Abstract: A pharmaceutical composition, a combination product including the composition and a method of manufacturing the composition. The composition includes a particulate complex of API bound with a crosslinked polysaccharide micro sponge. The combination product includes a dosage form of the particulate complex within a container. The amount of the particulate complex is selected to provide a defined dose of the API. The formulation may be heated within the container by a vaporizer device, vaporizing the API and dissociating the API from the micro sponge as vapour, which passes through apertures in the container facilitating administration of the API by inhalation of the vapour. The apertures are sized to facilitate passage of the vapour but prevent passage the particulate complex. The API may be hydrophobic such as phytocannabinoids or hydrophilic such as nicotine. The pharmaceutical composition may be manufactured by driving binding of the API to the micro sponge in solution.

Abstract: Disclosed are dendrimers of formula (I): and pharmaceutically acceptable salts thereof. Also disclosed are pharmaceutical compositions comprising the dendrimer of formula (I) and methods of using the same for treating cancer.

Abstract: Chlorotoxin derivatives containing amino acid sequence X0X1CMPCXS1XS2XS3DHXS4XS5ARRCX2X3CCGGYGX4CFGYQC LCX5X6X7X8 wherein (i) the N-terminal X0X1 cluster is AM, 0M, or 00; (ii) the solubility XS1XS2XS3XS4XS5 cluster is FTTQT, FTTES, SSSQT, SSSES, FSSQT, FSSES, or FSSQS; (iii) the internal X2X3X4 cluster is DKR, RDK, KDR, IKY, HKW, DRK, LKQ, KKK; and (iv) the C-terminal X5X6X7X8 cluster is N000, R000, NR00, NRG0, NRGY, NRRR, or RRRR; 0 denotes a position where no amino acid is present; the chlorotoxin derivative has a relative human MMP-2 binding that is at least 1.62 times higher than the wild-type chlorotoxin of SEQ ID NO: 1.

Abstract: Provided herein are methods of treating B-cell proliferative disorders (such as diffuse large B-cell lymphoma “DLBCL”) using immunoconjugates comprising anti-CD79b antibodies in combination with an immunomodulatory agent (such as lenalidomide) and an anti-CD20 antibody (such as obinutuzumab or rituximab).

Abstract: Compounds or salts thereof represented by formula (I): wherein X indicates a leaving group, Y indicates an affinity peptide having a binding region in a CH2 domain in an immunoglobulin unit comprising two heavy chains and two light chains, M indicates a trivalent group linking the carbon atom in C?O adjacent to M and the carbon atom in C?W via a main chain portion consisting of 3 to 5 carbon atoms, O indicates an oxygen atom, S indicates a sulfur atom, W indicates an oxygen atom or a sulfur atom, N3 indicates an azide group, La indicates a bond or a divalent group, and Lb indicates a bond or a divalent group; and antibodies or salts thereof which can be prepared using such a compound or salt thereof, are useful for controlling the bonding ratio of an antibody and a modifying group within a desired range.

Abstract: The present invention provides a novel anti-DLL3 antibody-pyrrolodiazepine derivative and a novel anti-DLL3 anti-body-pyrrolodiazepine derivative conjugate using the same.

Abstract: It is an object of the present invention to provide an antibody-drug conjugate of an antibody binding to DLL3 and a drug having antitumor activity, a pharmaceutical composition comprising the antibody-drug conjugate and having therapeutic effects on a tumor, a method for treating a tumor using the antibody-drug conjugate or the pharmaceutical composition, and the like. The present invention provides an antibody-drug conjugate of an antibody binding to DLL3 and a drug having antitumor activity, a pharmaceutical composition comprising the antibody or the antibody-drug conjugate, and a method for treating a tumor.

Abstract: The present invention relates in certain aspects to antibodies, binding polypeptides, and immunoconjugates specific for human EPH receptor A5 (EphA5).

Abstract: Agents having cytotoxic activity, as well as compositions, uses, and methods relating thereto, are described herein. Certain steroid acid-peptide conjugates or moieties have the ability to induce killing or inhibition of proliferation of mammalian cells, in vitro or in vivo upon administration to a subject. The steroid acid-peptide conjugates include bile acids and bile acid analogs and peptides that may include a nuclear localisation signal or a portion thereof. Also described herein is a method for treating cancer, an autoimmune disease, or any other disease or disorder ameliorated by treatment with an antiproliferative drug in a subject in a subject with the cytotoxic agents described herein.

Abstract: The invention relates generally to conjugated activatable antibodies that bind CD71 in their active form and methods of making and using these anti-CD71 conjugated activatable antibodies in a variety of therapeutic, diagnostic and prophylactic indications.

Abstract: The present disclosure relates to anti-Epidermal Growth Factor Receptor (EGFR) antibody drug conjugates (ADCs) which inhibit Bcl-xL, including compositions and methods using such ADCs, and methods for making such ADCs.

Abstract: The invention relates to a saponin derivative The invention also relates to a first pharmaceutical composition comprising the saponin derivative of the invention. In addition, the invention relates to a pharmaceutical combination comprising the first pharmaceutical composition of the invention and a second pharmaceutical composition comprising for example an ADC or AOC. The invention also relates to the first pharmaceutical composition or the pharmaceutical combination of the invention, for use as a medicament, or use in the treatment or prophylaxis of a cancer or an auto-immune disease. Furthermore, the invention relates to an in vitro or ex vivo method for transferring a molecule from outside a cell to inside said cell, comprising contacting said cell with the molecule and with a saponin derivative of the invention. The invention also relates to a saponin conjugate comprising a cell-surface molecule binding-molecule, capable of binding to a target cell, covalently bound to the saponin.

Abstract: Disclosed are compositions and methods for treating a disease or disorder such as cancer in a subject in need thereof. In some aspects, the method comprises administering to the subject a vector comprising a first nucleic acid sequence encoding a promoter operably linked to each of a second nucleic acid sequence encoding a therapeutic polypeptide, and a third nucleic acid sequence encoding a peptide domain that is stabilized when phosphorylated by kinase activity in a target tissue. The kinase activity can be elevated extracellular regulated kinase (ERK) activity.

Abstract: The disclosure provides nanoemulsion compositions and methods of making and using thereof to deliver a bioactive agent such as a nucleic acid to a subject. The nanoemulsion composition comprises a hydrophobic core based on inorganic nanoparticles in a lipid nanoparticle that allows imaging as well as delivering nucleic acids. Methods of using these particles for treatment and vaccination are also provided.

Abstract: Provided are compositions that include targeting peptides and methods for using the same to treat and/or prevent various diseases, disorders, and/or conditions. In some embodiments, the compositions and methods relate to liposomal compositions that include a liposome, the surface of which is conjugated to a peptide having an amino acid sequence as set forth in any of SEQ ID NOS: 3-38, optionally wherein the liposome encapsulates a therapeutic agent or a detectable agent. In some embodiments, the peptide has an amino acid sequence that is one of SEQ ID NOs: 14, 19, 20, 27, and 28. Also provided are methods treating or preventing fibrosis, for decreasing the incidence of a disease, disorder, or condition associated with chronic pancreatitis (CP), for targeting active agents to targets, including but not limited to collagen III-expressing cells and extracellular matrix, and for decreasing incidence of side effects associated with apigenin treatment.

Abstract: The instant disclosure relates to nucleic acid-lipid particles having 1,2-dioleoyl-3-trimethylammonium-propane (DOTAP), which preferentially localize and deliver associated cargoes to the lung and various lung tissues, as well as to tissues to which such particles are directly injected. The instant disclosure provides compositions comprising such lipid particles, optionally in association with a therapeutic agent (e.g., a therapeutic mRNA and/or nucleic acid controller system), as well as methods and kits for delivering a lipid particle-associated therapeutic agent and/or treating a disease or disorder, e.g., a lung disease or disorder, in a subject, using the lipid particle compositions provided herein.

Abstract: The present invention concerns an in vivo method for introducing a naked mRNA molecule into the cytosol of a cell(s) in a subject, by the use of photochemical internalization, wherein the photosensitising agent is a sulphonated meso-tetraphenyl chlorin, sulfonated tetraphenylporphine or a di- or tetrasulfonated aluminium phthalocyanine used in an amount of 0.000001-0.001 ?g and the cells(s) are contacted with the mRNA molecule and photosensitising agent for 30 seconds to 10 minutes before irradiation of the cell(s). The method may be used to express a polypeptide in the subject. The method is also directed to pharmaceutical compositions containing the photosensitising agents and the mRNA and uses of the molecules in therapy, e.g. to treat or prevent cancer or an infection.

Abstract: In various aspects and embodiments, the invention provides a method of treating a cardiovascular disease in a subject in need thereof, the method comprising administering an effective amount of a viral vector comprising a therapeutic polynucleotide directly into the heart of the subject. In various embodiments, the pharmaceutical composition is administered through a series of 15 injections at separate delivery sites in the heart of the subject, and wherein the viral vector diffuses through substantially all of the heart.

Abstract: Provided herein are polynucleotide sequences encoding human acid sphingomyelinase (SMPD1) and expression cassettes containing these coding sequences. Also provided are vectors, such as recombinant adeno-associated virus (rAAV) vectors having vector genomes that include an engineered SMPD1 coding sequence operably linked to one or more regulatory sequences. Further, compositions containing these expression cassettes and rAAV are provided, as well as methods for the use of these compositions for treatment of Niemann Pick Type A disease.

Abstract: The present disclosure provides a modified AAV capsid protein comprising a targeting peptide, optionally further comprising a liver-toggle mutation. The modified AAV capsid protein can form an rAAV, which has a preferred tropism, specificity or biodistribution in vivo or in vitro. The rAAV of the present disclosure can be used for gene therapies targeted at a specific tissue. The present disclosure also provides rAAV compositions comprising MTM1 coding sequences and their use to treat subjects suffering from X-linked myotubular myopathy (XLMTM).

Abstract: The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of Lysosomal Acid Lipase Deficiency (LAL-D) disorders such as Wolman Disease and cholesterol ester storage disease (CESD). The disclosed rAAV provide a wild type lipase A (LIRA) cDNA to a subject in need which results in expression of the wild type protein.

Abstract: Methods and materials for treating a mammal having a TDP-43 proteinopathy (a disorder characterized by the accumulation and/or aggregation of TDP-43 polypeptides in the central nervous system) are provided herein. For example, this document provides methods and materials for administering nucleic acids encoding polyadenylate-binding protein 4 (PABPC4) to a mammal having a TDP-43 proteinopathy, such that the level of PABPC4 in the central nervous system of the mammal is increased.

Abstract: There is described a vector for treating Dopamine Transporter Deficiency Syndrome, the vector comprising a promoter operably linked to a human SLC6A3 gene, wherein the promoter is selected from a human synapsin 1 promoter, a CAG promoter, a CMV promoter, a CAMKII promoter, a beta-actin promoter, and a human EF1-alpha promoter. Also described are methods and uses involving the vector for treating Dopamine Transporter Deficiency Syndrome.

Abstract: Provided herein are methods for treatment of cystic fibrosis (CF), including for patients with class I CFTR mutations. The methods may involve administration of a recombinant adeno-associated virus (rAAV) that includes an AV.TL65 capsid protein and a polynucleotide that includes an F5 enhancer and a tg83 promoter operably linked to a CFTR?R minigene, or a pharmaceutical composition thereof.

Abstract: The present disclosure generally relates to engineered Cluster Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated (Cas) 12a proteins and system, and methods for use in gene editing and gene modulation for application to gene therapy. Related systems and methods of gene modulation are also disclosed.

Abstract: This invention relates to the compositions and methods for labeling antibodies and other proteins and targeting agents with a helical bundle protein that is functionalized with cargo. Cargos can include but are not limited to fluorescent dyes, haptens (e.g. biotin), contrast agents (e.g. gadolinium, radionuclides), chelated metals, therapeutic agents, sensitizers, or other small molecules. Specifically, provided herein are compositions having a helical bundle, that has been labeled at precisely defined locations with cargo, and that can be conjugated, attached or fused to an antibody or other targeting agent.

Abstract: The present disclosure relates to polymers which contain a hydrophobic and hydrophilic segment which is sensitive to pH. In some aspects, the polymers form a micelle which is sensitive to pH and results in a change in fluorescence based upon the particular pH. In some aspects, the disclosure also provides methods of using the polymers for the imaging of cellular or extracellular environment or delivering a drug.

Abstract: Provided are proteins/peptides. The proteins or peptides comprise a sequence designed by the methods described herein. The proteins and peptides may have one or more trifluoroleucine (LTF, which may be referred to as TFL or LTF throughout) residues.

Abstract: A method (100) for generating an instantly-formed artificial saliva gel or an ultrasound gel, comprising: (i) providing (110) a first composition comprising a pH-sensitive polymer, wherein the first composition is maintained at an acidic pH; (ii) providing (110) a second composition comprising a pH agent configured to maintain the second composition at an alkaline pH; and (iii) combining (120) the first composition and the second composition to generate a single instantly-formed gel, wherein the pH of the single instantly-formed gel is at a level required to increase viscosity of the pH-sensitive polymer to a level suitable for an artificial saliva gel or an ultrasound gel; wherein either the first composition or second composition comprises a solvent, and wherein the acidic pH of the first composition and the alkaline pH of the second composition are configured such that the pH of the single instantly-formed gel is between approximately 6.5 and 7.5.

Abstract: This disclosure relates to compounds of Formula IV: for fibrin imaging, wherein the compounds comprise an imaging or therapeutic radioisotope.

Abstract: The present invention is related to a compound comprising a cyclic peptide of formula (I) and an N-terminal modification group A attached to Xaa1, wherein Xaa1, Xaa2, Xaa3, Xaa4, Xaa5 and Xaa6 are each residues of an amino acid, Xaa7 is a residue of an amino thiol or an amino acid of formula, Yc is a cyclization element which is either present or absent, and the N-terminal modification group A is either a blocking group Abl or an amino acid Aaa.

Abstract: A device including a housing that is substantially impermeable to ultraviolet radiation having a wavelength of from 280 nm to 400 nm, and at least one window defined in the housing, the window including a UV-C radiation band-pass mirror film having a multiplicity of alternating first and second optical layers collectively transmitting UV-C radiation at a wavelength from at least 100 nm to less than 280 nm and not transmitting UV-A and UV-B radiation at a wavelength of from 280 nm to 400 nm, and an ultraviolet radiation source positioned within the housing, the ultraviolet radiation source being capable of emitting ultraviolet radiation at one or more wavelength from 100 nm to 400 nm. The device optionally further includes an ultraviolet mirror film positioned within the housing so as to reflect ultraviolet radiation emitted by the ultraviolet radiation source. A method of disinfecting a material is also disclosed.

Abstract: A disinfecting mat device is provided. The device is comprised of a body with at least one, but preferably a plurality of layers with at least one UV light, at least one storage tank that stores at least one disinfecting agent, and at least one sensor. The device is designed to be used as a door mat, such that the device sanitizes the bottom of the shoes of the user as they step on the device by expelling sanitizing solution through the top surface. The device also simultaneously sanitizes the bottom of a user's shoes via the UV light when the user steps on the device.

Abstract: In some embodiments, a water ozonation system can include an ozone generator including a corona discharge unit configured to provide ozonated air as an output. The water ozonation system can further include a tubing having a first end coupled to the output of the corona discharge unit and configured to deliver the ozonated air to a second end. The water ozonation system can further include an injector assembly having a suction port coupled to the second end of the tubing and in communication with a Venturi tube having an input and an output, such that flow of liquid from the input to the output results in a suction being formed through the suction port to thereby draw the ozonated air into the liquid flowing through the Venturi tube.

Abstract: System and methods for disinfecting ultrasound probes including a disinfection module operably and physically coupled with an ultrasound-imaging system including an ultrasound probe. The disinfection module is configured to expose the external surface of the ultrasound probe to UV light to define a high-level disinfection process. Sensors determine when the ultrasound probe is disposed within a disinfecting cavity of the disinfection module and logic governs the operation of UV light sources. Logic determines a contamination level of the probe and defines an appropriate disinfection level. Exposing the probe to hydrogen peroxide is an alternative to the UV light. Logic may activate the high-level disinfection process upon placement of the probe within the cavity.

Abstract: A sterilization cabinet, comprising a top panel, at least two side panels, and a floor panel forming a part of a chamber of the sterilization cabinet; at least one door connected to at least one of the at least two side panels of the sterilization cabinet; a vent formed in at least one of the two side panels; at least one first filter covering the vent and a filter cover configured to hold the first filter against the vent; a drain positioned in the floor panel, wherein the floor panel has a slope configured to cause condensate within the chamber to flow into the drain and wherein the drain is the only outlet for the condensate along the floor panel; and a second filter covering the drain such that condensate flowing into the drain passes through the second filter.

Abstract: The substrate for diffusing volatile substances comprises a dispersible binder and a natural material, and the percentages by weight in the dry state are: dispersible binder: 15-45%; and natural material: 55-75%. It may also comprise a preservative. It provides a substrate that provides constant and durable air diffusion properties.

Abstract: A window includes: a base layer; and a fragrance-emitting layer on the base layer and including a plurality of fragrance-emitting capsules; and an anti-fingerprint layer on the fragrance-emitting layer.

Abstract: An aroma diffuser comprises a compression plug connected to an air pump, an oil tube, an atomizing chamber, a spray outlet base, and a rotary spray outlet cover. The atomizing chamber comprises a chamber, a spray outlet sleeve, and a spray outlet, a distance between the air outlet and a chamber wall of the atomizing chamber is 11-13 times an aperture diameter of the air outlet, the spray outlet of the atomizing chamber extends downward to form a spray outlet channel, the spray outlet sleeve is sleeved outside of the spray outlet channel, atomized essential oil enters into the gaps between the spray outlet sleeve and the spray outlet channel from the groove notch and is then sprayed out, a bottom of the spray outlet sleeve is obliquely disposed and comprises a first reflux hole, and the rotary spray outlet cover is rotatably connected to the spray outlet base.

Abstract: A full facial host mask is provided that may have a portion that may be removed and re-mated with the host mask. The removable portion may be, for example, a biological mask (e.g., a UV-C device that may include several UV-C light sources (e.g., UV-C LEDs) and such UV-C LEDs may have UV-C reflecting structures arranged to direct UV-Cina particular direction and at a particular size and shape). The UV-C generating device in the removable satellite mask ay be utilized in the breathing stream to provide a low-air resistance, long duration, high performance mask for biological (e.g., DNA-based, RNA-based, gram-positive bacteria, and gram-negative bacteria). The full facial mask may include mechanical filters (e.g., filters having 300 nm pores or smaller), chemical filters, and/or nuclear particulate filters. In doing so, a single mask may be issued to an entity (e.g., a warfighter) that can provide a full facial mask as well as partial face mask so various mission profiles may be achieved.

Abstract: A sterilizing apparatus and a home appliance comprising the same are provided.

Abstract: The subject invention provides topical therapeutic compositions and methods of their use for enhanced healing of wounds, including burns, and scars of the skin, wherein the compositions and methods utilize microbial biosurfactants. The invention reduces the healing time of skin wounds, reduces the appearance of scars, and improves other skin conditions such as acne, psoriasis and eczema.

Abstract: Proposed are a method of manufacturing a hemostatic medical material that has excellent biosafety and can be applied to various situations by controlling the decomposition rate as necessary, and a medical material manufactured thereby. The method includes pretreating a natural cellulose substrate by adjusting the pH of the substrate, causing the hemostatic agent to be adsorbed on the pretreated substrate, drying the substrate with the hemostatic agent adsorbed thereon, primarily modifying the dried substrate using monochloroacetic acid (MCA), secondarily modifying the primarily modified substrate using an organic acid aqueous solution, washing the secondarily modified substrate, and post treating the substrate by adjusting the pH of the washed substrate.

Abstract: Provided herein are hydrogel-hydrocolloid sealant compositions for use as soft-tissue sealants. The compositions can include a cross-linkable electrophilic component; a cross-linkable nucleophilic component; a swellable filler material including gelatin; and, a buffer having a pH in the range of about 9.0 to about 10.0. The compositions can have a first fluid state and a second crosslinked state, and in the crosslinked state, the electrophilic and nucleophilic components can crosslink to form a crosslinked hydrogel network where the swellable filler material including gelatin is disposed within the crosslinked network. Also provided are systems and methods of manufacturing the sealant compositions as well as methods of repairing soft tissue defects including applying the sealant compositions disclosed herein.

Abstract: A medical tissue adhesive and a preparation method thereof. The medical tissue adhesive includes: a functional component, an assistant crosslinker and a dispersing agent. The functional component is a polymer chain modified by tissue adhesive group or a micro nano particle modified by tissue-adhering functional groups. The tissue-adhering functional groups includes: o-nitrobenzyl photoplate group, active ester group or carbonyl group. The medical tissue adhesive not only uses the dispersing agent to expel the tissue fluid/blood on the tissue surface, so that the adhesive molecular segment quickly forms a chemical bond with the amino group on the tissue surface to form a strong tissue adhesive force. But the medical tissue adhesive can also trigger the adhesive molecular segment to react with the amino group on the assistant crosslinker, so that the medical tissue adhesive can quickly gel and solidify. Therefore, it has good bleeding performance, hemostatic performance and tissue adhesion performance.

Abstract: The present application relates to a lyophilized scaffold composition having at least one polysaccharide wherein said scaffold is substantially solid and capable of being formed into a desired shape; wherein the at least one polysaccharide has a protonation level resulting in controlled rehydration of said scaffold, such that when said scaffold is contacted with at least one of a neutral aqueous solution, blood, blood derived fluid and combinations thereof, said scaffold forms a microparticle dispersion and stimulates tissue remodeling and anabolic wound repair, a process for preparing a lyophilized scaffold composition and the use of a lyophilized scaffold composition for wound repair in a mammal.

Abstract: A method for producing a swellable body including bacterial cellulose includes providing a body made of bacterial cellulose, cleaning the body using at least one liquid medium, freezing the body at atmospheric pressure for at least six hours, freeze drying the body, and mechanically pressing the entire molded body or parts thereof after freeze drying of the body. A body of bacterial cellulose is improved regarding its swelling capability.

Abstract: Magneto-responsive properties are traditionally imparted to scaffold systems via integration of iron oxide-based magnetic nanoparticles (MNPs), yet poor understanding of long-term MNP toxicity presents a significant translational challenge. Given the demonstrated iron-binding capacity of silk fibroin (SF), passive chelation of ferric iron ions is explored herein as an alternative, MNP-free approach for magnetic functionalization of silk fibroin (SF)-based biomaterials. SF microfibers treated with aqueous ferric chloride (FeCl3) exhibit significantly increased iron content relative to the nascent protein.

Abstract: The present invention discloses a recombinant collagen protein and its use in the cartilage repair matrix. The present invention discloses a recombinant collagen protein which contains the sequence shown in SEQ ID No. 1; the amino acid sequence of the stated recombinant collagen protein comprises N basic repetitive units; the basic repetitive unit contains n1 amino acid sequences with the following characteristic: G-Xaa1-Xaa2-G-E-Xaa3; the 3? end and the 5? end of the basic repetitive unit are connected to form the amino acid sequence with the above characteristic. The recombinant collagen protein claimed by the present invention has significant integrin binding activity, has the effect of promoting cell adhesion, proliferation, differentiation, and repairing cartilage tissue defects, etc, and it has promising prospects for application.

Abstract: Compositions containing purified collagen biomaterial derived from tissues, for example, insoluble amnion, soluble amnion, soluble chorion of the human placenta, are provided. The collagen compositions can be used to promote wound healing, promote tissue regeneration, prevent or reduce scarring, reduce local inflammation, minimize tissue rejection, promote graft integration. Methods for using the collagen composition as a biomaterial implant for dermal filling, skin grafting, and hair transplantation are also provided.