Absstract of: AU2024279267A1
Disclosed is a pharmaceutical composition for inhalation, comprising lipid carriers comprising a pharmaceutical agent, the therapeutic uses thereof, and a method of making same.
Absstract of: AU2024275807A1
The present disclosure relates to a process for the preparation of tolerizing immune modifying nanoparticles encapsulating antigens associated with primary biliary cholangitis (PBC), compositions comprising the particles and use thereof for the treatment of PBC.
Absstract of: AU2024261346A1
The current invention relates to a delivery system to deliver one or more cargo to one or more cells, wherein the cargo delivery system comprises at least a calixarene, a phospholipid, an additional lipid such as sterol. The invention further relates to a method of delivering cargo to a subject using the delivery system and a pharmaceutical composition comprising the delivery system. The invention also relates to the use of a calixarene in an immunogenic composition, wherein said composition comprises an immunogenic component encapsulated in a lipid nanoparticle (LNP) comprising said calixarene and wherein said LNP has an adjuvant effect in said immunogenic composition. The invention also relates to a vaccine, wherein said vaccine comprises an immunogenic component encapsulated in a lipid nanoparticle, wherein said lipid nanoparticle comprises at least one calixarene molecule and said lipid nanoparticle acts as an adjuvant in said vaccine. The invention also relates to a method of preparing an immunogenic composition and a composition comprising a lipid nanoparticle (LNP) adjuvant comprising calixarene.
Absstract of: AU2024274301A1
Genetic constructs expressing a pro-inflammatory cytokine and a recombinant receptor, such as a chimeric antigen receptor (CAR), with a binding domain that binds STEAP1 are disclosed. The genetic constructs disclosed herein can be used in the treatment of prostate cancer, the Ewing family of tumors (EFT), bladder cancer, ovarian cancer, and rhabdomyosarcoma. The genetic constructs disclosed herein provide enhanced recombinant receptor cytotoxicity, the ability to bind and elicit cytotoxic effects even in low antigen density conditions, and enhanced interferon-gamma signaling which remodels the tumor microenvironment, further improving endogenous antitumor immunity.
Absstract of: AU2024263334A1
Disclosed herein are coronavirus (CoV) Spike (S) polypeptides, including naturally and non-naturally occurring polypeptides, and nanoparticles and immunogenic compositions comprising the same, which are useful for stimulating immune responses against various SARS-CoV-2 strains. 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.
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: WO2025251157A1
Provided herein is a method of treating or preventing or diagnosing a central nervous system central nervous system disease, disorder, trauma or injury. The method comprises use of a lipid nanoparticle comprising at least one nucleic acid encoding an antibody or antigen-binding fragment and the lysosomal enzyme glucocerebrosidase. Also provided is a lipid nanoparticle that can be used with the method disclosed herein.
Absstract of: KR20250173666A
본 발명은 핵산 전달체에 관한 것으로서, 보다 상세하게는 다수의 핵산을 포함하는 핵산 코어 및 상기 핵산 코어의 표면을 코팅하는 지질 코팅층을 포함하고, 상기 지질 코팅층은 양이온성 지질, 중성 지질 및 PEG화 지질을 포함하는 핵산 코팅용 지질 조성물을 포함함으로써 상기 핵산을 세포에 전달할 때의 독성을 최소화할 수 있는 핵산 전달체에 관한 것이다.
Absstract of: US2025375393A1
A method for preparing protein-based nanoparticle for self-packaging and delivering mRNA includes the following steps. A first donor plasmid, a second donor plasmid and a third donor plasmid are provided. A plasmid transposing step is performed. A recombinant virus preparing step is performed so as to obtain a first recombinant baculovirus, a second recombinant baculovirus and a third recombinant baculovirus. A transducing step is performed, wherein the first recombinant baculovirus, the second recombinant baculovirus and the third recombinant baculovirus are used to infect a producer cell so as to express a nucleocapsid protein, an envelope protein, an engineered envelope protein and a target RNA, and the nucleocapsid protein, the envelope protein, the engineered envelope protein and the target RNA are self-assembled to form a protein-based nanoparticle for self-packaging and delivering mRNA.
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: US2025375534A1
The present disclosure provides nucleic acid particles comprising an immunomodulator, RNA, and a cationic lipid or a cationic polymer, wherein nucleic acid particles described herein reduce inflammatory response and/or increase protein or antigen expression associated with previous formulations.
Absstract of: US2025375532A1
The present disclosure described bi-functional delivery particles with the ability to bind to two different cell types in a distinct fashion. Employing a first ligand with differential binding capabilities, the delivery particles may bind to a first target, such as cell, in a reversible fashion such that when they encounter a second target (e.g., cell) a second ligand that bind irreversibly to the second target will disrupt the binding to the first target. As such, the first target acts as a carrier to delivery the particle to a diagnostic or therapeutic second target. In particular aspects, the first and second cells are circulating cells.
Absstract of: US2025375537A1
Methods and compositions for genetically modifying a cell are provided.
Absstract of: US2025375392A1
Ionizable lipids having branched tails, nanoparticles containing the ionizable lipids, compositions containing the nanoparticles, and methods for using the ionizable lipids, nanoparticles, and compositions to deliver agents (e.g., RNAs) to cells, tissues, and/or organs are provided herein.
Absstract of: US2025375378A1
Composition and methods for treating Niemann-Pick disease type C are disclosed, utilizing lipid nanoparticles (LNPs) encapsulating mRNA sequences coding for NPC1 and/or NPC2 proteins.
Absstract of: US2025375391A1
The present disclosure provides lipid assemblies suitable for delivery of therapeutic agents to hematopoietic stem and progenitor cells (HSPCs), wherein the lipid assemblies comprise a neutral polymer surface lipid. The present disclosure also provides therapeutic and diagnostic uses related to the lipid assemblies.
Absstract of: WO2024160936A1
The present invention provides improved RNA-LNP formulations with lower amounts of RNA adduct, As well as methods and uses to reduce the amount of RNA adduct in RNA- LNP formulations, in particular mRNA-LNP formulations.
Nº publicación: EP4658250A1 10/12/2025
Applicant:
BIONTECH SE [DE]
BioNTech SE
Absstract of: CN120603581A
An aqueous dispersion having an aqueous mobile phase and a dispersed phase is described; wherein the dispersed phase comprises a lipid mixture comprising a cationically ionizable lipid; and the aqueous mobile phase comprises anions of an aqueous acid; wherein the aqueous dispersion is substantially free of inorganic cations, organic solvents, and RNA. Methods of making the aqueous dispersions, nucleic acid-lipid particles and methods of making them using the aqueous dispersions, and their use in medicine are disclosed.