Abstract: The invention provides a syringe for use in an ophthalmic injection. The syringe comprises a body, a stopper and a plunger. The body comprises an outlet at an outlet end and the stopper is arranged within the body such that a front surface of the stopper and the body define a variable volume chamber from which a fluid can be expelled though the outlet. The plunger comprises a plunger contact surface at a first end and a rod extends between the plunger contact surface and a rear portion. The plunger contact surface is arranged to contact the stopper but not couple thereto, such that the plunger can be used to force the stopper towards the outlet end of the body, reducing the volume of the variable volume chamber, but not to move the stopper away from the outlet end.

Abstract: Provided herein are formulations, processes, solid forms and methods of use relating to (E)-1-(4-(1-(((4-cyclohexyl-3-(trifluoromethyl)benzyl)oxy)imino)ethyl)-2-ethylbenzyl)azetidine-3-carboxylic acid.

Abstract: The present invention provides anti-VEGF antibodies formulated as high concentration, aqueous pharmaceutical compositions, suitable for an injection, preferably an intravitreal injection. The aqueous pharmaceutical compositions are useful for delivery of a high concentration of the antibody active ingredient to a patient without high levels of antibody aggregation and without a high level of sub-visible particulate matter. An aqueous composition of the invention comprises an antibody having a concentration of at least 50 mg/ml. An aqueous pharmaceutical composition of the invention includes a sugar, a buffering agent, and a surfactant.

Abstract: A pharmaceutical container may include a cylindrical hollow container body (100) defining a hollow chamber (111) to receive a pharmaceutical liquid, the container body (100) including a portion on a sidewall (121) of the container body (100), wherein the portion has a longitudinal extension along the longitudinal axis of the container body (100) and a circumferential extension along the circumference of the container body (100), thereby forming a window (122) through the sidewall (121) of the container body (100) into the hollow chamber (111), the window (122) comprising two long edges (124) and two short edges (125), and a Near Field Communication (NFC) antenna (110) arranged on the outer surface (120) of the sidewall (121) of the container, the antenna (110) including one or a plurality of nested antenna windings (150), each of them surrounding an interior periphery (140) including a substantially longitudinal portion (141) arranged along the two long edges (124) of the window (122), and a substantially cir

Abstract: The present disclosure provides CD2 binding molecules that specifically bind to CD2, including monospecific, bispecific and trispecific binding molecules, conjugates comprising the CD2 binding molecules, and pharmaceutical compositions comprising the CD2 binding molecules and the conjugates. The disclosure further provides methods of using the CD2 binding molecules to modulate CD2 signaling in order to treat a variety of immune (e.g., autoimmune), inflammatory and proliferative disorders. The disclosure yet further provides recombinant host cells engineered to express the CD2 binding molecules and methods of producing the CD2 binding molecules by culturing the host cells under conditions in which the CD2 binding molecules are expressed.

Abstract: The present invention is directed to genome editing systems, reagents and methods for immunooncology.

Abstract: The present invention relates to a pharmaceutical combination comprising (a) a Raf inhibitor as defined herein, or a pharmaceutically acceptable salt thereof and (b) a MEK inhibitor, particularly trametinib, particularly for use in the treatment of a proliferative disease. This invention also relates to uses of such combination for preparation of a medicament for the treatment of a proliferative disease; methods of treating a proliferative disease in a subject in need thereof comprising administering to said subject a jointly therapeutically effective amount of said combination; use of such combination for the treatment of proliferative disease; pharmaceutical compositions comprising such combination and commercial packages thereto.

Abstract: The present disclosure provides recombinant adeno-associated virus (AAV) virions comprising: a) a variant capsid protein; and b) a heterologous nucleic acid comprising one or more nucleotide sequences encoding one or more heterologous gene products. The rAAV virions are useful for delivery of gene products to a retinal cell. The present disclosure provides methods of delivering a gene product to a retinal cell in an individual.

Abstract: The invention relates to 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 method for treating, preventing, inhibiting, ameliorating, or eradicating the pathology and/or symptomology of cryptosporidiosis by administering such a compound.

Abstract: A compound of Formula (I) is provided that has been shown to be useful for treating a disease caused by a viral infection: (I) wherein R1, R2, R3, A, L, m, n, p and q are as defined herein.

Abstract: Pulmonary hypertension is a progressive disease of various origins that is associated with vascular remodelling and results in right heart dysfunction. Accumulating evidence indicates important roles of immune cells and inflammatory chemokines in the pathogenesis and progression of pulmonary hypertension. We have identified CCL21 as anti-remodelling efficacy biomarker for pulmonary hypertension. CCL21 was found to be highly sensitive and specific in discriminating pulmonary hypertension patients from matched controls. CCL21 was upregulated in pulmonary hypertension and down-regulated with treatment with an anti-remodelling agent.

Abstract: The present disclosure relates to monoclonal antibodies and antigen binding fragments thereof that bind to human Factor XI and activated Factor XI (“Factor XIa”), and pharmaceutical compositions and methods of treatment comprising the same.

Abstract: The present invention relates to a combination treatment which comprises (a) at least one ERK inhibitor preferably Compound B as described herein, and (b) a c-RAF inhibitor or a pharmaceutically acceptable salt thereof, preferably Compound A, which may be combined into a single pharmaceutical composition or prepared for separate or sequential administration. It includes a c-RAF inhibitor and an ERK inhibitor prepared for simultaneous, separate or sequential administration for the treatment of a proliferative disease, particularly an advanced solid tumor that harbors a Mitogen-activated protein kinase (MAPK) alteration, and includes methods of using these compounds in combination as well as a commercial package comprising such combination.

Abstract: The present disclosure provides for IL2 engrafted into the CDR sequences of an antibody having preferred therapeutic profiles over molecules known and used in the clinic. In particular, the provided antibody cytokine engrafted protein compositions increase or maintain CD8+ T effector cells while reducing the activity of Treg cells. Additionally, provided compositions convey improved half-life, stability and producibility over recombinant human IL2 formulations such as Proleukin®.

Abstract: Disclosed herein are antibodies, antigen binding fragments thereof, and antibody drug conjugates thereof that bind human CD48. Also disclosed are pharmaceutical compositions comprising the antibodies, antigen binding fragments thereof, and antibody drug conjugates thereof; and methods of making and using such pharmaceutical compositions for treating cancer in a patient in need of treatment.

Abstract: Provided are compositions and methods for treating diseases associated with expression of mesothelin. Also provided are a chimeric antigen receptor (CAR) specific to mesothelin, vectors encoding the same, and recombinant T cells comprising the mesothelin CAR. Further provided are methods of administering a genetically modified T cell expressing a CAR that comprises a mesothelin binding domain.

Abstract: The invention provides compositions including a fusion polypeptide and methods for making a fusion polypeptide that includes a COF1/CRBN-binding polypeptide, COF2/CRBN-binding polypeptide, or COF3/CRBN-binding polypeptide and a heterologous polypeptide of interest.

Abstract: Provided herein are crystalline and amorphous forms of a compound having structural formula (1). Also provided are pharmaceutical compositions comprising the crystalline and amorphous forms, methods for their manufacture, and uses thereof for treating a variety of diseases, disorders or conditions, associated with potassium channels.

Abstract: This application relates to various crystalline forms of (S)-3-amino-4-(5-(4-((5-chloro-3-fluoropyridin-2-yl)oxy)phenyl)-2H-tetrazol-2-yl)butanoic acid in its free form, as well as compositions, method of making and methods of using the same. In some embodiments the crystalline forms also contain water (“hydrates”). These materials are useful in the treatment of diseases and disorders which are typically ameliorated by the inhibition of LTA4H. Such diseases and disorders may include inflammatory and autoimmune disorders and pulmonary and respiratory tract inflammation.

Abstract: The invention relates to bivalent bispecific monoclonal antibodies (bbmAb) or variants thereof, and methods of manufacturing such antibodies by co-expressing modified Fc-mutated derivatives of two different monoclonal antibodies in mammalian cell lines.