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Molecular dynamics simulations examined tecovirimat across solvent and concentration conditions. Molecular conformations changed relatively little, while intermolecular association patterns differed substantially between benzene and ethyl acetate. Those associations resembled packing motifs in known anhydrous polymorphs IV and VI. The study offers a mechanistic computational explanation; it does not establish prospective experimental prediction of polymorph outcomes.
Amphista announced the first patient dose in BRAMLIE, a Phase 1 study of AMX-883 in acute myeloid leukemia and high-risk myelodysplastic syndrome. The oral candidate recruits DCAF16 to degrade BRD9. The study will evaluate safety, pharmacokinetics, and preliminary activity, moving a non-cereblon, non-VHL degradation approach beyond preclinical models and into patients.
Catalent and Mantra have signed a commercial supply agreement covering single-serve over-the-counter oral gels. Catalent will provide formulation development, manufacturing, and packaging at its St. Petersburg, Florida, facility for products addressing occasional sleeplessness, pain, and menstrual discomfort. The announcement establishes a development and supply relationship; it does not report new clinical efficacy data or regulatory approvals for the portfolio.
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.
Triastek and its Shanghai subsidiary entered a collaboration and license agreement with Janssen Pharmaceutica NV to develop oral medicines using 3D printing and delivery technologies for peptides and other modalities. JJDC will also make an equity investment. Financial terms and named collaboration candidates were undisclosed, and the agreement does not establish clinical performance for T25.
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.
A GEN commentary by a USP principal scientist argues that mRNA development is shifting toward precision delivery across cancer, protein replacement, encoded antibodies, regenerative medicine, and gene editing. It emphasizes reaching appropriate tissues and cell types while limiting unintended exposure. This is an industry perspective on development priorities, not a new clinical readout.
Researchers prepared myricetin cocrystals with diprophylline and theobromine and evaluated structure, stability, moisture sensitivity, dissolution, and in vivo absorption. Both forms improved in vitro solubility and reduced hygroscopicity. However, their pharmacokinetic behavior did not simply track the solubility gains, reinforcing the need to assess solid-form performance across laboratory and biological endpoints rather than a single property.
A Nature Chemical Biology study used high-throughput proteomics and machine learning to investigate toxicity in experimental androgen receptor degraders. Researchers linked off-target effects to mitochondrial complex I inhibition and identified small linker changes that reduced hepatotoxicity signals. Optimized compounds retained target degradation and showed improved activity in prostate cancer xenografts, providing preclinical evidence for a safer design workflow.
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