Abstract: Stabilized oral cannabidiol composition comprising cannabidiol (CBD) having a purity of at least 98%. The composition further contains ?-caryophyllene (BCP), at least one additional lipophilic solvent (e.g. medium chain triglycerides (MCT)), and at least one additional antioxidant (e.g. ?-tocopherol (vitamin E)). The composition is substantially free of THC. The CBD can be synthetic, biosynthetic or of botanical origin. Also disclosed are methods of making and the stabilized oral CBD composition.

Abstract: This invention features nucleic acid constructs which comprises reporter genes and a query sequence, wherein the query sequences encode or are RNA folded into a secondary structure and or RNA regulatory elements. These nucleic acid constructs can be used in massively parallel assay methods for perturbation profiling also disclosed herein. Such methods provide the ability to study the effect of chemical or genetic perturbations to modulate RNA within an intracellular context.

Abstract: The present invention relates to certain diaryl macrocyclic compounds, pharmaceutical compositions containing them, and methods of using them, including methods for treating cancer, pain, neurological diseases, autoimmune diseases, and inflammation.

Abstract: Described herein are novel piperidine urea derived compounds and their pharmaceutical compositions for the treatment of cancer as a monotherapy or a combination therapy with chemotherapeutic agents and/or checkpoint inhibitors.

Abstract: Provided herein are polypeptides that bind to a transferrin receptor, methods of generating such polypeptides, and methods of using the polypeptides to target a composition to a transferrin receptor-expressing cell.

Abstract: An apparatus for treating an aneurysm in a blood vessel includes an occlusion element including a first tubular mesh having a first end and a second end coupled together at a proximal end of the occlusion element such that an intermediate portion of the first tubular mesh between the first end and the second end includes a substantially 180 degree turn, the intermediate portion of the first tubular mesh extending distally from the proximal end of the occlusion element, wherein the intermediate portion of the first tubular mesh has a collapsed configuration and is configured to expand to an expanded. In some embodiments, the apparatus further includes a second tubular mesh having a first end and a second end coupled to the proximal end of the occlusion element such that an intermediate portion of the second tubular mesh between the first end and the second end includes a substantially 180 degree turn.

Abstract: Applicant provides methods of treating diseases including Cushing's syndrome and hormone-sensitive cancers by concomitant administration of a glucocorticoid receptor modulator (GRM) and steroidogenesis inhibitors, and by concomitant administration of a GRM and CYP3A inhibitors. The GRM may be, e.g., mifepristone; the CYP3A inhibitors or steroidogenesis inhibitors (collectively “inhibitors”) may be, e.g., ketoconazole or itraconazole. Inhibitors may cause toxicity or other serious adverse reactions; concomitant administration of inhibitors with other drugs may increase the risk of such toxicity and adverse reactions due to the inhibitors and/or the other drugs. Applicant has surprisingly found that GRMs may be administered to subjects receiving inhibitors without increasing the risk of adverse reactions; for example, Applicant has found that mifepristone may be concomitantly administered with ketoconazole or itraconazole, providing safe concomitant administration of the GRM and ketoconazole or itraconazole.

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 inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.

Abstract: The invention relates to psychoactive medicines including 2C-B, methylone, MBDB, their respective metabolites, isomers, enantiomers, polymorphs, and analogues (2C-series and cathinones); their preparation, formulations, intermediates, routes of administration, dosing and schedule for medical uses for psychiatric and neurological conditions and disorders.

Abstract: Provided herein is a compound of Formula (I) or a pharmaceutically acceptable salt thereof, wherein R19, R5, R3a, R1a, R1b, R2a, R2b, R4a, R4b, R6a, R6b, R7a, R7b, R11a, R11b, R12a, R12b, R18, RD, and q are defined herein. L is selected from the group consisting of: wherein A indicates the point of attachment at C17 and wherein X is selected from the group consisting of —C(0)N(R55a)(R55b), —N(R55a)(R55b), —N(R55b)C(O)(R55a), and R55C wherein R55c is carbon-bound substituted or unsubstituted heteroaryl or substituted or unsubstituted aryl. Also provided herein are pharmaceutical compositions comprising a compound of Formula (I) and methods of using the compounds, e.g., in the treatment of CNS-related disorders.

Abstract: The present technology relates to depots for the treatment of postoperative pain via sustained, controlled release of a therapeutic agent. In some embodiments, the depot may comprise a therapeutic region comprising an analgesic, and a control region comprising a bioresorbable polymer and a releasing agent mixed with the polymer. The releasing agent may be configured to dissolve when the depot is placed in vivo to form diffusion openings in the control region. The depot may be configured to be implanted at a treatment site in vivo and, while implanted, release the therapeutic agent at the treatment site for no less than 3 days.

Abstract: The present disclosure provides 6-heteroaryloxy benzimidazole and azabenzimidazole compounds and compositions thereof useful for inhibiting JAK2.

Abstract: This invention relates to the treatment of Pulmonary Hypertension with heart failure with preserved ejection fraction (PH-HFpEF). More specifically, embodiments of the invention provide compositions and methods useful for the treatment of PH-HFpEF employing the use of levosimendan.

Abstract: The compositions and methods described herein are directed to treating solid tumors using CAR T therapy and/or antibodies targeting GCC. The compositions include CAR comprising an extracellular domain that binds GCC.

Abstract: The disclosure provides compositions comprising novel adenosine base editors (e.g., ABE8) that have increased efficiency and methods of using these adenosine deaminase variants for editing a target sequence.

Abstract: Provided herein are tumor-infiltrating lymphocytes (TILs) engineered to express a membrane-bound interleukin 15 (mbIL15). The mbIL15 TILs can be expanded in vitro using a rapid expansion protocol without the use of exogenous interleukin 2 (IL2) and can be used in adoptive cell therapy without concomitant use of an exogenous cytokine such as IL2. The TIL can be further engineered such that the mbIL15 is operably linked to one or more drug responsive domains (DRDs), polypeptides that can regulate the abundance and/or activity of the IL15 upon binding of the DRD with a ligand. Also provided herein are components for making the modified TILs and methods for making and using the modified TILs.

Abstract: The invention features polynucleotides that encode bases editors having a heterologous intron for self-inactivation, compositions comprising such polynucleotides, and methods of inactivating a base editor encoded by such polynucleotides.

Abstract: The present disclosure provides compositions and methods for the treatment of ocular diseases associated with angiogenesis, particularly wet age-related macular degeneration.

Abstract: Formulations of tegavivint, methods of making such formulations, and methods of treatment of cancer by administering the formulations.

Abstract: The present disclosure relates to HIF-2? inhibitors and methods of making and using them for treating cancer. Certain compounds were potent in HIF-2? scintillation proximity assay, luciferase assay, and VEGF ELISA assay, and led to tumor size reduction and regression in 786-O xenograft bearing mice in vivo.