Abstract: The present disclosure relates to methods of determining potency of a therapeutic cell compositions in connection with cell therapy. The cells of the therapeutic cell composition can express recombinant receptors such as chimeric receptors, e.g., chimeric antigen receptors (CARs) or other transgenic receptors such as T cell receptors (TCRs). The methods provide an assay for identifying the potency, including relative potency, of a therapeutic cell composition.

Abstract: Provided herein are nucleic acid constructs, host insect cells, and methods for producing recombinant AAV capsids with high potency at high yield.

Abstract: The present invention provides novel systems, methods and compositions for making and using recombinantly engineered novel Cas9 enzymes optimized for human cells, for nucleic acid targeting and manipulation. The present invention is based on the discovery of novel Cas9 enzymes from Streptococcus constellatus, Sharpen spp. isolate RUG017, Veillonella parvula, Ezakiella peruensis, Lactobacillus fermentum strain AF15-40LB strain and Peptoniphilus sp. Marseille-P3761 bacteria that were codon-optimized and recombinantly produced for use in human cells. In some embodiments, novel Cas9 enzymes can be used for base editing. In some embodiments, the novel engineered Cas9 enzymes are used to treat human diseases.

Abstract: Disclosed is a body temperature monitoring system based on a smart watch, the system including: a baseline measuring unit configured to convert information on body temperature of a user, measured at predetermined intervals, into data; a personal variable input unit configured to store information on age and gender of the user; and a controller configured to compare and analyze a body temperature value converted by the baseline measuring unit and a normal body temperature range derived by the personal variable input unit. The controller is further configured to: determine whether the user's current body temperature falls within the normal body temperature range based on information produced from the baseline measuring unit and the personal variable input unit; and identify a disease type by analyzing a duration, a value, and a timing of deviation of the user's body temperature from the normal body temperature range.

Abstract: Provided are implantable and biodegradable drug delivery devices capable of delivering an active pharmaceutical ingredient (API) directly to a target pancreatic tumor tissue. The drug delivery devices can include at least two layers. One of the layers can include the API and a biodegradable polymer and a second layer can include another biodegradable polymer that degrades slower than the first biodegradable polymer. When the device is placed directly on the target tissue, the API layer can degrade thereby releasing the API towards the target tissue while the non-API layer can prevent the API from being released away from the target tissue onto non-target tissue.

Abstract: The present disclosure relates to methods of treating neurological diseases, comprising administering to a subject in need thereof, a therapeutically effective amount of a compound or pharmaceutical composition according to any embodiment described herein. The present disclosure relates to the methods of treating Parkinson's disease and Dementia with Lew Bodies, comprising administering to a subject in need thereof, a therapeutically effective amount of a compound or pharmaceutical composition according to any embodiment described herein.

Abstract: The present disclosure relates to an isolated polynucleotide comprising a sequence that encodes an interleukin (IL)-12 molecule, and a delivery system that can carry the polynucleotides. The disclosure also includes methods of making the polynucleotides and methods of treatment using the same.

Abstract: A method comprising obtaining a bodily fluid from a subject; contacting the bodily fluid with an adsorbent material comprising a synthetic carbon particle (SCP) to produce a first filtrate having a level of disease mediators (y); contacting the first filtrate with an adsorbent material comprising the SCP and an anion exchange resin where the ratio of SCP to anion exchange resin is from about 0.1:100 to 100:0.1 to produce a second filtrate; contacting the second filtrate with an adsorbent material comprising the SCP and a cation exchange resin where the ratio of SCP to cation exchange resin is from about 1:100 to produce a third filtrate; and administering the third filtrate to the subject.

Abstract: The disclosure relates to the use of atovaquone or a salt thereof, and, optionally, proguanil or a salt thereof, for the treatment of various human diseases and conditions, including gastrointestinal disorders and, in particular, those associated with inflammation, such as inflammatory bowel disease (IBD), ulcerative colitis (UC), and Crohn's disease (CD).

Abstract: The present disclosure provides methods, compositions and kits for measuring activity of a frataxin (FXN) protein, e.g., an FXN fusion protein. The present invention also provides methods for identifying compounds capable of modulating activity of an FXN protein, e.g., an FXN fusion protein.

Abstract: Compounds are provided according to Formula (I): and pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof; wherein X1, X2, X3, X4, Y, A, L1, L2, R1, R2, R5, m and n are as defined herein. Compounds of the present invention are contemplated useful for the prevention and treatment of a variety of conditions.

Abstract: Provided herein are methods and uses involving antibodies that specifically bind to a KIT receptor tyrosine kinase for managing, treating, or preventing an eosinophil or mast cell related disorder and/or one or more symptoms thereof, for example a mast cell related disorder of the nervous system, e.g., central nervous system, for example neuromyelitis optica (NMO), neuromyelitis optica spectrum disorder (NMOSD), multiple sclerosis (MS), and neurofibromatosis (NF).

Abstract: An apparatus for treating an aneurysm in a blood vessel includes an occlusion element configured to be releasably coupled to an elongate delivery shaft and configured to be delivered in a collapsed configuration through an inner lumen of a delivery catheter, the occlusion element including an inverted mesh tube having an outer layer and an inner layer, the outer layer transitioning to the inner layer at an inversion fold, wherein at least the outer layer is formed into a first expanded shape including: a smooth outer cylinder configured to engage a wall of an aneurysm throughout a 360° circumference; and a substantially distal-facing surface including one or more heat-formed undulations, the one or more undulations configured to apply a radial force on the wall of the aneurysm.

Abstract: Methods are provided for preventing, reducing, and/or treating contrast-induced acute kidney injury which include administering an inhibitor of fatty acid oxidation to a patient in need thereof. Also provided are methods involving use of trimetazidine or pharmaceutically acceptable salts thereof for the prevention and/or treatment of contrast-induced acute kidney injury. Methods are also provided for preventing and/or treating contrast-induced acute kidney injury which include administration of one or more of trimetazidine, etomoxir, oxfenicine, perhexiline, mildronate, or ranolazine, or pharmaceutically acceptable salts of any of the preceding.

Abstract: This invention provides a fluid therapeutic placental product comprising placental cells and a placental dispersion comprising placental factors. The placental cells and the placental dispersion are derived from placental tissue. A placental tissue can optionally be an amnion, chorion, or a trophoblast-depleted chorion. The placental product of the present invention is useful in treating a patient with a tissue injury (e.g. wound or burn) by applying the placental product to the injury. Similar application is useful with ligament and tendon repair and for engraftment procedures such as bone engraftment.

Abstract: The present invention provides pharmaceutical compositions comprising an iodine-containing compound and a steroid, useful for treating a clinical symptom in a patient's airway (e.g., nose, lung, and sinus), as well as methods for using the same.

Abstract: Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

Abstract: Disclosed herein are inhibitors of TACC and methods of treating certain diseases and disorders (e.g., diseases and disorders related to TACC).

Abstract: The present disclosure relates to ALK5 inhibitors and their uses.

Abstract: Provided herein are inhibitors of TNF-?, pharmaceutical compositions comprising the inhibitory compounds, and methods for using the TNF-? inhibitory compounds for the treatment of diseases or disorders.