Resumen de: FR3172798A1
La présente invention a pour objet un copolymère amphiphile comprenant :- un polymère hydrophile,- un polymère hydrophobe, et- un motif clivable situé entre le polymère hydrophile et le polymère hydrophobe,caractérisé en ce que le motif clivable comprend au moins un groupe fonctionnel F permettant de de limiter et/ou d’empêcher le clivage spontané du motif clivable dans des conditions physiologiques. L’invention vise également des nanoparticules comprenant au moins un tel copolymère, les procédés de fabrication du copolymère et des nanoparticules, les compositions les comprenant et leurs utilisations.
Resumen de: WO2026187804A1
Provided herein are gene editing systems and compositions directed to effectuate in vivo edits in the ANGPTL3 gene. Treatment or prevention of a diseases, such as cardiovascular disease, through disruption of the production of ANGPTL3 protein through genetic editing and the reduction of the blood ANGPTL3 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 ANGPTL3. 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 ANGPTL3 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.
Resumen de: US20260263371A1
The invention is situated in the field of RNA formulation, in particular to lyophilization of RNA. More specifically, the invention relates to a composition that is lyophilized and a method to obtain a lyophilized composition. Moreover, the present invention provides the use of a reconstituted composition according to the invention in human and/or veterinary medicine.
Resumen de: WO2026188162A1
The present invention provides compositions including an FNDC5 mRNAand a nanoparticle. The nanoparticle may be a lipid nanoparticle. The compositions may be incorporated into pharmaceutical compositions. Methods of using the compositions provided herein including methods of treating cancer, suppressing tumor growth and modulating the tumor microenvironment. The cancer or tumor may be oral squamous cell carcinoma.
Resumen de: WO2026187051A1
The present invention relates to a lipid nanoparticle preparation device to which an inline dilution channel is applied, and a lipid nanoparticle preparation method using same. In the lipid nanoparticle preparation device, dilution is continuously performed such that lipid nanoparticles can be prepared with a uniform diameter and excellent encapsulation rate, thereby enabling application to scale-up production. In addition, the present invention relates to the lipid nanoparticle preparation method using the lipid nanoparticle preparation device, and can smoothly perform, through efficient execution of dilution, conversion into scale-up production by applying tangential flow filtration (TFF) conditions and processes validated in the laboratory.
Resumen de: US20260263585A1
A universal influenza mRNA vaccine is used to encode a protein. The resulting protein encoded by the universal influenza mRNA vaccine has a matrix protein 2 extracellular domain (M2e), a hemagglutinin (HA) stem LAH region and a nucleoprotein (NP) of an influenza A virus. The universal influenza mRNA vaccine is used in a mouse animal model. The universal influenza mRNA vaccine can induce strong humoral immune and cellular immune responses, and can protect animal model mice against various influenza A viruses.
Resumen de: US20260263573A1
Disclosed is a composition comprising a closed-loop peptide comprising at least two methionines and at least two sulfonium centers; and a conjugate of a negatively charged immunoadjuvant and a peptide.
Resumen de: US20260263437A1
0000 A depot pharmaceutical formulation comprises a biocompatible hydrogel, the hydrogel encapsulating an effective amount of a colony-stimulating factor receptor (CSFR) inhibitor, wherein the CSFR inhibitor blocks CSF receptors on tumor associated macrophages (TAMs); and an effective amount of a platelet-conjugated anti-immune checkpoint inhibitor (ICI) antibody, wherein the platelet-conjugated anti-ICI antibody activates T-cells.
Resumen de: AU2025231263A1
The present invention provides a delivery system comprising of a first aqueous composition harbouring small particles and a second aqueous composition harbouring larger particles in which the particles of the first and second aqueous compositions may comprise the same or a different compound of interest, wherein said compound of interest preferably is a hydrophobic or amphiphilic compound. In both cases the small particles comprise a partly liquid oil phase at temperatures around about 4°C.
Resumen de: AU2024432366A1
A composition that includes a stem cell and a coated iron oxide nanoparticle. The coated iron oxide nanoparticle, being present in the cytoplasm of the stem cell, contains a superparamagnetic iron oxide core that is coated with one or more biocompatible polymers, each of which has a polyethylene glycol group, a silane group, and a linker covalently linking the polyethylene glycol group and the silane group. Also provided is a method for treating an inflammatory disorder in which stem cells are cultured in the presence of coated iron oxide nanoparticles and the cultured stem cells are administered to a subject suffering from an inflammatory disorder. Further disclosed is a method for tracking stem cells in vivo by labeling stem cells with coated iron oxide nanoparticles, administering the labeled stem cell to an individual, and obtaining one or more T2 weighted magnetic resonance images of the individual to track the stem cells.
Resumen de: AU2025223541A1
The present disclosure describes improved LNP-based RNA vaccines, nucleobase editing systems, and therapeutics for use in treating and/or immunization against disease. In particular, the disclosure describes improved LNPs, including novel and improved ionizable lipids for making LNPs, that enhance the targeted delivery of LNP-based RNA vaccines 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 vaccines and/or therapeutic agents.
Resumen de: AU2025223640A1
This disclosure provides, for instance, novel lipids suitable for use in lipid nanoparticles, for therapeutic delivery of nucleic acids. Also provided are methods of making and using the lipids.
Resumen de: US20260263575A1
Disclosed herein are mRNA therapeutic compositions including one or more polynucleotides encoding one or more tumor antigenic, immunogenic, or signaling polypeptides, formulated within a lipid reconstructed plant messenger packs (LPMPs) comprising natural lipids and an ionizable lipid. The disclosure also includes a method for making a mRNA therapeutic composition, comprising reconstituting a film comprising purified PMP lipids in the presence of an ionizable lipid to produce a LPMP comprising the ionizable lipid, and loading into the LPMPs with one or more polynucleotides encoding one or more tumor antigenic or immunogenic polypeptides.
Resumen de: AU2026220199A1
Abstract Provided are compositions containing extracellular vesicles from microalgae (MEVs) that are exogenously loaded with bioactive cargo or other cargo of interest, such as for therapeutic, industrial, diagnostic, and cosmetic uses. The MEVs have a variety of applications as therapeutics, including as vaccines, as anti-cancer therapeutics, as diagnostics, and other such uses Abstract
Resumen de: US20260265341A1
This invention relates to the field of peptide diagnostics and therapeutics, and more particularly to novel structures and uses of a ligand-modified species of immodulin peptides herein termed “episynchrons”—end-modified immodulin peptides capable of driving the enhanced formation of RXR heterodimers in the nucleus of living mammalian cells—to modulate immunoregenerative processes, among other uses. The covalent addition of the exemplified nuclear receptor ligands to the carboxyterminus of a peptide prior to full chain elongation during peptide synthesis is novel and relevant to the cost of production of these agents at scale. Moreover, the facile and organelle-specific ‘on-demand’ reprogramming of transcriptional sets within target tissues is a technical breakthrough relevant to the future of precision medicine. Pre-designed tissue-specific epigenetic synchrony made possible by the unique structure of episynchrons in combination with the formulation of episynchrons in zip-coded microparticles or nanoparticles delivered in a location-specific manner, is a powerful and novel concept for twenty-first century medicine. The compositions and methods provided herein demonstrate new and surprising biological activities for episynchrons that are useful in treating a wide range of dysfunctions and perturbations in mammals in the context of organ- and stress-specific precision medicine.
Resumen de: AU2025211367A1
The present invention relates to ionizable lipids for use in lipid nanoparticles, lipid nanoparticle formulations comprising these ionizable lipids, alone or in combination with other lipids and/or polymers. The lipid nanoparticles formulations may be formulated with nucleic acids for their delivery to target tissues after administration, in particular after parenteral administration such as intravenous, intramuscular, subcutaneous or intratumoral administration.
Resumen de: AU2025212867A1
Provided herein are methods to treat various neurodegenerative disorders comprising delivering a viral particles to the whole brain, including both superficial and deep structures. In some aspects, the viral particles are administered at low dose levels to the CSF, in conjunction with microbubbles followed by application of focused ultrasound (FUS) to a region of interest of the brain, thereby causing entry of the viral particles to the brain.
Resumen de: US20260263378A1
Disclosed is a complex including: cellulose nanocrystals; at least one β-cyclodextrin molecule; and at least one curcumin molecule, suitable for use in the treatment of any kind of peripheral neuropathies. Further disclosed is a pharmaceutical composition including at least the complex and at least one pharmaceutically acceptable excipient. Also disclosed is the use of the complex or the pharmaceutical composition, in particular in the form of a hydrogel, a subcutaneous implant, an implantable pump, an implanted biofunctionalized nerve conduit, to improve the treatment compliance, to allow an extended release of the complex and to obtain better pharmacokinetics.
Resumen de: US20260263377A1
Polymeric bile acid (pBA) nanoparticles and tolerogenic formulation containing polymeric bile acid nanoparticles for oral delivery and induction of antigen-specific tolerance in a subject may include immunosuppressants and/or disease-specific antigen. Oral delivery results in local organ accumulation as well as systemic delivery of the nanoparticles. Early intervention with the nanoparticles induces antigen-specific tolerance and prevents development of autoimmune disorders. Treatment with the nanoparticles results in long-term antigen-specific immune tolerance, even after cessation of treatment, in autoimmune diseases.
Resumen de: US20260265407A1
Disclosed herein are methods and agents for the treatment of cancer using p53-independent apoptosis to reduce the number of p53-depleted or p53-mutated cancer cells that have amplified HER2 gene. Also disclosed herein are methods and agents for the treatment of HER2-positive cancer in individuals with Li-Fraumeni Syndrome.
Resumen de: US20260263560A1
0000 Described herein are pharmaceutical compositions containing genes encoding a positive regulatory domain zinc finger protein 16, a peroxisome proliferator-activated receptor gamma coactivator 1-alpha protein, and a bone morphogenetic protein 7. Also disclosed are methods of treating or managing obesity and diabetes by delivering these pharmaceutical compositions using ultrasound-targeted microbubble destruction.
Resumen de: US20260263372A1
An aqueous dispersion having an aqueous mobile phase and a dispersed phase; wherein the dispersed phase comprises a lipid mixture including a cationically ionisable lipid; and the aqueous mobile phase comprises an anion of an aqueous acid; wherein the aqueous dispersion is substantially free of inorganic cations, organic solvents and RNA, is described. Methods of preparing the aqueous dispersion, nucleic acid-lipid particles and methods of preparing them using the aqueous dispersion, and their use in medicine are disclosed.
Resumen de: AU2025211997A1
Provided are an anti-albumin antibody or an antigen-binding fragment thereof and the use thereof, and an anti-albumin nanobody with improved affinity or an antigen-binding fragment thereof. The anti-albumin antibodies or antigen-binding fragments thereof can bind to albumins from different species with high affinity. In addition, the anti-albumin antibody can be linked to a bioactive effector molecule to form a fusion construct without affecting the activity of the bioactive effector molecule.
Resumen de: US20260263369A1
Provided herein are compositions comprising exosomes comprising CD47 on their surface, and further comprising a CRISPR system. Further provided are methods of using the exosomes for gene editing and the treatment of cancer by gene editing.
Nº publicación: US20260263380A1 10/09/2026
Solicitante:
MAA LABORATORIES INC [US]
MAA Laboratories, Inc.
Resumen de: US20260263380A1
This invention relates to methods of preparing nanotherapeutic compounds and compositions comprising nanotherapeutic compounds. The nanotherapeutic compounds prepared according to the methods provided herein are useful for the treatment of disease, for example, cancer, in a subject in need thereof.