Abstract: The present invention relates to methods, treatment regimens, uses, kits and therapies for prevention of graft rejection in solid organ transplantation, particularly solid organ xenotransplantation, by administering an anti-CD40 antibody or a combination of an anti-CD40 antibody and an anti-C5 antibody.

Abstract: The invention provides compounds of Formula (I): as described herein, along with pharmaceutically acceptable salts, pharmaceutical compositions containing such compounds, and methods to use these compounds, salts and compositions for treating viral infections.

Abstract: The present invention relates to the novel use of certain piperidinyl-indole derivatives in the treatment of patients suffering from renal diseases or disorders, and in particular for the treatment of patients suffering from C3G (C3 glomerulopathy) and IgAN (IgA nephropathy).

Abstract: The disclosure provides 32-deoxo-rapamycin derivatives of Formula (I), or a pharmaceutically acceptable salt thereof, pharmaceutical compositions and pharmaceutical combinations comprising a compound of Formula (I), and methods of making same. Also provided herein are methods of using a compound of Formula (I) or a pharmaceutically acceptable salt thereof for treating the diseases and disorders described herein.

Abstract: The present invention relates to a method for measuring at least one inhalation flow feature in an inhaler, wherein a capsule containing a formulation is located in the inhaler, the method comprising the steps of sensing an impact feature relating to impacts of the capsule on the inhaler and correlating the impact feature to at least one inhalation flow feature. The present invention also relates to an inhaler adapted to aerosolize a formulation contained in a capsule, wherein the inhaler comprises a sensor for sensing an impact feature relating to impacts of the capsule on the inhaler and a processor for correlating the impact feature to at least one inhalation flow feature.

Abstract: The present invention provides compounds of formula I: or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein the variables are as defined herein. The present invention further provides pharmaceutical compositions comprising such compounds, and methods of using such compounds for treatment of joint damage or joint injury in a mammal, and for inducing differentiation of mesenchymal stem cells into chondrocytes.

Abstract: The present disclosure provides BCMA binding molecules that specifically bind to human BCMA, conjugates comprising the BCMA binding molecules, and pharmaceutical compositions comprising the BCMA binding molecules and the conjugates. The disclosure further provides methods of using the BCMA binding molecules to treat cancers that express cell surface BCMA. The disclosure yet further provides recombinant host cells engineered to express the BCMA binding molecules and methods of producing the BCMA binding molecules by culturing the host cells under conditions in which the BCMA binding molecules are expressed.

Abstract: A solid support for use in non-aqueous DNA-conjugated molecule reactions, wherein the support comprises a solid body formed from a plurality of polyethylene glycol units, wherein the solid body includes at least one cationic moiety.

Abstract: The present invention provides 5-hydroxy-7-oxabicyclo[2.2.1]heptane-2-carboxamide derivatives of Formula (1) wherein the variables are as defined herein. The present invention further provides pharmaceutical compositions comprising such compounds. The compounds are used for inducing chondrogenesis, in methods of treating or preventing joint damage, resulting from joint injury or arthritis, and for inducing hyaline cartilage production. The present description discloses the preparation of exemplary compounds as well as pharmacological data thereof (examples 1 to 89; tables 1 and 2). An exemplary compound is e.g. rac-(1R,2R,3S,4R,5S)—N-(2-fluoro-5-(trifluoromethyl)phenyl)-5-hydroxy-3-(3-(trifluoromethyl)phenyl)-7-oxabicyclo[2.2.1]heptane-2-carboxamide (example 1).

Abstract: The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.

Abstract: The present invention describes specific crystalline forms of N-[4-(Chlorodifluoromethoxy)phenyl]-6-[(3R)-3-hydroxypyrrolidin-1-yl]-5-(1H-pyrazol-5-yl)pyridine-3-carboxamide. The present invention further relates to methods for preparing said crystalline forms, pharmaceutical compositions comprising said crystalline forms, and methods of using said crystalline forms and pharmaceutical compositions to treat disease.

Abstract: Antibody molecules that specifically bind to LAG-3 and bispecific antibody molecules with a first binding specificity for LAG-3 and a second binding specificity for one of PD-1, TIM-3, CEACAM-1, CEACAM-5, PD-L1, or PD-L2, are disclosed. The anti-LAG-3 antibody molecules can be used to treat, prevent and/or diagnose cancerous or infectious disorders.

Abstract: The present invention describes specific crystalline forms of N-[4-(Chlorodifluoromethoxy)phenyl]-6-[(3R)-3-hydroxypyrrolidin-1-yl]-5-(1H-pyrazol-5-yl)pyridine-3-carboxamide. The present invention further relates to methods for preparing said crystalline forms, pharmaceutical compositions comprising said crystalline forms, and methods of using said crystalline forms and pharmaceutical compositions to treat disease.

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), compositions and reaction mixtures comprising the same, and methods of treatment using the same.

Abstract: The invention relates to substituted tetrahydro-pyrido-pyrimidine derivatives of the formula (I), wherein Y, R1, R2 and m are as defined in the description. Such compounds are suitable for the treatment of a disorder or disease which is mediated by the activity of the PI3K enzymes.

Abstract: The invention relates to an in vitro quantative cell-based assay that uses a primary mouse cell model system permissive to viral vector infection and a quantitative high content image-based system for determining potency of a transgene-expressing viral vector drug product for lot disposition.

Abstract: The present invention relates to compounds of formula (I) or a pharmaceutically acceptable salt form thereof, wherein the substituents are as defined in the specification; to intermediates in the preparation of the compounds, to pharmaceutical compositions comprising the compounds and to the use of the compounds in the treatment of disease.

Abstract: The present disclosure provides CD19 binding molecules that specifically bind to CD19, including monospecific, bispecific and trispecific binding molecules, conjugates comprising the CD19 binding molecules, and pharmaceutical compositions comprising the CD19 binding molecules and the conjugates. The disclosure further provides methods of using the C19 binding molecules to treat diseases and disorders associated with expression of CD19. The disclosure yet further provides recombinant host cells engineered to express the CD19 binding molecules and methods of producing the CD19 binding molecules by culturing the host cells under conditions in which the CD19 binding molecules are expressed.

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 invention provides compositions and methods for treating diseases associated with expression of BCMA. The invention also relates to chimeric antigen receptor (CAR) specific to BCMA vectors encoding the same, and recombinant T cells comprising the BCMA CAR. The invention also includes methods of administering a genetically modified T cell expressing a CAR that comprises a BCMA binding domain.