Absstract of: US2025144037A1
Provided are a mRNA vaccine composition with improved storage stability and a method of improving storage stability of the vaccine composition.
Absstract of: US2025144035A1
Provided herein are devices and dosage forms useful in delivering macromolecular active ingredients or drugs, such as proteins, peptides and nucleic acids, through epithelial membranes, such as intestinal epithelium. Also provided are trans-epithelial drug delivery methods and methods of treatment of diabetes or insulin resistance, or to induce weight loss.
Absstract of: US2025144018A1
The bio-microbur therapeutic delivery platform is a three-dimensional (3D)-oriented nanoneedle platform shaped like a microscale version of a fruit bur. The bio-microbur may be used for drug delivery and other biological applications, including without limitation delivery of oral vaccines or other oral biologics. Similar to a fruit bur's ability to adhere to different surfaces, the bio-microbur therapeutic delivery platform adheres to biological tissue, cell membranes, and biological gels. The bio-microbur therapeutic delivery platform includes a core and a plurality of nanoneedles secured to the core and extending outwardly therefrom, adapted for carrying and delivering a therapeutic agent. The treating agent may be an SSRI, such as fluoxetine.
Absstract of: WO2025097132A1
The present invention is directed to a capsid containing (a) an endogenous Gag polypeptide, (b) one or more effector-polypeptide conjugates, where each effector-polypeptide conjugate independently comprises an endogenous Gag polypeptide, and (c) optionally a heterologous cargo, such as an mRNA or siRNA.
Absstract of: US2025144043A1
Among the various aspects of the present disclosure is the provision of compositions and methods for treating cancer and sensitizing cancer or tumors to treatments. An aspect of the present disclosure provides for a method of preventing treatment resistance in non-HPV-associated cancer comprising administering a pharmaceutical composition comprising HPV-associated DNA (e.g., HPV-associated exosomes). Another aspect of the present disclosure provides for a method of increasing response to cancer treatment, optionally, surgery, radiation therapy, and chemotherapy in a subject having, suspected of having, being treated for, having previously been treated for, or diagnosed with cancer comprising administering a pharmaceutical composition comprising HPV-associated DNA (e.g., HPV-associated exosomes).
Absstract of: US2025144042A1
Described are coated particles containing a core and a coating, and pharmaceutical formulations containing these coated particles. The core contains a polymer or a hydrophobic drug. The coating contains a glycoprotein or a combination of a sugar and a protein. The coating surrounds the core. The coated particles are effectively absorbed by mucosa such as intestinal mucosa, GI tissue, and/or vaginal mucosa, and show increased systemic uptake following oral or mucosal administration. Optionally, the coated particles contain one or more active agents encapsulated in the core and/or embedded in the coating of the particles, for systemic or local delivery.
Absstract of: US2025145975A1
Methods which make use of baculovirus vector (BV)-magnetic nanoparticle (BV-MNP) complexes to facilitate in vivo delivery of clustered regularly interspaced palindromic repeat (CRISPR) systems are provided. BV-MNP complexes carrying a CRISPR nuclease and a guide RNA having homology to an immune checkpoint gene can be administered to a subject to inhibit the immune checkpoint gene. Inhibition of the immune checkpoint gene can promote a desired immune response in the subject. Methods which leverage the contrast provided by MNPs in BV-MNP complexes in combination with magnetic resonance imaging (MRI) to detect in vivo delivery of CRISPR systems are also provided.
Absstract of: US2025145976A1
The present disclosure provides optimized mRNAs encoding a site-directed endonuclease for use in a CRISPR/Cas system. Also provided herein are delivery systems for use of the CRISPR/Cas system in methods of in vivo and ex vivo genome editing.
Absstract of: US2025146016A1
The present disclosure relates to circular, non-viral DNA vectors, compositions including one or more of the disclosed vectors, and methods for delivering and/or expressing one or more therapeutic genes (e.g., proteins) in mammals, e.g., human patients. In some embodiments, the present disclosure is directed to circular, non-viral DNA vectors, such as circular non-viral DNA vectors including at least two inverted repeat sequences, where the at least two inverted repeat sequences are separated by a non-repeated nucleotide sequence which is not part of the at least two inverted repeat sequences. In some embodiments, the disclosed circular, non-viral DNA vectors do not include a “DD element.” In some embodiments, the disclosed circular, non-viral DNA vectors do not include a “DD element,” but include at least a portion of a bacterial origin of replication.
Absstract of: WO2025096878A1
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: 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: 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: 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: 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: TW202430136A
The present invention provides a cycloalkane-based lipid compound of Formula 1 and a pharmaceutically acceptable salt thereof. The cycloalkane-based lipid compound is a compound having a form in which carbonyl-based substituents are bonded to the central structure of a cycloalkane. The cycloalkane-based lipid compound according to the present invention is used as an ionizable lipid, which is a component of a lipid nanoparticle for delivering a nucleic acid.
Absstract of: AU2023380329A1
Aspects disclosed herein include a material and formulations having the material, the material comprising functionalized artificial melanin nanoparticles, wherein: each functionalized artificial melanin nanoparticle comprises a surface functionalized with a modifier agent via a linker group; wherein the modifier agent comprises the linker group and a functional group attached to the linker group; and wherein the linker group is an amine group. Aspects also include a method of healing skin damage using the disclosed formulations.
Absstract of: AU2023379598A1
Novel ionizable lipids are provided. Also provided are novel lipid nanoparticle compositions for the delivery of nucleic acid material to cells in vitro and in vivo with different and improved pharmacokinetic profiles as compared to what is typically observed in the art. Also provided are methods for using the compositions in research and as therapeutics.
Absstract of: AU2023368775A1
Provided herein is a lipid nanoparticle comprising an encapsulated oligonucleotide molecule, wherein the oligonucleotide molecule is single-stranded or double-stranded and has a length of between 5 and 500 nucleotides; and 20 to 70 mol% of a neutral lipid content relative to total lipid present in the lipid nanoparticle, an ionizable lipid; a sterol; and optionally a hydrophilic polymer-lipid conjugate.
Absstract of: AU2023374051A1
The present disclosure features a method of preparing a population of payload-associated cell complexes (PACCs), as well as related methods of use thereof.
Absstract of: US2025144040A1
Composite particle including the following: (1) a nanocrystal of an organic compound, and (2) a block copolymer associated with one or more surfaces of the nanocrystal of an organic compound. The organic compound, in some embodiments, is a pharmaceutical compound, a therapeutic compound, and/or a bioactive compound. The block copolymer may be a block copolymer with at least one hydrophilic poly(2-oxazoline) block. Pharmaceutical compositions may include these composite particles. The pharmaceutical compositions may include a dispersion that has an aqueous or aqueous-based continuous phase and a dispersed phase that includes the composite particles. The dispersion may be administered to sick patients, including those diagnosed with or exhibiting symptoms of COVID-19. For example, the dispersion may be aerosolized, and a patient may inhale the aerosol.
Absstract of: US2025144232A1
Disclosed are compositions comprising a lipid nanoparticle and a modified biomolecular corona. In some embodiments, the modified biomolecular corona comprises a fused cell-specific binding domain. In some embodiments, the modified biomolecular corona protein has been additionally modified such that it does not bind substantially to its natural receptor. In some embodiments, the fused cell-specific binding domain binds to a target cell.
Absstract of: US2025144228A1
Provided herein are conjugate molecules comprising an antibody or antibody fragment capable of binding polyclonal immnunoglobulins within a subject. Such conjugates are useful for recruiting immune cells of the subject to one or more cell types that are targeted by the conjugate. Also provided herein are compositions comprising the conjugates, including pharmaceutical compositions that may be administered to a subject, for such a purpose as to treat or prevent a disease.
Absstract of: US2025144204A1
An adjuvant composition, comprising a complex comprising an adjuvant and microparticles of a biodegradable polymer and/or cyclodextrin, wherein the adjuvant is incorporated into the microparticles of the biodegradable polymer and/or encapsulated in the cyclodextrin.
Absstract of: US2025144200A1
Disclosed herein are intranasal immunogenic compositions comprising viral glycoprotein nanoparticles, which are suitable for use as vaccines. The nanoparticles present antigens from pathogens surrounded to and associated with a detergent core resulting in enhanced stability and good immunogenicity. Dosages, formulations, and methods for preparing the vaccines and nanoparticles are also disclosed. Methods for using the intranasal immunogenic compositions to stimulate an immune response in a subject against a virus are also disclosed.
Nº publicación: EP4549464A1 07/05/2025
Applicant:
UNIV FUDAN [CN]
Fudan University
Absstract of: EP4549464A1
Provided are a new nanobody targeting human HER2/ErbB2 (HER2-Nanobody, HER2-Nb), a method for preparing same, and use thereof. The monoclonal nanobody efficiently and specifically binds to a purified HER2 protein and HER2 on the surface of a tumor cell, blocks a signal pathway of tumor HER2, and can be used for preparing a therapeutic antibody, an antibody-fusion protein or an antibody-drug conjugate targeting HER2-positive tumors.