Oligonucleotide– tag –
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Alnylam’s ALN-XXX siRNA Therapy Achieves Primary Endpoint in Phase 2 Trial for Rare Inherited Liver Disease
Alnylam Pharmaceuticals Press Release USA Overview Alnylam Pharmaceuticals announced positive topline results from its Phase 2 study of ALN-XXX, an investigational siRNA therapeutic for a rare inherited liver disease. The trial successfu... -
FDA Approves Hepcludex, First Treatment for Chronic Hepatitis Delta Virus (HDV) Infection
FDA (Press Release), European AIDS Treatment Group USA Overview The U.S. FDA has approved Hepcludex (bulevirtide-gmod) injection as the first treatment for chronic hepatitis delta virus (HDV) infection in adults, with or without compensa... -
Tuning siRNA Packing Order in Lipid Nanoparticles Controls Oligonucleotide Functional Delivery
bioRxiv (Preprint) International Overview This preprint investigates how tuning the siRNA packing order within lipid nanoparticles (LNPs) can modulate oligonucleotide functional delivery, addressing a key challenge in RNA therapeutics: e... -
GSK’s Bepirovirsen for Chronic Hepatitis B Receives FDA Priority Review and Breakthrough Therapy Designation
GSK USA Overview GSK announced that its investigational antisense oligonucleotide (ASO), Bepirovirsen, a candidate for chronic Hepatitis B, has been granted both Priority Review and Breakthrough Therapy Designation (BTD) by the U.S. FDA.... -
Wave Life Sciences Reports Positive RNA Editing Clinical Data for WVE-006 in Alpha-1 Antitrypsin Deficiency (AATD)
BioPharm International USA Overview Wave Life Sciences announced favorable clinical data from the RestorAATion-2 trial of WVE-006, an RNA editing therapeutic for Alpha-1 Antitrypsin Deficiency (AATD). WVE-006, a GalNAc-conjugated RNA edi... -
Veritas In Silico Advances mRNA-Targeted Small Molecule Drug Discovery with AI Platform ‘aibVIS,’ Bolstering European Collaborations
Investing.com (Fisco提供) Japan Overview Japanese biotech Veritas In Silico (VIS) is spearheading mRNA-targeted small molecule drug discovery using its proprietary AI platform, "aibVIS." This platform integrates in silico RNA structure a... -
Avidity Biosciences Leads Muscle Disease Therapy with Antibody-Oligonucleotide Conjugates (AOCs): DM1 HARBINGER™ Trial Advances to Phase 3
Bitget Global Overview Avidity Biosciences is a leading company in Antibody-Oligonucleotide Conjugates (AOCs), developing novel RNA therapeutics for muscle diseases. Its lead candidate, delpacibart etedesiran (AOC 1001), is progressing i... -
Silexion Therapeutics Initiates GMP Manufacturing for KRAS-Driven Cancer siRNA Therapy SIL204, Secures Phase 2/3 Trial Approval
Manila Times (via GlobeNewswire) ケイマン諸島 Overview Silexion Therapeutics has commenced GMP clinical batch manufacturing for SIL204, its next-generation siRNA therapeutic targeting mutated KRAS oncogenes. Concurrently, the company rec... -
The Modern CDMO: A Strategic Blueprint for Pharmaceutical Leaders in a Complex Era
Agnopharma Global Overview This guide outlines the evolution of CDMOs from traditional CMOs, emphasizing their indispensable role in contemporary drug development. Modern CDMOs offer integrated solutions from process development to comme... -
siRNA Therapeutics: Advancements in Chemical Modification and Targeted Delivery Expand Clinical Horizons Beyond the Liver
MDPI Global Overview siRNA therapeutics are achieving significant clinical success driven by sophisticated chemical modifications and robust delivery systems. GalNAc conjugates have become the gold standard for liver-targeted therapies, ...
