MX2022004032A - Modificaciones químicas de arn interferente pequeño con mínimo contenido de flúor. - Google Patents

Modificaciones químicas de arn interferente pequeño con mínimo contenido de flúor.

Info

Publication number
MX2022004032A
MX2022004032A MX2022004032A MX2022004032A MX2022004032A MX 2022004032 A MX2022004032 A MX 2022004032A MX 2022004032 A MX2022004032 A MX 2022004032A MX 2022004032 A MX2022004032 A MX 2022004032A MX 2022004032 A MX2022004032 A MX 2022004032A
Authority
MX
Mexico
Prior art keywords
minimal
interfering rna
chemical modifications
small interfering
fluorine content
Prior art date
Application number
MX2022004032A
Other languages
English (en)
Spanish (es)
Inventor
Naim Nazef
Weimin Wang
Original Assignee
Dicerna 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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of MX2022004032A publication Critical patent/MX2022004032A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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 against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/533Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
MX2022004032A 2019-10-02 2020-10-02 Modificaciones químicas de arn interferente pequeño con mínimo contenido de flúor. MX2022004032A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962909278P 2019-10-02 2019-10-02
PCT/US2020/053999 WO2021067744A1 (en) 2019-10-02 2020-10-02 Chemical modifications of small interfering rna with minimal fluorine content

Publications (1)

Publication Number Publication Date
MX2022004032A true MX2022004032A (es) 2022-09-21

Family

ID=73038388

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022004032A MX2022004032A (es) 2019-10-02 2020-10-02 Modificaciones químicas de arn interferente pequeño con mínimo contenido de flúor.

Country Status (12)

Country Link
US (1) US20220389430A1 (https=)
EP (1) EP4038191A1 (https=)
JP (1) JP2022551269A (https=)
KR (1) KR20220069103A (https=)
CN (1) CN114761557B (https=)
AU (1) AU2020358016A1 (https=)
CA (1) CA3153026A1 (https=)
CL (1) CL2022000825A1 (https=)
IL (1) IL291841A (https=)
MX (1) MX2022004032A (https=)
TW (1) TW202126809A (https=)
WO (1) WO2021067744A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PE20250400A1 (es) 2021-04-12 2025-02-11 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir cetohexoquinasa
CA3209418A1 (en) * 2021-04-14 2022-10-20 Utsav SAXENA Compositions and methods for modulating pnpla3 expression
EP4372085A4 (en) * 2021-07-16 2026-01-21 Suzhou Ribo Life Science Co Ltd Nucleic acid, composition and conjugate, container, preparation process and use
EP4405368A1 (en) * 2021-09-22 2024-07-31 Sanegene Bio USA Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
WO2023069495A1 (en) * 2021-10-19 2023-04-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides with 2'-deoxy-2'-f-2'-c-methyl nucleotides
JP2024543195A (ja) 2021-12-01 2024-11-19 ディセルナ ファーマシューティカルズ インコーポレイテッド Apoc3発現を調節するための組成物及び方法
CN119183476A (zh) * 2022-04-26 2024-12-24 上海拓界生物医药科技有限公司 氘代化学修饰和包含其的寡核苷酸
TW202526017A (zh) * 2023-09-14 2025-07-01 美商Ionis製藥公司 用於減少apociii表現的化合物及方法
WO2025191337A2 (en) * 2024-03-11 2025-09-18 Synerk Biotech Limited Oligonucleotide terminal phosphate derivatives and method of use

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
DK2341943T3 (en) 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
US8691971B2 (en) 2008-09-23 2014-04-08 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
EP2928877B1 (en) 2012-12-06 2020-01-22 Merck Sharp & Dohme Corp. Disulfide masked prodrug compositions and methods
CA2950960A1 (en) 2014-06-06 2015-12-10 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
WO2019075419A1 (en) * 2017-10-13 2019-04-18 Dicerna Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION
UA128786C2 (uk) * 2017-10-20 2024-10-23 Дайсерна Фармасьютикалз, Інк Олігонуклеотид для лікування інфекції гепатиту в

Also Published As

Publication number Publication date
WO2021067744A1 (en) 2021-04-08
CA3153026A1 (en) 2021-04-08
AU2020358016A1 (en) 2022-04-21
CN114761557B (zh) 2024-11-15
CL2022000825A1 (es) 2023-01-27
CN114761557A (zh) 2022-07-15
JP2022551269A (ja) 2022-12-08
US20220389430A1 (en) 2022-12-08
KR20220069103A (ko) 2022-05-26
EP4038191A1 (en) 2022-08-10
TW202126809A (zh) 2021-07-16
IL291841A (en) 2022-06-01

Similar Documents

Publication Publication Date Title
MX2022004032A (es) Modificaciones químicas de arn interferente pequeño con mínimo contenido de flúor.
ZA202108348B (en) Angiotensinogen (agt) irna compositions and methods of use thereof
MX2025007610A (es) Composiciones de arni de 17beta-hidroxiesteroide deshidrogenasa tipo 13 (hsd17b13) y metodos de uso de las mismas
MX2021006745A (es) Constructos de iarn modificados quimicamente y usos de estos.
MX2021012126A (es) Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central.
CR20190294A (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO
MA40819A1 (fr) Variant d'arni
MX2019005816A (es) Agentes de arn modificados con efecto reducido fuera de la diana.
AR120341A1 (es) COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
BR112014011896A2 (pt) agentes de rnai, composições e métodos de uso destes para o tratamento de doenças associadas à transtirretina (ttr)
MX2024004750A (es) Composiciones de acido ribonucleico de interferencia (arni) contra el factor b del complemento (cfb) y sus metodos de uso.
EP2348112A3 (en) Stabilized immune modulatory RNA (SIMRA) compounds
NO20070263L (no) Immunostimulerende oligonukleotidmultimerer
MY162563A (en) Compositions and methods for treating hyperproliferative diseases
PH12021500009A1 (en) Rna molecules comprising non-canonical base pairs
CR7901A (es) Pirazolilcarboxanilidas para la lucha contra los microorganismos indeseados
ATE548454T1 (de) Nukleinsäurewirkstoffe zur stilllegung von h19 zwecks behandlung rheumatoider arthritis
BR112018008344A2 (pt) molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição
CL2013003307A1 (es) Agente que comprende ciazofamida o fenamidona; y su uso para tratar micosis en peces e invertebrados en todos sus estadios de desarrollo causada por hongos de los generos saprolegnia, achlya y aphanomyces (div.sol.n°1056-08).
PH12020500527A1 (en) Rna molecules
EA201792261A1 (ru) Нуклеозидные вещества для уменьшения вредной активности генов, содержащих удлиненные нуклеотидные повторы
MX2023011466A (es) Composiciones de agentes de ácido ribonucleico de interferencia (arni) de huntingtina (htt) y métodos de uso de estas.
MX2024002440A (es) Composiciones de acido ribonucleico de interferencia (arni) del efector b similar al factor de fragmentacion de adn subunidad alfa (dffa) que induce la muerte celular (cideb) y metodos de uso de estas.
BR112022004221A2 (pt) Tratamento de hidradenite com inibidores de jak
BR112022023408A2 (pt) Oligonucleotídeo de fita dupla e composição para tratar covid-19 contendo o mesmo