Nucleic acid molecules encoding modified factor VIII proteins

US9447168B2 · US · B2

Patent metadata
FieldValue
Publication numberUS-9447168-B2
Application numberUS-201314407008-A
CountryUS
Kind codeB2
Filing dateJun 12, 2013
Priority dateJun 12, 2012
Publication dateSep 20, 2016
Grant dateSep 20, 2016

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  1. Title

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  2. Abstract

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  3. Assignees and inventors

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  4. Key dates

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  5. First independent claim

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Abstract

Official abstract text for this publication.

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 hemophilia, e.g. hemophilia A, and a method for the preparation of a parvoviral gene delivery vector.

First claim

Opening claim text (preview).

The invention claimed is: 1. An isolated nucleic acid molecule comprising a nucleotide sequence encoding a Factor VIII protein, wherein the Factor VIII protein B domain spacer is replaced and the B domain comprises the amino acid sequence of SEQ ID NO: 4, and wherein the Factor VIII protein participates in blood coagulation via the coagulation cascade. 2. The nucleic acid molecule of claim 1 , wherein the B domain of the Factor VIII protein is between 30 and 37 amino acids in length. 3. The nucleic acid molecule of claim 1 , wherein the B domain of the Factor VIII protein is 31 amino acids in length. 4. The nucleic acid molecule of claim 1 , wherein the portions of the nucleotide sequence encoding for the A1, A2, A3, C1 and C2 domains of the Factor VIII protein are codon optimised compared to the wild type sequence. 5. The nucleic acid molecule of claim 1 , wherein the nucleic acid molecule comprises the nucleotide sequence of SEQ ID NO: 3. 6. The nucleic acid molecule of claim 1 , wherein the nucleotide sequence encodes for a protein comprising the amino acid sequence of SEQ ID NO: 6. 7. A vector comprising a nucleic acid molecule of claim 1 . 8. A host cell comprising the vector of claim 7 .

Assignees

Inventors

Classifications

  • A61K38/37Primary

    Factors VIII · CPC title

  • Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy · CPC title

  • Animal expressing industrially exogenous proteins · CPC title

  • Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents · CPC title

  • C07K14/755Primary

    Factors VIII {, e.g. factor VIII C (AHF), factor VIII Ag (VWF)} · CPC title

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What does patent US9447168B2 cover?
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 a…
Who is the assignee on this patent?
Ucl Business Plc, St Jude Children'S Res Hospital
What technology area does this patent fall under?
Primary CPC classification A61K38/37. Mapped technology areas include Human Necessities.
When was this patent published?
Publication date Tue Sep 20 2016 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
What related patents are in patentsdb?
We list 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).