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 ...

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.

Trump administration health officials assured drugmakers and researchers at a roundtable hosted by the FDA that they want to ...

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.

Researchers are trying to better understand how co-mutations and PD-L1 expression impact response to single-agent KRAS G12C ...

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 ...

Only 6 percent of patients carried a concerning DPYD variant, but nearly two-thirds of them were later hospitalized after ...

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.

As with the drug's 2023 approval in castration-resistant tumors, it's not clear if it works comparably in all homologous ...