The firms will screen compounds from natural sources for activity against gene variants linked to chronic diseases.

In his first term, pharmaceutical companies took the "most favored nation" policy to court over the White House's effort to slash costs for patients.

After seeing promising results in preclinical studies, the firm hopes to begin Phase I trials shortly after getting IND clearance.

From decoy DNA sequences to plasmapheresis, researchers are testing strategies to get around anti-AAV antibodies that hinder ...

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

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.

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

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

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.