Abstract: Provided herein are methods of treating multiple myeloma, comprising administering to a subject with multiple myeloma an inhibitor of Nuclear Receptor Binding SET Domain Protein 2 (NSD2). Also provided are methods of use wherein the multiple myeloma has previously been determined to have a 4:14 chromosome translocation (t(4;14)).

Abstract: Provided are transgenic mice whose genome comprises a nucleic acid sequence encoding human cereblon (CRBN) or a fragment thereof, wherein endogenous mouse CRBN is not expressed in the transgenic mice. Also provided are cells, cell lines, tissues, and organs derivable from the transgenic mice, and methods for producing and using such mice.

Abstract: Provided herein are compounds and compositions thereof for modulating S1P5. In some embodiments, the compounds and compositions are provided for treatment of neurological diseases.

Abstract: Provided herein are compounds of Formula (I) and pharmaceutically acceptable salts, tautomers, isotopologues, or stereoisomers thereof, wherein R1, R2, R3, R4 and R5 are as defined herein (referred to herein as “Heterocyclic Compounds”), compositions comprising an effective amount of a Heterocyclic Compound, as well as methods of preventing and/or treating cryptosporidiosis and/or inhibiting a parasite or parasitic activity.

Abstract: Provided herein are compounds and compositions thereof for modulating IRAK4. In some embodiments, the compounds and compositions are provided for treatment of inflammatory or autoimmune diseases.

Abstract: Provided herein are 4-aminoisoindoline-1,3-dione compounds having the following structure: wherein R, Ring A, and n are as defined herein, compositions comprising an effective amount of a 4-aminoisoindoline-1,3-dione compound, and methods for treating or preventing disorders.

Abstract: The present disclosure relates to methods of using antibody conjugates with binding specificity for BCMA (BCMA) and its isoforms and homologs in combination with a gamma secretase inhibitor (GSI), such as in therapeutic methods. Also provided are pharmaceutical compositions and kits comprising the antibody conjugates and GSI.

Abstract: Provided herein are compounds and compositions thereof for modulating S1P5. In some embodiments, the compounds and compositions are provided for treatment of neurological diseases.

Abstract: Provided are processes for the preparation of (S)-tert-butyl 4,5-diamino-5-oxopentanoate, or a salt, solvate, hydrate, enantiomer, mixture of enantiomers, or isotopologue thereof. Also provided are solid forms of various intermediates and products obtained from the processes.

Abstract: Provided herein are lyophilized formulations of 2-(4-chlorophenyl)-N-((2-(2,6-dioxopiperidin-3-yl)-1-oxoisoindolin-5-yl)methyl)-2,2-difluoroacetamide or a stereoisomer or mixture of stereoisomers, pharmaceutically acceptable salt, tautomer, prodrug, solvate, hydrate, co-crystal, clathrate, or polymorph thereof. Methods of using the formulations and dosage forms for treating, managing, and/or preventing cancer are also provided herein.

Abstract: Provided herein are compounds and compositions thereof for modulating BCL6. In some embodiments, the compounds and compositions are provided for treatment of cancer or an autoimmune disease.

Abstract: Provided herein are uses of chimeric antigen receptors (CARs) for treating a cancer (such as B cell related cancer, e.g., multiple myeloma).

Abstract: Provided herein are piperidine dione compounds having the following structure: wherein RN, R1, R2, R3, R4, L, V, m, and n are as defined herein, compositions comprising an effective amount of a piperidine dione compound, and methods for treating or preventing an androgen receptor mediated disease.

Abstract: Provided herein is a crystalline 4-amino-2-(2,6-dioxopiperidine-3-yl)isoindoline-1,3-dione hemihydrate. Pharmaceutical compositions comprising the crystalline 4-amino-2-(2,6-dioxopiperidine-3-yl)isoindoline-1,3-dione hemihydrate are also disclosed.

Abstract: Provided herein are uses of chimeric antigen receptors (CARs) for treating a tumor or a cancer (such as B cell related cancer, e.g., multiple myeloma). In addition, an optimal washout period for commencing a therapy for the treatment of a condition in a subject after a prior exposure can be determined by receiving, for each of a plurality of subjects, prior treatment history data. Left-censored data can then be derived from the prior treatment history data for each of the subjects that includes a washout period and event or censor. A time scale of the left-censored treatment data is then inverted to result in right-censored treatment data. The right-censored treatment data is then applied to a time-to-event (TTE) model that associates one or more variables of interest with a time since exposure to the prior exposure. A maximally selected log-rank statistic across a plurality of cutoffs within a pre-defined percentile range is computed for continuous variables within the one or more variables of interest.

Abstract: Provided herein are methods of generating T cells, e.g., cytotoxic T lymphocytes starting from peripheral blood mononuclear cells without a separate step of generating and isolating antigen-presenting cells, and with a single round of antigen stimulation. Also provided herein are methods of using said cytotoxic T lymphocytes, for example, to treat cancer and/or viral infection.

Abstract: Provided herein are methods of treating diseases associated with vascular calcification and/or cardiovascular disease in a subject by using the level of a biomarker, in particular, snail homolog 1 (Snai1), phosphosmad2, phosphosmad3, urinary protein, dickkopf homolog 1 (Dkk1), collagen type 1 alpha 1 (Col1a1), activin (e.g., free activin), runt-related transcription factor 2 (Runx2), alkaline phosphatase (Alp), bone-specific alkaline phosphatase (BSAP), C-terminal type 1 collagen telopeptide (CTX), osterix, Klotho, alpha-smooth muscle actin (alpha-SMA), myocardin (MYOCD), activin receptor type 2A (ActRIIA), axis inhibition protein 2 (Axin2), and/or smooth muscle protein 22-alpha (Sm22-alpha), as an indicator(s) of responsiveness of the subject to the treatment, efficacy of the treatment, or appropriate dosage for the treatment with an activin type II receptor signaling inhibitor.

Abstract: Provided herein are cells, e.g., T cells expressing artificial cell death polypeptides that cause death of a cell, e.g., cells (e.g., T lymphocytes) expressing the cell death polypeptide, when the cell death polypeptide is multimerized or dimerized. Also provided herein is use of such cells, e.g., T lymphocytes, to treat diseases such as cancer.

Abstract: Provided herein are compositions, methods and uses involving antibodies that specifically bind to human CD47. Also provided are uses and methods, such as therapeutic methods, diagnostic methods, and methods of making such antibodies.

Abstract: Provided herein are methods and intermediates for making (1R, 2R, 5R)-5-amino-2-methylcyclohexanol hydrochloride, which are useful for the preparation of compounds useful for the treatment of a disease, disorder, or condition associated with the JNK pathway.