Abstract: The present invention relates to compounds of formula (I), wherein R1 to R7, A1 and A2 are as described herein, and their pharmaceutically acceptable salt thereof, and compositions including the compounds and methods of using the compounds.

Abstract: Herein is reported a composition for the targeted delivery of large nucleic acids to the nucleus of a eukaryotic cell comprising non-covalent complexes of histones in form of assembled nucleosomes, a large nucleic acid, a hapten, and a bispecific binder that has a first binding specificity to the hapten and a second binding specificity to a cell-surface target present on the eukaryotic cell, wherein the histone and/or the nucleic acid is/are covalently bound/conjugated to the hapten, the histone and the large nucleic acid are associated (non-covalently) with each other/form a non-covalent complex, and the hapten and the bispecific binder are associated with each other/bound to each other by the first binding specificity of the bispecific binder.

Abstract: The present invention relates to compounds of formula (I), wherein R1 to R6, A1 and A2 are as described herein, and their pharmaceutically acceptable salt thereof, and compositions including the compounds and methods of using the compounds.

Abstract: Herein is reported a method for determining the introduction of a nucleic acid into the genome of a mammalian cell, whereby the mammalian cell comprises one or two transcriptionally active alleles of a DPH1, DPH2, DPH4 and/or DPH5 gene, comprising the steps of transfecting the mammalian cell with one or more plasmids comprising the nucleic acid to be introduced, and the elements required for gene editing of said DPH gene, cultivating the transfected cell in the presence of a DPH gene transcription sensitive toxin, and thereby determining the introduction of a nucleic acid into the genome of the mammalian cell if the transfected cells is viable in the presence of the toxin.

Abstract: Herein is reported an antibody binding to rabbit CD19 comprising (a) a HVR-H1 comprising the amino acid sequence of SEQ ID NO: 32 or 33 or 34, (b) a HVR-H2 comprising the amino acid sequence of SEQ ID NO: 35 or 36, (c) a HVR-H3 comprising the amino acid sequence of SEQ ID NO: 37, (d) a HVR-L1 comprising the amino acid sequence of SEQ ID NO: 38, (e) a HVR-L2 comprising the amino acid sequence of SEQ ID NO: 39, and (f) a HVR-L3 comprising the amino acid sequence of SEQ ID NO: 40, as well as methods of using the same, especially in the identification and selection of antibody producing rabbit B-cells.

Abstract: The present invention provides methods to prevent the formation of visible particles in aqueous protein formulations, as well as compositions and pharmaceutical products obtained with said method.

Abstract: Genotyping methods and compositions for selecting patients with cardiovascular disease who will benefit from treatment with HDL-raising or HDL mimicking agent, in particular with a CETP inhibitor/modulator.

Abstract: The present invention relates to a dosage and administration regimen of anti-C5 antibodies, particularly of the anti-C5 antibody Crovalimab, for use in a method of treating or preventing GBS in a subject. The dosage and treatment regimen of the present invention include the administration of an anti-C5 antibody, preferably of the anti-C5 antibody Crovalimab, with loading dose followed by the administration of (a) maintenance dose(s) of the anti-C5 antibody to the subject, wherein the initial administered loading dose is intravenously given to the subject and the maintenance doses are subcutaneously administered in a lower dosage as the intravenously administered loading dose.

Abstract: The present invention provides new bicyclic tetrahydroazepine derivatives having the general formula (I) wherein R1, R2 and R4 are as defined herein, compositions including the compounds, processes of manufacturing the compounds and methods of using the compounds.

Abstract: The present invention relates to a dosage and administration regimen of anti-C5 antibodies, particularly of the anti-C5 antibody Crovalimab, for use in a method of treating or preventing GBS in a subject. The dosage and treatment regimen of the present invention include the administration of an anti-C5 antibody, preferably of the anti-C5 antibody Crovalimab, with loading dose followed by the administration of (a) maintenance dose(s) of the anti-C5 antibody to the subject, wherein the initial administered loading dose is intravenously given to the subject and the maintenance doses are subcutaneously administered in a lower dosage as the intravenously administered loading dose.

Abstract: The present invention provides new bicyclic tetrahydrothiazepine derivatives having the general formula (I) wherein R1, R2 and R4 are as defined herein, compositions including the compounds, processes of manufacturing the compounds and methods of using the compounds.

Abstract: The invention relates to UBE3A protein targets and their usage as target engagement biomarkers for compounds that modulate ube3a expression.

Abstract: The invention provides novel compounds having the general formula: wherein R1, R2, R3, R4, R5, R6, X, Y, W and n are as described herein, compositions including the compounds and methods of using the compounds.

Abstract: The present invention relates to 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one for use in the treatment of spinal muscular atrophy (SMA), its pharmaceutical composition to be used in the treatment of SMA, its methods of treatment thereof.

Abstract: The present invention relates to compounds of formula (I), wherein R1 to R3 are as described herein, and their pharmaceutically acceptable salt thereof, and compositions including the compounds and methods of using the compounds.

Abstract: The invention relates to bispecific antibodies comprising a first antigen-binding domain that specifically binds to TfR and a second, and optionally a third, antigen-binding domain that specifically binds to PD1. The invention further relates to methods of producing these molecules, to methods of using the same, to pharmaceutical compositions thereof, and their use as medicaments for the treatment of cancer, of acute and chronic infections and of Graft-versus-host disease.

Abstract: The present disclosure relates to a computer-implemented method of a computer-implemented method of determining a patient's response to a treatment in multiple myeloma. The method comprises: providing results of a series of predefined consecutive tests on the patient, determining a response at time t as a function of a test result of the time t and a subsequent test result of a time t+1.

Abstract: The invention provides novel heterocyclic compounds having the general formula (I) or (II), and pharmaceutically acceptable salts thereof, wherein the variables are as described herein. Further provided are pharmaceutical compositions including the compounds, processes of manufacturing the compounds and methods of using the compounds as medicaments, in particular methods of using the compounds for the treatment or prevention of acute neurological disorders, chronic neurological disorders and/or cognitive disorders.

Abstract: The application relates to a compound of formula (I) containing a thiazole ring, an indazol ring and a 6,7-dihydro-5H-pyrrolo[1,2-c]imidazole ring system, to pharmaceutical compositions containing it and its medical use. The compounds are described as selective allosteric inhibitors of T790M/L858R, T790M/L858R/C797S, L858R, L858R/C797S containing EGFR mutants and thus useful for the treatment of cancer, in particular non-small cell lung cancer.