Ionis Pharmaceuticals is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The ...

The UPLC system was configured with an Acquity Premier Oligonucleotide C18 column (1.7 µm, 2.1 × 50 mm). The aqueous and ...

Emerging modalities such as self-amplifying and circular RNA, expanding oncology and rare-disease applications, and scalable manufacturing are creating high-growth investment avenues, supported by ...

Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing ...

FDA clearance of IND application for VO659 with plans to initiate US-based clinical trial later this yearNew data highlight distinctive mechanism of action of exon 1–directed CAG repeat–targeting ASO ...

FDA's Plausible Mechanism Framework streamlines individualized therapy approvals for ultra-rare diseases.

Companies developing therapies for rare diseases could find it easier to bring their treatments to market after the FDA ...

New study reveals how cytoplasmic and nuclear functions of USP30-AS1 converge to promote tumor growth Breast cancer progression is increasingly ...

Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company dedicated to restoring protein expression by harnessing ...

With big pharma signalling interest in novel RNA-targeted approaches, the term “druggable” is being redefined as technology advances.