Syndax Pharmaceuticals unveiled new data this week from the Phase I/II AUGMENT-101 trial of its menin inhibitor Revuforj (revumenib) in patients with relapsed or refractory NPM1-mutated ...

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.

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 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.

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