A Phase III trial evaluating nex-z in transthyretin amyloidosis with cardiomyopathy is underway, with plans for another ...

In a previous trial, patients with RSK2-high cancers had a significantly greater benefit from treatment with PMD-026 than those with RSK2-low cancers.

Blincyto plus chemo was approved for adult patients with Ph-negative, CD19-positive, and MRD-negative B-cell precursor acute lymphoblastic leukemia.

Bluebird Bio has commercialized three gene therapy products, including one of the first for sickle cell disease, but would soon run out of cash.

The firm plans to build on its organ and tissue modeling technology to develop more sophisticated tests, including full-body digital twins.

Researchers explored how Pluvicto is used in the community and academic settings and when given by oncologists or urologists.

The registrational trial will include 212 patients who are newly diagnosed with advanced head and neck cancer.

Boehringer Ingelheim, IP Group, UK Respiratory Gene Therapy Consortium, and OXB launched the first-in-human clinical trial, known as LENTICLAIR 1.

NEW YORK – Biogen and Stoke Therapeutics have entered into a collaboration to develop and commercialize zorevunersen, an antisense oligonucleotide (ASO) drug that Stoke has been developing for Dravet ...

NEW YORK – Boehringer Ingelheim on Wednesday said the US Food and Drug Administration accepted its new drug application for the irreversible tyrosine kinase inhibitor zongertinib as a treatment for ...

The bill's introduction by a Republican state representative earlier this month was met with opposition from genetic counselors.