Title:
ANTISENSE OLIGONUCLEOTIDES TARGETING ATXN3
Document Type and Number:
WIPO Patent Application WO/2022/117747
Kind Code:
A3
Abstract:
The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia.
Inventors:
FUNDER ERIK (DK)
HUDLEBUSCH HEIDI RYE (DK)
PEDERSEN LYKKE (DK)
SONDERGAARD CHRISTOFFER (DK)
STEPHAN ALEXANDER HERBERT (CH)
HUDLEBUSCH HEIDI RYE (DK)
PEDERSEN LYKKE (DK)
SONDERGAARD CHRISTOFFER (DK)
STEPHAN ALEXANDER HERBERT (CH)
Application Number:
PCT/EP2021/084018
Publication Date:
July 28, 2022
Filing Date:
December 02, 2021
Export Citation:
Assignee:
HOFFMANN LA ROCHE (US)
HOFFMANN LA ROCHE (US)
HOFFMANN LA ROCHE (US)
International Classes:
C12N15/113; A61K31/712
Domestic Patent References:
| WO2019217708A1 | 2019-11-14 | |||
| WO2016127002A1 | 2016-08-11 | |||
| WO2013138353A2 | 2013-09-19 | |||
| WO2018089805A1 | 2018-05-17 | |||
| WO2020245233A1 | 2020-12-10 |
Other References:
LAUREN R. MOORE ET AL: "Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models", MOLECULAR THERAPY-NUCLEIC ACIDS, vol. 7, 25 April 2017 (2017-04-25), US, pages 200 - 210, XP055674189, ISSN: 2162-2531, DOI: 10.1016/j.omtn.2017.04.005
ELENI KOURKOUTA ET AL: "Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide", MOLECULAR THERAPY-NUCLEIC ACIDS, vol. 17, 5 August 2019 (2019-08-05), US, pages 601 - 614, XP055712431, ISSN: 2162-2531, DOI: 10.1016/j.omtn.2019.07.004
NEVES-CARVALHO ANDREIA ET AL: "Polyglutamine spinocerebellar ataxias: emerging therapeutic targets", EXPERT OPINION ON THERAPEUTIC TARGETS, vol. 24, no. 11, 10 October 2020 (2020-10-10), UK, pages 1099 - 1119, XP055891636, ISSN: 1472-8222, Retrieved from the Internet DOI: 10.1080/14728222.2020.1827394
ELENI KOURKOUTA ET AL: "Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide", MOLECULAR THERAPY-NUCLEIC ACIDS, vol. 17, 5 August 2019 (2019-08-05), US, pages 601 - 614, XP055712431, ISSN: 2162-2531, DOI: 10.1016/j.omtn.2019.07.004
NEVES-CARVALHO ANDREIA ET AL: "Polyglutamine spinocerebellar ataxias: emerging therapeutic targets", EXPERT OPINION ON THERAPEUTIC TARGETS, vol. 24, no. 11, 10 October 2020 (2020-10-10), UK, pages 1099 - 1119, XP055891636, ISSN: 1472-8222, Retrieved from the Internet
Attorney, Agent or Firm:
RODRIGUEZ, Lorena (CH)
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