The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Jul. 29, 2025

Filed:

Jul. 27, 2021
Applicant:

Ucl Business Ltd, London, GB;

Inventors:

Amit Nathwani, London, GB;

Deepak Raj, London, GB;

Assignee:

UCL Business LTD, London, GB;

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); A61K 38/47 (2006.01); A61P 3/00 (2006.01); C12N 9/40 (2006.01); C12N 15/113 (2010.01); C12N 15/55 (2006.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0058 (2013.01); A61K 38/47 (2013.01); A61P 3/00 (2018.01); C12N 9/2465 (2013.01); C12N 15/86 (2013.01); A01K 2217/075 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01); C12N 2800/22 (2013.01);
Abstract

There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional α-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.


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