Abstract: The disclosure provides new, stable, pharmaceutically acceptable amorphous solid dispersions of 1-(4-fluoro-phenyl)-4-((6bR,10aS)-3-methyl-2,3,6b,9,10,10a-hexahydro-1H,7H-pyrido[3?,4?:4,5]pyrrolo[1,2,3-de]quinoxalin-8-yl)-butan-l-one, together with methods of making and using them, and pharmaceutical compositions comprising them.

Abstract: The present disclosure relates to methods of identifying subjects in need of treatment for, e.g., a coenzyme A reduction, elevation, sequestration, toxicity, or redistribution (CASTOR) disease such as, for example, defects in fatty acid oxidation enzymes, methylmalonic acidemia, glutaric acidemia, propionic academia, and HMG-CoA lyase, via small molecule modulators of CoA levels. The methods may comprise assessing levels of carnitines, CoA, and/or various metabolites and biomarkers and administration of therapeutics useful in the treatment of CASTOR disorders, metabolic diseases, and/or neurological diseases. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present inventions.

Abstract: The present invention relates to molecules represented with Formula (I), Formula (II), and Formula (III) and the use of these molecules in the treatment of SARS-CoV-2.

Abstract: Methods of administering a myosin inhibitor to a patient and related methods of risk mitigation including controls on distribution are described herein. Methods disclosed herein provide for safe administration of mavacamten and other myosin inhibitors, and mitigate the risk of heart failure due to systolic dysfunction.

Abstract: The invention of the present disclosure relates to methods for diagnosing and for treating a progressive lung disease in a subject. In various embodiments, the method for treating a progressive lung disease in a subject includes administering a pharmaceutical composition comprising a Human Epidermal Growth Factor Receptor 2 (HER2) blocking agent and a pharmaceutically acceptable carrier to the subject, wherein the method improves clinical outcome compared to an untreated control.

Abstract: An antiviral agent contains a xanthine oxidase inhibitor in combination with an active oxygen scavenger. A method for producing an antiviral agent includes combining a xanthine oxidase inhibitor-with an active oxygen scavenger. A method for treating a viral infectious disease, includes administering to a subject in need thereof a xanthine oxidase inhibitor in combination with an active oxygen scavenger.

Abstract: The present invention relates to methods, compositions, and oral dosage forms of a SHP2 inhibitor, a SOS1 inhibitor, an ERK1/2 inhibitor, a CDK4/6 inhibitor, an AKT inhibitor, an mTOR inhibitor, a pan-HER inhibitor, or an EGFR inhibitor in combination with a MEK inhibitor or a dual RAF/MEK inhibitor, for treating abnormal cell growth (e.g., cancer).

Abstract: The invention features methods of treating glioblastoma in a subject, the method comprising administering to the subject an effective amount of sepiapterin or a pharmaceutically acceptable salt thereof.

Abstract: This disclosure relates to formulations containing arginine and other ingredients useful in managing acute and chronic complications of coronavirus infections, including respiratory symptoms fatigue, hypercoagulable state, cardiac symptoms, conditions that result from endothelial dysfunction, altered T-cell function/immune dysregulation, mitochondrial dysfunction, and/or and other complications often associated with coronavirus or other viral infections.

Abstract: Methods for treating a Human Immunodeficiency Virus (HIV) infection comprising administering to a subject in need of treatment thereof an effective amount of a small molecule nSMase2 inhibitor.

Abstract: A pharmaceutical composition containing multi-target protein kinase inhibitor compounds, and the use thereof. The pharmaceutical composition contains compounds shown in formula II and formula A or formula B as active ingredients, and an excipient. The pharmaceutical composition has the characteristics of a simple preparation method, a smooth preparation process and suitability for industrial production. Moreover, an oral preparation prepared from the pharmaceutical composition, especially an oral solid preparation, has advantageous preparation properties such as dissolution rate and content uniformity, and excellent stability; and same is suitable for use and storage as a medicine.

Abstract: Compositions comprising, for example, caffeine, sodium benzoate and one or more of lactic acid, acetic acid, aspartic acid and glutamic acid, processes for making the compositions and methods of using the compositions. The methods include accelerating emergence from anesthesia and/or countering the effects of anesthesia, including reversing the effects of anesthesia. The methods also include treating opioid intoxication or overdose.

Abstract: The present disclosure relates to compounds. and to their pharmaceutical compositions. that inhibit dipeptidyl peptidase IV (DPP4). Hie compounds selectively promote the proliferation of alveolar type 2 cells (AEC2s) and are useful in therapeutic methods of treating diseases whose etiology. for example. derives from epithelial degeneration and maladaptive remodeling, such as pulmonary' diseases like idiopathic pulmonary fibrosis (IFF), acute respiratory' distress syndrome (ARDS), and infant respiratory' distress syndromes (IRDS).

Abstract: ALK abnormalities are widely found in a variety of pediatric malignant solid tumors, and ALK inhibitors against ALK abnormalities are considered an important target in the development of treatment in the field of pediatric oncology. According to the present invention, it is possible to provide a pharmaceutical composition for the treatment of childhood cancer with an ALK abnormality, with a novel dosage and administration, comprising alectinib or a salt thereof, and to be used in the treatment of cancer in children under the age of 2.

Abstract: A sterile aqueous pharmaceutical composition contains methylene blue as active ingredient and water, wherein an Azure B impurity content of the composition ranges from 2.0 wt. % to 3.0 wt. %, a pH of the composition is below 4.0, and a total metal content of the pharmaceutical composition is above 20 ppm.

Abstract: Disclosed are methods of synthesis and/or purification of certain 3,7-diamino-phenothiazin-5-ium compounds (“diaminophenothiazinium compounds”) including Methylthioninium Chloride (MTC) (Methylene Blue), and the resulting high purity characterized by a purity greater than 98%, and very low levels of heavy metals and organic impurities Azure A, B, C and MVB. Also disclosed are methods of treatment of a tauopathy or methemoglobinemia in a patient in need thereof, comprising administering to the patient a therapeutically effective amount of the high-purity diaminophenothiazinium compound.

Abstract: The present invention relates to products and methods for the treatment and/or prevention of neurological disorders characterized by neurodegeneration. More particularly, the invention provides a product comprising: (i) a Rho-associated protein kinase (ROCK) inhibitor (e.g. fasudil) or a pharmaceutically acceptable salt thereof; and (ii) a farnesyltransferase inhibitor (e.g. lonafarnib) or a pharmaceutically acceptable salt thereof; for use in treating or preventing a neurological disorder characterized by neurodegeneration in a subject.

Abstract: Methods and corresponding uses are provided for treating a cancer in a patient in need thereof, comprising administering an effective amount of sodium trans-[tetrachloridobis(1H-indazole)ruthenate(III)] (BOLD-100). BOLD-100 may be used in combination with an effective amount of an inhibitor of ataxia-telangiectasia mutated and Rad3-related protein kinase (ATRi).

Abstract: Provided herein are compositions for preventing or treating muscle conditions such as muscle damage, injury, or atrophy. In some embodiments, the compositions comprise a prostaglandin E2 (PGE2) compound and a myotoxin. In some embodiments, the muscle damage, injury, or atrophy is the result of a nerve injury, a surgical procedure, or a traumatic injury. Methods of promoting muscle regeneration and methods of increasing muscle mass are also provided herein.

Abstract: Estrogen and progesterone replacement therapies are provided herein. Among others, the following formulations are provided herein: solubilized estradiol without progesterone; micronized progesterone without estradiol; micronized progesterone with partially solubilized progesterone; solubilized estradiol with micronized progesterone; solubilized estradiol with micronized progesterone in combination with partially solubilized progesterone; and solubilized estradiol with solubilized progesterone.

Abstract: The present invention relates to methods of treating cancer comprising administering a bufalin derivative compound of Formula I, wherein the compound is administered at least once a week for at least two weeks

Abstract: An object of the present invention is to provide a therapeutic agent for pulmonary fibrosis which has high therapeutic effect. The invention provides a therapeutic agent for pulmonary fibrosis which comprises, as an active ingredient, a compound represented by the following formula (I): wherein R represents a linear or branched alkyl group having 1 to 30 carbon atoms, a linear or branched alkenyl group having 2 to 30 carbon atoms, or a linear or branched alkynyl group having 2 to 30 carbon atoms, which may contain a cycloalkane or aromatic ring; X and Y each independently represent an oxygen atom or a methylene group, provide that X and Y do not simultaneously represent a methylene group; and M represents a hydrogen atom or an alkali metal atom.

Abstract: This disclosure is directed, at least in part, to combination therapies comprising GPR119 agonists and GPR40 agonists useful for the treatment of conditions or disorders involving the gut-brain axis. In some embodiments, the condition or disorder is: a metabolic disorder, such as diabetes, obesity, or nonalcoholic steatohepatitis (NASH); a nutritional disorder, such as short bowel syndrome; or an eating disorder, such as binge eating disorder.

Abstract: Oral formulations comprising one or more proteasome inhibitors for use as a treatment of coronavirus infection in a subject. Also disclosed are the use of such for the treatment or prevention of SARS-CoV-2 infection in humans.

Abstract: [Problem] COVID-19 caused by coronavirus SARS-CoV-2, cytokine release syndrome, cytokine release syndrome with CAR (chimeric antigen receptor)-T therapy, sepsis, systemic inflammatory response syndrome, hemophagocytic syndrome, macrophage activation syndrome, influenza, GVHD, systemic vasculitis, Kawasaki disease, Takayasu arteritis, and other diseases, if severe, can cause acute respiratory distress syndrome, disseminated intravascular coagulation syndrome, acute circulatory insufficiency, multi-organ failure, and other symptoms which are one of the causes of death. [Solution to Problem] Compounds represented by formulas (1) to (4) are effective when taken orally and can be expressed in smaller amounts than mangiferin.

Abstract: Embodiments of therapeutic protocols to treat Post-Acute Sequelae SARS-CoV-2 infection (“PASC”), a.k.a. “long Covid,” are described. The PASC treatment protocols focus on a moderating a hyperimmune response; destroying and removing the SARS CoV-2 spike protein from the gut and body, detoxifying the body and the brain; replenishing key nutrients; mitigating depression and anxiety; and a regimen of physical and mental exercises. A standard six-week protocol and a shorter, three-week protocol are disclosed for those with a milder form of PASC are disclosed.

Abstract: The invention provides a method for treating biliary tract cancer in a patient in need thereof, comprising the step of intravenously administering to the patient a therapeutically effective amount of (i) devimistat, (ii) gemcitabine, and (iii) cisplatin, in order to treat the biliary tract cancer.

Abstract: The present invention relates to a PGC-1? expression promoting agent, a muscle building agent, or a mitochondria activating agent, which comprises at least one pyrimidine nucleotide or a precursor thereof as an active ingredient.

Abstract: The present invention provides a stable aqueous composition of Methisoprinol. In particular, it provides a stable aqueous injectable composition comprising 25 to 150 mg/ml of Methisoprinol, at least one alcohol, and at least one excipient selected from pH modifier or buffer, surfactant, solvents, co-solvent, preservative, antioxidants, chelating agent and a tonicity modifier. The alcohol is present in proportion up to 40% w/v, preferably up to 30% w/v. The invention also provides methods of preparation of such composition and uses thereof in the treatment or prevention of viral diseases and immune suppressed states.

Abstract: Disclosed herein are dinucleotide compounds that target telomers in cancer cells and methods for using the dinucleotide compounds to treat cancers alone or before administration of checkpoint inhibitors.

Abstract: Formulations of substances comprising at least one RNA stabilizing substance and at least one substance comprising extracellular RNA or based on RNA and methods of using the formulations to improve the storage and use stability of substances comprising extracellular RNA or based on RNA at temperatures of 4° C. and above in solution without lyophilization.

Abstract: The present disclosure provides small hairpin nucleic acid molecules capable of stimulating interferon production. The nucleic acid molecules of the present disclosure has a double-stranded section of less than 19 base pairs and at least one blunt end. In certain embodiments, the molecule comprises at least one 5?-triphosphate and/or at least one 5?-diphosphate. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection, and/or ameliorating, minimizing, reversing, and/or preventing persistent SARS-CoV-2 viral infection, and/or minimizing or preventing SARS-CoV-2 viral infection-derived mortality and/or lethality, in a subject. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection in a tumor-bearing subject.

Abstract: The present disclosure provides a method for treating and/or preventing hearing loss by using a Toll-like receptor 7 and/or 9 antagonist. The Toll-like receptor 7 and/or 9 antagonist of the present disclosure achieves the effect of treating and/or preventing hearing loss by attenuating noise-induced increased gene expression of cytokines and chemokines in the cochlea, attenuating the noise-induced increase of nuclear factor kappa-B expression in the cochlea, and decreasing noise-induced macrophage infiltration into the cochlea.

Abstract: Provided herein are methods for the treatment of Alport syndrome, using a modified oligonucleotide targeted to miR-21. In certain embodiments, the modified oligonucleotide targeted to miR-21 improves kidney function and/or reduces fibrosis in subjects having Alport syndrome. In certain embodiments, administration of the modified oligonucleotide targeted to miR-21 delays the onset of end-stage renal disease in a subject having Alport syndrome. In certain embodiments, the modified oligonucleotide targeted to miR-21 delays the need for dialysis or kidney transplant in a subject having Alport syndrome.

Abstract: Disclosed are antiseptic compositions for disinfecting tissues, in particular for ocular use. Methods and compositions disclosed herein include sodium chlorite, optionally in combination with a surfactant.

Abstract: The invention enables direct oral granules enabling controlled release of magnesium, having both extended-release and non-extended-release fractions. The granules include extended-release particles having a magnesium-containing core and a lipophilic coating, with at least the cores of these extended-release particles having a mandated abrasion resistance which is determined by means of vibration screening over a period of 60 minutes and which allows these cores to be melt-coated, more particularly melt-coated with lipids in a fluidized bed. The invention further provides a method for characterizing the magnesium-containing cores, and also a process for producing the extended-release particles by means of hotmelt coating.

Abstract: The present invention provides novel therapeutic products which are used for treating chronic non healing wounds. The present invention is related to novel therapeutic products generated from peripheral blood mononuclear cells. The present invention also provides a process of preparing the therapeutic products, an aqueous suspension for Injection, gel and patch generated from peripheral blood cells in vitro having wound healing properties.

Abstract: Provided are methods and compositions for obtaining functionally enhanced derivative effector cells obtained from the differentiation of genomically engineered iPSCs. The derivative cells provided herein have stable and functional genome editing that delivers improved or enhanced therapeutic effects. Also provided are therapeutic compositions and the use thereof comprising the functionally enhanced derivative effector cells alone, or with antibodies or checkpoint inhibitors in combination therapies.

Abstract: Described herein are methods and compositions for selective activation of cells using variant cytokine receptor and cytokine pairs, wherein the cytokine receptors comprise an extracellular domain (ECD) of granulocyte-colony stimulating factor receptor (G-CSFR). In certain embodiments, the methods and compositions described herein are useful for exclusive activation of cells for adoptive cell transfer therapy. Thus, included herein are methods of producing cells expressing variant receptors that are selectively activated by a cytokine that does not bind its native receptor. Also disclosed herein are methods of treating a subject in need thereof, comprising administering to the subject cells expressing an variant receptor comprising an extracellular domain of G-CSFR and co-administering a variant cytokine that activates the variant receptor.

Abstract: The present disclosure relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include purifying peripheral blood mononuclear cells (PBMC) from a source, stimulating the PBMC with at least one HIV-specific peptide, depleting at least one subset of cells from the PBMC, wherein the at least one subset of cells comprises any one or more of CD8+ T cells, CD4+ T cells, ?? cells, NK cells, B cells, T regulatory cells, and NKT cells, transducing the depleted PBMC with a viral delivery system encoding at least one genetic element, culturing the transduced PBMC for at least one day, and harvesting the cultured PBMC.

Abstract: Chimeric antigen receptors targeting CD20 and preparation methods thereof are provided. The antigen binding region of the chimeric antigen receptor may include a heavy chain variable region shown in SEQ ID NOs: 7, 9 or 33 and a light chain variable region shown in SEQ ID NOs: 11, 13 or 35.

Abstract: The invention provides a natural killer (NK) cell engineered to express a chimeric antigen receptor (CAR), wherein the CAR comprises the extracellular domain of CD27 or a CD70-binding portion thereof, as well as compositions comprising the engineered NK cell, methods for producing the engineered NK cell, and therapeutic applications of the engineered NK cell, such as for treating neoplastic diseases.

Abstract: The technology described herein is directed to Natural Killer (NK) cell CAR polypeptides comprising intracellular signaling domains, intracellular costimulatory domains, and/or transmembrane domains from NK-associated polypeptides. In various aspects, described herein are polynucleotides, vectors, or cells expressing said NK CAR polypeptides, and pharmaceutical compositions comprising said NK CAR polypeptides, polynucleotides, vectors, or cells. Also described herein are methods of using said NK CAR polypeptides, for example to treat various diseases and disorders, such as cancer or infectious diseases.

Abstract: An engineered immune receptor (e.g., a chimeric antigen receptor (CAR) or chimeric costimulatory receptor (CCR)) that contains one or more short linear motifs that bind to other intracellular signaling proteins are provided, as well as nucleic acids encoding the same, cells that contain the same and methods of use. Examples of such motifs include a PLC?1-binding motifs and TRAF binding motifs, but other motifs may be used. These motifs are thought to recruit other proteins to the engineered immune receptor, thereby altering cellular responses.

Abstract: The present disclosure relates to expansion and activation of T cells. In an aspect, the present disclosure relates to expansion and activation of ?? T cells that may be used for transgene expression. In another aspect, the disclosure relates to expansion and activation of ?? T cells while depleting ?- and/or ?-TCR positive cells. T cell populations comprising expanded ?? T cell and depleted or reduced ?- and/or ?-TCR positive cells are also provided for by the instant disclosure. The disclosure further provides for methods of using the disclosed T cell populations.

Abstract: An example antigen test device comprises a genetically engineered diagnostic cell comprising an exogenous polynucleotide sequence including a receptor element that encodes a chimeric antigen receptor (CAR) comprising an extracellular antigen binding domain operably linked to a transmembrane domain, and an intracellular signaling domain that recognizes an antigen on a surface of a pathogen-infected cell from a sample or on a surface of a virus particle associated with a pathogen from the sample, an actuator element that encodes a transcription factor binding site, and an effector element that encodes a detectable reporter protein, wherein, in response to the antigen binding domain of the CAR binding to the antigen, the genetically engineered diagnostic cell is configured to activate and to synthesize the detectable reporter protein.

Abstract: Provided is a chimeric antigen receptor (CAR) or a T cell receptor (TCR) comprising one or more of the antigen binding motifs disclosed herein. Aspects of the disclosure relate to a polynucleotide encoding a chimeric antigen receptor (CAR) or a T cell receptor (TCR) comprising one or more of the antigen binding motifs. Provided are antibodies and antigen binding systems that comprise a binding motif that binds CD20 and optionally a binding motif that binds CD19, and methods of producing and using the same. Antibodies and antigen binding systems of the present disclosure comprise CARs that comprise an anti-CD20 binding motif and an anti-CD19 binding motif. Provided are compositions, such as antibodies and CARs that are or comprise an anti-CD20/anti-CD19 antigen binding system of the present disclosure, and cell therapies comprising the same, are useful, e.g., in the treatment of cancer.

Abstract: Provided are adoptive cell therapy methods involving the administration of doses of cells for treating disease and conditions, including certain B cell malignancies. The cells generally express recombinant receptors such as chimeric antigen receptors (CARs). In some embodiments, the methods are for treating subjects with non-Hodgkin lymphoma (NHL). In some embodiments, the methods are for treating subjects with relapsed or refractory NHL. Also provided are articles of manufacture and prophylactic treatments in connection with adoptive therapy methods.

Abstract: Kidney tissue-derived stem cells or organoids according to the present invention are easy to apply for treatment, may be supplied in large amounts due to their high self-renewal capacity, have an excellent ability to differentiate into kidney cells, are less likely to form tumors, and have an excellent ability to regenerate damaged tissue when injected directly into lesions. Therefore, they may be used very suitably for regenerative therapy based on adult stem cells among these stem cells. In addition, a composition according to the present invention is capable of specifically selecting only kidney tissue-derived stem cells among kidney cells. Furthermore, kidney tissue-derived stem cells expressing Lrig1 protein or a gene encoding the same have excellent self-renewal and pluripotent abilities and are able to differentiate into nephrons, and thus they may be used very effectively for the prevention or treatment of kidney disease.

Abstract: A method for treating a cell damage-related disease, including administering a composition to a subject in need thereof. The composition contains, as an active ingredient, one or more selected from the group consisting of stem cells genetically engineered to overexpress a carcinoembryonic antigen-related cell adhesion molecule (CEACAM) family protein, cells differentiated from the stem cells, and components derived from the stem cells.