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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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Selective reactivation of genes on the inactive x chromosome
The General Hospital Corporation
November 16, 2017 - N°20170327823

Methods and compositions for selectively reactivating genes on the inactive x chromosome (xi) in a locus-specific manner, e. G., genes associated with x-linked diseases, e. G., rett syndrome, factor viii or ix deficiency, fragile x syndrome, duchenne muscular dystrophy, and pnh, in heterozygous females.
Factor viii polypeptide formulations
Bioverativ Therapeutics Inc.
October 05, 2017 - N°20170281734

The present invention provides a formulation of a factor viii polypeptide, e. G., fviii-fc, and methods of using the same. The fviii polypeptide can be a recombinant fviii protein, a short-acting fviii protein, or a long-acting fviii protein. The pharmaceutical formulation comprising a fviii polypeptide can be used for individual prophylaxis, weekly prophylaxis, episodic (on-demand) treatment, or perioperative management of ...
Antibody capable of neutralizing substance having activity alternative to function of coagulation factor viii (fviii)
Chugai Seiyaku Kabushiki Kaisha
September 28, 2017 - N°20170275376

The present inventors attempted to produce antibodies that neutralize the activity of a substance having an activity of functionally substituting for fviii to be used for the method of measuring the reactivity of fviii in the presence of a substance having an activity of functionally substituting for fviii. As a result, the inventors discovered that by using the produced antibodies, ...
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Systems for factor viii processing and methods thereof
Bioverativ Therapeutics Inc.
September 21, 2017 - N°20170267744

The present invention provides methods of reducing nonprocessed factor viii or a chimeric polypeptide comprising factor viii comprising co-transfecting in a host cell a polynucleotide encoding factor viii with a polynucleotide encoding a protein convertase, where the endogenous processing enzymes of the host cell are insufficient to convert all of the factor viii to its processed isoform; expressing a proprotein ...
Methods for treating a disease that develops or progresses as a result of decrease or ...
Chugai Seiyaku Kabushiki Kaisha
September 07, 2017 - N°20170253663

The inventors discovered that by administering a pharmaceutical composition comprising a bispecific antigen-binding molecule that recognizes blood coagulation factor ix and/or activated blood coagulation factor ix and blood coagulation factor x and/or activated blood coagulation factor x according to a given dosage regimen, diseases that develop and/or progress due to a decrease or deficiency in the activity ...
Factor viii formulation
Csl Limited
September 07, 2017 - N°20170252412

The invention relates to factor viii compositions and their use.
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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.
Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders
Spark Therapeutics, Inc.
August 03, 2017 - N°20170216408

Cpg reduced nucleic acid variants encoding fviii protein and methods of use thereof are disclosed. In particular embodiments, cpg reduced nucleic acid variants encoding fviii are expressed more efficiently by cells, are secreted at increased levels by cells over wild-type factor viii proteins, exhibit enhanced expression and/or activity over wild-type factor viii proteins or are packaged more efficiently into ...
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.
Modified von willebrand factor
Csl Limited
June 01, 2017 - N°20170152300

The present invention provides a modified polypeptide which binds factor viii. The modified polypeptide comprises a sequence as shown in seq id no:3 in which the sequence comprises at least a modification at position 1 or 3 such that the modified polypeptide binds to factor viii with an off rate at least 5 fold lower than a reference polypeptide comprising an unmodified seq ...
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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.
Stabilization of factor viii without calcium as an excipient
Advantech Bioscience FarmacÊutica Ltda
March 23, 2017 - N°20170080059

The present invention provides a novel albumin-free formulation of recombinant factor viii that does not require calcium ions as an added formulation excipient. The absence of calcium chloride in the formulation results in a number of improvements and benefits for a factor viii formulation. This invention allows a factor viii formulation that can be lyophilized more efficiently as the absence ...
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 ...
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