KR20210038589A - Atxn2 발현 감소용 화합물 및 방법 - Google Patents

Atxn2 발현 감소용 화합물 및 방법 Download PDF

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KR20210038589A
KR20210038589A KR1020217005202A KR20217005202A KR20210038589A KR 20210038589 A KR20210038589 A KR 20210038589A KR 1020217005202 A KR1020217005202 A KR 1020217005202A KR 20217005202 A KR20217005202 A KR 20217005202A KR 20210038589 A KR20210038589 A KR 20210038589A
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modified
seq
nucleobase
certain embodiments
modified oligonucleotide
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Korean (ko)
Inventor
수잔 엠. 프라이어
프리얌 싱
프랭크 리고
페이만 자파-네자드
홀리 코르다지에빅츠
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아이오니스 파마수티컬즈, 인코포레이티드
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Publication of KR20210038589A publication Critical patent/KR20210038589A/ko
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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KR1020217005202A 2018-07-25 2019-07-25 Atxn2 발현 감소용 화합물 및 방법 Pending KR20210038589A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862703240P 2018-07-25 2018-07-25
US62/703,240 2018-07-25
PCT/US2019/043424 WO2020023737A1 (en) 2018-07-25 2019-07-25 Compounds and methods for reducing atxn2 expression

Publications (1)

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KR20210038589A true KR20210038589A (ko) 2021-04-07

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US (3) US11078486B2 (https=)
EP (1) EP3826645A4 (https=)
JP (2) JP7411632B2 (https=)
KR (1) KR20210038589A (https=)
CN (1) CN112423767B (https=)
AR (1) AR115847A1 (https=)
AU (2) AU2019310097A1 (https=)
BR (1) BR112021000308A2 (https=)
CA (1) CA3106986A1 (https=)
CL (1) CL2021000159A1 (https=)
CO (1) CO2021000196A2 (https=)
CR (1) CR20210100A (https=)
EA (1) EA202190299A1 (https=)
IL (1) IL280123A (https=)
JO (1) JOP20210018A1 (https=)
MX (1) MX2021000922A (https=)
PE (1) PE20211398A1 (https=)
PH (1) PH12021500007A1 (https=)
SG (1) SG11202100077PA (https=)
TW (1) TWI843738B (https=)
UY (1) UY38316A (https=)
WO (1) WO2020023737A1 (https=)
ZA (1) ZA202100108B (https=)

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EP3119888B1 (en) 2014-03-19 2021-07-28 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
HUE050704T2 (hu) * 2014-04-01 2020-12-28 Biogen Ma Inc Összetételek a SOD-1 expressziójának modulálására
AU2016381174A1 (en) 2015-12-31 2018-05-31 Ionis Pharmaceuticals, Inc. Methods for reducing Ataxin-2 expression
US20200283800A1 (en) * 2017-10-23 2020-09-10 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative diseases
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
AR115847A1 (es) 2018-07-25 2021-03-03 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de la atxn2
EP4100527A2 (en) * 2020-02-07 2022-12-14 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
WO2021159008A2 (en) * 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
US20230212572A1 (en) * 2020-06-09 2023-07-06 Roche Innovation Center Copenhagen A/S Guanosine Analogues for Use in Therapeutics Polynucleotides
WO2022026531A1 (en) * 2020-07-29 2022-02-03 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases
EP4323008A4 (en) * 2021-04-14 2026-02-25 Aro Biotherapeutics Company FN3-ARNSI DOMAIN CONJUGATES AND THEIR USES
EP4441224A4 (en) * 2021-12-03 2026-03-11 Quralis Corp Antisense oligonucleotides with modified skeletal chemicals
WO2023107893A2 (en) * 2021-12-06 2023-06-15 The Board Of Trustees Of The Leland Stanford Junior University Treatment of neuorodegenerative diseases through the inhibition of ataxin-2
WO2023239747A2 (en) * 2022-06-06 2023-12-14 University Of Utah Research Foundation Methods of modulating atxn2 expression
KR20250028434A (ko) * 2022-06-24 2025-02-28 일라이 릴리 앤드 캄파니 Atxn2 rna 간섭 작용제
CA3267488A1 (en) * 2022-09-16 2024-03-21 King's College London COMPOSITIONS AND METHODS OF TREATMENT OF NEUROLOGICAL DISEASES
GB202302480D0 (en) * 2023-02-22 2023-04-05 Drishti Discoveries Ltd shRNA for the treatment of disease
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