2075results about "Microencapsulation based" patented technology

Compounds having the structure of Formula (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof; wherein R1, R2, R3, R4, R5, L1, L2, a and z are as defined herein, are disclosed. Use of these 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.

In accordance with the purpose(s) of the present disclosure, as embodied and broadly described herein, the disclosure, in one aspect, relates to lipid nanoparticles for use as drug delivery agents. The lipid nanoparticles are composed of lipopeptide conjugates, where a peptide is bonded to a lipid. The lipid nanoparticles described herein represent a new approach in drug delivery, addressing critical challenges in balancing biodegradability, biocompatibility, and organspecific targeting. The lipid nanoparticles can selectively target and deliver bioactive agents to specific tissues in a subject by modifying specific amino acids and ratios thereof in the peptide of the lipopeptide conjugate.

The disclosure provides, for example, double stranded DNA (dsDNA) molecules comprising one or more chemically modified nucleobases. In some embodiments, the dsDNA molecule is circular and comprises a first strand and a second strand, wherein the first strand comprises one or more chemically modified nucleobases, and the second strand is free of chemically modified nucleobases. In some embodiments, the dsDNA molecule comprises a promoter sequence and an effector sequence that encodes an effector.

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.

Provided are variant Cas12f4 polypeptides, fusion proteins containing variant Cas12f4 polypeptides, nucleic acids encoding variant Cas12f4 polypeptides and fusion proteins, compositions and methods related to those variant Cas12f4 polypeptides and fusion proteins, and modified host cells comprising the variant Cas12f4 polypeptides and / or encoding nucleic acids. The variant Cas12f4 polypeptides disclosed herein exhibit improved functionality, stability, and / or other desirable properties and may be used in combination with guide Cas12f4 RNAs that can be used in a variety of applications ex vivo, in vitro, and in vivo.

Lipid nanoparticles comprising ionizable lipids, helper lipids, neutral lipids, structural PEG-lipids, and anchor PEG-lipids are provided herein, together with targeted lipid nanoparticle compositions, and uses thereof. Methods of administering targeted lipid nanoparticles and targeted lipid nanoparticle compositions for the delivery of biologically active agents, are also provided. Such compositions and methods can be used, for example, to deliver a cargo (e.g., a mRNA cargo), to a cell population of interest.

The invention discloses a lipid compound capable of being used for gene delivery, a preparation method of the lipid compound and application of the lipid compound in gene delivery. Also disclosed herein is a lipid nanoparticle comprising the lipid compound, a gene delivery composition comprising the lipid compound or the lipid nanoparticle. The lipid compounds, lipid nanoparticles and delivery systems herein enable efficient complexation, protection, intracellular and targeted delivery and release of biomolecules, such as oligonucleotides and nucleic acids, in vitro and in vivo tissues and organs.

The present disclosure relates to cellular compositions that are modified to introduce a nucleic acid or vector expressing the same. Such compositions may be used to deliver nucleic acid to a target cell and treat disease such as cancer.

The present invention is directed to lipid nanoparticles using cationic cholesterol for topical delivery for nucleic acid delivery, and when administered locally, side effects caused by systemic drug delivery can be minimized and protein expression can be confined to the site of administration. In addition, the duration of protein expression at the site of administration can be increased, and thus the lipid nanoparticles can be useful in the technical field related to nucleic acid therapeutics.

Disclosed herein include methods, compositions, and kits suitable for use in e.g., nucleic acid delivery. Provided are compositions (e.g., nucleic acid compositions) comprising polynucleotide(s) encoding a fusion protein, a dimerization fusion protein, a soluble RBP, an adapter fusion protein, and / or a cell fusion protein. The compositions also comprise one or more polynucleotides comprising one or more RNA cargo molecules. The fusion protein, dimerization fusion protein, and / or adapter fusion protein can comprise an endosomal sorting complex required for transport (ESCRT)-recruiting domain (ERD). Recruitment of one or more ESCRT proteins results in secretion of enveloped nanoparticles (ENPs) from a cell in which at least one of a fusion protein, a dimerization fusion protein, and / or an adapter fusion protein are expressed. The ENPs can comprise the one or more RNA cargo molecules. There are also provided populations of ENPs in some embodiments.

Methods and compositions for modulating a target genome are disclosed. This disclosure relates to novel compositions, systems and methods for altering a genome at one or more locations in a host cell, tissue or subject, in vivo or in vitro. In particular, the invention features compositions, systems and methods for inserting, altering, or deleting sequences of interest in a host genome.

Compositions comprising microalgal extracellular vesicles (MEVs) formulated for intranasal delivery are provided, whereby upon intranasal administration, the MEVs are transported by a specific route, to specific regions in the brain via the olfactory nerve after intranasal administration, and to interconnected brain regions through the lateral olfactory tract (LOT). The MEVs are transported via neuronal axonal transport. The MEVs have the ability to cross synapses including: (i) synapses between olfactory sensory neurons (OSNs) and mitral / tufted neurons; (ii) synapses between mitral / tufted neurons and local neurons in multiple brain regions colonized by the lateral olfactory tract (LOT); and (iii) synapses between neurons in brain regions colonized by the LOT and neurons from the frontal cortex, hippocampus, thalamus, and hypothalamus. The compositions contain extracellular vesicles (MEVs) from microalgae loaded with bioactive cargo for the treatment, detection, diagnosis, or monitoring of diseases, disorders, or conditions of or involving the brain, particularly neuronal delivery of the cargo. The compositions and methods have multiple applications as therapeutic and diagnostic agents for the treatment, diagnosis, and monitoring of diseases, disorders, or conditions of or involving the brain. The compositions can be used in methods and uses for the treatment of cancers involving the brain, and can be used, for example, to deliver therapeutic agents for psychiatric diseases, disorders, conditions, and to deliver therapeutic agents for neurodegenerative diseases, disorders, and conditions.

The disclosure provides compositions, methods of treatment, and methods of making and using compositions to deliver a nucleic acid to a subject. Compositions described herein include lipid carriers, optionally including an inorganic particle, capable of admixing with nucleic acids. Nucleic acids provided herein include those encoding for cancer antigens (full length proteins or fragments) as well as antibodies. Methods of using the compositions as a therapeutic vaccine for the treatment of a cancer are also provided.

The disclosure relates to block copolymer nanoparticles for therapeutic delivery of nucleic acids, and methods therefor. More particularly, the invention relates to polymer nanoparticles, such as reversible addition-fragmentation chain transfer (RAFT) polymer compositions, for delivering miRNAs.

Embodiments disclosed herein provide methods, including computer-implemented methods, for designing guide sequence which may be incorporated into custom, large scale guide sequence libraries. The methods require only a list of target genes as input and utilize on target and off target scores to generate an optimal set of guide sequences for a set of target genes. In certain embodiments, the methods may also utilize multi-tissue RNA-sequencing data and / or protein annotation to design targets to genes that are highly expressed and / or contain a functional protein domain. The invention further comprises guide libraries, cells comprising said guide libraries. Computer-implemented embodiments further improve computer system function by reducing excessive user wait time through the use of data structures that reduce search from linear to logarithmic time.

Ionizable cationic lipids, methods for synthesizing the same, intermediates useful in synthesis of the ionizable cationic lipids, and methods of synthesizing the intermediates are disclosed. The ionizable cationic lipids are useful as a component of lipid nanoparticles (LNP), which in turn can be used for delivering nucleic acids into cells in vivo or ex vivo. LNP compositions are also disclosed, including LNP comprising a functionalized lipid to enable conjugation of a binding moiety, and targeted LNP (tLNP), that is an LNP in which a binding moiety has been conjugated to the functionalized lipid and can serve as a targeting moiety to direct the tLNP to a desired tissue or cell type.

The present invention relates to a novel ionizable lipid compound represented by formula (I) or a salt thereof, and lipid nanoparticles including the same. Lipid nanoparticles including a novel ionizable lipid compound according to the present invention have excellent nucleic acid encapsulation efficiency and high cell delivery efficiency of nucleic acids.

The present application disclosed a compound of formula (I) and a salt and a stereoisomer thereof, wherein each variable is as defined in the specification. The present application also disclosed a nanoparticle composition comprising the compound or a salt or a stereoisomer thereof, and the use of the nanoparticle composition for delivering active agents.

The present invention provides a method for producing nucleic acid-encapsulating lipid nanoparticles, including the following steps (a) and (b): step (a) of mixing an alcohol solution containing an ionic lipid having a tertiary amino group, a sterol, and a PEG lipid with a citrate buffer having pH 3 to 6.5 in which nucleic acid is dispersed to prepare a suspension of nucleic acid-encapsulating lipid nanoparticles; and step (b) of exchanging a dispersion medium of the aforementioned suspension for a Tris buffer having pH 5.2 to 9.0 by concentrating the suspension of nucleic acid-encapsulating lipid nanoparticles by ultrafiltration and diluting same with the aforementioned Tris buffer.

The present disclosure provides humanized antibodies and antigen binding domains thereof that bind CD8α and other antibody formats comprising these antigen binding domains, their use as a targeting moiety on lipid nanoparticles (tLNP) to deliver a therapeutic payload (such as a nucleic acid molecule) or other types of payloads. The present disclosure further relates to pharmaceutical compositions comprising the humanized anti-CD8α antibodies and CD8-targeted tLNP encapsulating a payload.

The invention relates to the technical field of viruses, in particular to a construction method and application of an African swine fever virus strain. According to the invention, the three virulence genes MGF110-9L, EP402R and I73R of the African swine fever virus are taken as targets for the first time to construct a virus strain with deletion of the three virulence genes, and the virus strain is found to be capable of effectively reducing the virus toxicity but retaining better immunogenicity, can improve the immunity of an organism to the African swine fever virus, and has a relatively great application prospect.

Relates to a modified delivery carrier and application thereof. The modified delivery carrier comprises a delivery carrier and a targeting domain coupled with the delivery carrier, the delivery carrier is used for loading a reagent, and the targeting domain can specifically target a surface antigen of an immune cell.

Disclosed herein are modified lipid compositions comprising (a) a structural component comprising one or more lipids selected from the group consisting of soybean-derived lipids, cardiolipin, sphingolipid, ceramide, glucosylceramide, lactosylceramide, galactosylcholesterol, glucosylcholesterol; and modified by (b) ionizable lipids. The present disclosure also includes a method for preparing a modified lipid composition, the method comprises reconstituting (a) a structural component comprising one or more lipids selected from the group consisting of soybean-derived lipids, cardiolipin, sphingolipid, ceramide, glucosylceramide, lactosylceramide, galactosylcholesterol and / or glucosylcholesterol in the presence of (b) ionizable lipids to produce the modified lipid composition, and loading one or more heterologous functional agents into the modified lipid composition.

The disclosure relates to block copolymer nanoparticles for therapeutic delivery of nucleotides, and methods therefor. More particularly, the invention relates to polymer nanoparticles, such as reversible addition-fragmentation chain transfer (RAFT) polymer compositions, for delivering miRNAs.

The disclosure provides, for example, a DNA molecule comprising a first strand of DNA, wherein the first strand is circular and single stranded over at least 90% of its length, and a second strand of DNA covalently linked to the first strand as described herein. The DNA molecule may comprise an effector sequence encoding a therapeutic effector. In some embodiments, the DNA molecule shows improved conversion to double-stranded DNA relative to a DNA molecule that lacks the second strand.

The present disclosure provides, at least in part, methods and compositions for in vivo fusosome delivery. In some embodiments, the fusosome comprises a combination of elements that promote specificity for target cells, e.g., one or more of a fusogen, a positive target cell-specific regulatory element, and a non-target cell-specific regulatory element. In some embodiments, the fusosome comprises one or more modifications that decrease an immune response against the fusosome.

The disclosure provides, for example, double stranded DNA (dsDNA) molecules comprising one or more chemically modified nucleobases. In some embodiments, the dsDNA molecule is circular and comprises a first strand and a second strand, wherein the first strand comprises one or more chemically modified nucleobases, and the second strand is free of chemically modified nucleobases. In some embodiments, the dsDNA molecule comprises a promoter sequence and an effector sequence that encodes an effector.