71 results about "Haemophilia A" patented technology

In a method of clotting blood in which the blood exhibits a reduced tendency to clot and may be from a person undergoing an anticoagulant therapy or having type A or B hemophilia or von Willebrand disease, a therapeutically effective amount of a composition comprising clay as the active ingredient is administered to a wound from which the blood emanates. Upon contacting the blood, this clay, which may be kaolin, bentonite, or any type of layered clay, causes the blood to clot. In a method of arresting blood flowing from a wound, a therapeutically effective amount of a composition comprising clay as the active ingredient is administered to the bleeding wound. In this method, the blood has a reduced tendency to clot and may be from a person undergoing an anticoagulant therapy or having at least one of hemophilia A or B or von Willebrand disease.

The invention relates to adenoviral vectors. More particularly, this invention relates to recombinant high capacity adenoviral vectors which can be employed in the treatment of hemophilia A, as well as methods and process for their creation and use.

The present invention provides agents for treating blood coagulation abnormalities, which contain as an active ingredient a lentiviral vector carrying a blood coagulation factor gene operably linked to a promoter which induces platelet-specific expression. Agents for treating hemophilia A or hemophilia B are provided by application of the gene encoding Factor VIII or Factor IX. Blood coagulation abnormalities can be treated by gene therapy by infecting hematopoietic stem cells or such with the therapeutic agents of the present invention.

There is provided a nucleic acid molecule comprising a nucleotide sequence encoding for a functional factor VIII protein, wherein the portion of the nucleotide sequence encoding for the B domain of the factor VIII protein is between 90 and 111 nucleotides in length and encodes for an amino acid sequence comprising a sequence having at least 85% identity to SEQ ID NO: 4 and which comprises six asparagine residues. Also provided is a functional factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating haemophilia, e.g. haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.

The invention relates to a recombinant factor VIII that includes one or more mutations at an interface of A1 and C2 domains of recombinant factor VIII. The one or more mutations include substitution of one or more amino acid residues with either a cysteine or an amino acid residue having a higher hydrophobicity. This results in enhanced stability of factor VIII. Methods for making the recombinant factor VIII, pharmaceutical compositions containing the recombinant factor VIII, and use of the recombinant factor VIII for treating hemophilia A are also disclosed.

The present invention relates to a recombinant factor VIII that is characterized by one or more mutations within a region surrounding an activated protein C cleavage site, which one or more mutations result in a reduced rate of inactivation by activated protein C. Isolated nucleic acid molecules, recombinant expression vectors, and host cells suitable for expression of the recombinant factor VIII are also disclosed. The recombinant factor VIII can be used for the treatment of clotting disorders, such as hemophilia A.

The present invention provides immune conjugates for inducing antigen specific immune tolerance to coagulation Factor VIII. The immune conjugates contain a FVIII protein or antigenic fragment that is conjugated to a binding moiety for a sialic acid binding Ig-like lectin (Siglec) expressed on B cells. The invention also provides methods of using the FVIII immune conjugates to induce immune tolerance to FVIII in a subject. Additionally provided in the invention are methods for treating bleeding disorders such as hemophilia A via the use of the FVIII immune conjugates and an unconjugated FVIII with coagulating activity.

This invention relates to methods and compositions for suppressing an immune response to Factor VIII in subjects suffering from hemophilia A and having preformed inhibitor antibodies against Factor VIII, and compositions and methods that advantageously render subjects amenable to standard treatments for hemophilia A, including Factor VIII replacement therapy.

The present invention is related to a monoclonal anti-idiotypic antibody directed against a Factor VIII inhibitor antibody binding to the domain A2 of Factor VIII, and to a cell line producing this monoclonal anti-idiotypic antibody, to the use of this monoclonal anti-idiotypic antibody as drug, and more particularly, to its use for the manufacturing of a drug to be used for the treatment of haemophilia A.

For the first time, the present invention provides antibodies that enhance the generation of activated blood coagulation factor VIII. The antibodies enhance the cleavage of blood coagulation factor VIII at the Arg of position 372 and suppress the cleavage at the Arg of position 336 by recognizing and binding to the A2 domain of blood coagulation Factor VIII. Such antibodies are expected to be useful in preventing or treating diseases that develop or progress due to decrease or loss of the blood coagulation factor VIII activity, for example, hemophilia A, acquired hemophilia, and von Willebrand's disease.

The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention are genetically engineered to interact with factor VIII (FVIII) target pre-mRNA so as to result in correction of clotting FVIII genetic defects responsible for hemophilia A. The compositions of the invention further include recombinant vector systems capable of expressing the PTMs of the invention and cells expressing said PTMs. The methods of the invention encompass contacting the PTMs of the invention with a FVIII target pre-mRNA under conditions in which a portion of the PTM is trans-spliced to a portion of the target pre-mRNA to form a RNA molecule wherein the genetic defect in the FVIII gene has been corrected. The methods and compositions of the present invention can be used in gene therapy for correction of FVIII disorders such as hemophilia A.

The present invention relates to improved human FVIII variants having at least one substitution in the A2 and/or C2 domain. The present invention also relates to their uses in the treatment of hemophilia A, particularly in patients with inhibitors.

The invention relates to an anti-idiotypical antibody targeting an antibody inhibiting the human factor VIII, said inhibiting antibody targeting the C2 region of the human factor VIII, the variable region of each of the light chains thereof being encoded by a sequence of nucleic acids of which at least 70% is identical to the murine sequence of nucleic acids SEQ ID NO: 1, and the variable region of each of the heavy chains thereof being encoded by a sequence of nucleic acids of which at least 70% is identical to the murine sequence of nucleic acids SEQ ID NO: 2, the constant regions of the light chains and the heavy chains being constant regions from a non-murine species. The invention also relates to the use of said antibody for activating the FcγRIII receptors of cytotoxic immune cells, and to the production of a medicament especially for the treatment of haemophilia A.