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Researchers separated the effects of circular structure, preparation quality, and nucleotide modification across two vaccine antigen systems. Cleaner circular RNA preparations caused less innate immune activation, but antibody and T-cell response levels were comparable with linear mRNA. Incorporating m6A was associated with higher antibody avidity, rather than a general increase in expression.
Researchers screened PEG-free hybrid particles containing a polylactic acid core and engineered lipid surface. A lead formulation preserved particle size and reporter activity after vibrating-mesh nebulization, outperforming the tested SM-102 reference under those conditions. Non-nebulized particles also transfected organoids and primary airway cultures. The authors identify mucus transport, aerosol deposition, and in vivo efficacy as unresolved next steps.
Serial blood samples from 73 vaccine recipients were analyzed using ddPCR for total and long-range-linked mRNA and LC-MS for ionizable lipids. RNA and lipid decay differed across three vaccine platforms. Cohorts differed in dose, vaccination history, and sampling schedules. Detecting circulating RNA fragments does not by itself establish continued protein expression, clinical toxicity, or comparative vaccine effectiveness.
The CAPSULIX consortium of OZ Biosciences, ExploRNA, and Miltenyi received Eurostars support for a three-year project with a total budget of €1,518,766. It will combine novel cap-analog mRNA or self-amplifying RNA with biodegradable LNPs for nonviral CAR-T manufacturing. GMP-compatible process development and preclinical validation are objectives. The total project budget is not necessarily the amount of grant funding.
BioNTech reported that AHEAD-MERIT, a Phase 2/3 trial of BNT113 plus pembrolizumab, did not meet its progression-free survival dual primary endpoint versus pembrolizumab alone. The study enrolled patients with previously untreated, unresectable recurrent or metastatic HPV16-positive, PD-L1-expressing head and neck cancer. Overall-survival follow-up continues; under the prespecified design, a significant survival benefit could still establish a positive trial outcome.
This repeat-dosing study examined whether changes in PEG chemistry, PEG concentration, RNA cargo, or administration route could overcome efficacy loss after an immunogenic nanoparticle dose. None reliably restored delivery in the tested setting, and reduced activity tracked anti-PEG antibodies. The results emphasize the interaction between ionizable lipids and innate immunity, without establishing that every PEG-containing product will behave identically.
Researchers combined a 520-member degradable lipid library with interpretable machine learning and a 10,920-member virtual design space. Independent mouse experiments identified PA97MD101 as a lead for hepatic mRNA expression, Cre-mediated recombination, and Cas9-based TTR suppression. The study links screening predictions with functional animal validation, without establishing clinical safety or efficacy.
RinuaGene announced an October 4 collaboration framework with the Medicines Patent Pool to explore development, potential licensing, technology transfer, and local manufacturing of investigational therapeutic mRNA candidate RG002 in selected low- and middle-income countries. Other mRNA candidates may also be assessed. This is an exploratory framework, not a completed license, completed technology transfer, or WHO endorsement of an approved product.
A design-of-experiments approach produced an SM-102-derived formulation with less hepatic reporter expression and preserved or improved expression after local administration. Experiments included intramuscular, intracerebroventricular, and intravitreal routes. Mechanistic analyses associated the change with altered protein binding, systemic exposure, and hepatocyte trafficking. These route-specific results support formulation optimization but do not demonstrate noninvasive access to the brain or eye.
A study designed ionizable lipids specifically for circular RNA, whose topology differs from linear mRNA. Lead lipid iL-19 combined aminoethoxy head groups with doubly branched tails and improved protein expression after intramuscular delivery in mice relative to several benchmark lipids. The results establish preclinical design principles, not clinical superiority.
A C12-200-based formulation was optimized for movement and cellular delivery in the protein-poor vitreous environment. It co-delivered RB1 mRNA and triptolide, improving antiproliferative activity and suppressing tumors in an orthotopic retinoblastoma model after intravitreal administration. Retinal evaluations supported tolerability during the study period. The combination remains preclinical and requires longer ocular safety assessment and pediatric translation studies.
Researchers selected eight hemagglutinin sequences to represent circulating swine influenza diversity and evaluated individual and multivalent mRNA vaccines in pigs. The multivalent formulation elicited responses against its component antigens and protected against lung lesions after the tested challenge. This large-animal veterinary study supports adaptable multivalent design, while protection across untested strains and direct relevance to human influenza remain unresolved.
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