Oligonucleotide– tag –
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Ractigen’s SOD1-ALS siRNA Therapy RAG-17 Shows Significant SOD1 Reduction and Favorable Safety in Phase 1, Published in Nature Medicine
BioSpace USA Overview Ractigen Therapeutics published preclinical and positive First-in-Human Phase 1 clinical data for RAG-17, an siRNA therapy for SOD1-ALS, in Nature Medicine. The trial met primary safety endpoints with no serious adv... -
UBC-Led Global Review Reaffirms mRNA Vaccine Safety & Efficacy, Accelerating Applications in Cancer and Beyond
UBC News - The University of British Columbia Canada Overview An extensive global review led by researchers at the University of British Columbia has unequivocally reaffirmed the safety and efficacy of COVID-19 mRNA vaccines. Based on bi... -
COVID-19 mRNA Vaccines Comprehensively Proven Safe and Effective, Establishing Foundation for Future mRNA Therapeutics
CIDRAP (Center for Infectious Disease Research and Policy) USA Overview Extensive real-world use and rigorous evaluation of COVID-19 mRNA vaccines have conclusively demonstrated their comprehensive safety and efficacy, establishing a rob... -
Patisiran and Inotersen Halt Polyneuropathy Progression in hATTR Amyloidosis; Next-Gen siRNA Vutrisiran Also FDA Approved
oneAMYLOIDOSISvoice USA Overview Patisiran (Onpattro) and inotersen (Tegsedi), TTR mRNA silencing therapies for hereditary ATTR amyloidosis (hATTR), have demonstrated the ability to halt the progression of polyneuropathy and even show im... -
Engineered Antibody Brainshuttle™ Technology Boosts Brain Drug Delivery of RNA-Based ASOs by Dual-Targeting TfR1 and CD98hc Receptors, Illuminating CNS Disease Treatment
EMJ Unknown Overview Antibody-based Brainshuttle™ technology has made significant strides in brain drug delivery for promising therapeutics like RNA-based antisense oligonucleotides (ASOs), which are otherwise unable to cross the blood-b... -
Ionis Enters Global Licensing Deal with Recordati for Alexander Disease ASO Zilganersen: Secures $30M Upfront Payment to Accelerate International Expansion
Rare Disease Report USA Overview Ionis Pharmaceuticals has entered a global licensing agreement with Recordati for zilganersen, an antisense oligonucleotide (ASO) for Alexander disease, covering territories outside the U.S. Ionis will re... -
Asahi Kasei’s Novel Forward Osmosis–Membrane Distillation System Installed at Peptistar API Manufacturing Facility to Reduce Pharmaceutical Manufacturing Time for Thermolabile Peptides and Oligonucleotides, Advancing Towards GMP Production
Asahi Kasei Japan Overview Asahi Kasei has implemented a novel forward osmosis–membrane distillation (FO-MD) system at Peptistar's API manufacturing facility to shorten production times for thermolabile APIs like peptides and oligonucleo... -
Focused Ultrasound BBB Opening Facilitates Limited LNP-modRNA Brain Delivery in Niemann-Pick Disease Type C Mouse Model but Fails to Induce Therapeutic Effect
bioRxiv Unknown Overview Efficient molecular therapeutic delivery to the central nervous system (CNS) remains a major barrier in neurogenetic disorders like Niemann-Pick Disease Type C (NPC). This preprint evaluated Focused ultrasound-me... -
EAACI 2026: Wave Life Sciences’ siRNA BW-20805 Shows 93-99% Attack Rate Reduction in Hereditary Angioedema Phase 2, with 60-83% Patients Remaining Attack-Free
EMJ Allergy & Immunology Unknown Overview At the EAACI 2026 conference, updated Phase 2 data for Wave Life Sciences' BW-20805, a plasma prekallikrein-targeting GalNAc-siRNA, revealed highly promising results in adult patients with heredi... -
Identifying Blood-Brain Barrier (BBB) Bottleneck: Nanomedicine Key to Overcoming Glioblastoma Brain Drug Delivery Challenges
Neuroscience News Unknown Overview The blood-brain barrier (BBB), a major obstacle to brain drug delivery, severely limits therapies for neurological disorders like glioblastoma. Recent research identified intracellular sorting after dru...
