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 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.

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

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.

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

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

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

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

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