	US 12,084,673 B2
	Vectors for the treatment of Friedreich's ataxia
Antoni Matilla Dueñas, Badalona (ES); Ivelisse Sánchez Díaz, Badalona (ES); and Eudald Balagué Cabasés, Badalona (ES)
Assigned to FUNDACIÓ INSTITUT D'INVESTIGACIÓ EN CIENCIES DE LA SALUT GERMANS TRIAS I PUJOL, Badalona. (ES); and GENTEC, S.A., Barcelona (ES)
Appl. No. 16/757,263
Filed by FUNDACIÓ INSTITUT D'INVESTIGACIÓ EN CIÈNCIES DE LA SALUT GERMANS TRIAS I PUJOL, Badalona (ES); and GENTEC, S.A., Barcelona (ES)
PCT Filed Oct. 17, 2018, PCT No. PCT/EP2018/078384 § 371(c)(1), (2) Date Apr. 17, 2020, PCT Pub. No. WO2019/076973, PCT Pub. Date Apr. 25, 2019.
Claims priority of application No. 17382691 (EP), filed on Oct. 17, 2017.
Prior Publication US 2021/0189423 A1, Jun. 24, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/864 (2006.01); A61K 48/00 (2006.01); A61P 25/00 (2006.01); A61P 25/14 (2006.01); C12N 15/86 (2006.01)

CPC C12N 15/86 (2013.01) [A61K 48/005 (2013.01); A61K 48/0058 (2013.01); A61K 48/0066 (2013.01); A61P 25/00 (2018.01); A61P 25/14 (2018.01); C12N 2750/14143 (2013.01); C12N 2830/48 (2013.01)]

11 Claims

1. An adeno-associated virus (AAV) vector comprising a nucleic acid, wherein the nucleic acid comprises:

(i) a nucleic acid sequence encoding frataxin;

(ii) a phospho-glycerate-kinase (PGK) promoter consisting of SEQ ID NO:1; and

(iii) a woodchuck hepatitis virus posttranscriptional regulatory element (WPRE);

wherein (ii) and (iii) are operably linked to and regulate the expression of (i), wherein there is an operationally functional linker between (i) and (ii), wherein said linker consists of SEQ ID NO: 6, and wherein the AAV vector is an AAV serotype 9 vector.