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Nucleic acid drugs

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

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Latest picks

61–80 of 80
Aug 25Tue
  1. WeChat88

    GSK's bepirovirsen wins world-first approval in Japan for functional cure of chronic hepatitis B

    Japan's Ministry of Health, Labour and Welfare has approved GSK's antisense oligonucleotide Hibsago (bepirovirsen) for adults with chronic hepatitis B who have received at least six months of nucleos(t)ide analogue therapy and meet predefined virological criteria, to achieve a functional cure. Announced on August 24, this is the drug's first approval anywhere in the world and Japan's only approved functional-cure treatment for chronic hepatitis B.

    Why it matters: First global approval of a functional-cure drug for chronic hepatitis B affects treatment options for a large patient population.

  2. WeChat80

    Japan Approves GSK Antisense Drug Bepirovirsen as First Functional Cure for Chronic Hepatitis B

    Japan's Ministry of Health, Labour and Welfare has approved GSK's investigational antisense oligonucleotide drug Hibsago (bepirovirsen) for adults with chronic hepatitis B virus infection, positioning it as a functional cure therapy. Functional cure means HBV DNA and hepatitis B surface antigen remain undetectable for at least 24 consecutive weeks after stopping all treatment. GSK announced the approval on August 24.

    Why it matters: First approved functional-cure therapy for chronic hepatitis B, a new treatment paradigm for a widespread infection.

  3. WeChat82

    GSK's antisense drug Bepirovirsen approved in Japan as first functional cure for chronic hepatitis B

    GSK's antisense oligonucleotide Bepirovirsen, licensed from Ionis, has been approved in Japan as a functional cure for chronic hepatitis B. It applies to adults with CHB infection who have received at least six months of nucleos(t)ide analogue therapy and meet viral marker criteria. The drug suppresses hepatitis B virus DNA replication, lowers HBsAg levels and stimulates a durable immune response. GSK filed for approval in China in May.

    Why it matters: First approved functional cure drug for chronic hepatitis B, with a China filing pending.

Aug 24Mon
  1. Science61

    De novo design of RNA pseudoknots with deep learning.

    Deep learning tools achieved accurate de novo design of RNA pseudoknot secondary structures, with generative AI matching experienced human designers on most blind challenges in an Eterna competition involving 57 pseudoknots. Designs were evaluated by single nucleotide-resolution chemical mapping, compensatory mutagenesis, and cryo-electron microscopy, and AI-generated molecules with accurate secondary structures formed well-ordered 3D folds stabilized by noncanonical tertiary interactions not modeled during design. Success was guided by an RNet foundation model trained on prior chemical mapping data, indicating that difficult RNA design tasks may be tractable without first solving RNA 3D structure prediction.

    Why it matters: AI matches expert human designers on blind RNA pseudoknot design without requiring 3D structure prediction.

  2. Cell65

    Functional atlas of vertebrate viral RNA elements that stabilize RNA and enhance translation.

    Massively parallel reporter assays covering roughly 200,000 genomic segments from 297 vertebrate-infecting viral genera identified viral RNA elements in 19 genera, grouped into six subclasses, that enhance RNA stability and translation via TENT4-mediated mixed tailing. Diverse TENT4-independent elements also emerged, including Potamipivirus Pt1, which stabilizes linear mRNA to levels comparable to circular RNA by recruiting PAPγ/α for cytoplasmic polyadenylation. The work charts viral RNA regulation and points to elements with RNA therapeutic potential.

    Why it matters: Large-scale virome survey yields catalogued RNA stability elements and a candidate tool for RNA therapeutics.

Aug 22Sat
  1. WeChat79

    Moderna and Merck Announce intismeran Plus KEYTRUDA Adjuvant Melanoma Trial Hits RFS and DMFS Endpoints

    Merck and Moderna jointly announced that the Phase III INTerpath-001 trial of intismeran autogene (V940/mRNA-4157) combined with KEYTRUDA (pembrolizumab) as adjuvant therapy for completely resected stage IIB-IV melanoma met both its primary endpoint of recurrence-free survival (RFS) and key secondary endpoint of distant metastasis-free survival (DMFS). The companies plan to present detailed data at an upcoming international medical conference.

    Why it matters: INTerpath-001 met both RFS and DMFS in adjuvant melanoma, informing head-to-head comparison of individualized neoantigen mRNA therapy with PD-1 monotherapy adjuvant.

Aug 21Fri
  1. WeChat77

    Personalized Neoantigen mRNA Cancer Vaccine Plus Keytruda Meets Pivotal RFS Primary Endpoint, Filing Planned

    A pivotal trial of a personalized neoantigen mRNA cancer vaccine combined with Keytruda met its primary endpoint of recurrence-free survival (RFS) and the key secondary endpoint of distant metastasis-free survival (DMFS) at a prespecified interim analysis. The combination showed statistically significant and clinically meaningful improvement over Keytruda monotherapy as adjuvant therapy in resected melanoma.

    Why it matters: The interim analysis provides RFS and DMFS data readers can compare directly with existing Keytruda monotherapy adjuvant data in resected melanoma.

  2. WeChat76

    Moderna Personalized mRNA Cancer Vaccine Succeeds in Phase III INTerpath-001 Randomized Trial

    Moderna's personalized mRNA cancer vaccine met its endpoint in the phase III randomized controlled INTerpath-001 trial. The study enrolled 1,137 patients with stage IIB-IV cutaneous melanoma after complete surgical resection and no prior systemic therapy. It is the first personalized neoantigen therapy to succeed in a phase III randomized controlled trial.

    Why it matters: The source gives the 1,137-patient stage IIB-IV post-surgery enrollment range and safety readout for comparison with prior phase II efficacy signals.

  3. WeChat71

    Moderna and Merck intismeran plus pembrolizumab posts positive Phase III topline in melanoma adjuvant therapy

    Moderna and Merck jointly announced that the Phase III INTerpath-001 trial of the personalized mRNA neoantigen therapy intismeran (V940/mRNA-4157) combined with pembrolizumab as adjuvant therapy for melanoma met positive topline results. Each intismeran course encodes up to 34 neoantigens, tailored to each patient's tumor mutation profile; Moderna shares surged 177% on the news.

    Why it matters: Reports the positive INTerpath-001 topline readout and Moderna's 177% share surge as a market reference for personalized mRNA neoantigen therapy in melanoma adjuvant treatment.

Aug 19Wed
  1. STAT74

    STAT+: Moderna and Merck say mRNA cancer vaccine succeeded in late-stage melanoma trial

    Moderna and Merck announced that the personalized mRNA neoantigen vaccine intismeran combined with Keytruda met its primary endpoint in a Phase 3 adjuvant melanoma trial, delaying recurrence and distant metastasis. The release did not provide the HR, confidence intervals, or detailed endpoint results; those await a full readout and comparison with existing adjuvant immunotherapy regimens.

    Why it matters: Topline result for a personalized mRNA vaccine plus Keytruda in adjuvant melanoma, but HR, confidence intervals, and stratification details were not released, so it serves only as a preliminary reference for Phase 3 neoantigen vaccine readouts.

  2. Nature71

    Enhanced antitumor immunity of mRNA vaccines by bioorthogonal-like delayed activation of exogeneous STING

    The paper reports a strategy for enhancing the antitumor immunity of mRNA vaccines using bioorthogonal-like delayed activation of exogenous STING. The approach delays STING activation so that it coincides with vaccine-induced antigen expression rather than acting immediately on delivery. No results or quantitative outcomes are stated in the material itself.

    Why it matters: It proposes a timed-activation STING adjuvant strategy for improving mRNA cancer vaccines.

  3. Cell56

    RNA structures regulate norovirus life cycle and enable rational attenuation in vivo

    Genome-wide mapping of RNA structure in murine norovirus reveals that disrupting specific structural elements impairs viral replication in cells and in vivo. The disruption yields a genetically stable virus that elicits protective immunity in vivo, providing a rational strategy for RNA virus vaccine design.

    Why it matters: Disrupting viral RNA structural elements to build live attenuated vaccines offers a rational strategy applicable to other RNA viruses.

Aug 8Sat
  1. WeChat72

    Phase 3 SHASTA-3 and SHASTA-4 Data Show siRNA Lipid-Lowering Drug Cut Median Triglycerides Nearly 80%

    An siRNA lipid-lowering drug achieved deep triglyceride reductions in the phase 3 SHASTA-3 and SHASTA-4 trials, with median decreases from baseline of 79% and 81% respectively. Safety and tolerability remained stable, with no new safety risk signals. On the day of the announcement the company's shares rose more than 20%, and the drug targets a population of about 3 million patients with severe hyperlipidemia.

    Why it matters: The brief gives median triglyceride reductions of 79% and 81% in SHASTA-3 and SHASTA-4, allowing comparison with other siRNA and lipid-lowering therapies.

  2. WeChat73

    FDA Approves Moderna Flu mRNA Vaccine mFLUSIVA for Adults 50 and Older

    The US FDA approved Moderna's flu mRNA vaccine mFLUSIVA (mRNA-1010) on August 5, 2026 for seasonal influenza in all adults aged 50 and older. The quadrivalent vaccine targets multiple influenza A and B strains. It is Moderna's fourth FDA-approved mRNA product and the third mRNA vaccine type to win regulatory approval beyond coronavirus and RSV vaccines.

    Why it matters: FDA approval expands approved mRNA vaccine classes beyond coronavirus and RSV.

Aug 7Fri
  1. WeChat82

    FDA Approves Moderna's mFLUSIVA, the First mRNA Flu Vaccine Worldwide

    The FDA has approved Moderna's seasonal influenza vaccine mFLUSIVA (mRNA-1010) for adults aged fifty and older, making it the first authorized mRNA flu vaccine in the US and worldwide. The approval rests on late-stage trials conducted across multiple countries in adults over fifty. The vaccine is Moderna's fifth approved product and will reach US retail pharmacies in the coming weeks for the approaching flu season.

    Why it matters: First-ever mRNA influenza vaccine approval affects seasonal flu prevention options for older adults.

  2. WeChat74

    mFLUSIVA Becomes First Approved mRNA Influenza Vaccine Worldwide

    mFLUSIVA has been approved as the world's first marketed mRNA influenza vaccine, extending the mRNA technology route in infectious disease prevention beyond the earlier coronavirus vaccines. The approval marks the platform's shift from an emergency tool for public health crises to a routine technology for seasonal infectious diseases. Seasonal influenza causes hundreds of thousands of deaths and millions of hospitalizations worldwide each year.

    Why it matters: First regulatory approval of an mRNA influenza vaccine extends the platform beyond coronavirus vaccines.

  3. WeChat77

    FDA Approves Moderna's mRNA Flu Vaccine mFLUSIVA for Adults 50 and Older

    The FDA has approved Moderna's mRNA flu vaccine mFLUSIVA (mRNA-1010) for adults aged 50 and older. It is the first mRNA flu vaccine licensed in the United States and the first mRNA product approved in a routine vaccine setting. Supply to US retail pharmacies is expected within the coming weeks.

    Why it matters: First mRNA influenza vaccine approval in the US extends mRNA technology beyond the pandemic use.

  4. WeChat72

    FDA Approves Moderna's mRNA Flu Vaccine mFLUSIVA for Adults 50 and Older

    On August 5, 2026, the FDA approved Moderna's mRNA flu vaccine mFLUSIVA (mRNA-1010) for adults aged 50 and older. It is the first mRNA flu vaccine approved in the US and Moderna's fifth product approved globally, its fourth FDA-approved product in the US. The approval extends mRNA technology from its pandemic use into influenza.

    Why it matters: First mRNA influenza vaccine cleared in the US, extending the mRNA platform into a new indication.

  5. WeChat75

    FDA Approves Moderna's mFLUSIVA, First Influenza mRNA Vaccine, for Adults 50 and Older

    The FDA has approved Moderna's mFLUSIVA (mRNA-1010), a seasonal influenza mRNA vaccine, for all adults aged 50 and older in the United States. mFLUSIVA becomes Moderna's fifth approved product globally and its fourth FDA approval, and the material describes it as the world's first approved influenza mRNA vaccine.

    Why it matters: FDA approval of the first influenza mRNA vaccine affects vaccination options for adults 50 and older.

Aug 4Tue
  1. WeChat68

    Nebulized Engineered Exosomes Suppressing Ferroptosis Ease PM2.5-Induced Pulmonary Fibrosis in Mice

    A team led by Zhang Rong and Bao Lei at Hebei Medical University published a study in Redox Biology describing engineered exosomes encapsulating miR-218-5p and modified with the iRGD targeting peptide, which significantly alleviated PM2.5-induced pulmonary fibrosis in mice when delivered by inhalation. In vitro experiments showed that exosomes secreted by PM2.5-exposed macrophages induced ferroptosis in epithelial cells, driving epithelial-mesenchymal transition, and targeted inhibition of HO-1 reversed this phenotype.

    Why it matters: It demonstrates an inhaled engineered exosome strategy for delivering miRNA to inhibit ferroptosis, suggesting a potential therapy for environmentally induced pulmonary fibrosis.

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