Patent classifications
C12Y304/23005
METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYLOIDOSIS
Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.
METHOD OF DETECTION OF ANALYTE ACTIVE FORMS AND DETERMINATION OF THE ABILITY OF SUBSTANCES TO BIND INTO ANALYTE ACTIVE SITES
A method for detection of active form of analytes in a sample and/or for determination of ability of tested substances to bind to the active site of these analytes has the following steps: a) analyte or group of analytes from the sample is immobilized on the surface of a solid carrier; b) analyte or group of analytes is incubated with a detection probe; c) then the solid carrier is washed to remove unbound detection probe; and subsequently, the amount of bound detection probe is determined.
METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYLOIDOSIS
Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.
ENGINEERED CELLS WITH MODIFIED HOST CELL PROTEIN PROFILES
Mammalian cell lines genetically engineered to have reduced or eliminated expression of specific host cell proteins, and methods for using the engineered mammalian cell lines for the production of recombinant proteins having low levels of residual host cell protein contamination.
TREATMENT OF NEURONAL CEROID LIPOFUSCINOSIS
The invention provides for novel treatments of lysosomal storage diseases, in particular of Neuronal Ceroid Lipofuscinosis (NCL) or synucleinopathy or a disease characterized by block of autophagic flow. The treatment comprises administration of human pro-Cathepsin, in particular human pro-Cathpsin D, B or L to patients in need thereof.
Conjugates for the treatment of cancer targeted at intracellular tumor-associated antigens
The present disclosure relates to conjugates, preferably, antibody-drug conjugates, directed against select non-transmembrane tumor antigens that are normally intracellular but can be secreted from cancer cells, such as human cathepsin D, and can be targeted in a way that enables the selective delivery of the conjugate to cancer cells. The design and mechanism of action disclosed enable the preferential delivery of the conjugate prodrug to cancer cells over normal cells for the purpose of selectively killing cancer cells. The uses of such conjugates for the treatment of cancer are described.
AGENT FOR ACTIVATING A NEURON
Developing an agent for activating a neuron which activates a brain neuron. The agent is suitable for promoting cell transplantation for a neural disease, survival of a transplanted cell, differentiation of the transplanted cell into a neuronal cell, and/or maturation of the neuron. The agent for activating the neuron contains a protein selected from the group consisting of Lysozyme (LYZ), Secreted phosphoprotein 1 (SPP1), Progranulin (PGrn), Cathepsin D (Ctsd), Cathepsin S (Ctss), Apolipoprotein D (Apod), Sparc, CD52, Macrophage expressed gene 1 (MPEG1), C-C motif chemokine ligand 2 (CCL2), Cystatin F (CST7), Ras family small GTPase 2 (RAC2), Serpin family A member 3 (SERPINA3), Anosmin 1 (ANOS1), Kallikrein-related peptidase 6 (KLK6), Alpha-2-macroglobulin (A2M), GM14295, Galectin 3-binding protein (LGALS3BP), Apolipoprotein C1 (APOC1), and orthologs thereof, or an active fragment thereof, or a nucleic acid encoding the protein or active fragment thereof, and an agent for assisting cell transplantation containing the agent.
REDUCTION OF LIPASE ACTIVITY IN PRODUCT FORMULATIONS
The invention relates a method for producing a stable recombinant protein, comprising growing a non-naturally occurring host cell in a culture medium to produce a recombinant protein, and making a composition comprising the recombinant protein and a polysorbate. The production of endogenous lipoprotein lipase by the host cell is reduced. The endogenous lipoprotein lipase is present in the composition in a small amount, and is capable of degrading the polysorbate. The invention also relates to the relevant host cells and compositions, and preparation thereof.
METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYLOIDOSIS
Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.
Treatment of synucleinopathies
This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.