Methods of delivering factor VIII encoding nucleic acid sequences

US10124041B2 · US · B2

Patent metadata
FieldValue
Publication numberUS-10124041-B2
Application numberUS-201615254984-A
CountryUS
Kind codeB2
Filing dateSep 1, 2016
Priority dateJun 12, 2012
Publication dateNov 13, 2018
Grant dateNov 13, 2018

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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 a factor VIII protein, wherein a B domain portion of the factor VIII protein is encoded by a nucleotides sequence between 90 and 111 nucleotides in length and has an amino acid sequence that is at least 85% identical to SEQ ID NO: 4 which comprises six asparagine residues. Also provided is a factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating Haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.

First claim

Opening claim text (preview).

The invention claimed is: 1. A method for administering to a subject a nucleic acid molecule encoding a Factor VIII protein, comprising: intravenously administering to the subject a nucleic acid molecule encoding a Factor VIII protein, wherein the Factor VIII protein B domain comprises a spacer that is replaced and wherein the B domain comprises the amino acid sequence set forth in SEQ ID NO:4. 2. The method of claim 1 , wherein the B domain of said Factor VIII protein is between 30 and 37 amino acids in length. 3. The method of claim 1 , wherein the B domain of said Factor VIII protein is 31 amino acids in length. 4. The method of claim 1 , wherein the B domain of said Factor VIII protein comprises the amino acid sequence of SEQ ID NO:5. 5. The method of claim 1 , wherein the B domain of said Factor VIII protein consists of the amino acid sequence of SEQ ID NO:5. 6. The method of claim 1 , wherein the Factor VIII protein encoded by the nucleic acid molecule comprises domains A1, A2, A3, C1 and C2, and wherein the nucleic acid molecule is codon optimised compared to a corresponding wild type sequence. 7. The method of claim 1 , wherein said Factor VIII protein comprises the amino acid sequence of SEQ ID NO:6. 8. The method of claim 1 , wherein said nucleic acid molecule encoding said Factor VIII protein comprises the nucleic acid sequence of SEQ ID NO:3.

Assignees

Inventors

Classifications

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

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

  • Murine · CPC title

  • Animal expressing industrially exogenous proteins · CPC title

  • Genetically modified vertebrates, e.g. transgenic · CPC title

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What does patent US10124041B2 cover?
There is provided a nucleic acid molecule comprising a nucleotide sequence encoding a factor VIII protein, wherein a B domain portion of the factor VIII protein is encoded by a nucleotides sequence between 90 and 111 nucleotides in length and has an amino acid sequence that is at least 85% identical to SEQ ID NO: 4 which comprises six asparagine residues. Also provided is a factor VIII protein,…
Who is the assignee on this patent?
Ucl Business Plc, St Jude Childrens 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 Nov 13 2018 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 3 related publications on this page (citations in our corpus or others sharing the same primary CPC).