JOP20210018A1 - مركبات وطرق لتقليل التعبير الوراثي عن atxn2 - Google Patents

مركبات وطرق لتقليل التعبير الوراثي عن atxn2

Info

Publication number
JOP20210018A1
JOP20210018A1 JOP/2021/0018A JOP20210018A JOP20210018A1 JO P20210018 A1 JOP20210018 A1 JO P20210018A1 JO P20210018 A JOP20210018 A JO P20210018A JO P20210018 A1 JOP20210018 A1 JO P20210018A1
Authority
JO
Jordan
Prior art keywords
methods
compounds
atxn2
reduce
genetic expression
Prior art date
Application number
JOP/2021/0018A
Other languages
Arabic (ar)
English (en)
Inventor
Paymaan Jafar-Nejad
Holly Kordasiewicz
Priyam Singh
Susan M Freier
Frank Rigo
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of JOP20210018A1 publication Critical patent/JOP20210018A1/ar

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/46—Ingredients of undetermined constitution or reaction products thereof, e.g. skin, bone, milk, cotton fibre, eggshell, oxgall or plant extracts
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Botany (AREA)
  • Inorganic Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
JOP/2021/0018A 2018-07-25 2019-07-25 مركبات وطرق لتقليل التعبير الوراثي عن atxn2 JOP20210018A1 (ar)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
JOP20210018A1 true JOP20210018A1 (ar) 2021-01-21

Family

ID=69182364

Family Applications (1)

Application Number Title Priority Date Filing Date
JOP/2021/0018A JOP20210018A1 (ar) 2018-07-25 2019-07-25 مركبات وطرق لتقليل التعبير الوراثي عن atxn2

Country Status (23)

Country Link
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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CN106103717A (zh) 2014-03-19 2016-11-09 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
RS60707B1 (sr) * 2014-04-01 2020-09-30 Biogen Ma Inc Kompozicije za modulaciju ekspresije sod-1
US20190002887A1 (en) 2015-12-31 2019-01-03 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
BR112020013994A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
BR112021000308A2 (pt) 2018-07-25 2021-04-13 Ionis Pharmaceuticals, Inc. Compostos e métodos para redução da expressão de atxn2
WO2021159008A2 (en) * 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
EP4100527A2 (en) * 2020-02-07 2022-12-14 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
US20240294906A1 (en) * 2020-07-29 2024-09-05 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases
BR112023021318A2 (pt) * 2021-04-14 2023-12-19 Aro Biotherapeutics Company Conjugados de domínio fn3-sirna e usos dos mesmos
WO2023102188A1 (en) * 2021-12-03 2023-06-08 Quralis Corporation Gapmer antisense oligonucleotides with modified backbone chemistries
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
EP4536236A2 (en) * 2022-06-06 2025-04-16 University of Utah Research Foundation Methods of modulating atxn2 expression
US20250179491A1 (en) * 2022-06-24 2025-06-05 Eli Lilly And Company Atxn2 rna interference agents
WO2024056902A2 (en) * 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases
GB202302480D0 (en) * 2023-02-22 2023-04-05 Drishti Discoveries Ltd shRNA for the treatment of disease
WO2025067482A1 (zh) * 2023-09-28 2025-04-03 大睿生物 调控atxn2表达的dsrna分子

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