Absstract of: US2025312482A1
Described herein are lipid nanoparticle (LNP) formulations with demonstrated tropism towards smooth muscle cells. Also described herein are LNPs conjugated with peptides that can target tissue or cell surface receptors. The formulations of the disclosure include amounts of DOTAP, an ionizable lipid, amounts of a neutral lipid; amounts of cholesterol; and amounts of one or more PEG-lipids with preferential tropism towards vascular smooth muscle cells (vSMCs). Also described herein are peptides that target receptors highly expressed on the surface of vSMCs (IL-6R, CD63 and GAL-3) or that target proteins in the extracellular matrix adjacent to vSMCs (Col-IV) increasing the uptake into these cells.
Absstract of: US2025312481A1
A genetic delivery nanoparticle includes gelatin, a lipidoid, and a genetic molecule payload. The genetic molecule payload can be an RNA, DNA or Crispr system payload. SiRNA is an example payload and can be encapsulated with the gelatin and lipidoid and covalently conjugated to a surface of the nanoparticle. The nanoparticle surface can also include an antibody and PEG conjugated to it. A method for forming genetic delivery nanoparticle includes forming an adduct of gelatin, the lipidoid and a genetic payload; and cross-linking the gelatin to form the genetic delivery nanoparticle. Varying the size of a carbon chain in the lipidoid controls the size of the formed genetic delivery nanoparticle.
Absstract of: US2025312430A1
Lipid nanoparticle formulations with cell type specific transfection activity and capable of producing Th1 and/or Th2 response in vivo and their use for plasmid DNA or mRNA delivery is disclosed.
Absstract of: US2025312442A1
The disclosure relates to EBV ribonucleic acid vaccines as well as methods of using the vaccines and compositions comprising the vaccines.
Absstract of: US2025313857A1
Provided herein are compositions and methods for gene therapy for disorders of arterial calcification as well as Generalized Arterial Calcification of Infancy (GACI). The methods include a gene addition strategy to deliver a DNA construct to target tissues (such as liver and smooth muscle cells) to express soluble recombinant ENPP1 (srENPP1) or transmembrane full-length recombinant ENPP1 (rENPP1).
Absstract of: US2025313830A1
The disclosure features a polynucleotide encoding a polypeptide, which polynucleotide comprises a 5′ UTR, a coding region encoding a polypeptide, and a 3 UTR, and lipid nanoparticles comprising the same. The polynucleotides and/or lipid nanoparticles of the present disclosure can increase the level and/or activity of the polypeptide by increasing the half-life and/or duration of expression of the polynucleotide encoding the polypeptide. Also disclosed herein are methods of treating a disease or disorder in a subject using the lipid nanoparticles of the present disclosure.
Absstract of: US2025312455A1
The present invention relates to plasmonic metal-collagen hybrid nanoparticles that have excellent protein loading efficiency and excellent biocompatibility and can be utilized as protein delivery vehicles. In the present invention, a plasmonic metal-collagen hybrid nanoparticle for protein delivery, comprising a collagen of 1 kDa to 30 kDa, a thermosensitive polymer monomer, a plasmonic metal nanoparticle, and a photoinitiator, and a method for preparing the same are provided.
Absstract of: US2025312483A1
The present invention relates to silica particles comprising nucleic acids encapsulated inside the same. Furthermore, the present invention relates to a method for producing said particles and the uses thereof in gene transfer or cell marking and as a medicinal product, specifically as a medicinal product for protein/enzyme replacement therapy.
Absstract of: AU2024243121A1
The present invention relates to stabilizing an oil in water nanoemulsion in a pressurized container. The nanovesicles comprised in the nanoemulsion are particularly stable in regard to vesicle size and vesicle size homogeneity after long-term storage at different temperatures.
Absstract of: AU2024253808A1
The present invention relates to oil in water nanoemulsions which are essentially free of propylene glycol. The nanovesicle formulations are particularly stable in regard to shelf life at different storage temperatures.
Absstract of: AU2024252371A1
The present disclosure relates to novel compounds, methods, and cell-targeting formulations, e.g.. a lipid nanoparticle (LNP) for targeted delivery to a tissue or a cell type. The compound and formulation provided herein are designed to have a targeting moiety configured to provide selective delivery features for the formulation and a lipid tail for being incorporated into the bilayer membrane of the formed lipid nanoparticle.
Absstract of: AU2024236558A1
The present disclosure describes improved LNP-based nucleobase editing systems and therapeutics for use in treating a disease. In particular, the disclosure describes improved LNPs, including novel and improved ionic lipids for making LNPs, that enhance the targeted delivery of LNP-based nucleobase editing systems and therapeutics based on linear and/or circular mRNAs. The improved LNPs protect linear and/or circular mRNA payloads from degradation and clearance while achieving targeted systemic or local delivery for use as enhanced nucleobase editing systems and/or therapeutic agents.
Absstract of: MX2025009878A
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Absstract of: AU2024225887A1
The present application discloses compositions comprising nanoparticles of vitamin K2, and their methods of use.
Absstract of: WO2025212858A1
Disclosed are in vitro transcribed (IVT) messenger RNAs (mRNAs) comprising a polynucleotide that encodes human TJP2 gene, a 5' terminal cap, and a poly-adenylation (poly- A) tail at the 3' terminal end. In aspects, the polynucleotide is substantially free of uridine bases and comprises pseudouridine bases. Methods, compositions and uses employing the IVT mRNA for treating or preventing progressive familial intrahepatic Cholestasis type IV (PFIC type 4) in a patient in need thereof is also disclosed.
Absstract of: WO2025212932A1
Described herein are methods of synthesizing ionizable amine lipids, such as Lipid A: Formula (I) with increased overall purity, process robustness and an applied impurity control strategy, leading to a substantially pure Lipid A product. Intermediate compounds and methods of synthesizing intermediate compounds are also described.
Absstract of: WO2025212530A1
The present disclosure provides nanoparticle compositions in which individual nanoparticles comprise payloads, as well as methods of making and using such nanoparticle compositions, and various compositions and/or technologies relating to such nanoparticle compositions, their production, and/or their use.
Absstract of: WO2025210521A1
The present disclosure provides lipid nanoparticles that are particularly beneficial in inducing an immune response in a subject, particularly by stimulating CD8+ T cells associated with protein or protein fragments of interest. The lipid nanoparticles can have a negative zeta potential and/or particular configurations and/or relative amounts of a phospholipid, an ionizable lipid, and a conjugated lipid. The disclosure also provides vaccines, other pharmaceutical compositions, and methods including the provided lipid nanoparticles.
Absstract of: WO2025208819A1
The present invention relates to polymer microspheres loaded with metal nanoparticles, a preparation method therefor, and use thereof. The present invention pertains to the technical field of tumor embolization therapy. The polymer microspheres loaded with metal nanoparticles are characterized by comprising a polymer microsphere framework and metal nanoparticles distributed in the polymer microsphere framework; the metal nanoparticles are manganese nanoparticles or iron nanoparticles; the particle size of the metal nanoparticles is 600 nm-1000 nm. The particle size of the polymer microspheres meets the requirements of embolization therapy. The polymer microspheres can embolize local blood vessels, achieving an embolization therapy effect. Due to the addition of the metal nanoparticles, the microspheres have a higher rigidity and a better embolization effect than traditional polymer microspheres. The preparation yield of the polymer microspheres loaded with metal nanoparticles of the present invention is significantly higher (2-3 times) than the yield of polymer microspheres loaded with active metal microparticles such as magnesium microparticles, the production efficiency and the production safety are higher, and the production cost is lower.
Absstract of: WO2025213139A1
Provided herein are compositions and methods for gene therapy for disorders of arterial calcification as well as Generalized Arterial Calcification of Infancy (GACI). The methods include a gene addition strategy to deliver a DNA construct to target tissues (such as liver and smooth muscle cells) to express soluble recombinant ENPP1 (srENPP1) or transmembrane full-length recombinant ENPP1 (rENPP1).
Absstract of: WO2025210520A1
The present disclosure provides lipid nanoparticles that are particularly beneficial in inducing an immune response in a subject, particularly by stimulating production of antibodies by the subject. The lipid nanoparticles can include one or more of a low conjugated lipid concentration, a neutral phospholipid, and a concentration of an ionizable lipid and/or sterol that satisfy certain thresholds dependent on the conjugated lipid concentration. The disclosure also provides vaccines, other pharmaceutical compositions, and methods including the provided lipid nanoparticles.
Absstract of: WO2025213135A1
Described herein are lipid nanoparticle (LNP) formulations with demonstrated tropism towards smooth muscle cells. Also described herein are LNPs conjugated with peptides that can target tissue or cell surface receptors. The formulations of the disclosure include amounts of DOTAP, an ionizable lipid, amounts of a neutral lipid; amounts of cholesterol; and amounts of one or more PEG-lipids with preferential tropism towards vascular smooth muscle cells (vSMCs). Also described herein are peptides that target receptors highly expressed on the surface of vSMCs (1L-6R, CD63 and GAL-3) or that target proteins in the extracellular matrix adjacent to vSMCs (Col-IV) increasing the uptake into these cells.
Absstract of: WO2025212420A1
A supraparticle includes and can consist of, ultrasmall Au nanoparticles, a stabilizing matrix, and a lipidoid. The supraparticle can advantageously include a labile molecule payload. A method is disclosed to self-assemble the components into ordered multi-unit structures. The method provides a self-limiting assembly process that results in finite-size supraparticles that can store and transport sensitive labile molecule payloads (for example, labile RNAs) and release them selectively upon exposure to biochemical-stimuli within cells when introduced into cells.
Absstract of: WO2025212287A1
The invention provides novel antibody-nanogel conjugates with encapsulated protein degraders and related methods for controlled and targeted delivery of degrader molecules as therapeutic agents.
Nº publicación: WO2025212705A1 09/10/2025
Applicant:
MASSACHUSETTS INSTITUTE OF TECH [US]
MASSACHUSETTS INSTITUTE OF TECHNOLOGY
Absstract of: WO2025212705A1
Viruses, bacteria, and parasites frequently cause infections in the gastrointestinal (GI) tract, but traditional vaccination strategies typically elicit little or no mucosal antibody responses. Disclosed herein is a vaccine comprising a nanoemulsion and an immunogen non-covalently conjugated to the surface of the nanoemulsion by an amphiphilic linker, wherein the vaccine is suitable for inducing a mucosal antibody response in the gastrointestinal tract. Also disclosed are methods of using the vaccine to immunize a subject by intraperitoneal administration of an effective amount of the vaccine, alone or with an adjuvant.