Title:
HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/256290
Kind Code:
A3
Abstract:
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods of inhibiting expression of a C9orf72 gene and methods of treating subjects having a C9orf72-associated disease or disorder, e.g., C9orf72 amyotrophic lateral sclerosis, frontotemporal dementia or Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.
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Inventors:
DANG LAN (US)
MCININCH JAMES (US)
NGUYEN TUYEN (US)
DUBOSE BRITTANY (US)
FRENDEWEY DAVID (US)
SHARMA-KANNING AARTI (US)
MCININCH JAMES (US)
NGUYEN TUYEN (US)
DUBOSE BRITTANY (US)
FRENDEWEY DAVID (US)
SHARMA-KANNING AARTI (US)
Application Number:
PCT/US2022/031519
Publication Date:
January 19, 2023
Filing Date:
May 31, 2022
Export Citation:
Assignee:
ALNYLAM PHARMACEUTICALS INC (US)
REGENERON PHARMA (US)
DANG LAN THI HOANG (US)
MCININCH JAMES D (US)
NGUYEN TUYEN M (US)
DUBOSE BRITTANY (US)
REGENERON PHARMA (US)
DANG LAN THI HOANG (US)
MCININCH JAMES D (US)
NGUYEN TUYEN M (US)
DUBOSE BRITTANY (US)
International Classes:
C12N15/113; A61K31/713; A61P25/28
Domestic Patent References:
| WO2020205605A2 | 2020-10-08 | |||
| WO2021081236A1 | 2021-04-29 | |||
| WO2017079291A1 | 2017-05-11 | |||
| WO2019032607A1 | 2019-02-14 | |||
| WO2019217459A1 | 2019-11-14 | |||
| WO2020227395A2 | 2020-11-12 |
Other References:
HU JIAXIN ET AL: "Engineering Duplex RNAs for Challenging Targets: Recognition of GGGGCC/CCCCGG Repeats at the ALS/FTD C9orf72 Locus", CHEMISTRY & BIOLOGY, CURRENT BIOLOGY, LONDON, GB, vol. 22, no. 11, 12 November 2015 (2015-11-12), pages 1505 - 1511, XP029306633, ISSN: 1074-5521, DOI: 10.1016/J.CHEMBIOL.2015.09.016
ANDREW G.L. DOUGLAS: "Non-coding RNA in C9orf72-related amyotrophic lateral sclerosis and frontotemporal dementia: A perfect storm of dysfunction", NON-CODING RNA RESEARCH, vol. 3, no. 4, 10 September 2018 (2018-09-10), pages 178 - 187, XP055730876, ISSN: 2468-0540, DOI: 10.1016/j.ncrna.2018.09.001
MAYL KEITH ET AL: "Disease Mechanisms and Therapeutic Approaches in C9orf72 ALS-FTD", BIOMEDICINES, vol. 9, no. 6, 25 May 2021 (2021-05-25), pages 601, XP055962130, DOI: 10.3390/biomedicines9060601
ANDREW G.L. DOUGLAS: "Non-coding RNA in C9orf72-related amyotrophic lateral sclerosis and frontotemporal dementia: A perfect storm of dysfunction", NON-CODING RNA RESEARCH, vol. 3, no. 4, 10 September 2018 (2018-09-10), pages 178 - 187, XP055730876, ISSN: 2468-0540, DOI: 10.1016/j.ncrna.2018.09.001
MAYL KEITH ET AL: "Disease Mechanisms and Therapeutic Approaches in C9orf72 ALS-FTD", BIOMEDICINES, vol. 9, no. 6, 25 May 2021 (2021-05-25), pages 601, XP055962130, DOI: 10.3390/biomedicines9060601
Attorney, Agent or Firm:
ZACHARAKIS, Maria, Laccotripe et al. (US)
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