Title:
ANTISENSE SEQUENCES FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS
Document Type and Number:
WIPO Patent Application WO/2021/205005
Kind Code:
A3
Abstract:
The present invention relates to antisense sequences, nucleic acid constructs and vectors comprising said antisense sequences, and their use for treating a C9orf72 hexanucleotide repeat expansion associated disease such as amyotrophic lateral sclerosis or frontotemporal dementia.
Inventors:
BIFERI MARIA-GRAZIA (FR)
CAPPELLA MARISA (FR)
BARKATS MARTINE (FR)
CAPPELLA MARISA (FR)
BARKATS MARTINE (FR)
Application Number:
PCT/EP2021/059313
Publication Date:
November 18, 2021
Filing Date:
April 09, 2021
Export Citation:
Assignee:
ASSOCIATION INST DE MYOLOGIE (FR)
INST NAT SANTE RECH MED (FR)
UNIV SORBONNE (FR)
INST NAT SANTE RECH MED (FR)
UNIV SORBONNE (FR)
International Classes:
C12N15/113; A61K31/7088; A61P21/00
Domestic Patent References:
| WO2016167780A1 | 2016-10-20 | |||
| WO2019032607A1 | 2019-02-14 | |||
| WO2015057727A1 | 2015-04-23 | |||
| WO2015054676A2 | 2015-04-16 | |||
| WO2014062736A1 | 2014-04-24 | |||
| WO2016024205A1 | 2016-02-18 | |||
| WO2016112132A1 | 2016-07-14 | |||
| WO2018064600A1 | 2018-04-05 | |||
| WO2019094694A1 | 2019-05-16 |
Other References:
GIULIETTA RIBOLDI ET AL: "Antisense Oligonucleotide Therapy for the Treatment of C9ORF72 ALS/FTD Diseases", MOLECULAR NEUROBIOLOGY, vol. 50, no. 3, 1 December 2014 (2014-12-01), US, pages 721 - 732, XP055508816, ISSN: 0893-7648, DOI: 10.1007/s12035-014-8724-7
JIANG JIE ET AL: "Pathogenic Mechanisms and Therapy Development for C9orf72 Amyotrophic Lateral Sclerosis/Frontotemporal Dementia", NEUROTHERAPEUTICS, ELSEVIER INC, US, vol. 16, no. 4, 1 October 2019 (2019-10-01), pages 1115 - 1132, XP036999711, ISSN: 1933-7213, [retrieved on 20191030], DOI: 10.1007/S13311-019-00797-2
MIS MARIA SARA ET AL: "Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders", MOLECULAR NEUROBIOLOGY, HUMANA PRESS, US, vol. 54, no. 6, 28 June 2016 (2016-06-28), pages 4466 - 4476, XP036276831, ISSN: 0893-7648, [retrieved on 20160628], DOI: 10.1007/S12035-016-9993-0
JIANG JIE ET AL: "Gain of Toxicity from ALS/FTD-Linked Repeat Expansions inC9ORF72Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs", NEURON, CELL PRESS, US, vol. 90, no. 3, 21 April 2016 (2016-04-21), pages 535 - 550, XP029531466, ISSN: 0896-6273, DOI: 10.1016/J.NEURON.2016.04.006
CAPPELLA M ET AL: "AAV-mediated expression of antisense oligonucleotides for the treatment of C9orf72-ALS", RVSV(M DELTA 51)-M3 IS AN EFFECTIVE AND SAFE ONCOLYTIC VIRUS FOR CANCER THERAPY; 27TH ANNUAL CONGRESS OF THE EUROPEAN SOCIETY OF GENE AND CELL THERAPY BRITISH SOCIETY FOR GENE THERAPY, ESGCT 2019 20191022 TO 20191025 BARCELONA,, vol. 30, no. 11, 1 November 2019 (2019-11-01), pages A98, XP009522981, ISSN: 1557-7422, DOI: 10.1089/HUM.2019.29095.ABSTRACTS
CHRISTOPHER J. DONNELLY ET AL: "RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention", NEURON, vol. 80, no. 2, 1 October 2013 (2013-10-01), US, pages 415 - 428, XP055649589, ISSN: 0896-6273, DOI: 10.1016/j.neuron.2013.10.015
CHRISTOPHER J. DONNELLY ET AL: "RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention. Supplemental Information", NEURON, 16 October 2013 (2013-10-16), XP055508871, Retrieved from the Internet [retrieved on 20180921]
C. LAGIER-TOURENNE ET AL: "Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES (PNAS), vol. 110, no. 47, 19 November 2013 (2013-11-19), US, pages E4530 - E4539, XP055228419, ISSN: 0027-8424, DOI: 10.1073/pnas.1318835110
JIANG JIE ET AL: "Pathogenic Mechanisms and Therapy Development for C9orf72 Amyotrophic Lateral Sclerosis/Frontotemporal Dementia", NEUROTHERAPEUTICS, ELSEVIER INC, US, vol. 16, no. 4, 1 October 2019 (2019-10-01), pages 1115 - 1132, XP036999711, ISSN: 1933-7213, [retrieved on 20191030], DOI: 10.1007/S13311-019-00797-2
MIS MARIA SARA ET AL: "Development of Therapeutics for C9ORF72 ALS/FTD-Related Disorders", MOLECULAR NEUROBIOLOGY, HUMANA PRESS, US, vol. 54, no. 6, 28 June 2016 (2016-06-28), pages 4466 - 4476, XP036276831, ISSN: 0893-7648, [retrieved on 20160628], DOI: 10.1007/S12035-016-9993-0
JIANG JIE ET AL: "Gain of Toxicity from ALS/FTD-Linked Repeat Expansions inC9ORF72Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs", NEURON, CELL PRESS, US, vol. 90, no. 3, 21 April 2016 (2016-04-21), pages 535 - 550, XP029531466, ISSN: 0896-6273, DOI: 10.1016/J.NEURON.2016.04.006
CAPPELLA M ET AL: "AAV-mediated expression of antisense oligonucleotides for the treatment of C9orf72-ALS", RVSV(M DELTA 51)-M3 IS AN EFFECTIVE AND SAFE ONCOLYTIC VIRUS FOR CANCER THERAPY; 27TH ANNUAL CONGRESS OF THE EUROPEAN SOCIETY OF GENE AND CELL THERAPY BRITISH SOCIETY FOR GENE THERAPY, ESGCT 2019 20191022 TO 20191025 BARCELONA,, vol. 30, no. 11, 1 November 2019 (2019-11-01), pages A98, XP009522981, ISSN: 1557-7422, DOI: 10.1089/HUM.2019.29095.ABSTRACTS
CHRISTOPHER J. DONNELLY ET AL: "RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention", NEURON, vol. 80, no. 2, 1 October 2013 (2013-10-01), US, pages 415 - 428, XP055649589, ISSN: 0896-6273, DOI: 10.1016/j.neuron.2013.10.015
CHRISTOPHER J. DONNELLY ET AL: "RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention. Supplemental Information", NEURON, 16 October 2013 (2013-10-16), XP055508871, Retrieved from the Internet
C. LAGIER-TOURENNE ET AL: "Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES (PNAS), vol. 110, no. 47, 19 November 2013 (2013-11-19), US, pages E4530 - E4539, XP055228419, ISSN: 0027-8424, DOI: 10.1073/pnas.1318835110
Attorney, Agent or Firm:
CABINET BECKER ET ASSOCIES (FR)
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