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218
results.
Updated on
23/12/2025 [06:52:00]
Results 150 to 175 of 218
Absstract of: CN120641126A
Modified single-stranded DNA molecules, as well as methods of cell-free synthesis thereof and their use as therapeutic agents, are disclosed.
Absstract of: MX2025006656A
The invention relates to the field of diseases caused by high levels of LDL-C and/or fibrinogen, such as cardiovascular disease. The invention involves oligonucleotides for RNA editing technology in deaminating target adenosine nucleotides, such as the adenosine at position 1055, in transcripts of the human <i>B4GALT1 </i>gene.
Absstract of: EP4628494A1
A phosphatidylamine compound including a plurality of tertiary amino group structures and the composition and use thereof are provided. The phosphatidylamine compound is a phospholipid compound including two or more tertiary amino group structures, the structure of which is represented by the following formula (I), where, the definition of each substituent is detailed in the instructions. The compound works together with other lipid components such as cholesterol, DSPC/DOPE, DMG-PEG2000, and other helper lipids to form lipid nanoparticles (LNPs), which may be used for efficient delivery of drug molecules such as nucleic acids (siRNA, mRNA, pDNA), thereby realizing diagnosis and treatment of diseases such as cancer, fibrosis (e.g., liver, lung, kidney).
Absstract of: WO2024123461A1
Provided herein are compositions and methods for treating retinal degeneration, such as an inherited retinal degeneration. In some examples, the compositions include a pigment epithelium-derived factor (PEDF) protein or a peptide thereof. Compositions including a PEDF 17merH105A peptide in an eye drop formulation or an adeno-associated virus vector including a nucleic acid encoding a PEDF protein or PEDF 17merH105A peptide are provided. Methods for treating retinal degeneration include administering a provided eyedrop formulation or adeno-associated virus vector to an eye of a subject with retinal degeneration.
Absstract of: CN120435484A
The present application relates to a peptide having a cartilage regeneration effect and a use thereof, and provides a peptide comprising an amino acid sequence represented by SEQ ID NO: 1 or Glu (E)-Asp (D)-Asp (D), a composition for cartilage regeneration comprising the peptide, and a pharmaceutical composition for preventing or treating cartilage diseases, comprising the composition for cartilage regeneration as an active ingredient.
Absstract of: US2025288534A1
The present disclosure provides a lipid nanoparticle to treat, prevent or diagnose a central nervous system disease, disorder, trauma or injury, the lipid nanoparticle comprising: a non-cationic helper lipid; a sterol; a hydrophilic polymer-lipid conjugate; an ionizable, amino amino lipid having a pKa between 5.0 and 7.0; and an mRNA having a nucleic acid sequence encoding for a secretory polypeptide for treating, preventing or diagnosing the central nervous system disease, disorder, trauma or injury, the secretory polypeptide being capable of secretion from a cell of the central nervous system into a interstitial and/or cerebrospinal fluid of a subject. Further provided are methods for administration of the lipid nanoparticles to treat, prevent or diagnose the central nervous system disease, disorder, trauma or injury and uses of such lipid nanoparticles.
Absstract of: WO2025252942A1
The invention relates to new calibrated-size gas-filled microvesicles bearing an overall negative charge, to their method of manufacturing and to their use. Said gas-filled microvesicles have a geometric standard deviation (GSD) value of at least 1.20 or lower and a stabilizing envelope comprising a phospholipid selected from phosphatidic acid, phosphatidylserine or a mixture thereof and a pegylated phospholipid.
Absstract of: WO2025255027A1
The instant disclosure relates to lipid particles that harbor cationic lipids, the particles found to be capable of delivering associated cargoes - particularly nucleic acid cargoes when formulated as nucleic acid-lipid particles - intracellularly to skin tissue cells when administered topically to a subject. The instant disclosure provides compositions comprising such lipid particles, optionally in association with a therapeutic agent (e.g., a therapeutic mRNA and/or nucleic acid controller system), as well as methods and kits for delivering a lipid particle-associated therapeutic agent and/or for treating or preventing a disease or disorder, e.g., a skin disease or disorder, in a subject, using one or more lipid particle compositions provided herein.
Absstract of: WO2025254653A1
Particles are provided that include (a) a liposome having a negatively charged outer surface; (b) a first layer comprising poly-L-arginine (PLR), wherein the PLR is non-covalently associated with the negatively charged outer surface of the liposome; (c) a second layer, comprising hyaluronate (HA), wherein the HA is non-covalently associated with the first layer; and (d) a blood brain barrier-targeting peptide layer electrostatically coupled to the second layer; as are particles that are loaded with a therapeutic and their use for treating a brain cancer.
Absstract of: WO2025255534A1
Compounds are provided having the following structure: (I) or (II), or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein X, 1r, 2r, 3r, R11, R12, R21, R22, R31 and R32 are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.
Absstract of: WO2025254014A1
This preparation for delaying or preventing gingivitis and bone resorption associated with periodontal disease includes particles of a nano-sized or micro-sized biodegradable polymer containing a macrolide-based antimicrobial agent.
Absstract of: WO2025253270A1
An immunogenic composition that includes an adjuvant and a soluble protein. The adjuvant may be a nanoparticle, such as a zinc chitosan nanoparticle. The soluble protein may comprise a tag, such as a His tag. A method for inducing an immune response in a subject in need thereof, breaking an immune tolerance in a subject in need thereof, and/or for active immunization to prevent a disease in a subject by administering the immunogenic composition. A system for inducing an immune response in a subject in need thereof, breaking an immune tolerance in a subject in need thereof, and/or for active immunization to prevent a disease in a subject that includes the immunogenic composition and a delivery system.
Absstract of: WO2025254371A1
In some embodiments, a lipid compound has aliphatic chain-containing tail moieties that have an asymmetric structure. The lipid compound can be used as an ionizable lipid compound and stabilizes a pharmaceutically active substance, such as a nucleic acid-based therapeutic agent or vaccine. The expression efficiency of a target molecule encoded in a nucleic acid molecule is improved by using the ionizable lipid compound. The lipid compound can be applied to nucleic acid-based therapeutic agents or vaccines.
Absstract of: WO2025251519A1
The present invention relates to the field of nanomedicine. Disclosed are an intravenous oncolytic virus capable of enhancing the radiotherapy efficacy, and a preparation method therefor and a use thereof. In the present invention, PEI-Se-Se-PEG is used to modify the surface of a negatively charged oncolytic virus, thereby developing a "stealth" oncolytic virus suitable for intravenous injection, referred to as AD@PSSP. Compared with the prior art, intravenous injection of AD@PSSP can significantly prolong the circulation time of the oncolytic virus in blood and improve the safety; moreover, the inhibitory effect of radiotherapy on the growth of tumors of whole body is effectively improved, and long-lasting immunological memory can also be activated, thereby facilitating the inhibition of tumor recurrence.
Absstract of: WO2025251574A1
A cage-like nanocarrier for targeted delivery of siRNA, and a preparation method therefor and the use thereof. The preparation method comprises: (A) mutating a negatively charged or uncharged amino acid on the inner surface of a ferritin into a positively charged amino acid; and any one or more of the following steps: (B) coupling the N-terminus of the ferritin to a functional peptide having nucleic acid affinity; (C) coupling the N-terminus of the ferritin to a functional peptide promoting lysosomal escape; (D) truncating the E-helix at the C-terminus of the ferritin; (E) coupling the C-terminus of the ferritin to a functional peptide having nucleic acid affinity; and (F) coupling the C-terminus of the ferritin to a functional peptide promoting lysosomal escape. A new nucleic-acid-loaded protein nanocage carrier is constructed by means of modifying a negatively charged inner cavity of ferritin to make same positively charged. By means of electrostatic adsorption, a negatively charged siRNA can be efficiently loaded into a ferritin nanocage, thereby significantly improving the in-vivo and in-vitro delivery stability of siRNA, lysosomal escape functions, and efficacy of targeted therapy.
Absstract of: WO2025251149A1
A self-adjuvanting extracellular vesicle (SAEV) is provided comprising an antigen and a STING activator, wherein the SAEV comprises a recombinant polypeptide which comprises the antigen and the STING activator, or the SAEV comprises a recombinant polypeptide comprising one of the antigen and the STING activator and the other of the antigen or the STING activator is co-loaded in the vesicle independently of the recombinant polypeptide. Methods of vaccination with a SAEV are also provided.
Absstract of: US2025375464A1
The present disclosure relates to compositions and methods for reducing expression of MYC gene in a cell. In some embodiments, an expression repressor comprises a targeting moiety that binds a MYC promoter, anchor sequence, or super-enhancer. In some embodiments, the expression repressor comprises an effector moiety that represses transcription or methylates DNA. Systems comprising two expression repressors are also disclosed. The compositions can be used, for example, to treat cancers such as HCC.
Absstract of: AU2024213596A1
A method of treating a pulmonary condition in a subject having or at risk of having the pulmonary condition generally includes administering to the subject a therapeutic composition in an amount effective to treat the pulmonary' condition. Generally, the therapeutic composition includes purified exosome product (PEP) exosomes and a pharmaceutically acceptable carrier. In one or more embodiments, the PEP exosomes are modified to include at least one exogenous active agent. In one or more embodiments, the therapeutic composition is formulated for delivery directly to a potion of tire pulmonary tract.
Absstract of: WO2024163754A1
The invention provides compositions of matter comprising a lipid enveloped virus capsid protein and a ribonucleic acid, as well as methods for using such compositions. In illustrative compositions, the lipid enveloped virus capsid protein envelops the ribonucleic acid so as to for a capsid that can inhibit the degradation of the ribonucleic acid (e.g. by RNAses). A method of delivering a ribonucleic acid into the cytoplasm of a mammalian cell is also provided. Typically, the method comprises the steps of combining the mammalian cell with a composition of matter described herein under conditions selected to allow the lipid enveloped virus capsid to contact the mammalian cell and deliver the ribonucleic acid into the cytoplasm of a mammalian cell.
Absstract of: MX2025008776A
A composition for treating hyperprocalcitonemia is described. The composition comprises a lipophilic or hydrophobic component, an amphiphilic emulsifier, a polar liquid carrier, and with or without one or more electrolytes, where the amphiphilic emulsifier forms micelles having a lipophilic or hydrophobic core comprising the lipophilic or hydrophobic component in the polar liquid carrier, and /or liposomes organized as a lipid bilayer and/or other particle configurations.
Absstract of: EP4660189A1
The present invention addresses the problem of creating a carbamoyl lipid or a urea lipid each having a cyclic amino and developing lipid nanoparticles, and thereby providing a pharmaceutical composition for nucleic acid therapeutics and others. The present inventors have discovered a compound that is a carbamoyl lipid or a urea lipid each having a cyclic amino or a salt of the compound, and have studied on lipid nanoparticles that can be used in various pharmaceutical compositions. As a result, it was revealed that lipid nanoparticles can be formed using the compound of the present invention, which is a lipid, or a salt of the compound. By using the lipid nanoparticles containing the carbamoyl lipid or the urea lipid each having a cyclic amino according to the present invention, it is expected that a pharmaceutical composition containing the lipid nanoparticles each encapsulating a nucleic acid therein can be used as a prophylactic or therapeutic agent for astrocyte-related diseases.
Absstract of: WO2024161416A1
The present invention relates to a caprolactone based biodegradable compound(s) of formula (I) comprising photo-cleavable groups, photo-sensitive groups or combination thereof. The present invention also relates to a method of preparing the caprolactone based biodegradable compound(s) of formula (I). In addition, the present invention relates the caprolactone biodegradable compound(s) of formula (I) useful in the pharmaceutical field in controlled release systems, drug delivery system/carrier, bioimaging, implants, etc.
Absstract of: US2025352490A1
Particles made of a polymeric matrix having associated therewith a therapeutically active agent usable in treating a medical condition associated with an overexpression of P-selectin in a subject in need thereof and featuring a P-selectin selective targeting moiety represented by Formula I as defined and described in the specification and claims, compositions comprises these particles and uses thereof, are provided.
Absstract of: WO2024161249A1
Compounds are provided having the following structure Formula (I) or a pharmaceutically acceptable salt, N-oxide, tautomer or stereoisomer thereof, wherein G1, G2, G3, L1, L2, R1, R2, R3, W and Y are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a nucleic acid, compositions comprising the compounds and methods for their use and preparation are also provided.
Nº publicación: EP4658313A1 10/12/2025
Applicant:
GENZYME CORP [US]
GENZYME CORPORATION
Absstract of: AU2024214423A1
Provided are ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to cells (e.g., HSC), and methods of making and using such lipids and targeted lipid. nanoparticles.
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