Skip to content
中文EN

Nucleic acid drugs

siRNA, ASO, mRNA therapeutics and vaccines: delivery, indication expansion and safety.

80 picksRelatedGene therapy & editingRare diseaseCardiometabolic

Latest picks

21–40 of 80
Sep 28Mon
  1. Medical Xpress56

    Experimental mRNA therapy dramatically extends treatment window for acetaminophen overdose in preclinical models

    Lipid nanoparticle delivery of mRNA encoding the SRXN1 protein reduced liver injury in mouse models of acetaminophen overdose and still worked when given six hours after overdose, whereas N-acetylcysteine gives complete protection at one hour, partial protection at two hours and none beyond that in mice. The work traces the effect to a newly identified SRXN1-USP7-HO-1 pathway that stabilizes the antioxidant-defense molecule HO-1. SRXN1 levels rose in liver samples from patients who died of acetaminophen overdose, and mice lacking the protein suffered worse injury.

    Why it matters: It identifies a new liver-protection pathway and an mRNA therapy that extends the treatment window in mice.

  2. WeChat65

    mRNA-Encoded CD19×CD3 T-Cell Engager ABO2203 Drives Complete Remission in 3 Refractory Secondary ITP Patients

    An mRNA-encoded CD19×CD3 T-cell engager, ABO2203, achieved complete remission within one month in all 3 treated patients with refractory secondary ITP, and the responses held through 6 months of follow-up. The patients had systemic lupus erythematosus, Sjögren's syndrome, or antiphospholipid syndrome and had failed steroids, immunosuppressants, TPO-RA, and telitacicept. The work, from Ruijin Hospital and Abogen Biosciences, published in Cell.

    Why it matters: First-in-human use of an mRNA-encoded T-cell engager producing complete remissions in refractory secondary ITP.

  3. Nature56

    AI-guided optimization for thermostable mRNA vaccines

    AGENT, an AI-driven framework combining high-throughput experimentation with Bayesian optimization, engineers thermostable solid-state mRNA vaccines. The formulations retained full bioactivity after 2 months at 37 °C and elicited immune responses non-inferior to fresh vaccines. Microneedle patches enabled cold-chain-free delivery in rodents and nonhuman primates.

    Why it matters: Cold-chain-free mRNA vaccines with retained bioactivity could change vaccine distribution logistics.

  4. Nature42

    Accelerated discovery of thermostable mRNA–lipid nanoparticle vaccines using data-efficient AI

    The paper presents a data-efficient AI workflow, Bayesian optimization implemented in the AutODEx framework, for accelerating the discovery of thermostable mRNA-lipid nanoparticle vaccines. Candidate formulations were optimized toward stability, using a SARS-CoV-2 RBD sequence deposited at GenBank (OP839194). Datasets and source code are publicly released on Zenodo and GitHub.

    Why it matters: It shows an AI-driven route to thermostable mRNA-LNP vaccines, a known cold-chain bottleneck.

  5. bioRxiv57

    RNA isoform-resolved multiplexed sequencing with bioorthogonal barcoding

    Bobcodes, bioorthogonal barcodes attached at multiple internal positions along cellular RNAs via acylation and click chemistries, enable multiplexed sequencing that preserves splice junction information. Template switching onto tethered bobcodes during cDNA synthesis occurs with greater than 99% accuracy in species mixing experiments, and priming choice selects between 3'-end counting and full-length isoform capture. A BOB-seq v0.1 drug screen detects transcriptome-wide on- and off-target RNA splicing effects and outperforms existing multiplexing RNA sequencing methods in workflow simplicity, sample-to-sample variability, and barcoding accuracy.

    Why it matters: It adds isoform resolution to scalable multiplexed RNA sequencing, enabling transcriptome-wide splicing drug screens.

Sep 23Wed
  1. FierceBiotech58

    Roche’s phase 3 win tees up fight with Novartis for blockbuster kidney disease market

    Sefaxersen, a monthly subcutaneous antisense oligonucleotide inhibiting complement factor B, met the primary endpoint of a phase 3 trial in primary IgA nephropathy, significantly reducing proteinuria versus placebo at the Week 37 interim analysis. Safety and tolerability were consistent with earlier trials, and Roche plans regulatory talks, aiming for accelerated approval in a market where Novartis' oral factor B inhibitor Fabhalta is already fully authorized.

    Why it matters: Positive phase 3 proteinuria data position sefaxersen as a challenger to Fabhalta in the growing IgAN market.

  2. Science47

    The nucleoside analog kamuvudine-9 shows protective and therapeutic efficacy in a mouse model of multiple sclerosis

    The nucleoside analog kamuvudine-9 showed protective and therapeutic efficacy in a mouse model of multiple sclerosis. Published in Science Translational Medicine, the study indicates that kamuvudine-9 effectively alleviates disease symptoms and has therapeutic potential.

    Why it matters: Reports protective and therapeutic efficacy of a nucleoside analog in a multiple sclerosis mouse model.

Sep 22Tue
  1. WeChat70

    FDA Approves Ionis Antisense Drug Zanvastro as First Alexander Disease Therapy

    The FDA approved Ionis Pharmaceuticals' Zanvastro (zilganersen, development code ION373), an antisense oligonucleotide, for treating Alexander disease in children and adults. It is the first FDA-approved therapy for the disease and the first disease-modifying therapy directly targeting accumulation of the causal protein. The approval came on September 3, 2026, nearly three weeks ahead of its PDUFA date of September 22, 2026.

    Why it matters: First FDA-approved therapy for Alexander disease, a rare indication with no prior treatment.

Sep 18Fri
  1. WeChat43

    ABO2203: mRNA Encoding CD19×CD3 Bispecific Tested Against Refractory Immune Thrombocytopenia

    ABO2203 is an in vivo T-cell engager approach: an mRNA encoding a CD19×CD3 bispecific antibody, developed for refractory immune thrombocytopenia. It aims to deliver B-cell-directed bispecific therapy without the manufacturing burden, lymphodepletion and genomic-integration risks of CAR-T, and without the short half-life and continuous infusion required by protein TCEs such as blinatumomab. The work targets patients who respond inadequately to glucocorticoids, TPO receptor agonists, rituximab, BTK inhibitors or splenectomy.

    Why it matters: It introduces an mRNA-encoded CD19×CD3 bispecific, ABO2203, as an off-the-shelf option for refractory immune thrombocytopenia.

Sep 15Tue
  1. Nature Biotechnology56

    Moderna flu vaccine wins FDA approval.

    The FDA has approved Moderna's mRNA seasonal influenza vaccine mFlusiva for people aged 50-64. It is the first seasonal flu vaccine to use mRNA-based production, encoding full-length hemagglutinin from three WHO-recommended strains in lipid nanoparticles, allowing rapid updates to match circulating strains. In two clinical trials, mFlusiva outperformed GlaxoSmithKline's four-strain vaccine Fluarix in preventing infection or eliciting antibody responses.

    Why it matters: First mRNA-based seasonal flu vaccine approval sets a precedent for rapidly updated influenza vaccines.

Sep 14Mon
  1. Nature69

    Creating bottom-up RNA transfer vehicles from synthetic protein assemblies.

    AI-designed protein assemblies combined with natural protein domains yielded more than 100 bottom-up RNA transfer vehicles with unique sizes and shapes, surpassing the RNA transfer efficiency of widely used delivery vehicles by several orders of magnitude. Tropism was programmed with computationally designed peptide binders, enabling delivery of therapeutically relevant cargo RNAs across cellular models. One vehicle's in vivo biodistribution was mapped in a mouse at near-single-cell resolution, confirmed as safe, and used for a gene-editing treatment strategy for Duchenne muscular dystrophy in patient-derived cells and a pig.

    Why it matters: Generative AI-designed protein assemblies outperform established RNA delivery vehicles and work in vivo.

  2. 顶刊论文（社媒热度）47

    Amplification of RNA for identification of Zika and HCV in whole blood.

    A blood drying protocol with thermal treatments and primer-limited reverse transcription stabilizes RNA in whole blood, allowing direct amplification without extraction, purification, or cold-chain logistics. Detection reached 10 copies per microliter for Zika virus and 1 international unit per microliter for HCV, and larger blood volumes achieved single-copy sensitivity without sample loss. Zika and HCV were detected with a portable fluorometer, supporting field-deployable point-of-care diagnostics.

    Why it matters: It removes extraction and cold-chain requirements from whole-blood RNA testing, enabling point-of-care Zika and HCV diagnosis.

Sep 12Sat
  1. WeChat69

    Inhaled siRNA ARO-RAGE Shows Dose-Dependent RAGE Silencing in Human Lungs

    Inhaled ARO-RAGE safely and durably silenced the RAGE target in the lung in its first human trial, published in Nature Medicine by Matthias Salathe's group at the University of Kansas Medical Center with Arrowhead. In healthy volunteers and asthma patients, soluble RAGE in serum and bronchoalveolar lavage fluid fell in a dose-dependent manner. This is the first randomized controlled trial to demonstrate safe and effective pulmonary gene silencing by an inhaled siRNA in humans, though disease benefit still requires more data.

    Why it matters: First randomized controlled human trial proving an inhaled siRNA can safely silence a lung gene.

Sep 11Fri
  1. 顶刊论文（社媒热度）56

    Targeting tumor-associated macrophages using mRNA lipid nanoparticles for cytotoxic T lymphocyte-mediated cancer immunotherapy.

    Antibody-coated lipid nanoparticles targeting TREM2 on tumor-associated macrophages delivered mRNA encoding CXCL9 together with a Toll-like receptor agonist, reversing immunosuppression and increasing tumor infiltration and activity of cytotoxic T lymphocytes. Adding PD-L1 and CTLA-4 checkpoint inhibitors produced a CTL-favoring immunological environment and durable memory immunity, supporting TAM reprogramming as a route to better immunotherapy response.

    Why it matters: It offers a targeting solution for mRNA cytokine delivery to macrophages and shows synergy with checkpoint inhibitors.

  2. BioCentury51

    Novartis DM1 miss raises stakes for next wave of DMPK readouts

    Novartis' del-desiran (delpacibart etedesiran) missed the primary endpoint of video hand opening time (vHOT) versus placebo in the 150-patient Phase III HARBOR study in myotonic dystrophy type 1. The company reports evidence of activity in secondary and exploratory measures, including muscle strength and daily living activities, but has not disclosed numerical results or a presentation date. Four upcoming DMPK-targeting readouts will now test whether other candidates can translate molecular effects into clinical benefit.

    Why it matters: The Phase III miss for the most advanced DMPK-targeting therapy leaves the mechanism's clinical translatability unresolved ahead of four readouts.

Sep 10Thu
  1. Nature65

    Moderna flu vaccine wins FDA approval

    The FDA has approved Moderna's mFlusiva, an mRNA-based seasonal flu vaccine, for people aged 50–64. It is the first seasonal influenza vaccine to use mRNA production, encoding full-length hemagglutinin from three WHO-recommended strains delivered in lipid nanoparticles. In two clinical trials, mFlusiva outperformed GlaxoSmithKline's four-strain vaccine Fluarix in preventing influenza infection or eliciting antibody responses.

    Why it matters: First mRNA-based seasonal flu vaccine approval sets a regulatory precedent for Moderna's mRNA platform beyond its earlier vaccines.

Sep 9Wed
  1. Nature44

    Inhaled siRNA therapy targeting RAGE for pulmonary inflammation: a first-in-human randomized trial

    Inhaled siRNA directed against RAGE was safe and well tolerated in a first-in-human randomized trial, its first test in people after preclinical development. The lung-epithelium-targeted therapy reduced RAGE levels in both serum and bronchoalveolar lavage fluid, supporting gene silencing delivered by inhalation as a route to treating pulmonary inflammation.

    Why it matters: First-in-human evidence that inhaled siRNA can safely silence a pulmonary inflammation target in lung and blood.

  2. BioCentury56

    The factors that could have contributed to Novartis’ Lp(a) miss

    Pelacarsen, Novartis' antisense oligonucleotide targeting LPA, failed to improve cardiovascular outcomes in the Phase III HORIZON secondary prevention trial in patients with elevated Lp(a) levels. The miss undermines the company's first-in-class Lp(a) ambitions. Depending on the full data and on the fate of subsequent programs, the outcome could carry implications for antisense oligonucleotides, other RNA modalities and cardiovascular R&D more broadly.

    Why it matters: A Phase III failure for the first-in-class Lp(a)-lowering antisense pelacarsen could reshape RNA-directed cardiovascular drug development.

Sep 7Mon
  1. WeChat64

    Japan Approves GSK Antisense Therapy Hibsago for Functional Cure of Chronic Hepatitis B

    Japan's Ministry of Health, Labour and Welfare has approved GSK's antisense oligonucleotide therapy Hibsago (bepirovirsen) as the first treatment in Japan aimed at achieving a functional cure for chronic hepatitis B. It is indicated for adults who have received at least six months of nucleos(t)ide analogue therapy and meet predefined virological marker criteria. The approval adds an ASO-based option alongside recent siRNA advances in functional cure efforts.

    Why it matters: First approved functional-cure therapy for chronic hepatitis B in Japan, relevant to clinicians treating the infection.

Sep 6Sun
  1. Artif Cells Nanomed Biotechnol32

    Network pharmacology, and molecular docking studies for two flavonoid compounds isolated from Dodonaea viscosa towards SARS-CoV-2 infection and loaded in lipid nanoparticles

    Santin and viscosine isolated from Dodonaea viscosa, loaded into spanlastic nanovesicles, showed anti-SARS-CoV-2 activity in an MTT assay, with IC50 of 4.483 µg/ml for V1 and 9.795 µg/ml for S1. V1 inhibited virus adsorption while S1 showed 55.7% virucidal activity. Network pharmacology identified EGFR as the top annotated gene with 7 edges, and docking plus ADMET profiling supported drug-like, non-toxic behaviour.

    Why it matters: It links two plant flavonoids in spanlastic nanovesicles to measured anti-SARS-CoV-2 activity and mechanism.

CBACBA Frontier Insights (CFI)Chinese Biopharmaceutical Association (CBA)www.cba-usa.org