MA51707A - Acides nucléiques pour inhiber l'expression d'un gène cible comprenant des liaisons phosphorodithioate - Google Patents

Acides nucléiques pour inhiber l'expression d'un gène cible comprenant des liaisons phosphorodithioate

Info

Publication number: MA51707A
Authority: MA; Morocco
Prior art keywords: bindings; inhibit; expression; nucleic acids; target gene
Prior art date: 2017-11-13

Application number

MA051707A

Other languages

English (en)

Inventor

Lucas Bethge

Christian Frauendorf

Judith Hauptmann

Adrien Weingärtner

Original Assignee

Silence Therapeutics Gmbh

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.)

2017-11-13

Filing date

2018-11-13

Publication date

2020-09-23

2018-01-16 Priority claimed from GBGB1800679.1A external-priority patent/GB201800679D0/en

2018-11-13 Application filed by Silence Therapeutics Gmbh filed Critical Silence Therapeutics Gmbh

2020-09-23 Publication of MA51707A publication Critical patent/MA51707A/fr

Links

NAGJZTKCGNOGPW-UHFFFAOYSA-K dioxido-sulfanylidene-sulfido-$l^{5}-phosphane Chemical compound [O-]P([O-])([S-])=S NAGJZTKCGNOGPW-UHFFFAOYSA-K 0.000 title 1
108020004707 nucleic acids Proteins 0.000 title 1
150000007523 nucleic acids Chemical class 0.000 title 1
102000039446 nucleic acids Human genes 0.000 title 1
108090000623 proteins and genes Proteins 0.000 title 1

Classifications

- 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
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
- 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
- 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
- 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
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/313—Phosphorodithioates
- 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
- 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
- C12N2310/32—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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
- 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
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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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
- 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
- C12N2310/3521—Methyl
- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
- 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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised

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Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Engineering & Computer Science (AREA)
Genetics & Genomics (AREA)
Biomedical Technology (AREA)
Molecular Biology (AREA)
Chemical & Material Sciences (AREA)
Organic Chemistry (AREA)
Biotechnology (AREA)
General Engineering & Computer Science (AREA)
Bioinformatics & Cheminformatics (AREA)
Zoology (AREA)
Wood Science & Technology (AREA)
General Health & Medical Sciences (AREA)
Biophysics (AREA)
Microbiology (AREA)
Biochemistry (AREA)
Plant Pathology (AREA)
Physics & Mathematics (AREA)
Epidemiology (AREA)
Pharmacology & Pharmacy (AREA)
Medicinal Chemistry (AREA)
Animal Behavior & Ethology (AREA)
Public Health (AREA)
Veterinary Medicine (AREA)
Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Saccharide Compounds (AREA)

MA051707A 2017-11-13 2018-11-13 Acides nucléiques pour inhiber l'expression d'un gène cible comprenant des liaisons phosphorodithioate MA51707A (fr)

Applications Claiming Priority (3)

Application Number	Priority Date	Filing Date	Title
EP17201408		2017-11-13
GBGB1800679.1A GB201800679D0 (en)	2018-01-16	2018-01-16	Phosphorodithioate linkages
EP18165913		2018-04-05

Publications (1)

Publication Number	Publication Date
MA51707A true MA51707A (fr)	2020-09-23

Family

ID=64316546

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
MA051707A MA51707A (fr)	2017-11-13	2018-11-13	Acides nucléiques pour inhiber l'expression d'un gène cible comprenant des liaisons phosphorodithioate

Country Status (14)

Country	Link
US (2)	US11873489B2 (fr)
EP (1)	EP3710585B1 (fr)
JP (1)	JP7461886B2 (fr)
KR (1)	KR102822091B1 (fr)
CN (1)	CN111527206A (fr)
AU (1)	AU2018363837B2 (fr)
BR (1)	BR112020009092A2 (fr)
CA (1)	CA3081910A1 (fr)
IL (1)	IL274611B2 (fr)
MA (1)	MA51707A (fr)
MX (1)	MX2020004898A (fr)
PH (1)	PH12020550244A1 (fr)
SG (1)	SG11202003082UA (fr)
WO (1)	WO2019092280A1 (fr)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
WO2021060440A1 (fr)	2019-09-26	2021-04-01	日油株式会社	Composition lyophilisée de nanoparticules lipidiques
EP4240851A1 (fr)	2020-11-04	2023-09-13	Universität Bern	Acides nucléiques pour inhiber l'expression de pros1 dans une cellule
TW202237840A (zh) *	2020-11-04	2022-10-01	瑞士伯恩大學	用於在細胞中抑制pros1之表現的核酸
WO2022184852A1 (fr) *	2021-03-03	2022-09-09	Silence Therapeutics Gmbh	Acides nucléiques conjugués comprenant un phosphorodithioate pour inhiber l'expression génique dans une cellule
CA3214660A1 (fr) *	2021-03-24	2022-09-29	Atalanta Therapeutics, Inc.	Arnsi double brin ayant des modifications chimiques a motifs
US20250304613A1 (en) *	2021-07-09	2025-10-02	Alnylam Pharmaceuticals, Inc.	Aminooxy click chemistry (aocc): a versatile conjugation approach
US11879125B2 (en)	2022-03-16	2024-01-23	Empirico Inc.	GalNAc compositions for improving siRNA bioavailability
AU2023245603A1 (en)	2022-03-28	2024-11-07	Empirico Inc.	Modified oligonucleotides
GB202311334D0 (en)	2023-07-24	2023-09-06	Astrazeneca Ab	Multivalent cargo-carrying complexes and uses thereof
WO2025021831A1 (fr)	2023-07-24	2025-01-30	Astrazeneca Ab	Complexes multivalents de transport de cargo et leurs utilisations
GB202311324D0 (en)	2023-07-24	2023-09-06	Astrazeneca Ab	Multivalent cargo-carrying complexes and uses thereof

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NL9201440A (nl)	1992-08-11	1994-03-01	Univ Leiden	Triantennaire clusterglycosiden, hun bereiding en toepassing.
US6506559B1 (en)	1997-12-23	2003-01-14	Carnegie Institute Of Washington	Genetic inhibition by double-stranded RNA
WO2003070910A2 (fr)	2002-02-20	2003-08-28	Ribozyme Pharmaceuticals, Incorporated	Inhibition induite par interference d'arn du facteur de croissance endothelial vasculaire et expression genetique du recepteur de facteur de croissance endothelial vasculaire au moyen d'acides nucleiques interferents courts (sina)
BRPI0313202A8 (pt) *	2002-08-05	2016-08-16	Atugen Ag	Formas adicionais para interferir com as moléculas de rna
WO2004090105A2 (fr) *	2003-04-02	2004-10-21	Dharmacon, Inc.	Polynucleotides modifies utilisables pour l'interference arn
CN102124107A (zh) *	2006-07-17	2011-07-13	瑟纳治疗公司	使用短干扰核酸(siNA)的RNA干扰介导的前蛋白转化酶枯草杆菌蛋白酶Kexin9(PCSK9)基因表达的抑制
RU2430740C2 (ru)	2006-08-18	2011-10-10	Ф.Хоффманн-Ля Рош Аг	Поликонъюгаты для введения in vivo полинуклеотидов
CN101500548A (zh)	2006-08-18	2009-08-05	弗·哈夫曼-拉罗切有限公司	用于体内递送多核苷酸的多缀合物
CA2910760C (fr)	2007-12-04	2019-07-09	Muthiah Manoharan	Lipides de ciblage
US9102938B2 (en)	2010-04-01	2015-08-11	Alnylam Pharmaceuticals, Inc.	2′ and 5′ modified monomers and oligonucleotides
EP2751269B1 (fr) *	2011-08-29	2016-03-23	Ionis Pharmaceuticals, Inc.	Procédés et composés utiles dans des pathologies associées à des expansions répétées
US10125362B2 (en)	2012-05-22	2018-11-13	Olix Pharmaceuticals, Inc.	RNA-interference-inducing nucleic acid molecule able to penetrate into cells, and use therefor
KR20240148947A (ko) *	2012-07-13	2024-10-11	웨이브 라이프 사이언시스 리미티드	키랄 제어
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EP3228326A1 (fr)	2016-04-05	2017-10-11	Silence Therapeutics GmbH	Acide nucléique lié à un glycoconjugué trivalent

2018
- 2018-11-13 CN CN201880085033.7A patent/CN111527206A/zh active Pending
- 2018-11-13 MA MA051707A patent/MA51707A/fr unknown
- 2018-11-13 MX MX2020004898A patent/MX2020004898A/es unknown
- 2018-11-13 IL IL274611A patent/IL274611B2/en unknown
- 2018-11-13 BR BR112020009092-1A patent/BR112020009092A2/pt unknown
- 2018-11-13 SG SG11202003082UA patent/SG11202003082UA/en unknown
- 2018-11-13 CA CA3081910A patent/CA3081910A1/fr active Pending
- 2018-11-13 WO PCT/EP2018/081101 patent/WO2019092280A1/fr not_active Ceased
- 2018-11-13 AU AU2018363837A patent/AU2018363837B2/en active Active
- 2018-11-13 US US16/763,399 patent/US11873489B2/en active Active
- 2018-11-13 JP JP2020544149A patent/JP7461886B2/ja active Active
- 2018-11-13 EP EP18803641.2A patent/EP3710585B1/fr active Active
- 2018-11-13 KR KR1020207016885A patent/KR102822091B1/ko active Active
2020
- 2020-04-08 PH PH12020550244A patent/PH12020550244A1/en unknown
2023
- 2023-12-04 US US18/527,920 patent/US20240200071A1/en active Pending

Also Published As

Publication number	Publication date
PH12020550244A1 (en)	2021-02-15
JP7461886B2 (ja)	2024-04-04
CA3081910A1 (fr)	2019-05-16
IL274611B2 (en)	2024-10-01
IL274611A (en)	2020-06-30
IL274611B1 (en)	2024-06-01
MX2020004898A (es)	2020-10-05
RU2020112641A3 (fr)	2022-04-08
BR112020009092A2 (pt)	2020-10-20
EP3710585A1 (fr)	2020-09-23
JP2021502411A (ja)	2021-01-28
EP3710585B1 (fr)	2026-02-25
AU2018363837B2 (en)	2024-11-14
WO2019092280A1 (fr)	2019-05-16
CN111527206A (zh)	2020-08-11
SG11202003082UA (en)	2020-05-28
RU2020112641A (ru)	2021-12-15
US11873489B2 (en)	2024-01-16
US20200392495A1 (en)	2020-12-17
KR20200098521A (ko)	2020-08-20
AU2018363837A1 (en)	2020-05-28
US20240200071A1 (en)	2024-06-20
KR102822091B1 (ko)	2025-06-17

Publication	Publication Date	Title
MA51707A (fr)	2020-09-23	Acides nucléiques pour inhiber l'expression d'un gène cible comprenant des liaisons phosphorodithioate
EP3352795A4 (fr)	2019-03-13	Compositions et méthodes de modification d'acides nucléiques cibles
EP3864173C0 (fr)	2025-08-27	Capture de surface des molecules acides de nucleicques cibles
MA47824A (fr)	2020-01-15	Administration polyanionique d'acides nucléiques
IL271547A (en)	2020-02-27	T7 rna polymerase variants
EP3490562A4 (fr)	2020-04-22	Complexes de pénétration cellulaire immolateurs pour l'administration d'acides nucléiques
EP3417062A4 (fr)	2019-12-11	Excision de séquences d'acides nucléiques rétrovirales
EP3491149A4 (fr)	2019-12-25	Traitement d'acide nucléique indépendant
EP3504229A4 (fr)	2020-02-12	Régulation de l'expression génique à l'aide de nucléases modifiées
IL282290A (en)	2021-05-31	Methods for targeted insertion of dna in genes
IL271553A (en)	2020-02-27	T7 rna polymerase variants
EP3008181A4 (fr)	2016-12-28	Méthodes et compositions pour la modification d'adn cible
EP3381438A4 (fr)	2019-08-14	Composition pour l'injection d'acide hyaluronique, contenant un dérivé de l'acide hyaluronique et une fraction adn, et utilisation de cette dernière
MA48050A (fr)	2020-02-12	Lipides biodégradables pour l'administration d'acides nucléiques
DK3364997T5 (da)	2024-09-30	Aspartoacylase genterapi til behandling af canavans sygdom
EP3414327A4 (fr)	2019-10-16	Détection d'acides nucléiques
EP3377656A4 (fr)	2019-03-27	Chargement d'acides nucléiques sur des substrats
MA43570A (fr)	2018-11-14	Thérapie génique pour traiter l'hypercholestérolémie familiale
EP3734293A4 (fr)	2021-09-15	Piston de cartouche d'extraction d'acide nucléique
EP3651775A4 (fr)	2021-04-07	Méthodes d'inhibition de l'expression génique d'hao1 (hydroxyacide oxydase 1 (glycolate oxydase))
EP3380637A4 (fr)	2019-05-01	Détermination de méthylation d'acides nucléiques
EP3393468A4 (fr)	2019-07-31	Méthodes pour le traitement d'une maladie immunodéficiente
EP3402883A4 (fr)	2019-09-18	Compositions et méthodes pour le séquençage d'acides nucléiques
EP3710109A4 (fr)	2021-06-30	Suivi tridimensionnel d'une cible dans un corps
EP3628003A4 (fr)	2021-01-20	Acides nucléiques p-éthoxy pour l'inhibition de igf-1r