GalNAc exploits millions of rapidly recycling receptors on hepatocytes to deliver siRNA with remarkable efficiency, enabling durable gene silencing after subcutaneous dosing while highlighting the unresolved challenge of extrahepatic RNA delivery.
Discover why Ionis' REVEAL enrollment turns obudanersen into a decisive Phase 3 test for Angelman syndrome treatment.
Tryngolza is no longer confined to the rare familial chylomicronemia syndrome market. Its broader United States approval gives clinicians the first therapy specifically shown to reduce acute pancreatitis risk in adults with severe hypertriglyceridemia, although questions surrounding dose selection, hepatic monitoring, reimbursement and long-term use will influence how quickly the treatment changes practice.
Find out how ARTHEx Biotech’s ATX-01 data could reshape RNA-targeted therapy for myotonic dystrophy today!
Explore why Codexis, Inc.’s latest RNA manufacturing advances could influence scalability, stereochemical control, and future siRNA production economics.
GLP-1 drugs dominate obesity. GSK is betting SiranBio’s SA030 can attack visceral fat risk from a different angle.
Vivatides Therapeutics raised $54 million to advance extrahepatic RNA therapeutics. Read why this platform bet could reshape RNA drug development.
Argo Biopharmaceutical Co., Ltd. (Argo Biopharma), a Shanghai-based clinical-stage RNA interference therapeutics company, has received Fast Track Designation from the U.S. Food and Drug Administration for BW-20805, its investigational small interfering RNA therapy targeting prekallikrein for the prevention of hereditary angioedema attacks. The designation comes as the company runs a global Phase II open-label study
NEJM publication of Avidity Biosciences delpacibart etedesiran data raises new questions for DM1 treatment. Discover what this changes next.
Avidity Biosciences sets spin-off record date for Atrium ahead of Novartis deal. Find out what this carve-out reveals about RNA platform risk and M&A strategy.