Abstract: The invention describes ultrasound-responsive particles having a core containing one or more therapeutic or diagnostic agents, and a polypeptide shell. The surface of the particle has one or more indentations which are generally able to entrap a gas bubble. The particles are capable of generating inertial cavitation on exposure to ultrasound. The inertial cavitation properties of the particles can be used to enhance delivery of the therapeutic or diagnostic agents to target sites in vivo.

Abstract: The invention is inter alia directed to a pharmaceutical composition or vaccine for multidose administration comprising lipid-based carriers encapsulating an RNA, wherein the composition comprises at least one antimicrobial preservative selected from an aromatic alcohol, a sugar alcohol, thiomersal, or a combination thereof. The present invention is also directed to a kit or kit of parts for preparing and/or administering the pharmaceutical composition or vaccine for multidose administration. Also provided are methods of treating or preventing a disorders or a diseases, and first and second medical uses of the pharmaceutical composition or vaccine. Further provided is the use of aromatic alcohols, sugar alcohols, and/or thiomersal for preserving and/or preparing a composition or vaccine comprising lipid-based carriers encapsulating an RNA.

Abstract: Compositions of polynucleotide(s) are disclosed. A polynucleotide may encode for a polypeptide, protein, or functional fragment thereof associated with primary ciliary dyskinesia (PCD), such as dynein axonemal intermediate chain 1 (DNAI1). Pharmaceutical compositions, kits, and methods for treating a disease or condition associated with cilia maintenance and function, and impaired function of the axoneme are also disclosed. The polynucleotide may be assembled with a lipid composition for delivery to an organ, such as the lung, of a subject. The lipid composition may comprise an ionizable cationic lipid. The polynucleotide can be expressed within cells of the organ of the subject.

Abstract: Ionizable cationic lipids, methods for synthesizing them, as well as intermediates useful in synthesis of these lipids and methods of synthesizing the intermediates are disclosed. The ionizable cationic lipids are useful as a component of lipid nanoparticles (LNP), which in turn can be used for the delivery of nucleic acids into cells in vivo or ex vivo. LNP compositions are also disclosed, including LNP comprising a functionalized lipid to enable conjugation of a binding moiety, and targeted LNP (tLNP), that is a LNP in which a binding moiety has been conjugated to the functionalized lipid and can serve as a targeting moiety to direct the tLNP to a desired tissue or cell type.

Abstract: Disclosed herein are nanoparticle compositions including a polymer component and a lipid component. The disclosure also relates to lipid nanoparticles (LNPs) having the nanoparticle composition for nucleic acid delivery, and methods of making and using the same. The LNPs can be used for fast, efficient and safe delivery of nucleic acid (e.g., DNA/RNA).

Abstract: The present application provides methods of treating a CNS disorder (such as glioblastoma and epilepsy) in an individual, comprising systemically (e.g., intravenously or subcutaneously) administering to the individual an effective amount of a composition comprising nanoparticles comprising an mTOR inhibitor (such as a limus drug, such as sirolimus or a derivative thereof) and an albumin, optionally further comprising administering a second agent (such as an anti-VEGF antibody, a proteasome inhibitor, or an alkylating agent).

Abstract: A method of synthesizing and a method of curing cancers using a nano device are disclosed, the nano device comprising: a framework comprising a structure formed by Si—O—Si covalent bonds and a plurality of mesopores configured to store drug cargos therein by disulfide covalent bonds; and an independent and separate chitosan layer, deposited directly on the framework. The chitosan layer is designed to coat and protect said framework layer from leaking the drug cargos in a first condition and to degrade so as to expose the drug cargos in a second condition. The framework releases the drug cargos by allowing a predetermined concentration of glutathione to break the disulfide (S—S) covalent bonds.

Abstract: A use of a compound or a medicinal derivative thereof in inhibiting absent in melanoma 2 (AIM2) protein activity is provided and belongs to the field of protein inhibitory medicines. Compared with traditional broad-spectrum immunomodulators, the compound has a strong targeting effect, and is more accurate, rapid, effective, safe and stable. Moreover, the compound has a strong binding force with human AIM2 protein and mouse AIM2 protein, and binding constants are as high as KD=1.56E?6M and KD=5.12E?6M respectively. Compared with traditional biological inhibitors, the compound has advantages of easy storage, stable activity, small molecular weight, lower production cost and easy absorption.

Abstract: The methods include treating cancer by the administration of an effective amount of acylfulvene to a subject in need thereof and an effective amount of radiation. The irradiation occurs before or concurrently with the administration of the effective amount of acylfulvene.

Abstract: Provided are methods of inhibiting spreading depolarization in a subject by administering a sigma-1 agonist or a sigma-1 positive modulator to the subject. In some cases the subject is administered fenfluramine as the sigma-1 positive modulator. In some cases, the subject has been diagnosed with epilepsy, traumatic brain injury, migraines, stroke, ischemic attacks, hypoxia or an increased risk of sudden unexpected death in epilepsy (SUDEP), or a combination thereof.

Abstract: Described herein are methods for treating, ameliorating, and/or preventing ulcerative colitis (UC) as well as other inflammatory gastrointestinal disorders using amiselimod. The method of treatment includes dosing amiselimod at a larger initial dose as compared to the eventual maintenance dose.

Abstract: Disclosed herein is a method of safely treating a nervous system condition with a combination of dextromethorphan and bupropion. This method is intended for patients having a neurological condition or a psychiatric condition, such as major depressive disorder, and a CYP2D6 poor metabolizer genotype or a CYP2D6 poor metabolizer phenotype.

Abstract: This disclosure relates to administration of a combination of: 1) about 100-110 mg, about 104-106 mg, or about 105 mg of bupropion hydrochloride, or a molar equivalent amount of a free base form or another salt form of bupropion; and 2) about 40-50 mg, about 44-46 mg, or about 45 mg of dextromethorphan hydrobromide, or a molar equivalent amount of a free base form or another salt form of dextromethorphan in certain patient populations, such as patients having moderate renal impairment, patients receiving a concomitant strong CYP2D6 inhibitor, patients who are known CYP2D6 poor metabolizers, those in need of an NMDA antagonist that does not cause dissociation, and those at risk of QT prolongation.

Abstract: This invention is directed to pharmaceutical compositions and methods for increasing immunogenicity in a cancer cell by contacting a cancer cell with epigenetic inhibitors to prompt the cancer cell's production of MHC-I-associated tumor antigens.

Abstract: A method of treating cancer includes a combination of a therapeutically effective amount of an illudin or an illudin analog thereof, derivative, or a pharmaceutically acceptable salt thereof, and a therapeutically effective amount of a PARP inhibitor or an analog, derivative, or a pharmaceutically acceptable salt thereof. Compositions and kits of the same are included herein.

Abstract: Compositions and methods for protecting a subject against, or treating a subject with, a pathogenic infection are presented. A method includes administering a composition including a cellular energy inhibitor having the structure according to formula I wherein X is selected from the group consisting of: a nitro, an imidazole, a halide, sulfonate, a carboxylate, an alkoxide, and amine oxide; and R is selected from the group consisting of: OR?, N(R?)2, C(O)R??, C1-C6 alkyl, C6-C12 aryl, C1-C6 heteroalkyl, a C6-C12 heteroaryl, H, and an alkali metal; where R? represents H, alkali metal, C1-C6 alkyl, C6-C12 aryl or C(O)R??, R? represents H, C1-C6 alkyl, or C6-C12 aryl, and R?? represents H, C1-C20 alkyl or C6-C12 aryl, at least one sugar to stabilize the cellular energy inhibitor and a biological buffer present in an amount sufficient to at least partially deacidify the cellular energy inhibitor and neutralize metabolic by-products of the cellular energy inhibitor.

Abstract: The present application relates to an anticoccidial composition comprising coumaric acid and use thereof. A composition comprising coumaric acid and/or a salt thereof, according to an embodiment, has excellent effects of inhibiting the cell penetration of protozoa that can induce coccidiosis and/or inhibiting the proliferation of the protozoa in cells, has excellent effects of preventing, ameliorating and treating coccidiosis in vivo, and remarkably reduces oocyst excretion in feces, thus being able to reduce secondary coccidiosis infection.

Abstract: The present invention relates to succinic acid and its derivative(s) of Formula (I), their pharmaceutically acceptable salts, polymorphs, solvates, hydrates, co-crystals, isomers and solvates thereof as an active pharmaceutical ingredient for the treatment of haematological disorder(s). The present invention further relates to compounds of Formula (I), for the treatment of disorders selected from dengue, idiopathic thrombocytopeniarpura (ITP), anaemia and chemotherapy induced myelosuppression. The present invention also relates to use of succinic acid for the treatment of haematological disorder(s), wherein succinic acid is extracted from Papaya leaves. The present invention also provides pharmaceutical compositions comprising succinic acid and its derivative(s) of Formula I and methods of treating haematological disorder(s).

Abstract: Disclosed herein is a method of treating a squamous cell carcinoma (SCC), a SCC associated with a disease, for example, recessive dystrophic epidermolysis bullosa (RDEB). The method of the disclosure describes administering a therapeutically-effective amount of (E)-2-(5-((2,4,6-trimethoxystyrylsulfonyl)methyl)-2-methoxyphenylamino)acetic acid, or a pharmaceutically-acceptable salt or zwitterion thereof, and derivatives thereof.

Abstract: Provided herein are compounds and their pharmaceutically acceptable salts, lipid particles comprising such compounds or pharmaceutically acceptable salts thereof and compositions of the foregoing that can be used to reduce immune intolerance in a subject, for example, to treat autoimmune disorders, or in combination with an antigenic therapy, such as a protein or gene therapy, to improve the efficacy of the antigenic therapy. The compounds have the following structural formula: wherein values for the variables (e.g., Ring A, L, R1, R2, R3, m) are as described herein.

Abstract: Compositions comprising 15-hydroxyeicosapentaenoic acid (15-HEPE) in free acid form or a pharmaceutically acceptable ester, conjugate, or salt thereof; and therapeutic methods of treating a subject comprising administering a composition comprising 15-HEPE to treat diseases including red blood cell disorders, hemoglobin disorders, bleeding disorders, and blood cancer in a subject in need thereof.

Abstract: The invention relates to the field of retinoid X receptor (RXR) signaling and a novel vitamin A pathway called Vitamin AS pathway. Compounds which are useful to provide (R)-9-cis-13,14-dihydroretinoic acid, an endogenous RXR ligand, are claimed as well as their uses and method for preparation thereof. The compounds of the invention are useful for pharmaceutical and nutritional uses.

Abstract: The present disclosure provides compositions containing medium chain triglycerides (MCTs) and also containing a food matrix into which at least a portion of the MCTs are formulated. The compositions include specific gums which provide stability and good mouthfeel. The compositions can improve cognitive functioning, support memory and/or recall, provide energy and/or ketones to the brain, and/or prevent and/or treat mild cognitive impairment (MCI).

Abstract: Disclosed are pharmaceutically acceptable topical formulations having enhanced potency in the alleviation of pain. Such formulations can be useful in the treatment of conditions such as back, skeletal, and joint pain, where the pharmaceutically acceptable topical formulations are applied topically to the site of pain.

Abstract: A stable pharmaceutical composition containing a non-steroidal anti-inflammatory drug derivative, at least comprising a separated solid part and liquid part, the solid part comprising a therapeutically effective dose of non-steroidal anti-inflammatory drug derivative and the liquid part being a pharmaceutically acceptable solvent. After a long-term stability study (25° C.±2° C., 12 months), the stability of the solid part was good, and met the requirements of the pharmaceutical industry.

Abstract: A ((((1r,3R,5S,7r)-3,5-dimethyladamantan-1-yl)carbamoyl)oxy)-methyl benzoate injection preparation contains ((((1r,3R,5S,7r)-3,5-dimethyladamantan-1-yl)carbamoyl)oxy)-methyl benzoate, a suspension agent, a stabilizer, an osmotic pressure adjustment agent, a pH regulator and a solvent; and the preparation may be a ready-to-use liquid injection and may also be a lyophilized powder for injection. The preparation has noticeable slow-release effects after injection, so that the drug action time can be prolonged, the frequency of dose can be reduced, the compliance of patients can be enhanced, and bioavailability can be increased. A method for preparing the above-mentioned ((((1r,3R,5S,7r)-3,5-dimethyladamantan-1-yl)carbamoyl)oxy)-methyl benzoate injection preparation is simple, economical and suitable for industrial production.

Abstract: This invention relates to pharmaceutical chemistry and specifically to the pharmaceutical composition of the compounds, which show multi-targeted action on the cholinergic, glutamatergic and mitochondrial systems that are affected in Alzheimer's Disease (AD), whose general Formula (I) is I, wherein the substituents R1 and R2 are set out in the description and claims. The formulation of these compounds increases effectiveness and tolerance in oral, sublingual, parenteral, transdermal and nasal administration. They can be used on their own, as monotherapy, as a replacement of the multi-therapy currently used for AD. The formulation of these compounds, their salts, hydrates, enantiomers, isomers, metabolites, prodrugs for administration to humans, as active ingredients for the treatment of AD, increases bioavailability, residence time of the active ingredient and adequate excretion, which increases the effectiveness, biosafety, adherence and tolerance to the treatment.

Abstract: Disclosed herein are shelf-stable formulations comprising benzoyl peroxide in a formulation effective to reduce or prevent degradation of the benzoyl peroxide to benzene. Further provided herein are methods of preventing or reducing degradation of benzoyl peroxide to benzene in a drug product, as well as methods of identifying drug products containing benzoyl peroxide that form high levels of benzene.

Abstract: Disclosed herein are shelf-stable formulations comprising benzoyl peroxide in a formulation effective to reduce or prevent degradation of the benzoyl peroxide to benzene. Further provided herein are methods of preventing or reducing degradation of benzoyl peroxide to benzene in a drug product, as well as methods of identifying drug products containing benzoyl peroxide that form high levels of benzene.

Abstract: The present disclosure targets West Nile virus, Respiratory Syncytial Virus, Influenza viruses such as Avian influenza, and other disease-causing microbes, including viruses, bacteria, fungi, and parasites. It does this using agents and methods with little toxicity compared to existing therapies.

Abstract: Described herein are compositions which can be used to alleviate vaginal dryness and vaginal atrophy. The compositions comprise a cannabinoid and a phytoestrogen. Optionally, the composition further comprises a prebiotic agent, preferably ?-glucan oligosaccharide. Optionally the composition further comprises a lubricating agent, preferably hyaluronic acid.

Abstract: Provided is a composition for suppressing muscle damage, comprising kaempferol and/or a glycoside thereof.

Abstract: Compositions in the form of a thermoformed extrudate, possibly packaged in pellet, flake, granule, powder, effervescent tablet or not, in injectable solution or not, in drink, in suspension, in gel, in ointment or in any other suitable form allowing administration to an animal or a human being, comprising at least one natural or synthetic dibenzo-?-pyrone as active substance and at least one natural or synthetic support.

Abstract: The invention involves the use of formulations of positive, negative or other allosteric modulators of primarily 5ht2a receptors, but also those of: 5ht1a/b/c/d, 5ht2b/c, 5ht3, 5ht4, 5ht7, and other receptors and systems, in combination with phenethylamines and other compounds; to treat nicotine dependency (addiction) such as tobacco-use (smoking, vaping, sublingual, oral) and/or tobacco smoking cessation.

Abstract: Disclosed in the present invention is the use of Crassifolin A in promoting mitophagy and treating neurodegenerative diseases. According to the present invention, experiments show that the diterpene compound Crassifolin A can significantly increase mitophagy levels, promote the removal of damaged mitochondria, maintain homeostasis in cells, and has potential effects of treating neurodegenerative diseases and other related diseases; moreover, Crassifolin A is a small-molecule compound that can easily penetrate through the blood-brain barrier to reach the brain so as to play a role, can be extracted from a Thickleaf croton root medicinal material, has the advantages of abundant medicinal sources, easy availability, low drug toxicity and few side effects, and has a good application prospect in the treatment of neurodegenerative diseases.

Abstract: The present disclosure provides methods and compositions for modified delivery of mycophenolic acid active agents, including sodium mycophenolate, in veterinary subjects. Presently disclosed methods and compositions are useful, for example, to treat autoimmune diseases, blood disorders associated with IMPDH activity, and immune rejection related to transplant or graft procedures.

Abstract: The present disclosure relates to treatment of a subject comprising administering to the subject a compound of formula (I); or a pharmaceutically acceptable salt thereof.

Abstract: The present disclosure relates to use of a pharmaceutical composition in the manufacture of a medicament for inhibiting and/or reversing aging in a subject. The pharmaceutical composition comprises an effective amount of ginkgolide B (GB) or a derivative thereof.

Abstract: A dietary supplement bead may include a body comprising iron and vitamin C. A dietary supplement bead may include a microencapsulating material in the body, the microencapsulating material facilitating a sustained release of the iron and the vitamin C simultaneously within a small intestine of a user.

Abstract: Disclosed herein are compositions and methods for treating or preventing Huntington's disease. In one aspect, the disclosed methods relate to targeting arachidonate 5-lipoxygenase (ALOX5) and 5-lipoxygenase-activating protein (FLAP). The compositions and methods disclosed herein can be used as disease modifying therapies to enable treatment of Huntington's disease and related disorders earlier in disease progression and improve clinical outcomes.

Abstract: The present disclosure relates to methods of treating a ciliopathy, the method comprising administering to a patient in need thereof a therapeutically effective amount of a compound or pharmaceutical composition thereof, wherein the compound modulates a TRPML ion channel. The disclosure also relates to pharmaceutical compounds of Formula (III) and subformulas thereof, or a pharmaceutically acceptable salt or composition thereof, and methods of their use for modulating TRPML ion channels, and for treating disorders.

Abstract: The present invention provides compounds for use in the treatment or prevention of leukaemia which are based on a 2-amino-[1,1?]-biphenyl or corresponding carbazole scaffold, in particular, it provides the following compounds of formula (I), their stereoisomers, and their pharmaceutically acceptable salts for use in such treatment: (I) (I) wherein: Y is selected from C=0 and —CR9 (in which R9 is H, —OH or —O-alkyl, (e.g., —O—C1-6 alkyl)); Z is selected from C=0 and —CR10 (in which R10 is H, —OH, —O-alkyl, (e.g., —O—C1-4 alkyl) or —O—C(O)RA wherein RA is H or alkyl (e.g. C1-4 alkyl)); R1 is —NO2 or —NR11R12 (wherein R11 and R12 are both H, or R11 is H and R12 is a group of the formula —C(O)RA in which RA is H or alkyl (e.g. C1-6 alkyl)); each of R2, R4, and R6 to R8 are independently selected from H, alkyl (e.g. C1-6 alkyl), —O-alkyl (e.g. —O—C1-6 alkyl), and halogen; R3 and R5 are independently selected from H, —O-alkyl (e.g.

Abstract: Provided herein are oral dosage forms comprising a) a core tablet comprising (i) a drug layer comprising apremilast and hypromellose acetate succinate (HPMCAS) in an amorphous solid dispersion; and (ii) a swellable layer comprising one or more swellable polymers; and b) a coating layer disposed on the core tablet, wherein the oral dosage form surface comprises at least one drug release orifice. The disclosed oral dosage forms provide once-a-day dosing of apremilast and are suitable for treating diseases or disorders ameliorated by inhibiting phosphodiesterase subtype IV (PDE4).

Abstract: Aspects disclosed herein include a method for treatment of a subject, the method comprising: topically administering a melanin formulation having an artificial melanin material (thereby administering artificial melanin material) to (or, to a site of) damaged skin of the subject; wherein the administered artificial melanin material comprises extracellular artificial melanin material at (or, at the site of) the damaged skin; and facilitating skin healing in the wound via at least the extracellular artificial melanin material; wherein the step of facilitating skin healing comprises at least a portion of the extracellular artificial melanin material performing a therapeutic extracellular activity.

Abstract: The present disclosure provides SETD2 protein inhibitors, and methods, uses, compositions, and kits for treating diseases, disorders, or conditions in a subject with a SETD2 protein inhibitor and a Second Therapeutic Agent, wherein the Second Therapeutic Agent comprises one or more BTK inhibitors, one or more anti-CD20 monoclonal antibodies, one or more alkylating agents, one or more topoisomerase II inhibitors, one or more vinca alkaloids, one or more platinum-based drugs, one or more nucleoside anticancer agents, one or more PI3K inhibitors, one or more CDK4/6 inhibitors, one or more CARM1 inhibitors, one or more inhibitors of enzymes of DNA damage repair, one or more SYK inhibitors, or one or more MEK inhibitors, or a combination thereof.

Abstract: Disclosed herein is a method of treating a substance use disorder in a subject suffering from sustained substance exposure, comprising: administering to the subject a therapeutically effective amount of 5-methoxy-N,N-dimethyltryptamine (5-MeO-DMT), or a pharmaceutically acceptable derivative or salt thereof.

Abstract: Provided herein are compositions and methods for cancer therapy. In particular, provided herein are compositions and methods for targeting SLC6A14 in cancer therapy using an agent that inhibits one or more activities of SLC6A14. Also, provided herein are compositions and methods for harnessing tumor SLC6A14 and SLC6A14-directed stiffness to overcome hypoxia-induced immune resistance and sensitize subjects to immunotherapy.

Abstract: The present disclosure relates in some aspects to methods for modulating and improving the subjective experience of N,N-dimethyltryptamine (DMT) by administering DMT after a long-acting tryptamine such as psilacetin, and in some embodiments, together with a benzodiazepine and/or ketamine, or in some further embodiments, together with a second long-acting psychedelic. In some aspects, disclosed methods are useful for treating medical conditions, such as mental health disorders and neurodegenerative disorders. In some aspects, disclosed methods are useful for improving health and wellbeing, such as in healthy people.

Abstract: Provided herein are, inter alia, methods and composition for the treatment of cancers that are recalcitrant to treatment and/or become resistant to certain drug treatments. The methods provided may, inter alia, be used to treat cancer in subjects having elevated STAT1 activity levels.

Abstract: Disclosed is a pharmaceutical combination comprising a glucokinase activator or a pharmaceutically acceptable salt thereof, an isotopic label thereof, a crystalline form thereof, a hydrate, a solvate, a diastereomeric or enantiomeric form thereof, and a DPP-IV inhibitor. Disclosed are a pharmaceutical composition and a fixed dose of a compound preparation, and the methods for preparing the pharmaceutical composition and the fixed dose of the compound preparation and the uses thereof.