Absstract of: US20260263627A1
This disclosure relates to compositions and methods for treating lung disorders, including, for example, Acute Respiratory Distress Syndrome (ARDS), Ventila-tor-Induced Lung Injury (VILI), Acute lung injury (ALI), and other acute and chronic lung disorders. Further, wherein a lipid nanoparticle, comprises: a) a VCAM-1 targeting molecule; and b) an RNA molecule encoding Kriippel-like Factor 2 (KLF2).
Absstract of: US20260263584A1
0000 The present disclosure provides methods and compositions for modulating the innate immunity to exogenous ribonucleic acid (RNA) when administered to a subject in need thereof. More particularly, the disclosure provides compositions comprising RNA encoding at least one antigen or therapeutic protein, and at least one compound that modulates the innate immunity to such RNA when administered to a subject in need thereof. The disclosure further provides methods of using such compounds to modulate the innate immunity of such compositions.
Absstract of: WO2026186810A1
Provided is a novel polymer that includes a repeating unit that is represented by general formula (I) (in which L1 represents a C1–3 alkylene group, L2 represents a C1–3 alkylene group or a group that is represented by formula (I-1), and q is 0 or 1). The polymer can be applied to a drug delivery system.
Absstract of: WO2026185840A1
The present invention relates to compositions in the form of colloids or emulsions for stabilizing growth factors, including a non-animal polysaccharide-based particle capable of binding to a cellulose binding domain (CBD); a fusion protein comprising a CBD linked by a short linker to a growth factor (GF) or a GF-derived agent, wherein the CBD is bound to the non-animal polysaccharide-based particle; and optionally water and oil, and to methods for preparation of oil-containing emulsions of the invention.
Absstract of: WO2026187803A1
Provided herein are gene editing systems and compositions directed to effectuate in vivo edits in the PCSK9 gene. Treatment or prevention of cardiovascular disease through disruption of the production of the PCSK9 protein through genetic editing and the reduction of the blood PCSK9 protein concentration is disclosed herein. Disclosed are nickase-based gene editing systems designed to effectuate the installation of insertions and/or deletions (indel variants) and/or non-synonymous variants in the coding sequence of PCSK9. The nickase-based gene editing systems generally comprise one or more mRNAs that encode one or more nickases and a paired guide oligonucleotides (e.g., gRNAs) that target the nickase to opposing strands of the PCSK9 gene and may be delivered in vivo to a mammalian subject in need thereof via a suitable delivery system, such as lipid nanoparticles (LNPs) (with or without GalNAc) intravenously, or otherwise, administered to a patient. The manufacturing, use, and formulation of the gene editing systems and compositions are also disclosed.
Absstract of: WO2026187805A1
Provided herein are gene editing systems and compositions directed to effectuate in vivo edits in the APOC3 gene. Treatment or prevention of cardiovascular disease through disruption of the production of apoC-III through genetic editing is disclosed herein. Disclosed are nickase-based gene editing systems designed to effectuate the installation of insertions and/or deletions (indel variants) and/or non-synonymous variants in the coding sequence of APOC3. The nickase-based gene editing systems generally comprise one or more mRNAs that encode one or more nickases and paired guide oligonucleotides (e.g., gRNAs) that target the nickase to opposing strands of the APOC3 gene and may be delivered in vivo to a mammalian subject in need thereof via a suitable delivery system, such as lipid nanoparticles (LNPs) (with or without GalNAc) intravenously, or otherwise, administered to a patient. The manufacturing, use, and formulation of the gene editing systems and compositions are also disclosed.
Absstract of: WO2026187823A1
This invention, in embodiments, relates to a method for inducing an immune response against a tumor in a subject which includes intratumorally (IT) administering into the tumor an immunotherapy composition comprising at least one immunoadjuvant, at least one immune checkpoint inhibitor, and a carrier. The carrier allows for visualization and/or controlled delivery, diffusion and/or retention of the at least one immunoadjuvant and the at least one immune checkpoint inhibitor into the tumor. This invention, in embodiments, further relates to an immunotherapy composition for intratumoral administration comprising at least one immunoadjuvant, at least one immune checkpoint inhibitor, and a carrier.
Absstract of: WO2025215034A1
The present invention provides novel conjugates comprising a magnetic particle and a multitude of viral structures covalently bound to said particle. These conjugates are suited for efficient extracorporeal removal of antibodies from blood. In particular, the conjugates are suited as adjuvant treatment in gene therapy.
Absstract of: EP4803097A1
0001 Disclosed is a Mycobacterium tuberculosis vaccine. Specifically, the vaccine comprises an immune composition comprising an antigen component and a particulate protein component. The particulate protein component comprises nanoparticle protein. The antigen component and the particulate protein component are covalently bound by means of a binding peptide 1 and a binding peptide 2 to form an immunogenic complex. The vaccine has excellent cell immunogenicity and antibody immunogenicity. The present invention also relates to a preparation method for the mycobacterium tuberculosis vaccine.
Absstract of: EP4803104A1
0001 The present invention relates to a use, as a drug delivery vehicle, of a gene carrier comprising a metal nanoparticle and a double-stranded nucleic acid molecule bound to a surface of the metal nanoparticle, in particular to a use of the carrier for the treatment of a metabolic disease, and to a pharmaceutical composition for the prevention or treatment of a metabolic disease comprising the same.
Absstract of: WO2025096329A1
The present disclosure is directed to methods of preparing peptide nanoparticle formulations, and in particular, peptide nanosuspensions, using low shear milling. More specifically, the disclosure is directed to methods of preparing peptide nanosuspensions by applying low frequency acoustic energy to an admixture comprising a peptide, an aqueous dispersion medium comprising a surface-active polymer and optionally a surfactant, and milling media, until the peptide has been milled to nanoparticle size. Also described are stable peptide nanosuspensions prepared by the methods.
Absstract of: WO2025096848A1
The disclosure relates to microparticles and nanoparticles comprising a porous polymer matrix comprising an uncapped polymer for sustained delivery of a net positively charged therapeutic agent. More particularly the disclosure relates to particles comprising PLGA or PLA which have a first state with relatively more interconnected pores at a first pH and a second state with relatively less interconnected pores at a second pH. Methods of making the particles and administering the particles are also provided.
Absstract of: WO2025097049A1
Disclosed herein are compositions and methods for CMC production of RNA therapeutic complexes (nanostructures) that contain SN-38 (7-Ethyl-10-hydroxycamptothecin) and/or Irinotecan. In particular, disclosed herein is an RNA nanoparticle having at least three synthetic RNA nucleotides coupled to each other, wherein the at least three synthetic RNA oligonucleotides form a central ore domain and at least three double-stranded arms arranged around the core domain and extending away from the central core domain, wherein at least one of the three double-stranded arms is conjugated with Irinotecan and/or SN-38 with an esterbond that is cleavable by esterase in cancer tissue or cancer cells.
Absstract of: WO2025096824A1
Disclosed are lipid nanoparticle (LNP) delivery systems that specifically target T cells. The LNP delivery system comprises antibodies conjugated to the surface of the LNP, e.g., via maleimide chemistry, that target at least two T cell surface proteins, e.g., CD3 and CD28. The LNP delivery system can have a single population of LNP conjugated to either a bispecific antiCD3/antiCD28 antibody, or two monospecific antiCD3 and antiCD28 antibodies, or two populations of LNP wherein each population comprises a monospecific antibody. The payload of the LNP delivery system can be, e.g., mRNA encoding chimeric antigen receptors (CAR), a linear DNA fragment or a plasmid encoding chimeric antigen receptors (CAR) or therapeutic proteins such as antibodies, components of a gene editing systems (e.g., CRISPR-Cas), small molecules, antibody-drug conjugates (ADC), and any combination thereof, either encapsulated in the LNP or attached to its surface (e.g., conjugated). Also provided are lipids, pharmaceutical compositions, kits, and methods of treatment.
Absstract of: EP4803532A1
An immunogenic fragment of African swine fever virus CD2V protein, a recombinant protein, an immunogenic composition, and a use thereof. The provided immunogenic fragment of African swine fever virus CD2V protein or a variant thereof with immunogenicity can greatly improve the expression quantity while retaining the strong immunocompetence.
Absstract of: EP4803629A1
0001 Provided is an isolated mRNA molecule, including a nucleotide sequence encoding a chimeric immunogenic polypeptide, where the chimeric immunogenic polypeptide includes an immunogenic fragment of hemagglutinin HA of influenza A H5N1, an immunogenic fragment of hemagglutinin HA of influenza A H1N1, and an immunogenic fragment of hemagglutinin HA of influenza B Victoria linked together. Further provided are a composition and a vaccine including the mRNA, a fusion protein encoded by the mRNA, and a method for inducing an immune response against an influenza virus in a subject using the mRNA, the composition, the vaccine, and the fusion protein.
Absstract of: WO2025096980A1
Disclosed are methods and compositions for functional genetic modifications at selected genomic sites such as KLKB1 gene. Also provided are cell populations, which comprise the functional genetic modification at one or more selected gene loci.
Absstract of: US20250144234A1
0000 This disclosure provides improved RNA molecules, including mRNA molecules that can be produced by in vitro transcription and are suitable for in vivo transfection using an appropriate delivery vehicle, such as a lipid nanoparticle (LNP) or targeted lipid nanoparticle (tLNP). The improved RNA include particular combinations of 5′ untranslated region (UTR) and 3′ UTR, particular 3′ UTRs, or particular open reading frame sequences. Also provided herein are compositions of the LNP, or tLNP with an antibody as a targeting moiety, such as anti-CD8 antibodies that are used as targeting moiety.
Absstract of: WO2025096940A1
The present invention relates to lipids that include novel triazole, and alkyl- and alkene-substituted amine compounds, intermediates in their synthesis, and uses of such triazole, and alkyl- and alkene-substituted amine compounds in, for example, lipid nanoparticle compositions for drug delivery and cancer treatments.
Absstract of: WO2025093745A1
The present invention relates to glycoengineered extracellular vesicles, as well as methods of production of such glycoengineered extracellular vesicles, methods of glycoengineering extracellular vesicles, and uses of glycoengineered extracellular vesicles.
Absstract of: WO2025091377A1
The present disclosure relates generally to lipids, lipid nanoparticle formulations, and methods of using the same for delivering nucleic acids, such as mRNA.
Absstract of: WO2025093665A1
The present disclosure relates generally to functionalized particles containing moieties capable of binding Immunoglobulin D (IgD), methods for producing them, and to pharmaceutical compositions containing them and their uses in medicine.
Absstract of: US20250144233A1
0000 Nanoparticle compositions for delivery of nucleic acids to subjects including aminoalkyl branched lipid-like molecules as carriers, and therapeutic or immunogenic nucleic acid agents enclosed within the nanoparticle containing are described. Also provided are methods for treating or preventing diseases or conditions in a subject by administering the nanoparticle compositions that provide immune responses and synergistic therapeutic or preventive effects.
Absstract of: WO2025095526A1
The present invention relates to a synthetic nucleic acid molecule comprising 3'-UTR polynucleotide with improved translation efficiency and a vaccine composition comprising the same, and more particularly to a synthetic nucleic acid molecule comprising 3'-UTR with improved translation efficiency manufactured by inclusion of specific motif and a codon-optimized signal sequence and an antigen encoding sequence, and a vaccine composition comprising the same. The synthetic nucleic acid molecule according to the present invention comprises a 3'-UTR polynucleotide with improved translation efficiency, which can effectively induce the expression of an antigenic polypeptide, which is useful for vaccine development because it can be expected to increase immunogenicity as a vaccine.
Nº publicación: EP4803075A1 09/09/2026
Applicant:
AGS THERAPEUTICS SAS [FR]
AGS Therapeutics SAS
Absstract of: EP4803075A1
0001 Provided are compositions and drug delivery systems containing extracellular vesicles from microalgae (MEVs) that are loaded with bioactive cargo. The MEVs are formulated and administered by a variety of routes of administration and have a variety of applications as therapeutics, including as vaccines, as anti-cancer therapeutics, as therapeutics for psychiatric diseases, disorders, and conditions as diagnostics, and other such uses.