Abstract: The present invention belongs to the field of biotechnology, recombinant protein production, molecular biology, microbiology and microbial genetics. It provides a modified eukaryotic cell that is modified to the effect that the modified eukaryotic cell is not able to provide an SSN6-like related protein or an SSN6-like protein that exerts its wildtype function and/or wildtype activity, the amount of SSN6-like related protein or of SSN6-like protein being present in the modified eukaryotic cell differs from the amount of SSN6-like related protein or of SSN6-like protein being present in its wildtype form, and/or essentially no SSN6-like related protein or SSN6-like protein is present in the modified cell. Additionally, the present invention provides a polynucleotide sequence comprising a modified ssn6-like related gene or modified ssn6-like gene, and a vector comprising said polynucleoptide.

Abstract: The present invention provides a compound of Formula (I) or a pharmaceutically acceptable salt thereof; (I) a method for manufacturing the compounds of the invention, and its therapeutic uses. The present invention further provides a combination of pharmacologically active agents and a pharmaceutical composition.

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: A dip tube (10) for extracting a fluid (102) from a bottle (104) comprises an adapter (12) configured to be at least partially inserted into a neck (106) of the bottle (104) and being provided with a connecting port (38) which is configured to be connected to a suction device (118), an elongated hollow withdrawal tube (14) which has a cross-sectional area that is smaller than a cross-sectional area of the adapter (12) and which is configured to protrude from the adapter (12) into a fluid receiving space (108) of the bottle (104), when the adapter (12) is inserted in the bottle neck (106), and which is arranged in fluid communication with the connecting port (38) of the adapter (12), and a sealing disc (52) extending from an outer circumferential surface (54) of the withdrawal tube (14) and being configured to abut against an inner surface (128) of the bottle (104) so as to seal the fluid receiving space (108) of the bottle (104).

Abstract: The invention relates to a Bcl-2 inhibitor for use in the treatment of a Bcl-2 mediated cancer carrying at least 1, 2, 3, 4, 5 or all of the following mutations: (i) Gly101Val; (ii) Asp103Tyr; (iii) Asp103Val; (iv) Asp103Glu; (v) Arg129Leu and (vi) Ala113Gly; wherein the Bcl-2 inhibitor is N-(4-hydroxyphenyl)-3-{6-[((3S)-3-(4-morpholinylmethyl)-3,4-dihydro-2(1H)-isoquinolinyl)carbonyl]-1,3-benzodioxol-5-yl}-N-phenyl-5,6,7,8-tetrahydro-1-indolizine carboxamide (Compound A) or 5-(5-chloro-2-{[(3S)-3-(morpholin-4-ylmethyl)-3,4-dihydroisoquinolin-2(1H)-yl]carbonyl}phenyl)-N-(5-cyano-1,2-dimethyl-1H-pyrrol-3-yl)-N-(4-hydroxyphenyl)-1,2-dimethyl-iH-pyrrole-3-carboxamide (Compound B), or a pharmaceutically acceptable salt thereof.

Abstract: This invention provides messenger RNA (mRNA) molecules comprising an open reading frame that encodes a protein of interest, wherein said modified RNA comprises a modified nucleoside selected from the group consisting of: (I), (II), and (III), gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of mRNA molecules.

Abstract: The invention relates to the use of a H3R inverse agonist for the treatment of shift work disorder.

Abstract: In one aspect, compounds of Formula AA, or a pharmaceutically acceptable salt thereof, are featured. The variables shown in Formula AA are as defined in the claims. The compounds of formula AA are NLRP3 activity modulators and, as such, can be used in the treatment of metabolic disorders (e.g. Type 2 diabetes, atherosclerosis, obesity or gout), a disease of the central nervous system (e.g. Alzheimer's disease, multiple sclerosis, Amyotrophic Lateral Sclerosis or Parkinson's disease), lung disease (e.g. asthma, COPD or pulmonary idiopathic fibrosis), liver disease (e.g. NASH syndrome, viral hepatitis or cirrhosis), pancreatic disease (e.g. acute pancreatitis or chronic pancreatitis), kidney disease (e.g. acute kidney injury or chronic kidney injury), intestinal disease (e.g. Crohn's disease or Ulcerative Colitis), skin disease (e.g. psoriasis), musculoskeletal disease (e.g. scleroderma), a vessel disorder (e.g. giant cell arteritis), a disorder of the bones (e.g.

Abstract: The present invention provides new protease resistant polypeptides, as well as compositions and methods for treating, ameliorating or preventing conditions related to joint damage, including acute joint injury and arthritis.

Abstract: The present invention provides antibody drug conjugates, wherein an antibody or antibody fragment that specifically binds to human cKIT is linked to a drug moiety, optionally through a linker. The present invention further provides pharmaceutical compositions comprising the antibody drug conjugates; and methods of making and using such pharmaceutical compositions for ablating hematopoietic stem cells in a patient in need thereof.

Abstract: The invention provides methods for predicting therapeutic responsiveness of a subject suffering from cognitive impairments or dysfunctions, psychotic and/or neurodegenerative disorders to an alpha 7 nicotinic acetylcholine receptor activator treatment.

Abstract: The present invention relates to myostatin antagonists, for the treatment of cancer cachexia, and cancer cachexia due to chemotherapeutic treatment. In particular, the myostatin antagonist bimagrumab was found to be beneficial in the treatment of cancer cachexia by reducing body weight loss. The present invention also relates to combinations and uses of a myostatin antagonist and an mTOR inhibitor for treating cancer cachexia by reducing, maintaining or increasing body weight loss or for use in treating age-related conditions.

Abstract: A platform and method for enabling collaboration on data analysis of life sciences data across disparate databases are disclosed. The collaboration platform may allow for performing exploratory analysis for drug discovery and development. The collaboration platform may include a search and graph module for generating a user project and determining and displaying one or more matching data assets and one or more potential collaborators; a collaboration module for coordinating a collaboration between the user and one or more selected collaborators; a data management module for receiving a schema for one or more producer projects, receiving data from the one or more selected data assets, and ingesting the received data using common standards and an ontology; and an insight application for generating disease specific inferences relating to a scientific question using the ingested received data, and receiving a feedback from the user and/or the selected collaborators to improve the search and graph module.

Abstract: A method for preparing purified yeast is disclosed, where the squalene source is a yeast that hyperproduces squalene. The squalene is useful for pharmaceutical purposes. For instance, it can be used to prepare an oil-in-water emulsion, and the emulsion is particularly suitable for use as an immmological adjuvant.

Abstract: The disclosure is directed to methods, treatment regimens, uses, kits and therapies for treating Generalized Pustular Psoriasis (GPP). These methods, treatment regimens, uses, kits and therapies utilize, inter cilia, administration of an IL-17 antagonist, e.g., an IL-17 antibody, such as secukinumab. Additionally disclosed are improved methods for treating plaque-type psoriasis that utilize up-titration and down-titration of the IL-17 antagonist, e.g., an IL-17 antibody, such as secukinumab, as well as modification of dose frequency. Further disclosed are methods of treating palmoplantar pustular psoriasis using the disclosed IL-17 antagonists, e.g., IL-17 antibodies, such as secukinumab.

Abstract: The present disclosure relates to a system (100) for use of a pharmaceutical product. The system comprises a container (102) accommodating a pharmaceutical product, the container (102) comprising a wireless communication unit (112) and a memory (116) which stores pairing-specific information for one or more medical devices (104), and a medical device (104) enabled for wireless communication with the wireless communication unit (112) of the container (102) and configured to read the pairing-specific information from the memory (116) of the container (102) to verify whether the container (102) is permitted to be used by the medical device (104).

Abstract: The invention provides methods and compositions for the treatment of cartilage damage or arthritis by administration of a protease-resistant ANGPTL3 polypeptide with chondrogenic activity.

Abstract: The invention provides methods of making immune effector cells (e.g., T cells, NK cells) that can be engineered to express a chimeric antigen receptor (CAR), and compositions and reaction mixtures comprising the same.

Abstract: The invention relates, in part, to systems and methods for scoring a sample containing tumor tissue from a cancer patient. The score is representative of a nearness between at least one pair of cells, a first member of the least one pair of cells expressing a first biomarker and a second member of the at least one pair of cells expressing a second biomarker that is different from the first biomarker. The score obtained from these methods can be indicative of a likelihood that a patient may respond positively to immunotherapy.

Abstract: Disclosed is a combination comprising an immunomodulator and a second therapeutic agent for use in treating cancer, wherein the immunomodulator is an inhibitor of an immune checkpoint molecule or an activator of a costimulatory molecule, or a combination thereof; and the second therapeutic agent is chosen from one or more of: 1) a c-MET inhibitor; 2) a CDK4/6 inhibitor; 3) a PI3K inhibitor; 4) a BRAF inhibitor; 5) an FGFR inhibitor; 6) a MEK inhibitor, or 7) a BCR-ABL inhibitor. The combination therapies can be used to treat or prevent cancerous conditions and/or disorders.