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

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.

NEW YORK – UTR Therapeutics is planning to start a Phase I clinical trial of UTRxM1-18, its investigational mRNA destabilizer for treating c-MYC-driven cancers, in 2026, if the US Food and Drug ...

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

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.

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

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

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

NEW YORK – Haya Therapeutics on Thursday said it has raised $65 million in a Series A funding round, which it will put toward accelerating development of its therapeutic pipeline, including its long ...