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

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.

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

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 ...