673results about "Drug compositions" patented technology

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

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

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.

Provided are methods for the treatment of congenital adrenal hyperplasia.

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.

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.

Compositions and methods for treating enzyme-deficiency diseases are disclosed. Multidomain therapeutic proteins containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The multidomain therapeutic proteins are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.

A substantially animal-protein free (APF) composition comprises a botulinum toxin serotype A neurotoxin having a molecular weight of 150 kDa, comprising one nanogram or less of residual nucleic acid for each milligram of the botulinum neurotoxin component, wherein the botulinum toxin serotype A neurotoxin is obtainable by a process comprising the steps of (a) providing a substantially APF fermentation medium; (b) fermenting Clostridium botulinum bacteria in the fermentation medium, and; (c) recovering the biologically active botulinum neurotoxin from the fermentation medium by contacting the fermentation medium with an anion exchange chromatography media followed by contacting an eluent from the anion exchange chromatography medium with a cation exchange chromatography media.

The present invention relates to a chemically modified adeno-associated virus (AAV), a method for preparing the same, and its use in gene therapy. The chemically modified AAV is preferably prepared by incubating the AAV with a chemical reagent having an N-substituted luminol moiety under conditions conducive to reaction of the chemical reagent with tyrosine residues exposed on the surface of the AAV capsid, preferably by electrochemical methods.

In the production of ethyl esters using oils and fats containing highly unsaturated fatty acids as constituent fatty acids as raw materials, the present invention provides ethyl esters in which environmental pollutants, particularly dioxins, contained in the oils and fats are reduced. [Solution] Highly unsaturated fatty acids or ethyl esters of highly unsaturated fatty acids, manufactured using oils containing highly unsaturated fatty acids as constituent fatty acids as raw materials, with reduced levels of environmental pollutants, and containing less than 0.05 pg-TEQ / g of polychlorinated dibenzo-paradioxins (PCDDs) and polychlorinated dibenzofurans (PCDFs) among the dioxins, and coplanar PCBs (Co-PCBs) Highly unsaturated fatty acids or ethyl esters of highly unsaturated fatty acids having a TEQ of less than 0.03 pg-TEQ / g, and highly unsaturated fatty acids or ethyl esters of highly unsaturated fatty acids with reduced environmental pollutants obtained by removing free fatty acids and environmental pollutants from raw oil by thin-film distillation, ethyl esterifying the obtained oil, and purifying it by rectification and column chromatography.

The present disclosure provides LNPs comprising payload molecules, eg, mRNA therapeutics, for the treatment of diseases or disorders that would benefit from delivery of the payload molecule to airway cells.

The present invention provides small molecules that act as binders for the BBB-crossing through the receptor carbonic anhydrase IV (CA-IV).

To provide a composition containing a cyclic polyribonucleotide and its use.SOLUTION: The present invention relates generally to pharmaceutical compositions and preparations of cyclic polyribonucleotides and uses thereof. In one aspect, the present invention provides: At least one structural element selected from a) an encryptogen; b) a staggerelement; c) a regulatory element; d) a replication element; f) a pseudo-double-stranded secondary structure; and g) an expression sequence; and at least one functional property selected from a) higher translation efficiency than a linear equivalent; b) stoichiometric translation efficiency of multiple translation products; c) lower immunogenicity than an equivalent lacking an encryptogen; d) increased half-life over a linear equivalent; and e) persistence during cell division.SELECTED DRAWING: Figure 1

This invention relates to fusion proteins comprising at least one serum albumin-binding protein. The fusion proteins of this invention are characterized by (i) high thermal stability (particularly above 70°C Tm), (ii) high binding affinity (in the double-digit nanomolar range) for human serum albumin, mouse serum albumin, and rat serum albumin, and (iii) long-term stability (at least 48 h) in human and mouse serum. This invention also relates to the use of the fusion protein or compositions comprising the fusion protein for medical applications.

Improved compositions and processes for preparing mRNA-loaded lipid nanoparticles (mRNA-LNPs) are provided.SOLUTION: (i) mixing a mRNA solution with a lipid solution in the presence of 0.05% to less than 3% of a poloxamer prior to removal, wherein the lipid solution comprises one or more cationic lipids, one or more non-cationic lipids, and less than 0.5% of one or more PEG-modified lipids or PEG, and (ii) removing the poloxamer, wherein upon removal less than 0.05% of the poloxamer remains.SELECTED DRAWING: Figure 2

A pharmaceutical liquid dosage form of Trientine and / or pharmaceutically acceptable salts thereof is provided, which are suitable for oral administration. A process of preparing the liquid dosage form and use for treatment of Wilson's disease and related diseases are also provided.

To provide a method of purifying albumin-fusion proteins.SOLUTION: Disclosed is a method of purifying albumin-fusion proteins to reduce the level of oxidation of susceptible amino acid residues. The method comprises an affinity matrix chromatography step and an anion exchange chromatography step. The purified albumin-fusion proteins have low levels of oxidation and retain their enhanced half-lives in vivo and retain bioactivity. In some embodiments, the albumin-fusion proteins comprise a scaffold, such as a human Tenascin C scaffold. Compositions comprising the albumin-fusion proteins are further disclosed.SELECTED DRAWING: Figure 9

This peptide includes an amino acid sequence represented by formula A1 or an amino acid sequence in which one or more amino acid residues in the amino acid sequence represented by formula A1 have been substituted, deleted, added, or inserted. A1: X1-X2-X3-X4-X5-X6-X7-X8-X9-X10-X11-X12-X13-X14-X15

This invention relates to intermediates for producing oligonucleotide analogs or salts thereof, and to methods for producing the same. Specifically, it relates to compounds of formula VIII and methods for producing the same, with the definitions of each substituent being as defined in the specification. JPEG2026520829000135.jpg30170

The present disclosure relates to a method of treating or preventing premature infants by intravenously administering a therapeutic amount of a composition comprising IGF-1 and IBGBP-3 in combination with a therapy selected from the group including caffeine, fentanyl, fluconazole, gentamicin, insulin, midazolam, morphine, low dose norepinephrine, vancomycin at a concentration of 5 mg / ml or less, parenteral nutrition (or a composition for use therein), and combinations thereof.

The invention discloses an up-conversion nano-composite with a core-shell structure, a preparation method and application, and belongs to the technical field of biomedical bimodal nano-probes. According to the nanocomposite, up-conversion nanoparticles (UCNPs) serve as a core, metal-organic frameworks (MOFs) serve as a shell layer, horse radish peroxidase (HRP) is loaded on the shell layer, and UCNP (at) MOF / HRP is formed. Subsequent biological functional modification, such as coupling of an antibody, an antigen, nucleic acid and other substances, can be carried by an activating group or a metal coordination bond carried by a shell ligand of the upconversion nanocomposite MOF with the core-shell structure, so that the functions of specific recognition, targeted capture, specific loading and the like are realized.

Disclosed herein are prenylated flavone compounds having the structure of Formula (I), or a pharmaceutically acceptable salt and / or hydrate or solvate thereof. Also disclosed herein are methods for producing prenylated flavone compounds, and uses for such compounds, including in medical treatments.

The invention discloses application of luteolin in reduction of indole production in ruminant rumen or in vitro. The invention also discloses a method for reducing or inhibiting the production of indole in a ruminant rumen or in an in-vitro fermentation system. It is found for the first time that luteolin can inhibit the activity of tryptophanase and reduce generation of indole. The invention has important application value in improving ruminant rumen microenvironment and reducing emission of malodorous substances.

The patent discloses an antibody against human IgG, a preparation method and application thereof, a heavy chain variable region of the antibody comprises heavy chain complementarity determining regions HCDR1, HCDR2 and HCDR3, and amino acid sequences are respectively shown as SEQ ID NO:1-3; a light chain variable region comprises light chain complementarity determining regions LCDR1, LCDR2 and LCDR3, and amino acid sequences are respectively shown as SEQ ID NO:4-6. The application efficiently prepares a high-activity monoclonal antibody which can combine with different subtypes of human IgG Fc by using an in-vitro culture method of B cells, and specifically relates to two monoclonal antibodies which can specifically combine with human IgG1 protein, and provides a powerful tool for detecting human IgG1 protein and researching diseases related to human IgG1 protein.