Two family members with a germline ALK-mutant neuroblastoma both experienced complete response to ALK inhibitors in a case study.

If the agency approves its application, the firm will begin a Phase I clinical trial in c-MYC-driven cancers in 2026.

The funds will support a Phase I/II trial of its gene therapy candidate in an aggressive form of dilated cardiomyopathy due to LMNA gene mutations.

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

The agency approved the treatment based on data from 57 patients with previously treated KRAS-mutant recurrent low-grade serous ovarian cancer.

The firm will test LP-184 as a monotherapy and in combination with a PARP inhibitor in patients who have DNA damage repair gene mutations.

NEW YORK – Sangamo Therapeutics is hoping to submit a biologics license application to the US Food and Drug Administration in 2026 and seek approval to market its gene therapy for Fabry disease, ...

NEW YORK – AstraZeneca on Wednesday said it will share with regulators data from the DESTINY-Breast11 trial showing that the antibody-drug conjugate Enhertu (trastuzumab deruxtecan), followed by a ...

The study will include two cohorts of breast cancer patients: triple-negative breast cancer and HR-positive, HER2-negative breast cancer.

The firm develops RNA-guided therapeutics that target the regulatory genome, composed of genes that regulate expression of other genes.