[go: up one dir, main page]

WO2024059881A3 - Products and compositions - Google Patents

Products and compositions Download PDF

Info

Publication number
WO2024059881A3
WO2024059881A3 PCT/US2023/074512 US2023074512W WO2024059881A3 WO 2024059881 A3 WO2024059881 A3 WO 2024059881A3 US 2023074512 W US2023074512 W US 2023074512W WO 2024059881 A3 WO2024059881 A3 WO 2024059881A3
Authority
WO
WIPO (PCT)
Prior art keywords
products
nucleobase sequence
compositions
agt gene
modulate
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US2023/074512
Other languages
French (fr)
Other versions
WO2024059881A2 (en
Inventor
Dmitry Samarsky
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sirnaomics Inc
Original Assignee
Sirnaomics 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 Sirnaomics Inc filed Critical Sirnaomics Inc
Priority to EP23866589.7A priority Critical patent/EP4587573A2/en
Priority to CN202380066528.6A priority patent/CN120303402A/en
Publication of WO2024059881A2 publication Critical patent/WO2024059881A2/en
Publication of WO2024059881A3 publication Critical patent/WO2024059881A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

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
    • C12N15/113—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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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]
    • 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
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/321—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
    • 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
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (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)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Nucleic acid products are provided that modulate, in particular interfere with or inhibit AGT gene expression. The products can be oligomeric compounds that comprise at least a first region of linked nucleosides having at least a first nucleobase sequence that is at least partially complementary to at least a portion of RNA transcribed from an AGT gene, wherein said first nucleobase sequence is selected from SEQ ID NOs 1 to 100.
PCT/US2023/074512 2022-09-16 2023-09-18 Products and compositions Ceased WO2024059881A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP23866589.7A EP4587573A2 (en) 2022-09-16 2023-09-18 Products and compositions
CN202380066528.6A CN120303402A (en) 2022-09-16 2023-09-18 Products and Compositions

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263407353P 2022-09-16 2022-09-16
US63/407,353 2022-09-16

Publications (2)

Publication Number Publication Date
WO2024059881A2 WO2024059881A2 (en) 2024-03-21
WO2024059881A3 true WO2024059881A3 (en) 2024-05-16

Family

ID=90275874

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/074512 Ceased WO2024059881A2 (en) 2022-09-16 2023-09-18 Products and compositions

Country Status (3)

Country Link
EP (1) EP4587573A2 (en)
CN (1) CN120303402A (en)
WO (1) WO2024059881A2 (en)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015179724A1 (en) * 2014-05-22 2015-11-26 Alnylam Pharmaceuticals, Inc. Angiotensinogen (agt) irna compositions and methods of use thereof
US20190160090A1 (en) * 2015-10-08 2019-05-30 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
US20220153775A1 (en) * 2020-11-18 2022-05-19 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
US20220213475A1 (en) * 2009-09-22 2022-07-07 Alnylam Pharmaceuticals, Inc. DUAL TARGETING siRNA AGENTS

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220213475A1 (en) * 2009-09-22 2022-07-07 Alnylam Pharmaceuticals, Inc. DUAL TARGETING siRNA AGENTS
WO2015179724A1 (en) * 2014-05-22 2015-11-26 Alnylam Pharmaceuticals, Inc. Angiotensinogen (agt) irna compositions and methods of use thereof
US20190160090A1 (en) * 2015-10-08 2019-05-30 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
US20220153775A1 (en) * 2020-11-18 2022-05-19 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression

Also Published As

Publication number Publication date
CN120303402A (en) 2025-07-11
EP4587573A2 (en) 2025-07-23
WO2024059881A2 (en) 2024-03-21

Similar Documents

Publication Publication Date Title
WO2022221441A3 (en) Products and compositions
JP2023002559A5 (en)
WO2023018523A3 (en) Products and compositions
Ulaganathan et al. Genome engineering for breaking barriers in lignocellulosic bioethanol production
CN104093850B (en) Methods and kits for reducing nonspecific nucleic acid amplification
Beaudry et al. An efficient strategy for the synthesis of circular RNA molecules
Kuwahara et al. Direct PCR amplification of various modified DNAs having amino acids: Convenient preparation of DNA libraries with high-potential activities for in vitro selection
US10457982B2 (en) Method for nucleic acid amplification
MY144014A (en) Method for in vitro recombination
WO2023240190A3 (en) Products and compositions
WO2023245126A3 (en) Products and compositions
US9193994B2 (en) Polynucleotide and use thereof
US20070254343A1 (en) Method of generating long nucleic acid molecules of defined sequence
JP2010532979A5 (en)
JPWO2020206143A5 (en)
WO2024059881A3 (en) Products and compositions
AU2022356440B2 (en) Polymerase variants for template-independent enzymatic nucleic acids synthesis and kit comprising the same
Auxilien et al. Specificity shifts in the rRNA and tRNA nucleotide targets of archaeal and bacterial m5U methyltransferases
Sýkorová et al. Transition between two forms of heterochromatin at plant subtelomeres
WO2022266486A3 (en) Products and compositions
WO2022272108A3 (en) Products and compositions
US20240352433A1 (en) Enzymes with hepn domains
US12006519B2 (en) Polymerase mutants and use with 3′-OH unblocked reversible terminators
JP5216203B2 (en) Method for polymerase chain reaction using a DNA polymerase with proofreading properties
Nawale et al. Incorporation of 4′-C-aminomethyl-2′-O-methylthymidine into DNA by thermophilic DNA polymerases

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 23866589

Country of ref document: EP

Kind code of ref document: A2

WWE Wipo information: entry into national phase

Ref document number: 202380066528.6

Country of ref document: CN

WWE Wipo information: entry into national phase

Ref document number: 2023866589

Country of ref document: EP

NENP Non-entry into the national phase

Ref country code: DE

ENP Entry into the national phase

Ref document number: 2023866589

Country of ref document: EP

Effective date: 20250416

121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 23866589

Country of ref document: EP

Kind code of ref document: A2

WWP Wipo information: published in national office

Ref document number: 202380066528.6

Country of ref document: CN

NENP Non-entry into the national phase

Ref country code: JP

WWP Wipo information: published in national office

Ref document number: 2023866589

Country of ref document: EP