10 results about "Factor IX" patented technology

To provide a method for treating reversible hemophilic arthropathy of a joint in a human having hemophilia.SOLUTION: Uses of a chimeric protein or composition comprising a coagulation factor and an Fc region are provided. The coagulation factor is selected from the group consisting of Factor VII (FVII), Factor VIIa (FVIIa), Factor VIII (FVIII), Factor IX (FIX), Factor X (FX), von Willebrand Factor (VWF), an antigen-binding portion thereof that specifically binds to FIX and FX, or any combination thereof.SELECTED DRAWING: None

This invention relates to Factor IX variant polypeptides for administration to soft tissues.

To provide nucleic acid sequences, expression vectors (e.g., vector genomes) and plasmids, compositions and viral vectors in which the nucleic acid encodes Factor IX (e.g. human Factor IX).SOLUTION: A nucleic acid sequence encodes human Factor IX protein, where the nucleic acid has a reduced number of CpG di-nucleotides compared to a wild-type sequence encoding human Factor IX.SELECTED DRAWING: None

The application provides a completely humanized anti-human blood coagulation factor IX monoclonal antibody or an antigen binding fragment thereof and application thereof. The antibody provided by the application has high affinity to human blood coagulation factor IX and significant inhibitory activity, and belongs to a neutralizing antibody, and has a good treatment prospect for thrombus diseases. The functional antibody is directly screened from blood plasma of hemophilia B patients by a mass spectrometry + de novo sequencing technology, and has the advantages of complete human origin, high safety and the like.

The invention provides a completely humanized anti-human coagulation factor IX monoclonal antibody or an antigen binding fragment thereof and application thereof. The antibody provided by the invention has high affinity and remarkable inhibitory activity on the human coagulation factor IX, belongs to a neutralizing antibody, and has a good treatment prospect on thrombosis diseases. The functional antibody is directly screened from the blood plasma of the hemophilia B patient through the'mass spectrum + de novo sequencing 'technology, and the method has the advantages of being completely humanized, high in safety and the like.

The present disclosure provides methods and compositions for culturing, engineering B-lineage cell populations that produce Factor IX (FIX), and administering engineered B-lineage cell populations.

This invention provides Factor IX variants, molecules comprising the variants, nucleic acids encoding the variants, compositions comprising the variants or the nucleic acids encoding the variants, and their use in methods for the modulation of hemostasis, for example in the prophylaxis or treatment of hemophilia B. The Factor IX variants have improved biological properties relative to other Factor IX variants and / or relative to wild-type Factor IX.

The present invention provides a pharmaceutical composition for treating bleeding disorders such as hemophilia (e.g., hemophilia B). [Solution] A pharmaceutical composition comprising a lentiviral vector for use in a method to reduce transduction into macrophages in a target that requires it, wherein the lentiviral vector comprises a specific nucleotide sequence encoding a polypeptide having factor IX (FIX) activity and a lipid coat containing the CD47 protein, and the method comprises 2 × 10 9 The pharmaceutical composition comprises administering a lentiviral vector to a subject in a dose of less than transduction units / kg (TU / kg), wherein the lentiviral vector is packaged in HEK293T cells overexpressing CD47 and contains a higher level of surface CD47 protein on its lipid coating than a control lentiviral vector produced in unmodified HEK293T cells.

A pharmaceutical composition comprising: a. transgenic Factor VII and b. a multispecific antibody directed against Factor IX and Factor X. The patent application contains 10 more claims.

The present disclosure provides lentiviral vectors comprising a nucleic acid sequence encoding a polypeptide with factor IX (FIX) activity, and methods of using such lentiviral vectors. The liver-targeted lentiviral vectors disclosed herein can be used for gene therapy, wherein the lentiviral gene delivery enables stable integration of the transgene expression cassette into the genome of targeted cells (e.g., hepatocytes) of pediatric (e.g., neonatal) or adult subjects, achieving an improvement in FIX expression at low lentiviral vector doses. The present disclosure also provides methods of treating bleeding disorders such as hemophilia (e.g., hemophilia B) comprising administering to a subject in need thereof a liver-targeted lentiviral vector comprising a nucleic acid sequence encoding a polypeptide with FIX activity sequence at low dosages.