334results about "Microencapsulation based" patented technology

Provided herein are compositions and methods of reducing adduct formation.

Provided are compositions and drug delivery systems containing extracellular vesicles from microalgae (MEVs) that are loaded with bioactive cargo. The MEVs are formulated and administered by a variety of routes of administration and have a variety of applications as therapeutics, including as vaccines, as anti-cancer therapeutics, as therapeutics for psychiatric diseases, disorders, and conditions as diagnostics, and other such uses.

The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and / or nanomaterials and methods of their use, including compounds of Formula (I) or a pharmaceutically acceptable salt thereof.

The application provides a group of small interfering RNAs (siRNAs) targeting pyroptosis-related inflammatory reaction genes and application thereof. The pyroptosis-related inflammatory reaction genes include IL1A, IL1B, IL6, HMGB1, S100A8, S100A9 and BACH1. The application designs and synthesizes specific siRNA sequences for the above genes, and verifies that the siRNAs can efficiently and specifically inhibit the mRNA expression level of the corresponding genes through cell transfection and real-time fluorescent quantitative PCR. The application also provides a composition containing the siRNAs, a pharmaceutical composition and application thereof in the preparation of a drug for treating diseases mediated by pyroptosis-related inflammatory reaction genes (especially inflammatory diseases). The siRNAs and the composition thereof provide a new effective strategy for treating diseases related to excessive activation of pyroptosis-related inflammatory reactions, and have a wide application prospect.

The invention provides the use of formulation of pharmaceutical ingredients that combine various types of nucleic acids with hydroxychloroquine or its analogs. The use of these pharmacological combinations can reduce excessive inflammation in vitro and in vivo. This approach of reducing excessive inflammation can be exploited in various fields, where nucleic acid-based drugs are used as therapeutics or as a preventive medicine approach.

The present application relates to the field of biotechnology and the field of RNA technology. In particular, the present application relates to an engineered tRNA, isolated nucleic acid molecules expressing the same, compositions comprising the same, delivery compositions and host cells. The present application also relates to their use in the manufacture of a medicament and a formulation.

The present disclosure relates to lymphatic endothelial cell (LEC)-specific lipid nanoparticle comprising a sterol, an ionizable lipid, a PEGylated lipid, and a phospholipid. Also disclosed herein are compositions comprising the LEC-specific lipid nanoparticle and a therapeutic agent. The present disclosure further relates to methods of delivering an agent to a lymphatic system in a subject, comprising administering to the subject an effective amount of the composition of the LEC-specific lipid nanoparticle and therapeutic agent.

The present disclosure provides methods and compositions for targeting lipid bilayer particles, such as secreted extracellular vesicles, and cargo entities contained therein, to recipient cells.

The disclosure relates to methods of manufacturing lipid nanoparticles comprising DNA for administration of gene therapy vectors or inducing antigen-specific immune responses in a subject by allowing the lipid nanoparticles to self-assemble under a fluid flow rate from about 1 mL per minute to about 10 mL per minute.

Provided herein are compositions, systems, and methods comprising effector proteins and uses thereof. These effector proteins may be characterized as engineered CRISPR-associated (Cas) proteins. Various compositions, systems, and methods of the present disclosure may leverage the activities of these effector proteins for the editing, detecting and / or engineering of nucleic acids.

The application discloses a liver-targeted gene editing system based on endogenous promoter hijacking and application, and belongs to the field of biological medicine. The system is composed of an LNP-wrapped modified Cas nuclease mRNA (first component) and a promoter-free viral vector carrying a therapeutic transgene donor (second component). The system uses LNP to realize the transient burst expression of Cas nuclease in the liver, mediates the generation of double-strand breaks at the site of endogenous high-expression genes, induces the site-specific integration of therapeutic transgenes without exogenous promoters, and hijacks the expression driven by endogenous promoters by using the splice acceptor (SA) mechanism. The application solves the risk of carcinogenesis caused by random integration of exogenous strong promoters and the immunotoxicity of long-term expression of nucleases through a "double safety lock" design. Experimental results prove that the system has high editing efficiency, long-term stability and no off-target, and can be used for various liver-derived metabolic diseases such as hemophilia, hypercholesterolemia and the like.

A composition for treating cancer is provided, comprising a DDB2 inhibitor and a chemotherapeutic agent, wherein the DDB2 inhibitor comprises three RNA fragments derived from lincRNA-p21. Furthermore, the DDB2 inhibitor enhances the chemosensitivity of cancer to chemotherapeutic agents for the treatment of cancers that are unresponsive to chemotherapy.

The present invention relates generally to the field of ionizable (also called cationic) lipids, and in particular provides a novel type of such lipid represented by formula (I). The present invention further provides a method for producing such lipids and their uses, particularly in the preparation of nanoparticle compositions, more particularly nanoparticle compositions containing nucleic acids. The present invention further provides vaccine formulations comprising the nanoparticle compositions based on the ionizable lipids disclosed herein.

This invention relates to the field of biotechnology, specifically to a method for screening highly efficient and low-toxicity mRNA delivery vectors based on an ionizable lipid library. The method includes: S1. Providing a lipid library containing at least 100 structurally diverse ionizable lipids; S2. Using an automated microfluidic platform, mixing each ionizable lipid in the lipid library with helper lipids, cholesterol, PEG-lipids, and reporter gene mRNA, respectively, to prepare a lipid nanoparticle (LNP) library in parallel; S3. Performing high-throughput in vitro screening on the LNP library; S4. Selecting the ionizable lipids corresponding to the LNPs with the top 10% efficacy scores and cell viability values ​​greater than 80% as Hit (initial positive candidates); S5. Performing rapid in vivo validation of the Hit; S6. Determining the final selected lipids based on the criteria of in vivo liver expression intensity > 200% of the positive control and ALT < 100 U / L. This method for screening highly efficient and low-toxicity mRNA delivery vectors based on an ionizable lipid library can more effectively screen for non-toxic mRNA delivery vectors.

The present disclosure relates generally to compositions and methods for gene therapy, and more specifically to delivering mRNA-based therapeutics to immune cells in vivo.

Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell. In some cases, the cell can be a lung cell of a particular cell type. Also described herein are pharmaceutical compositions comprising a therapeutic or prophylactic agent assembled to a lipid composition. The lipid composition can comprise an ionizable cationic lipid, and a selective organ targeting lipid. The lipid composition can further comprise a phospholipid. Further described herein are high-potency intravenous dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.

The present invention is directed to a method for treating cancer by delivering lipid-nanoparticles (LNPs) encapsulating immunostimulant mRNA and bispecific antibody mRNA to cancer cells or intratumorally. The method comprising the step of administering mRNA-encapsulated LNPs to a tumor lesion of a subject having cancer. The immunostimulant mRNA-LNP activates immune cells around tumor, which work together with mRNA-LNP encoding bispecific antibody and PBMC to effectively target local and distant metastatic tumors. Preferred immunostimulants are GM-CSF and IL-12, and its combination.

The present specification provides compositions, methods, and kits for inducing an immune response in a subject. In an aspect, a lipid composition is described that includes at least one ionizable lipid containing a charge (N), at least one peptide, and a nucleic acid molecule containing a charge (P). In an aspect, a method for delivery of a payload to immune cells is provided that uses a lipid composition including at least one ionizable lipid, at least one endosome-releasing peptide, and a payload. TIFF2025524562000039.tif140144

The present disclosure is directed to compositions comprising mitochondrial optogenetics-based gene therapies and their methods of use. In some embodiments, a composition described herein comprises an expression vector comprising a first nucleic acid sequence encoding a channelrhodopsin fusion protein and a second nucleic acid sequence encoding a luciferase protein. In some cases, the first nucleic acid sequence and the second nucleic acid sequence are operably linked to an expression control sequence. In some instances, the channelrhodopsin fusion protein comprises a channelrhodopsin protein linked to an inner mitochondrial membrane-mitochondrial localization signal (IMM-MLS). In some implementations, when the expression vector is expressed, the luciferase protein is localized to the cytosol. In some cases, the IMM-MLS comprises a leading sequence from a mitochondrial inner membrane protein selected from ABCB10, ABCB140, Cytochrome C, and renal outer medullary potassium channel (ROMK).

Disclosed herein, in aspects, are compositions, methods, kits, and systems relating to delivery of payload into cells, for instance, for in vivo delivery via lipid delivery particles that comprise a chimeric envelope protein.

The present specification provides a gene repressor system comprising a fusion protein containing a DNA binding domain such as a TALE, a zinc finger, or a CRISPR protein without catalytic activity and a guide nucleic acid (gRNA), which is useful for suppressing the precursor protein convertase subtilisin / kexin type 9 (PCSK9) gene. Also provided is a method of using such a system to suppress the transcription of PCSK9.

The present disclosure relates to compositions and methods for increasing the level of Klotho in a cell or in a subject and in particular to compositions and methods for treating diseases or conditions associated with Klotho. The methods described herein involve supplementing the level of Klotho protein in a cell by administering to the cell a nucleic acid encoding the Klotho protein.

An mRNA composition for treating cancer is provided. The mRNA composition for treating cancer includes an mRNA encoding a CD47-targeted chimeric antigen receptor (CAR) and an mRNA encoding interleukin-12 (IL-12).

The present application belongs to the field of nucleic acid drugs, and particularly relates to an optimized 3'UTR and a nucleic acid construct or mRNA molecule comprising the same. The present application also relates to the use of the optimized 3'UTR, the nucleic acid construct or the mRNA molecule in the prevention or treatment of diseases.

The present invention relates to the inhibition of the expression of interferon regulatory factor-4 (IRF4) using a multivalent combination of RNA interference, chemically modified oligonucleotides, and / or chimeric siRNAs. The present invention further relates to methods of treating IRF4-related diseases such as multiple myeloma.

An siRNA molecule for suppressing the expression of plasminogen activator inhibitor 1 (PAI-1) is disclosed. The siRNA molecule may comprise a sense strand and an antisense strand. In some embodiments, the sense strand has at least 80% sequence identity to one of SEQ ID NO:3, SEQ ID NO:5, SEQ ID NO:7, or SEQ ID NO:9. In some embodiments, the antisense strand has at least 80% sequence identity to one of SEQ ID NO:4, SEQ ID NO:6, SEQ ID NO:8, or SEQ ID NO:10. The sense strand and / or antisense strand may comprise one or more modified nucleotides. The sense strand and / or antisense strand may comprise a ligand. Lipid nanoparticles comprising the siRNA molecule are also disclosed. Methods for treating subjects suffering from at least one PAI-1 related condition, disease, or disorder using the siRNA molecule or lipid nanoparticles comprising the siRNA molecule are also disclosed.