#rna-therapeutics

All articles tagged with "rna-therapeutics"

Preclinical RNA therapy points to a treatment path for an ultrarare neurodevelopmental disorder

St. Jude researchers report that an antisense oligonucleotide therapy reversed key features of HNRNPH2-related neurodevelopmental disorder in preclinical models, offering mechanistic support for a move toward human study

Key Takeaways

St. Jude researchers used an antisense oligonucleotide to suppress aberrant HNRNPH2 in preclinical models.
The treatment also boosted HNRNPH1 expression and reduced multiple symptoms of the disorder.

DT Editorial Team·Apr 26, 2026·via medicalxpress.com

Biotech Briefing: Vivatides Raises $54 Million as Wegovy Distribution and Gilead Dealmaking Move Forward

A new China-U.S. biotech raised $54 million for preclinical RNA therapies, while Wegovy distribution rules changed in Europe and Gilead exercised a $45 million Kymera option.

Key Takeaways

Vivatides Therapeutics raised a $54 million Series A for preclinical RNA-focused R&D.
Endpoints reports Wegovy is dropping cold-chain requirements in the EU.

DT Editorial Team·Apr 10, 2026·via endpoints.news

Zodasiran Posts Early Trial Gains in Triglycerides and LDL-Linked Risk Markers

A phase 1 trial in Nature Medicine reports that zodasiran, an ANGPTL3-targeting small interfering RNA, lowered triglycerides and sharply reduced circulating ANGPTL3 across multiple dyslipidemia groups without serious and

Key Takeaways

Phase 1 data showed no serious treatment-related adverse events.
All study cohorts reported major ANGPTL3 and triglyceride reductions by week 16.

DT Editorial Team·Apr 8, 2026·via nature.com