Abstract: Therapeutic compositions, primarily for topical application, and methods of making and using the composition. Pharmaceutical compositions formulated for specific forms of administration are also provided.

Abstract: Therapeutic compositions, primarily for topical application, and methods of making and using the composition. Pharmaceutical compositions formulated for specific forms of administration are also provided.

Abstract: Described herein is anhydrous sodium thiosulfate, methods for synthesizing anhydrous sodium thiosulfate, pharmaceutical compositions thereof, and methods of treating ototoxicity. Anhydrous sodium thiosulfate is synthesized from sodium sulfite, sulfur, and cetylpyridinium chloride. The anhydrous sodium thiosulfate is formulated into a pharmaceutical composition comprising a buffer and solvent. These compositions are useful for eliminating or reducing ototoxicity in pediatric patients receiving platinum-based chemotherapeutics.

Abstract: The present invention is directed to the following: methods of treating or preventing stent thrombosis using pharmaceutical compositions comprising cangrelor and optionally bivalirudin; methods of reducing mortality in a subject undergoing stent implantation using pharmaceutical compositions comprising cangrelor and optionally bivalirudin; medicaments comprising cangrelor and optionally bivalirudin useful for treating or preventing stent thrombosis, or useful for reducing mortality in a subject undergoing stent implantation; pharmaceutical compositions comprising cangrelor and bivalirudin; and methods of preparing a medicament comprising cangrelor and optionally bivalirudin useful for treating or preventing stent thrombosis, or useful for reducing mortality in a subject undergoing stent implantation.

Abstract: Some aspects of this disclosure provide clinical media that support viability, re-plating efficiency, and repopulation capacity of cells and tissues during storage for up to 48 hours or longer. The clinical media provided herein are also useful for clinical irrigation. Cell or tissue preparations comprising a cell population or tissue and a clinical medium as provided herein are also provided, as are methods for generating such preparations. Methods for using the clinical media and cell and tissue preparations provided herein, for example, for administering an effective amount of cells or tissue to a subject in need thereof, are also disclosed.

Abstract: A new typepolyethylene glycol lipid and the use thereof. The lipid is free of in-vivo cleavable bonds, and can deliver a bioactive substance to a target cell or organ more stably. In addition, the new typepolyethylene glycol lipid can be positively charged in a specific pH environment, and can more easily form stable particles with the bioactive substance, so that the bioactive substance plays a role in the target cell or organ.

Abstract: A drug carrier composition includes the following components in parts by weight: 10-25 parts of sesame oil, 2-8 parts of beeswax, and 0.6-1.4 parts of plant polysaccharide; the plant polysaccharide is one or more selected from the group consisting of aloe polysaccharide, Gardenia polysaccharide, and Trametes robiniophia murr polysaccharide.

Abstract: The present invention relates to a solid oral pharmaceutical with a novel, well defined method to enhance solubility of active pharmaceutical ingredients (API) with low solubility in aqueous media and use those enhanced API in a new approach of preparing modified and sustained release pharmaceutical formulation for API which are regularly not suitable for sustained release formulations because of their low solubility. This invention is applicable for small molecule API (molecular weight <1000) for which the solubility can be enhanced by formation of a e.g., Cyclodextrin Complex regardless which Cyclodextrin or derivate thereof is employed according to the method described in this invention. Small molecule API falling in this category can be found in a wide range of indications.

Abstract: The present invention relates to a protein degradation agent, and a preparation method therefor and a use thereof. The protein degradation agent can degrade various proteins comprising c-Myc protein, and can therefore be used for the prevention and treatment of diseases related to dysregulation of various proteins comprising c-Myc protein, such as cancer, cardiovascular and cerebrovascular diseases, and viral infection-related diseases.

Abstract: Disclosed herein are stereocomplexes for the delivery of one or more anti-cancer agents. The stereocomplexes exhibit low toxicity and are biodegradable while also providing for controlled release of one or more anti-cancer agents at tumor sites. The stereocomplexes can be designed such that the anti-cancer agents operate synergistically and may optionally include additional targeting groups and functionalities. The stereocomplexes disclosed herein can be combined with pharmaceutically-acceptable carriers and/or excipients to form pharmaceutical compositions. By varying the amount of each anti-cancer agent in the stereocomplex, specific types of tumors and cancer cell lines can be treated.

Abstract: Compounds useful as biologically active compounds are disclosed. The compounds have the following structure (I): or a stereoisomer, tautomer or salt thereof, wherein L1, L2, L3, R1 R2, M, p, q, m, and n are as defined herein. Additional compounds, methods of preparation, pharmaceutical compositions, and methods of treatment related to compounds of Structure (I) are also provided.

Abstract: Described herein are nanoparticle compositions that can be used to target specific regions of the blood brain barrier (BBB). Such nanoparticle compositions can be used to deliver therapeutics to or across the BBB or to image the BBB or the permeability thereof.

Abstract: The invention relates to agents capable of sensitising STING activation, compositions comprising said agents and medical uses thereof. The agents may be peptides and compositions thereof. Methods of treatment of a disease or disorder, for instance cancer, by administration of the agents of the invention to a subject in need thereof is also provided.

Abstract: Protein conjugates comprising a protein carrier comprising a plurality of amine groups, a biological payload that interacts with an intracellular target and a linker linking them, wherein at least a portion of the amine groups are bound to a protecting group are provided. Pharmaceutical compositions comprising the protein conjugates as well as methods of using and producing the protein conjugates are also provided.

Abstract: Disclosed are compositions and methods of treating and diagnosing immune-related disorders characterized by the presence of aberrant immune cells that over-express certain proteins or that express a protein not expressed on normal immune cells.

Abstract: A linker, comprising an Lb structure fragment, the Lb structure being selected from the following formula, wherein subscript q is selected from any integer from 1 to 20, preferably from 3 to 10, and most preferably from 5 to 8. The present application also relates to a use of the linker in the preparation of a linker-drug and a ligand-drug conjugate.

Abstract: The invention relates to LAG-3 binding agents, in particular particularly variable heavy chain (VH) sdAbs and bispecific agents that target both LAG-3 and PD-1, and the use of such binding agents in the treatment, prevention and detection of disease.

Abstract: The present invention generally relates to methods of preventing methionine oxidation in immunoconjugates. The present invention also relates to pharmaceutical compositions of immunoconjugates in which the amount of methionine oxidation is minimized.

Abstract: A delivery (transport) of therapeutic enzymes, which is applicable in medicine, is disclosed. A compound containing a therapeutic enzyme and a transport element that are coupled to one another directly or by a linker is described, the transport element being a Fab fragment of immunoglobulin IgG specific to an insulin receptor epitope, and to the use of said compound to produce a pharmaceutical composition for treating diseases, as well as to the use of said compound for the treatment and prophylaxis of diseases, in particular lysosomal storage diseases, inter alia, for the treatment and prophylaxis of the enzyme deficiency characteristic of the respective lysosomal storage disease, such as mucopolysaccharidosis, in particular mucopolysaccharidosis types I and II.

Abstract: Provided herein are therapeutic PPSU nanoparticles for targeting specific cell types and delivering therapeutic compounds. Therapeutic PPSU nanoparticles include nanoparticles comprising midostaurin and anti-siglec antibodies for targeting mast cells. Also provided herein are methods for preparing the therapeutic PPU nanoparticles and using them to treat malignant mast cell disorders.

Abstract: Disclosed are novel conjugates and methods for the preparation thereof. One of the methods for the preparation of a conjugate comprises a step of: acting a compound of formula (I), with a thiol-containing molecule of formula (II), wherein represents an amino acid, a peptide, a protein, an antibody, a nucleotide, an oligonucleotide, a saccharide, a polysaccharide, a polymer, a small molecule, an optionally substituted C1-C8-alkyl, an optionally substituted phenyl, or an optionally substituted aromatic 5- or 6-membered heterocyclic system; resulting in a compound of formula (III).

Abstract: The present invention relates to a method for generating an antibody-payload conjugate by means of a microbial transglutaminase (MTG). The method comprises a step of conjugating a linker having a primary amine residue, said linker having the peptide structure (shown in N->C direction) (Aax)m-(Aax)(NH2)-(Aax)n-B-(Aax)o, or (Aax)m-B-(Aax)n-(Aax)(NH2)-(Aax)o, to a Gln residue comprised in the heavy or light chain of an antibody.

Abstract: The present invention relates to a linker of the following formula (I) or a salt thereof: The present invention relates to a linker-drug conjugate of the following formula (II) or a salt thereof: The present invention relates also to a binding unit-drug conjugate, such as an antibody-drug conjugate, of the following formula (III) or (IV) or a salt thereof: as well as a pharmaceutical composition comprising such a binding unit-drug conjugate and its use in the treatment of cancer.

Abstract: A method for preparing a protein conjugate, including contacting a fucose derivative donor Q-Fuc*? with a protein containing an oligosaccharide in a presence of a catalyst to obtain the protein conjugate. A protein conjugate obtained by the method. A method for preventing or treating disease, including administering the protein conjugate.

Abstract: Pharmaceutical compositions, kits, and methods are disclosed for use in treating or reducing the occurrence of a viral infection in a patient. The compositions, kits, and methods utilize at least one anti-Pathogenic Host Response (anti-PHR) agent, either alone or in combination with at least one anti-viral agent.

Abstract: Compositions and methods are disclosed for enhancing the bioavailability and/or increasing the stability of at least one agent by modifying the at least one agent with cyclodextrin. Pharmaceutical compositions including the cyclodextrin-modified agents are disclosed. Methods of manufacturing the cyclodextrin-modified agents as well as methods of using the cyclodextrin-modified agents are also disclosed.

Abstract: Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.

Abstract: The present invention provides compositions, systems, kits, and methods for generating expression of one or more proteins and/or biologically active nucleic acid molecules in a subject (e.g., at therapeutic levels for extended periods required to produce therapeutic effects). In certain embodiments, systems and kits are provided that comprise a first composition comprising a first amount of polycationic structures, and a second composition comprising a therapeutically effective amount of expression vectors (e.g., non-viral expression vectors not associated with liposomes) that are CpG-free or CpG-reduced, where the expression vectors comprise a first nucleic acid sequence encoding: i) a first therapeutic protein or proteins, and/or ii) a first biologically active nucleic acid molecule or molecules.

Abstract: The present application relates to the fields of gene therapy and molecular biology. More specifically, the present invention relates to an isolated modified capsid protein VP1 from adeno-associated virus serotype 9 (AAV9) comprising one or more amino acid substitutions compared to the wild-type AAV9 capsid protein VP1, which substitutions increase the efficiency of production (assembly) of the vector based on recombinant adeno-associated virus serotype 9 (rAAV9), to a capsid and a vector based on the above VP1, as well as to uses thereof.

Abstract: The invention provides persistent expression of ?-globin gene by using cell and/or gene therapy based administration of nucleotide sequence encoding ?-globin gene to treat thalassemia and sickle cell anemia. Lentivirus (LV) based viral vector system containing an expression cassette of ?-globin gene. Whereas, the lentivirus particle is packed with genes expressing functional ?-globin gene in erythroid cell lineage specifically.

Abstract: RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-18936 and compositions, methods, and uses thereof.

Abstract: Provided herein are compositions and methods for high efficiency genome editing by targeting novel genetic loci.

Abstract: The present invention features method and composition that can be used to facilitate intracellular delivery of DNA to a subject. The provided methods and compositions employ a nanoparticle for intracellular DNA delivery and a cytosolic DNA-sensing inhibitor. The cytosolic DNA-sensing inhibitor is provided to decrease the subject's immune response that can be stimulated by the DNA.

Abstract: Disclosed are a bornavirus vector comprising a nucleic acid encoding an antibody or antibody fragment capable of binding to a mutant SOD1 protein, the antibody or antibody fragment comprising a heavy-chain variable region comprising a heavy-chain CDR1 consisting of an amino acid sequence GFSLNTSGMG (SEQ ID NO: 1), a heavy-chain CDR2 consisting of an amino acid sequence IWWDDDK (SEQ ID NO: 2), and a heavy-chain CDR3 consisting of an amino acid sequence ARLGYAMDY (SEQ ID NO: 3), the heavy-chain variable region optionally having 3 or fewer amino acid substitutions, and/or a light-chain variable region comprising a light-chain CDR1 consisting of an amino acid sequence ONVGTN (SEQ ID NO: 4), a light-chain CDR2 consisting of an amino acid sequence SAS, and a light-chain CDR3 consisting of an amino acid sequence QQYYIYPYT (SEQ ID NO: 5), the light-chain variable region optionally having 3 or fewer amino acid substitutions; a recombinant virus comprising RNA encoded by the bornavirus vector; a cell infected with the

Abstract: Methods and compositions for treating leukemia involving administering a therapeutically effective amount of an inhibitor of indoleamine 2,3 dioxygenase (IDO1). The leukemia may be is acute myeloid leukemia or acute lymphoid leukemia. The inhibitor can be a small molecule such as indiximod, epacadostat, BMS-986205, navoximod, PF-0684003, KHK2455 or LY3381916 or epacadostat. The inhibitor can be used alone or in conjunctions with other chemotherapeutic agents. IDO1 can also be inhibited using a CRISP-CAS system. The inhibitor can be administered orally, intravenously, intramuscularly, topically, arterially, or subcutaneously.

Abstract: The present invention relates to novel hybrid promoters. The invention further relates to expression cassettes and vectors containing said hybrid promoters. Also disclosed herein are methods implementing these hybrid promoters, in particular methods of gene therapy.

Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a target sequence. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). Also disclosed are methods of using the nucleic acid molecules in gene therapy applications.

Abstract: The present disclosure provides codon optimized Factor VIII sequences, vectors, and host cells comprising codon optimized Factor VIII sequences, polypeptides encoded by codon optimized Factor VIII sequences, and methods of producing such polypeptides.

Abstract: The present invention is directed to methods for the treatment or prevention of oxidative stress in a cell, e.g., photoreceptor cell, and methods for the treatment and prevention of disorders associated therewith by the administration of an agent, e.g., a nucleic acid molecule, which increases the expression and/or activity of an antioxidant defense protein.

Abstract: The invention described herein provides a microdystrophin-encoding, codon optimized polynucleotide with reduced CPG island, and use thereof in the treatment of muscular dystrophy such as DMD/BMD.

Abstract: The present disclosure relates to CCL20 promoters derived from the regulatory region of the CCL20 gene that stimulate gene expression in response to inflammation. These CCL20 promoters, which can be stimulated by an endogenous or exogenous cytokine, can be used to control the timing of the expression of a heterologous gene. The present disclosure also relates to expression cassettes or vectors comprising the CCL20 promoters of the present disclosure operably linked to a nucleic acid sequence encoding, e.g., a polypeptide of interest, as well as delivery systems (e.g., viral particles, lipid vesicles, or nanoparticles) or cells comprising such expression cassettes or vectors. The present disclosure also relates to the use of the promoters, expression cassettes, vectors, delivery systems, or cells in the treatment of diseases related to inflammation, or for recombinant gene expression.

Abstract: Lipid-based vesicles, typically herein called transfection competent vesicles (TCVs), configured to safely and efficiently deliver DNA, RNA, other nucleic acid and protein cargoes into target cells. The safety and efficiency are each, and both, achieved in part by eliminating organic solvents such as ethanol and detergents such as sodium dodecyl sulfate from the TCV loading processes (i.e., inserting a cargo into the TCV), TCV storage processes, and/or TCV delivery processes. The cargoes can also comprise nucleic acids complexed with a protein, such as a ribonucleoprotein (RNP). The systems, compositions, devices and methods, etc., herein, in some embodiments, can provide empty TCVs that can if desired be loaded at the bench without use of specialized equipment.

Abstract: The present disclosure relates to prostate specific membrane antigen (PSMA) targeted compounds conjugated to near-infra red (NIR) dyes and methods for their therapeutic and diagnostic use. More specifically, this disclosure provides compounds and methods for diagnosing and treating diseases associated with cells and/or vasculature expressing prostate specific membrane antigen (PSMA), such as prostate cancer and related diseases. The disclosure further describes methods and compositions for making and using the compounds, methods incorporating the compounds, and kits incorporating the compounds.

Abstract: The present disclosure describes hyperpolarized materials for use in nuclear magnetic resonance, magnetic resonance imaging, or similar applications. The present disclosure describes methods for producing hyperpolarized materials for use in nuclear magnetic resonance, magnetic resonance imaging, or similar applications. The present disclosure describes precursor compounds for use in producing hyperpolarized materials for use in nuclear magnetic resonance, magnetic resonance imaging, or similar applications.

Abstract: The present disclosure relates to methods to create a robust procedure capable of supplying commercial quantities of a radioactive diagnostic agent indicated for use with positron emission tomography (PET) for localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult patients.

Abstract: The present invention generally relates to the field of radiopharmaceuticals and their use in nuclear medicine as tracers, imaging agents and for the treatment of various disease states of prostate cancer. Thus, the present invention concerns compounds that are represented by the general Formulae (Ia) or (Ib).

Abstract: A compound is provided comprising a melanocortin 1 receptor (MC1R) targeting peptide (MC1RTP), a radiolabeling group, and a linker joining the MC1RTP to the radiolabeling group. The MC1RTP is linear or cyclized, and comprises a sequence of Formula I or Formula II: Xaa1-Xaa2a-Xaa3-Xaa4-Xaa5-Xaa6-Xaa7a (I) or Xaa1-Xaa2b-Xaa3-Xaa4-Xaa5-Xaa6-Xaa7b (I). Xaa1 is L- /D-Nle, L-/D-Nle, L-/D-Ala, L-/D-Leu, L-/D-Ile, D-Ile, L-/D-Cys, L-/D-Met, L-/D-Phe, L-/D-Trp, L-/D-Val, L-/D-Nal, L-/D-2-Nal, Gly, L-/D-?-aminobutryic acid, L-/D-norvaline, or L-/D-homonorleucine. Xaa2a and Xaa7b are L-/D-Cys, L-/D-Asp, L-/D-Glu, L-/D-2-Aad, L-/D-3-Aad, L-/D-Pra, L-/D-Hpg, or L-/D-Bpg. Xaa2b and Xaa7a are L-/D-Cys, L-/D-Lys, L-/D-Orn, L-/D-Dab, L-/D-Dap, L-/D-Lys(N3), L-/D-Om(N3), L-/D-Dab(N3), L-/D-Dap(N3), L-/D-2-(5?-azidopentyl)alanine, or L-/D-2-(6?-azidohexyl)alanine. Xaa3 is L-/D-His, Pro, beta-(1,2,3-triazol-4-yl)-L-alanine, beta-(1,2,3-triazol-4-yl)-D-alanine, 1,2,4-triazole-3-alanine, or 1,2,4-triazole-3-D-N alanine.

Abstract: The present disclosure provides compounds or pharmaceutically acceptable salts thereof, e.g., radioimmunoconjugates including a chelating moiety or a metal complex thereof, a linker, and an antibody or antigen-binding fragment thereof targeting STEAP2. The present disclosure also provides pharmaceutical compositions of such compounds or pharmaceutically acceptable salts thereof and methods of treatment for conditions, e.g., cancer, using such compounds, pharmaceutically acceptable salts thereof, or pharmaceutical compositions.

Abstract: Provided is a method of preparing an aqueous ascorbic acid solution having a pH of 2.0 to 4.0, the method comprising: providing an initial aqueous solution of ascorbic acid and a base, wherein the initial solution has a pH of 5.0 to 8.0; and combining the initial solution with a second acid to obtain an ascorbic acid solution having a pH of 2.0 to 4.0. Also provided is the use of an aqueous ascorbic acid solution having a pH of 2.0 to 4.0 prepared by a described method as a radiostabiliser of a radio-labelled compound.

Abstract: The present disclosure features radiopharmaceutical compositions including a radionuclide and an imaging agent encapsulated within, embedded in, or conjugated to a nanoparticle (e.g., via a complexing moiety, such as a chelator), and methods of their use. The radiopharmaceuticals may be formulated for direct administration to the diseased tissue, with minimal or no washout of the radiopharmaceutical to non-target sites within a subject. The radiopharmaceuticals of the present disclosure allow for efficient treatment of diseased tissue proximal to the site of administration with minimal to no damage to the surrounding tissue.