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

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

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

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.

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.

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.

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 company said it took financial hits from the Inflation Reduction Act's Medicare Part D redesign, which included higher ...