Abstract: Disclosed are compounds and pharmaceutically acceptable salts and stereoisomers thereof that are suitable for cellular labeling or the treatment of cancer. Also disclosed are pharmaceutical compositions containing same, and methods of making and using the compounds.

Abstract: The present invention relates to methods for treating a disease or disorder associated with EP300 dependency and elevated CRBN expression levels (e.g., cancer (e.g., neuroblastoma)).

Abstract: The present disclosure provides novel heteroaryl compounds of formula (IX). Such compounds are useful for the treatment of cancers.

Abstract: The present invention is generally directed to identify interacting cells in the tumor microenvironment and using the identified interactions to enhance anti-tumor immunity in cancer. Identified interactions can be modulated using therapeutic agents. Immune cells resistant to suppression can be used for adoptive cell transfer. The present invention is also generally directed to cell types and genes that are correlated to time of tumor growth and tumor size.

Abstract: In some embodiments, a mass spectrometry tag may comprise a linker region, a mass balance region, and a reporter region. The mass spectrometry tag may be configured to fragment in a mass spectrometer via an energy dependent process to produce multiple reporter molecules. For example, the reporter region of the tag may be configured to produce at least two reporter molecules via fragmentation. In some embodiments, one or more regions of the tag may comprise at least one heavy isotope. In some such embodiments, the ability to fragment into multiple reporter molecules as well as the placement and/or number of heavy isotope(s) allows the mass spectrometry tag to be distinguished from other similar mass spectrometry tags. In some such embodiments, the ability to distinguish between tags having the same or substantially similar total mass to charge ratio and reporter region mass may allow the system to have a greater multiplexing capacity than conventional systems.

Abstract: The present application is directed to bispecific polypeptides comprising a first domain binding an antigen on an antigen presenting cell (ARC) and a second domain binding an antigen on an immune cell expressing a chimeric antigen receptor (CAR). Nucleic acids, vectors and host cells used in producing the polypeptide of the invention are also disclosed. Compositions comprising the bispecific polypeptides and methods of treating cancer and stimulating activation and expansion of immune cells in vivo and in vitro are also disclosed.

Abstract: The disclosure provides methods of reprogramming polyclonal T cells to maintain long-term persistence. The disclosure further provides methods of treatment, such as adoptive T cell transfer therapies, that harness Tscms for development of tumor-reactive T cells. In some embodiments, the disclosure provides methods and compositions for positive modulation of the Tscm-producing phenotype, e.g., positive modulation of TCF7 expression. Positive modulation of TCF7 expression allows for maintenance of an increased T number of stem-like T cells capable of both self-renewal and generation of differentiated, cytolytic progeny.

Abstract: Presented are methods of assessing the teratogenicity of agents by measuring the degradation of SALL4, and related compounds with reduced teratogenicity. Provided herein is a method for assessing the teratogenicity of an agent comprising: contacting an agent with SALL4; and measuring levels of SALL4, wherein the agent is teratogenic if SALL4 levels are substantially reduced in the presence of the agent relative to in the absence of the agent.

Abstract: The present invention provides methods for screening and diagnosing prostate cancer based on a correlation between cancer cell growth, cancer lethality or recurrence and the expression level of ZFP36 or NEDD9 and PTEN in conjunction with ZFP36. The disclosure also provides methods for screening and diagnosing prostate cancer based on a correlation between cancer lethality and the genotype of rs1910301 SNP. Also provided are methods for treating prostate cancer comprising providing a treatment or monitoring based on the expression level of ZFP36, the expression level of NEDD9, and the expression level of ZFP36 in conjunction with PTEN levels or the genotype of rs1910301 SNP.

Abstract: The presently disclosed subject matter provides for antigen-recognizing receptors that specifically target uPAR and cells comprising such uPAR-targeted antigen-recognizing receptors. The presently disclosed subject matter further provides uses of the uPAR-targeted antigen-recognizing receptors for treatment.

Abstract: Described herein are compositions and methods of using single-cell RNA-sequencing to identify treatment resistance in patients with ovarian cancer. Also, described herein are compositions and methods for treatment targeting resistance in patients with ovarian cancer.

Abstract: Provided herein are STING and SPARCS genes as biomarkers for determining an effective therapy for treating cancer. Further provided are methods for treating cancer using said biomarkers.

Abstract: The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.

Abstract: The present invention provides compositions and methods for treating cancers with reduced or absent CDH1 using a RHOA dominant negative polypeptide or biologically active fragment thereof, and/or a nucleic acid encoding a RHOA dominant negative polypeptide or biologically active fragment thereof.

Abstract: Provided herein are nanoparticles which enhance the immunogenicity of antigens for use in Beta coronavirus (e.g., MERS-CoV, SARS-CoV-1, or SARS-CoV-2) vaccines, as well as immunogenic compositions comprising the antigen nanoparticles and additional adjuvants for further enhancing immunogenicity.

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 provides a novel anti-DLL3 antibody-pyrrolodiazepine derivative and a novel anti-DLL3 anti-body-pyrrolodiazepine derivative conjugate using the same.

Abstract: The disclosure relates to cell-permeable stabilized peptide modules and methods of use for e.g., cellular delivery of cargoes.

Abstract: The application relates to a compound having Formula X: which modulates the activity of HER2 and/or a mutant thereof, and/or EGFR and/or a mutant thereof, a pharmaceutical composition comprising the compound, and a method of treating or preventing a disease in which HER2 and/or a mutant thereof, and/or EGFR and/or a mutant thereof, plays a role.

Abstract: The present invention relates to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathies, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to the use of novel classes of compounds, i.e. inhibitors of RSK (p90S6K); inhibitors of p70S6K; and inhibitors of rps6, to treat ribosomal disorders and ribosomopathies. In some embodiments, the invention relates to the use of specific Chk2 inhibitors and to the use of specific phenothiazine derivatives to treat ribosomal disorders and ribosomopathies, e.g. DBA.