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.

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.

A "productive" meeting with the FDA has given the firm confidence in its regulatory filing and commercialization plans for ST-920.

Patients on Enhertu followed by a chemotherapy and dual anti-HER2 regimen fared better than those on standard therapy.

Phase II trial results indicated that treatment with the firm's antisense oligonucleotide led to stable or improved best-corrected visual acuity.

The team is exploring whether biomarker testing could also identify patients who benefit more from immune checkpoint inhibitors after radiation.