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Factor VIII

Factor VIII-related patent applications - as published by the U.S. Patent and Trademark Office (USPTO).


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An improved expression cassette for packaging and expression of variant factor viii for the treatment ...
The Children's Hospital Of Philadelphia
August 17, 2017 - N°20170233456

Factor viii variants and methods of use thereof are disclosed. In particular embodiments, factor viii variants are expressed more efficiently by cells over wild-type factor viii proteins, are secreted at increased levels by cells over wild-type factor viii proteins, exhibit enhanced activity over wild-type factor viii proteins and are packaged more efficiently into viral vectors.
Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
Baxalta Gmbh
August 17, 2017 - N°20170233455

The present disclosure provides, among other aspects, codon-altered polynucleotides encoding factor viii variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia a.
Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
Baxalta Gmbh
August 10, 2017 - N°20170226188

The present disclosure provides, among other aspects, codon-altered polynucleotides encoding factor viii variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia a.
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Improved factor viii preparations suitable for therapeutic use and processes to obtain these
Csl Behring Gmbh
July 27, 2017 - N°20170209546

The present invention relates to pharmaceutical preparations comprising two-chain factor viii that are essentially free of factor viii-light chains (fviii-lcs) which are not associated with factor viii-heavy chains (fviii-hcs) having higher purity and which are less immunogenic. The invention also relates to methods to test the suitability of a pharmaceutical factor viii preparation and to methods to reduce in pharmaceutical ...
Protecting compositions for recombinantly produced factor viii
Octapharma Ag
July 06, 2017 - N°20170189494

A histidine-free composition comprising: a high purity factor viii (r-factor viii); arginine and/or sucrose; a surface-active agent to prevent or at least inhibit surface adsorption of factor viii; an amount of calcium chloride for specific stabilization of factor viii.
Factor viii polymer conjugates
Baxalta Gmbh
June 08, 2017 - N°20170157259

The invention is a proteinaceous construct comprising a factor viii molecule which is conjugated to a water-soluble polymer via carbohydrate moieties of factor viii, and methods of preparing same.
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Antibody substituting for function of blood coagulation factor viii
Csl Limited
May 25, 2017 - N°20170145111

The present inventors produced a variety of bispecific antibodies that specifically bind to both f. Ix/f. Ixa and f. X, and functionally substitute for f. Viiia, i. E., have a cofactor function to promote f. X activation via f. Ixa. Among these antibodies, the antibody a44/b26 reduced coagulation time by 50 seconds or more as compared to that observed ...
Method of purifying therapeutic proteins
Csl Limited
May 25, 2017 - N°20170143805

Also provided are solutions and pharmaceutical formulations comprising the fibrinogen and/or factor viii and/or vwf recovered by such methods, and uses thereof.
Liver-specific constructs, factor viii expression cassettes and methods of use thereof
Sangamo Biosciences, Inc.
May 04, 2017 - N°20170119906

Described herein are constructs used for liver-specific expression of a transgene.
Method of administering porcine b-domainless fviii
Baxalta Incorporated
May 04, 2017 - N°20170119857

The present invention provides a method of administering porcine b-domainless factor viii (obi-1) to a patient having factor viii deficiency to provide more rapid and effective protection against bleeding episodes, compared to formerly available methods, or to provide more effective protection to such patients during non-bleeding periods. This invention is based on the discovery that the recombinant b-domainless porcine fviii, ...
Adeno-associated virus factor viii vectors
Biomarin Pharmaceutical Inc.
April 06, 2017 - N°20170095538

The invention provides improved adeno-associated virus (aav) factor viii (fviii) vectors, including aav fviii vectors that produce a functional factor viii polypeptide and aav fviii vectors with high expression activity.
Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
Biomarin Pharmaceutical Inc.
March 30, 2017 - N°20170087219

The invention provides adeno-associated virus (aav) factor viii (fviii)-encoding/expressing vectors and virus, including aav fviii vectors with high expression activity and aav fviii vectors that express full-length or truncated functional fviii protein. The invention also relates to methods of making the herein described aav fviii vectors, recombinant aav fviii virus particles comprising or expressing such vectors, associated pharmaceutical ...
Factor viii formulations
University Of Connecticut
March 30, 2017 - N°20170087218

A factor viii (fviii) composition formulated such that nacl is not present in the final formulation or is present in trace amounts, which allows for a concomitant reduction in the lyophilization cycle time and increased stability of the lyophilized fviii.
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Factor viii chimeric proteins and uses thereof
Biogen Ma Inc.
March 16, 2017 - N°20170073393

The present invention provides a chimeric protein comprising a first polypeptide which comprises a fviii protein and a first ig constant region or a portion thereof and a second polypeptide which comprises a vwf protein comprising the d′ domain and d3 domain of vwf, a xten sequence having less than 288 amino acids in length, and a second ig constant ...
Enhancement of recombinant protein expression with copper
Advantech Bioscience Farmacêutical Ltda
March 09, 2017 - N°20170067013

The present invention provides a novel use of copper (cupric ion) for improved cell expression of recombinant proteins, particularly coagulation proteins such as recombinant factor viii, b domain deleted recombinant factor viii, recombinant factor ix and rfvii or rfviia. The use of such cell culture supplement results in higher productivity and robustness of the manufacturing process. This invention results in ...
Factor viii variants having a decreased cellular uptake
Sanquin Blood Supply Foundation
March 09, 2017 - N°20170066813

The present invention relates to modified coagulation factors. In particular, the present invention relates to modied factor viii molecules having decreased cellular uptake.
Stable factor viii formulations with low sugar-glycine
Advantech Bioscience Farmacêutica Ltda
March 09, 2017 - N°20170065683

The present invention provides a novel albumin-free preparation of factor viii has a high amount of salt and a low concentration of sugar and of glycine. The high salt provides stability and an elegant cake structure as the major crystalline component. The sugar and glycine provide a complex amorphous matrix together with some amorphous non-crystallized salt for stabilization of factor ...
Factor viii b cell epitope variants having reduced immunogenicity
Bloodworks
February 23, 2017 - N°20170051041

Provided herein are methods and compositions for preventing or reducing an initial immune response to factor viii in patients suffering from hemophilia a and for reducing the intensity of the immune response in patients having pre-formed inhibitor antibodies against factor viii.
Factor viii sequences
St. Jude Children's Research Hospital
February 23, 2017 - N°20170049859

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 ...
Nucleic acid encoding bispecific antibodies
Chugai Seiyaku Kabushiki Kaisha
January 26, 2017 - N°20170022293

Various bispecific antibodies that specifically bind to both blood coagulation factor ix/activated blood coagulation factor ix and blood coagulation factor x and functionally substitute for the cofactor function of blood coagulation factor viii, that is, the function to promote activation of blood coagulation factor x by activated blood coagulation factor ix, were produced. From these antibodies, multispecific antigen-binding molecules ...
Factor viii molecules with reduced vwf binding
Novo Nordisk A/s
January 26, 2017 - N°20170020992

The present invention relates to a recombinant factor viii molecule, wherein said molecule has reduced vwf binding capacity, and wherein said molecule is covalently conjugated with at least one side group.
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