Abstract: Compounds are provided according to Formula (I): and pharmaceutically acceptable salts thereof, and pharmaceutical compositions thereof; wherein R1, R2, R3, R4, R6, R7, R8 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: Room-temperature stable dosage forms for oral delivery of calcitonin are disclosed herein. Dosage forms for oral delivery of calcitonin with low water content are also disclosed herein. Further disclosed herein are methods for producing such room-temperature stable dosage forms and low-water content dosage forms. Methods of treatment comprising the administration of such room-temperature stable dosage forms and low-water content dosage forms are also disclosed herein.

Abstract: Systems and methods for utilizing machine learning to generate a trans-diagnostic classifier that is operative to concurrently diagnose a plurality of different mental health disorders using a single trans-diagnostic questionnaire that includes a plurality of questions (e.g., 17 questions). Machine learning techniques are used to process labeled training data to build statistical models that include trans-diagnostic item-level questions as features to create a screen to classify groups of subjects as either healthy or as possibly having a mental health disorder. A subset of questions is selected from the multiple self-administered mental health questionnaires and used to autonomously screen subjects across multiple mental health disorders without physician involvement, optionally remotely and repeatedly, in a short amount of time.

Abstract: Provided herein are chimeric antigen receptors (CARs), such as those specific for BCMA, that have improved properties, including increased CAR T cell binding to BCMA and improved CAR T cell killing of BCMA-expressing cancer cells. Use of the CARs in immune cells (e.g., T cells), compositions (e.g., CARs and nucleic acid constructs encoding the same), and methods are also contemplated.

Abstract: The invention provides compositions and methods for use in the treatment and prevention of cholestatic diseases.

Abstract: Provided herein are compositions comprising amino acids, e.g., hydroxyamino acids, thioamino acids, or pharmaceutically acceptable salts thereof, that can be used to reduce immune intolerance in a subject. The compositions can be used, 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.

Abstract: The present disclosure provides compounds, compositions thereof, and methods of using the same for the inhibition of CDK2, and the treatment of CDK2 related diseases and disorders.

Abstract: Provided herein are compounds that selectively inhibit HDAC6, a protein whose activity is associated with a variety of diseases (e.g., cancer, neurological disorders). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating HDAC6-related diseases and disorders (e.g., Alzheimer's disease, cancer) with the compounds in a subject, by administering the compounds and/or compositions described herein.

Abstract: Provided herein are vectors comprising a polycistronic expression casstte comprising a polynucleotide that encodes a TCR alpha chain, a polynucleotide that encodes a TCR beta chain, and a polynucleotide that encodes a cytokine, wherein the polynucleotides are separated by polynucleotide sequences that comprise 2A elements.

Abstract: Provided are stapled antimicrobial peptides (i.e., StAMPs) and methods of using the same (e.g., for treating bacterial infections caused by Gram-negative bacteria). In certain embodiments, the stapled peptides are based on the amino acid sequence of the antimicrobial peptide Magainin II, but include certain modifications that have been found to confer advantageous properties (e.g., improved antimicrobial activity, selectivity for killing Gram-negative bacteria, and/or reduced toxicity).

Abstract: The present invention relates, inter alia, to certain hepcidin peptide analogues, including peptides and dimers thereof, and to the use of the peptides and peptide dimers in the treatment and/or prevention of a variety of diseases, conditions or disorders, including treatment and/or prevention of iron overload diseases, which include hereditary hemochromatosis and iron-loading anemias, and other conditions and disorders described herein.

Abstract: Disclosed are small molecule antagonists of human ?4?7 integrin, and methods of using them to treat a number of diseases and conditions.

Abstract: The present application provides constructs comprising an antibody moiety that specifically binds to PSMA (e.g., PSMA expressed on the surface of a cell). Also provided are methods of making and using these constructs.

Abstract: The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.

Abstract: The present disclosure relates to compounds of Formula (I), and pharmaceutically acceptable salts thereof, wherein the variable are as described herein, and compositions and uses of same for treating neurological disorders.

Abstract: An automated medication dispenser system is disclosed. The system includes a dispenser device comprising a medication dispensing and storage module, a dispensing drive and control mechanism, and a communications interface. A third party communications host is in communication with the dispenser device, and has administration software with executable instructions for control of the dispensing drive and control mechanism to dispense medication from the medication dispensing and storage module. The communications interface is in communication with the third party communications host.

Abstract: Gene therapy compositions and methods are provided that make use of novel transposases and/or chimeric transposases for targeted transposition.

Abstract: The invention relates generally to compositions and methods for conjugating antibodies and activatable antibodies, and methods of partially reducing antibodies and/or activatable antibodies prior to conjugation, e.g., thiol-based conjugation, with an agent, e.g., a therapeutic and/or diagnostic agent.

Abstract: This invention provides Degronimers that have carbon-linked E3 Ubiquitin Ligase targeting moieties (Degrons), which can be linked to a targeting ligand for a protein that has been selected for in vivo degradation, and methods of use and compositions thereof as well as methods for their preparation.

Abstract: The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.