Alerta

FORMULATIONS FOR RNA-MEDIATED INSERTION OF TRANSGENES

Resumen de: WO2026174106A1

The present invention relates to compositions comprising lipid nanoparticles that encapsulate a first nucleic acid comprising (i) a 5' untranslated region (UTR) sequence and a 3' untranslated region (UTR) sequence, and (ii) a cargo sequence; and a second nucleic acid encoding an R2 retroelement polypeptide. The lipid nanoparticles comprise a plurality of lipids wherein 0.1 to 1, or 2.0 to 2.5 mol percent of the plurality of lipids is PEGylated, or comprise amino lipids such as SM-102, have N:P ratios of 1.5 to 6, and/or have diameters of about 40 to 200 nm. The compositions are used RNA-mediated insertion of transgenes in a cell.

Lipid nanoparticles and formulations thereof for CAR mRNA delivery

Resumen de: AU2026213957A1

The present invention relates to lipid nanoparticles (LNP) or compositions thereof for delivery of mRNA molecules encoding CAR, nucleic acid molecule, and/or therapeutic agents to selected targets, such as cells. Thus, in various aspects, the present invention also provides methods of preventing or treating diseases or disorders in a 5 subject in need thereof using the said LNPs or compositions thereof. ug u g

J-AGGREGATION-INDUCED EMISSION PHOTOSENSITIZER, PHARMACEUTICAL COMPOSITION THEREOF, PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: WO2026170677A1

Disclosed in the present invention are a J-aggregation-induced emission photosensitizer with NIR-II emission, and a pharmaceutical composition constructed thereby, a preparation method therefor and the use thereof. The photosensitizer can produce cytotoxic type I active oxygen upon light irradiation, and exhibits excellent fluorescence imaging capability in an NIR-II region and superior phototherapeutic performance. The photosensitizer is further co-assembled with sorafenib into a pharmaceutical composition, which has J-aggregation-induced secondary self-assembly property, enables long-term imaging at a tumor site, fulfills dual functions of NIR-II fluorescence imaging and tumor therapy, and exhibits good stability and low toxic and side effects, thereby holding a wide prospect for clinical application.

PREPARATION METHOD FOR CELLULOSE NANOCRYSTAL-BASED HYDROGEL, AND APPLICATION THEREOF

Resumen de: WO2026171285A1

Provided are a preparation method for a cellulose nanocrystal-based hydrogel, and an application thereof. The hydrogel preparation method comprises: (1) adding cerium ions to a cellulose nanocrystal (CNC) suspension, and controlling the pH to be neutral, to obtain a cerium oxide-modified CNC suspension; (2) mixing the modified CNC suspension in step (1) with a CNC suspension which has not undergone the modification in step (1), to obtain a CNC mixed suspension; and (3) mixing the CNC mixed suspension in step (2) with a prepolymer, and irradiating with ultraviolet light, to obtain a hydrogel.

HMPV FUSION PROTEIN F VARIANT, MRNA VACCINE, AND USE THEREOF

Resumen de: WO2026171211A1

Disclosed are a hMPV fusion protein F variant, an mRNA vaccine, and a use thereof. The hMPV fusion protein F variant has an amino acid residue difference at one or more sites relative to an hMPV fusion protein F having the accession number AGJ74096.1 in the GenBank database, wherein the amino acid sequence of the hMPV fusion protein F variant is as shown in SEQ ID NO: 1 or 2. The mRNA vaccine designed on the basis of the hMPV fusion protein F variant can induce a strong specific antibody response, and the persistence of an antibody in vivo is strong. The induced antibody can better neutralize an hMPV strain and control the amplification of the hMPV in the lungs. Therefore, the specific antibody induced by the mRNA vaccine provides an effective basis for prevention of hMPV infection-related diseases and development of combination vaccines.

COMPOSITIONS AND METHODS FOR PREVENTION AND TREATMENT OF GENETIC DISEASE

Resumen de: US20260240889A1

Methods and compositions for the transamniotic delivery of mRNA encoding alpha-1 antitrypsin protein to a fetus for the prenatal treatment of alpha-1 antitrypsin deficiency.

FORMULATIONS AND DOSES OF PEGYLATED URICASE

Resumen de: US20260242769A1

0000 Provided herein are methods and compositions and kits related to identification and/or treatment of subjects, including subjects with hyperuricemia, gout or a condition associated with gout, and for preventing gout flare.

Near-Infrared Dyes And Conjugates For Targeting Tumors

Resumen de: US20260241040A1

0000 The design, synthesis, and functionalization of a conjugate including a tumor-targeting near-infrared (NIR) dye and a therapeutic agent and/or a diagnostic agent, whereby the NIR dye can function to target the therapeutic agent and/or diagnostic agent to tumor cells.

COMPOSITIONS AND METHODS FOR COMPARTMENT-SPECIFIC CARGO DELIVERY

Resumen de: AU2026213966A1

Abstract Fusosome compositions and methods are described herein. Abstract ug u g

NOVEL MEANS TO MODULATE NMDA RECEPTOR-MEDIATED TOXICITY

Resumen de: US20260242440A1

0000 The present invention relates to the field of neurodegenerative processes and means to provide protection against the same. In particular, the present invention relates to polypeptides, fusion proteins, and other compounds interacting with the N-terminal domain of transient receptor potential melastatin subfamily member 4 (TRPM4), which are capable of interfering with NMDA receptor mediated neurotoxicity. The present invention also relates to nucleic acids encoding the aforementioned polypeptides or fusion proteins, compositions comprising the same and the use of said polypeptides, fusion proteins, and other compounds in methods for treating or preventing a disease of the human or animal body, for example in a method of treating diseases like Alzheimer's disease (AD), amyotrophic lateral sclerosis (ALS), Huntington's disease (HD) or stroke.

DEXAMETHASONE PRODRUG COMPOSITIONS AND USES THEREOF

Resumen de: US20260240776A1

The disclosure features pharmaceutical compositions formed from prodrug dimers for the extended delivery of a drug and for the treatment of a disease or condition.

Dimerization strategies and compounds for molecular imaging and/or radioimmunotherapy

Resumen de: AU2026210866A1

Abstract The present invention provides a multivalent compound for targeted molecular imaging and/or targeted drug delivery, wherein two components or targeting molecules each interacts with one or more biomarkers on a cell. The present invention further provides a multifunctional chelator to combine the targeting molecules. The present invention also provides an in vitro high-throughput screening assay to determine the length of the spacer molecules. The present invention also relates to compounds/probes, kits and methods for use in targeted molecular imaging and/or targeted drug delivery. ug u g

SURFACE FUNCTIONALIZED, HOST-GUEST POLYMER NANO-ASSEMBLIES AND METHODS THEREOF

Resumen de: US20260240786A1

0000 The invention generally relates to polymer-based nano-structures. More particularly, the invention relates to novel, surface-functionalized, guest-host polymer nano-assemblies and nano-delivery vehicles useful in diverse fields including drug delivery, diagnostics and specialty materials. The nano-assemblies and nano-delivery vehicles of the invention are afforded via simplify and reliable approaches.

NOVEL DRUG DELIVERY SYSTEMS AND METHODS OF MAKING AND USING SAME

Resumen de: US20260240789A1

0000 A method of preparing a nanoparticle-active pharmaceutical ingredient adduct comprising contacting a nanoparticle with a solvent under conditions suitable for the formation of a solubilized nanoparticle; contacting the solvent-nanoparticle mixture with an active pharmaceutical ingredient under conditions suitable for forming a solubilized nanoparticle-active pharmaceutical ingredient adduct; contacting the solubilized nanoparticle-active pharmaceutical ingredient adduct with an adduct stabilizing agent to form a stabilized nanoparticle-active pharmaceutical ingredient adduct; and; recovering the stabilized nanoparticle-active pharmaceutical ingredient adduct.

REPRESSOR FUSION PROTEIN SYSTEMS

Resumen de: US20260240958A1

Provided herein are gene repressor systems comprising repressor fusion proteins, such as repressor fusion proteins comprising DNA binding proteins, in some cases catalytically dead CRISPR proteins and guide nucleic acids (gRNA), useful in the repression of genes. Also provided are methods of using such systems to repress transcription of genes.

ANTI-TRANSFERRIN RECEPTOR ANTIBODIES AND USES THEREOF

Resumen de: US20260242498A1

The present disclosure provides anti-transferrin receptor antibodies, compositions comprising the same and methods of use for delivery of cargo to brain tissue. This disclosure also provides polynucleotides and vectors encoding the anti-transferrin receptor antibodies and cells comprising the same, methods of making the antibodies, and molecules comprising the antibodies.

RESPIRATORY VECTORS AND USES THEREOF

Resumen de: US20260242803A1

The application provides circular DNA vectors comprising promoters, scaffold matrix attachment region (S/MAR) sequences, and transgenes derived from proteins expressed in respiratory epithelium and which lack drug resistance genes. The application provides pharmaceutical compositions comprising therapeutic amounts of the DNA vectors.

METHODS FOR IN VIVO EDITING OF KLKB1

Resumen de: US20260240959A1

The first systemic administration of a CRISPR/Cas9-based therapeutic for in vivo editing of a liver KLKB1 gene in a clinical trial is described. Described herein are methods for in vivo editing of a liver KLKB1 gene by systemically administering a lipid nanoparticle composition comprising an mRNA encoding a Cas9 nuclease and a guide RNA that targets the gene. Assessment of biosafety metrics and clinical efficacy metrics, as well as methods of treatment, are also described herein.

COMPOSITIONS AND METHODS FOR THE TARGETING OF PCSK9

Resumen de: US20260241032A1

0000 Provided herein are systems comprising Class 2, Type V CRISPR proteins and guide nucleic acids (gRNA) useful in the modification of a PCSK9 gene. The systems are also useful for introduction into cells, for example eukaryotic cells. Also provided are methods of using such systems to modify cells having such mutations.

MULTIFUNCTIONAL HYBRID CELLULOSE NANOCRYSTALS-GOLD NANOPLATFORM AND USE THEREOF IN MEDICINE

Resumen de: US20260241030A1

The present invention refers to a multifunctional CNC*-Au-based nanoplatform, related conjugates and/or composition containing thereof.The present invention also refers to the use of such a multifunctional CNC*-Au-based nanoplatform, related conjugates and/or composition containing thereof in medicine

METHOD FOR PREPARING NANOSYSTEMS ENCAPSULATING ONE OR MORE ACTIVE INGREDIENTS

Resumen de: US20260240788A1

The present invention deals with a method for preparing a nanosystem vehicle encapsulating one or more active ingredients, preferably selected from pharmaceutical active ingredients, subsequently comprising cryomilling one or more amphiphilic compounds, one or more of said active ingredients and a frozen aqueous medium in order to obtain a uniform frozen mixture, and forming said nanosystem vehicle upon thawing. The invention further relates to a nanosystem vehicle obtainable by the aforementioned method and its uses for therapeutic and non-therapeutic applications.

POLYGLYCEROL-CONJUGATED LIPIDS AND LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME

Resumen de: WO2026174151A1

The present disclosure provides novel polymer-conjugated lipids conjugated to a polyglycerol or a polyglycerol derivative. The present disclosure also provides lipid nanoparticles (LNPs) formulation using the polymer-conjugated lipids and methods of treating a disease by administering the LNP formulations, including multiple doses of the LNP formulations.

PEPTIDE HYDROGELS AND THEIR USE FOR SKELETAL MUSCLE REGENERATION AS STIMULATORS OF MYOBLAST MIGRATION AND FUSION

Resumen de: EP4792824A1

0001 A peptide-based hydrogel for skeletal muscle regeneration, comprising: a) a self-assembling RADA16-I peptide forming a nanofibrous hydrogel matrix; and b) at least one bioactive peptide selected from: peptide fragment of stromal cell-derived factor 1 (SDF-1): SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 5, SEQ ID NO: 6, or peptide fragment of interleukin-4 (IL-4): SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO: 7, SEQ ID NO: 8, wherein the bioactive peptide is covalently linked to the RADA16-I sequence via a matrix metalloproteinase (MMPs) cleavable peptide linker and wherein cleavage of the linker by an MMPs results in controlled, localized release of the bioactive peptide in the microenvironment of regenerating muscle tissue.

JUNCTION OPENER PROTEIN AND NANOPARTICLE COMPOSITIONS AND USES THEREOF

Resumen de: WO2025081013A1

The disclosures provides compositions comprising nanoparticles and a junction opener protein; and nanoparticles comprising one or more bioactive agents and a junction opener protein that is conjugated to the surface of the nanoparticles. Various bioactive agents are described, such as squalene, squalane, and dehydroisosqualene, among others. Various nanoparticle compositions are provided, including lipid carriers comprising a hydrophobic core. Methods for treating a subject with cancer by administering the compositions provided herein are also described.

POLYMERIC NANOCARRIERS FOR DELAYING ONSET OF TYPE I DIABETES

Nº publicación: EP4791393A1 19/08/2026

Solicitante:

UNIV NORTHWESTERN [US]
Northwestern University

Resumen de: WO2025081185A1

Disclosed herein are methods for delaying the onset of type I diabetes and preventing nosocomial infections by administering PEG-b-PPS nanocarriers loaded with rapamycin. This invention aims to reduce the frequency of visits to a transfusion clinic, reduce the costs of treatments, and reduce adverse side effects, without reducing the effects of the islet transplant. This is accomplished by the use of a nanocarrier which targets treatment to the desired location.