Abstract: The present invention is directed to immune adjuvants containing IAP inhibitors, including Smac mimetics. The invention further provides pharmaceutical compositions and vaccines containing an IAP inhibitor and an antigen. Methods of enhancing an immune response by administration of an IAP inhibitor, methods of treating or preventing cancer, methods of treating or preventing infections, methods of treating autoimmune disorders, and methods of potentiating cytokine or antibody production are also provided.

Abstract: Compounds of formula (I), formula (I), processes for their production and their R use as pharmaceuticals.

Abstract: The present invention relates to methods for identifying Richter's transformation patients who benefit from PD-1 blockade.

Abstract: The application relates to a compound of Formula (I) which modulate the amount of STK4, a pharmaceutical composition comprising the compound, and a method of treating or preventing a disease or disorder associated with the modulation of STK4.

Abstract: The present invention provides methods for treating cancer using combinations of bromodomain and extra-terminal (BET) protein inhibitors and certain chemotherapeutic drugs.

Abstract: Provided according to an embodiment of the present invention is an antibody that binds specifically to B-cell maturation antigen (BCMA) and comprises a heavy chain variable domain (VH domain) composed of a sequence having a homology of 80% or higher with any one of the amino acid sequences of SEQ ID NOS: 1 to 20, or a fragment thereof.

Abstract: The present disclosure features methods and compositions for treating amyotrophic lateral sclerosis (ALS). The disclosed methods comprise administering to a subject having or suspected of having ALS a hematopoietic stem progenitor cell expressing at least one neuroprotective agent. The compositions disclosed comprise hematopoietic stem progenitor cells transduced to express a neuroprotective agent.

Abstract: Presently described are compositions and methods for performing genome-wide enrichment of DNA microsatellites (e.g., homopolymers) containing deletions and facilitating their detection via sequencing or other downstream methodologies. Additionally, disclosed herein are compositions and methods for selecting the genomic fraction containing poly-adenine/poly-thymidine homopolymer repeats and other A:T-rich sequences of low melting temperature from the genome, so that very little sequencing is required to detect the enriched homo-polymer deletions.

Abstract: Disclosed are bispecific compounds (degraders) that target CDK7 for degradation. Also disclosed are pharmaceutical compositions containing the degraders and methods of using the compounds to treat disease.

Abstract: The invention features methods for characterizing a cancer as sensitive or resistant to Bcl6 therapies, as well as compositions and methods for inducing the degradation or polymerization of a polypeptide of interest.

Abstract: The present invention provides dihydrooxydiazinone compounds of general formula (I): in which R1, R2, R3, and R4, are as defined herein, methods of preparing said compounds, intermediate compounds useful for preparing said compounds, pharmaceutical compositions and combinations comprising said compounds and the use of said compounds for manufacturing pharmaceutical compositions for the treatment or prophylaxis of diseases, in particular of hyperproliferative diseases, as a sole agent or in combination with other active ingredients.

Abstract: The present invention provides methods of increasing the efficiency of genome editing.

Abstract: The present invention is based, in part, on the discovery of monoclonal antibodies, and antigen-binding fragments thereof, that specifically bind to MICA/B ?3 domain, as well as immunoglobulins, polypeptides, nucleic acids thereof, and methods of using such antibodies for diagnostic, prognostic, and therapeutic purposes.

Abstract: The present disclosure provides nucleic acid-based nanoswitch catenanes and methods of use. A nanoswitch catenane may include a single-stranded nucleic acid comprising a first and second terminal domain linked to each other to form a host ring by one of a first, second or third switchable bridges, wherein the first switchable bridge is formed in the presence of a reaction agent through the reaction of two cognate functional groups, each linked to a terminal domain of the single-stranded nucleic acid, wherein the second switchable bridge is formed in the presence of a biomolecule of interest through binding of the bio-molecule of interest to two cognate antibodies, each linked to a terminal domain of the single stranded nucleic acid, and wherein the third switchable bridge is a link between two cognate functional groups that breaks in the presence of a dissociation agent. A nanoswitch catenane may also include a circular nucleic acid guest ring catenated with the host ring.

Abstract: The disclosure relates to methods for treating cancers (e.g., cancers having a BRCA1 and/or BRCA2 mutation(s)) by administering to the subject an effective amount of a ubiquitin-specific protease 1 (USP1) inhibitor.

Abstract: Provided are compositions for use in methods for treating diseases associated with expression of mesothelin comprising administering a cell that expresses a chimeric antigen receptor (CAR) specific to mesothelin in combination with a PD-L1 inhibitor.

Abstract: The present invention provides compounds of Formula (I), and pharmaceutically compositions thereof. Compounds of Formula (I) are binders of bromodomains and/or bromodomain-containing proteins (e.g., bromo and extra terminal (BET) proteins). Also provided are methods, uses, and kits using the compounds and pharmaceutical compositions for inhibiting the activity (e.g., increased activity) of bromodomains and/or bromodomain-containing proteins and for treating and/or preventing in a subject diseases associated with bromodomains or bromodomain-containing proteins (e.g., proliferative diseases, cardiovascular diseases, viral infections, fibrotic diseases, metabolic diseases, endocrine diseases, and radiation poisoning). The compounds, pharmaceutical compositions, and kits are also useful for male contraception.

Abstract: Disclosed are compounds that possess inhibitory activity against FAK. Also disclosed are pharmaceutical compositions containing the compounds and methods of using the compounds to treat cancer mediated by aberrant FAK activity.

Abstract: Provided herein are compounds of Formulae (I) and (II), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. The compounds described herein bind an E3 ubiquitin ligase (e.g., Cereblon) and induce degradation of a transcription factor (e.g., IKZF1, IKZF3). Also provided are pharmaceutical compositions comprising the compounds, and methods of treating and/or preventing diseases (e.g., proliferative diseases (e.g., cancers (e.g., carcinoma); leukemia, lung cancer, breast cancer, liver cancer, pancreatic cancer, gastric cancer, ovarian cancer, colon cancer, colorectal cancer, multiple myeloma, and acute myeloid leukemia (AML))). Further provided are methods of inducing the degradation of a transcription factor (e.g., IKZF1, IKZF3) by administering a compound or composition described herein to a subject and/or to a biological sample (e.g., cell or tissue).

Abstract: Disclosed are bispecific compounds (degraders) that target EP300/CBP for degradation. Also disclosed are pharmaceutical compositions containing the degraders and methods of using the compounds to treat disease.