Japan-based Ono Pharmaceuticals has secured the global rights to sapablursen, an RNA-targeting therapy developed by Ionis ...

Avidity Biosciences, Inc. (NASDAQ:RNA) has emerged as a frontrunner in ... Del-zota: Designed for DMD exon 44 skipping, this therapy has generated considerable excitement and could potentially ...

Alternative RNA splicing is like a movie editor cutting and rearranging scenes from the same footage to create different ...

Researchers at Case Western Reserve University have discovered molecules that present the potential to drive the development ...

Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) released topline results from Part 2 of a Phase 1/2 study of ARO-C3, the ...

A new review article highlights the transformative role of circular RNA (circRNA) in cancer, revealing its potential as both a key player in tumor biology and a promising avenue for future therapies.

With further testing, discovery may offer new target for early detection and treatmentResearchers at Case Western Reserve ...

The one-time therapy appears to correct the mutation that causes the disease, known as Alpha-1 antitrypsin deficiency (AATD), ...

Researchers at Case Western Reserve University have discovered molecules that present the potential to drive the development of gastric cancer-among the world's deadliest forms of the disease.

Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program ...

While initial study results suggest Beam's technology can correct alpha-1 antitrypsin deficiency's genetic roots, shares fell ...