1207results about "Drug compositions" patented technology

The invention discloses a method for improving beef quality, an action mechanism and an experimental method, in a cattle body, vitamin A can activate the expression of EBF2 through an active metabolite RA of the vitamin A, and the EBF2 can inhibit the transcription process of CYP26B1 in a targeted manner to maintain the activity of a retinol signal channel, so that PPAR gamma and downstream lipid metabolism related genes thereof are activated, and the activity of the retinol signal channel is improved. Fatty acid transport and lipid accumulation in fat cells in the cattle muscle are promoted, fat deposition in the cattle muscle is promoted, and the beef quality is improved. By constructing the molecular network for regulating and controlling the formation of the fat in the cattle muscle, a complex regulation and control mechanism for controlling the fat deposition in the cattle muscle can be understood more deeply, a method for improving the beef quality based on regulating and controlling the vitamin A mediated key factor EBF2 is provided, and a new way is provided for improving the beef quality in a targeted manner.

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.

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the KLKB1 gene are provided. Compositions and methods for treating subjects having hereditary angioedema (HAE), are provided.

Antibodies, antigen-binding proteins and Fc-fusion proteins that comprise recombinant polypeptides containing a chimeric heavy chain constant region sequence are provided that bind to certain Fc receptors however have reduced effector functions. Methods of making constructs for expression of such chimeric Fc-containing antibodies, antigen-binding proteins and Fc-fusion proteins in cell systems, and methods of producing and isolating the chimeric Fc-containing proteins are provided.

The present invention relates to a composition comprising a compound represented by general formula (1), a pharmaceutically acceptable salt thereof, or a pharmaceutically acceptable solvate thereof, and a surfactant. [wherein R1, P1, R2, P3, P4, R5, P6, R10, P10 and P11 are C1 to C6 alkyl or the like; R3 and P9 are hydrogen or the like; R7 is phenethyl optionally substituted by halogen or the like; R8 forms a 4-to 7-membered saturated heterocyclic ring together with a carbon atom bonded to P8 and R8 and a nitrogen atom bonded to P8; R9 forms a 3- to 8-membered alicyclic ring together with Q9 and a carbon atom bonded to R9 and Q9; and R11 is di-C1 to C6 alkylaminocarbonyl or the like].

The present invention relates to compositions for in vitro and in vivo delivery of nucleic acids, in particular messenger RNAs (mRNAs), into a target cell and their applications. The present invention is directed to a composition comprising (A) at least one nucleic acid and (B) at least one lipid nanoparticle (LNP) comprising (i) at least one ionizable lipid; (ii) at least one phospholipid; (iii) at least one sterol, especially neutral sterol; (iv) at least one poly(ethyleneglycol)-lipid (PEG-lipid); and (v) an imidazolium-based cationic lipid of formula (I), wherein R1, R2, R3, R4, R5, R6, R7, R8 and R9, Y and A− are as defined in the description. The present invention also relates to a method for in vitro or in vivo transfection of live cells and uses of said composition.

The present invention relates to methods for producing a preparation comprising recombinant AAV (rAAV), such methods comprising a step of mechanical lysis on mammalian producer cells, and methods for increasing the viral genome titre and / or capsid titre of a preparation comprising recombinant adeno-associated virus, related uses, and preparations obtained by or obtainable by the methods.

To provide an apple extract-containing composition that is less prone to sticking during a formulation process.SOLUTION: An apple extract-containing composition contains (A) an apple extract of 3 wt.% or more and 35 wt.% or less and (B) ang-khak, with a weight ratio of (A) to (B) (A / B) of 0.1 or more and 3.5 or less.SELECTED DRAWING: None

Recipe for a double microencapsulated powder, characterized in that the recipe comprises the following components in parts by weight: 5-60 parts by weight of core material of group A, 5-40 parts by weight of core material of group B, 5-16 parts by weight of protein shell material, 3-19 parts by weight of polysaccharide shell material, 0.1-2 parts by weight of immobilizing enzyme, 1-10 parts by weight of filler, 1-4 parts by weight of pH regulator and 0-2 parts by weight of antioxidant.

The invention discloses a functional beverage for removing heavy dampness of a human body, the formula of the functional beverage comprises raw astragalus membranaceus, lotus leaves, coix seeds, fresh couch grass roots and other traditional Chinese medicinal materials, and the functional beverage aims at solving the technical problems that an existing dampness removing product is single in effect and poor in palatability. The beverage is based on medicinal and edible raw materials, and realizes double breakthrough of systematic dampness clearing and convenient drinking through scientific compatibility and process innovation.

Provided herein are inhibitors of AKT1, pharmaceutical compositions comprising the inhibitory compounds, and methods for using the AKT1 inhibitory compounds for the treatment of disease.

Lipid nanoparticle compositions for the delivery of nucleic acids are provided.SOLUTION: In various embodiments, the lipid nanoparticle comprises an ionizable lipid of Formula (I). Also provided are methods of using such lipid nanoparticle compositions to achieve targeted delivery of therapeutic cargo without the need for a targeting ligand.SELECTED DRAWING: None

In a non-limiting embodiment, the present invention relates to antigen-binding molecules comprising two or more antigen-binding domains which are linked with each other. In a non-limiting embodiment, the antigen-binding molecules of the present disclosure have activity of holding two or more antigen molecules at spatially close positions, activity of regulating interaction between two or more antigen molecules, activity of regulating activation of two or more antigen molecules which are activated by association with each other, resistance to protease cleavage, or such.

To provide a flow path structure capable of promoting mixing of two fluids, a flow path structure unit, and a production method of a lipid particle.SOLUTION: A flow path structure of an embodiment includes a first flow path, a second flow path, and a third flow path. The second flow path is connected to the first flow path, and the third flow path is connected to the first flow path and the second flow path. When a distance between a top surface and a bottom surface of the flow path is defined as a flow path depth, the flow path depth of the second flow path in an opening through which the first flow path is connected with the second flow path is not constant, and the maximum value of the flow path depth of the second flow path in the opening is smaller than the flow path depth of the first flow path.SELECTED DRAWING: Figure 1

Provided herein are multispecific antigen-binding polypeptide constructs comprising at least two different heterodimers, each comprising a heavy chain and a light chain. At least one heterodimer comprises a Fab region comprising a lambda light chain and at least one heterodimer comprises a Fab region comprising a kappa light chain. One or more of the immunoglobulin heavy and light chains that form the antigen-binding polypeptide construct comprise amino acid modifications that promote correct pairing between the heavy and light chains to form the desired multispecific antigen-binding polypeptide construct. The amino acid modifications may be in the CH1 and / or CL domains, in the VH and / or VL domains, or a combination thereof.

The invention relates to the field of biological medicine, and particularly discloses a regulatory element combination for driving nucleic acid expression based on ITR-enhancer and application of the regulatory element combination. The regulatory element combination for driving nucleic acid expression based on an ITR-enhancer comprises at least one inverted terminal repeat (ITR) and at least one enhancer, the regulatory element combination is delivered through an AAV vector. The invention also provides a recombinant nucleic acid molecule for improving specific delivery of the target gene. The recombinant nucleic acid molecule comprises the regulatory element combination, an exogenous target gene and polyadenylic acid. The AAV vector provided by the invention can be used for driving the expression of a target gene without the action of a traditional promoter, and has the advantages of enhancer tissue specificity, short sequence (50-100bp), pathological microenvironment response and the like, thereby breaking through the capacity, targeting and dynamic limitation of the traditional AAV vector, improving the DNA (Deoxyribose Nucleic Acid) carrying capacity of the AAV vector, and simultaneously improving the expression of the target gene. The tissue and spatio-temporal expression specificity of a target gene is realized.

Described herein are compositions and methods for performing plasmapheresis. The compositions and methods for performing plasmapheresis are innovative at least in their application towards the treatment and prevention of aging and conditions associated with aging. Plasmapheresis compositions and methods described herein are directed towards reducing or eliminating conditions associated with aging.

The present disclosure provides methods of increasing cellular production of Niemann-Pick Disease, Type-C, Protein 1 (NPC1) in a patient in need thereof by administering a therapeutically effective amount of arimoclomol in combination with miglustat. The present disclosure further provides methods of reducing and / or delaying progression of Niemann-Pick Disease, Type-C in said patients.

Provided are methods for the treatment of congenital adrenal hyperplasia.

In particular, the present disclosure provides oligonucleotides and compositions thereof. In some embodiments, the provided oligonucleotides and compositions are useful for adenosine modification. In some embodiments, the present disclosure provides methods for treating various conditions, disorders, or diseases that may benefit from adenosine modification.

Described herein are hyaluronidase particles, compositions comprising the same, and methods of making and using the same. The compositions of the invention may comprise hyaluronidase particles and may be a suspension. Granules comprising hyaluronidase may have a moisture content of less than about 15% by weight of the particle and / or a water activity of less than about 0.9. In some embodiments, the methods of the invention may comprise administering to a subject a particle comprising hyaluronidase subcutaneously, intramuscularly, and / or intradermally, where the particle has a moisture content of less than about 15% by weight of the particle and / or a water activity of less than about 0.9.

A method for making the benzoate salt of 4-[5-[(3S)-3-aminopyrrolidine-1-carbonyl]-2-[2-fluoro-4-(2-hydroxy-2-methyl-propyl)phenyl]-2-fluoro-benzonitrile, designated compound (A), and methods for using compound (A) are provided.

Provided herein is a method for expressing and delivering a polynucleotide that encodes a protein of interest.Specifically, the protein of interest can be a nuclease associated with a gene editing system, in which the half-life of the mRNA that encodes the engineered nuclease is increased, so that the protein level and gene editing efficiency of the engineered nuclease are increased.In particular, the mRNA comprises a specific combination of 5'UTR sequence, Kozak sequence, and 3'UTR sequence.Further provided herein are pharmaceutical compositions that include polynucleotides, and methods for modifying the genome of eukaryotic cells using the polynucleotides disclosed herein.

Defined multi-conjugate oligonucleotides can have predetermined sizes and compositions. For example, in various embodiment, defined multi-conjugate oligonucleotides can have advantageous properties, for example in the form of defined multi-conjugate siRNA (i.e., including two, three or more siRNA) having enhanced intracellular delivery and / or multi-gene silencing effects. In various embodiment, the defined multi-conjugate oligonucleotides can be synthesized via new synthetic intermediates and methods. The defined multi-conjugate oligonucleotides can be used, for example, in reducing gene expression, biological research, treating or preventing medical conditions, or to produce new or altered phenotypes in cells or organisms.

The invention discloses application of beta-nicotinamide mononucleotide in regulation and control of gene editing efficiency, belongs to the field of gene editing treatment, and finds that the beta-nicotinamide mononucleotide (NMN) can efficiently inhibit the activity of CRISPR-Cas9, CRISPR-Cas12 and CRISPR-Cas13 systems in a broad-spectrum manner for the first time. The application comprises emergency blocking of off-target effect in gene editing clinical treatment, biological safety prevention and control of a virus vector gene editing system, and CRISPR activity regulation and control of in-vitro non-diagnostic purpose. Experiments show that NMN can inhibit CRISPR-mediated gene damage and cell death in a cell model, the inhibition efficiency in an in-vitro enzyme digestion system reaches 68.7%, and cell growth or transfection efficiency is not affected. The invention provides an innovative solution for safe application of CRISPR (clustered regularly interspaced short palindromic repeats) technology, and the NMN is approved to be taken orally as a health care product, so that the NMN has extremely strong clinical application potential. Compared with the existing CRISPR (clustered regularly interspaced short palindromic repeats)-resistant protein or synthetic small-molecule inhibitor, the NMN has the advantages of endogenous property, high biocompatibility, good oral safety and the like.

The present invention relates to a hydrogel-based drug delivery system that can efficiently deliver the required amount of a drug to a desired location for a longer period of time. The hydrogel-based drug delivery system includes: an amphipathic block copolymer including a mixture of a first poloxamer and a second poloxamer that have different molecular weights; and a protein extract (containing elastin and collagen. A drug delivery composition for promoting tissue regeneration, according to the present invention, is mixed with a drug and introduced into the body, thus increasing the excretion time of the drug in the body, has phase transition properties of a liquid (sol) state at room temperature while being gelled at body temperature, thus being convenient to inject as an injectable agent, can be applied to various body parts.

This disclosure addresses methods and applications of codon optimization of gene products. In particular, this disclosure features codon optimization methods by reducing the frequency of m6A modifications to promote increased half-life and stability of mRNA for the purpose of enhancing protein production. Additional methods for delivering codon-optimized gene products are disclosed. The methods disclosed are clinically relevant for gene therapy, cell therapy, and vaccine development.

Provided herein are compositions, systems, and methods for the creation, identification, and characterization of effector domains for activating and silencing gene expression. In particular, a high-throughput system for discovering and characterizing effector domains is provided.