BR112023001957A2 - Composições e métodos para inibir expressão de lpa - Google Patents

Composições e métodos para inibir expressão de lpa

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Publication number
BR112023001957A2
BR112023001957A2 BR112023001957A BR112023001957A BR112023001957A2 BR 112023001957 A2 BR112023001957 A2 BR 112023001957A2 BR 112023001957 A BR112023001957 A BR 112023001957A BR 112023001957 A BR112023001957 A BR 112023001957A BR 112023001957 A2 BR112023001957 A2 BR 112023001957A2
Authority
BR
Brazil
Prior art keywords
compositions
methods
expression
inhibit lpa
lpa expression
Prior art date
Application number
BR112023001957A
Other languages
English (en)
Inventor
Dale Brown Bob
T Dudek Henryk
Abrams Marc
Han Wen
Turanov Anton
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 BR112023001957A2 publication Critical patent/BR112023001957A2/pt

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    • 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
    • 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)
    • C12Y304/21007—Plasmin (3.4.21.7), i.e. fibrinolysin
    • 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 N.A.
    • 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
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
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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
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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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/351—Conjugate
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    • 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
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    • 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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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

COMPOSIÇÕES E MÉTODOS PARA INIBIR EXPRESSÃO DE LPA. São fornecidos neste documento oligonucleotídeos que inibem a expressão da apolipoproteína(a) (LPA). Também são fornecidas composições incluindo os mesmos e usos das mesmas, particularmente usos relacionados a tratamento de doenças, distúrbios e/ou condições associadas à expressão de LPA.
BR112023001957A 2020-08-05 2021-08-05 Composições e métodos para inibir expressão de lpa BR112023001957A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063061676P 2020-08-05 2020-08-05
US202063074779P 2020-09-04 2020-09-04
PCT/US2021/071109 WO2022032288A1 (en) 2020-08-05 2021-08-05 Compositions and methods for inhibiting lpa expression

Publications (1)

Publication Number Publication Date
BR112023001957A2 true BR112023001957A2 (pt) 2023-04-25

Family

ID=77519885

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023001957A BR112023001957A2 (pt) 2020-08-05 2021-08-05 Composições e métodos para inibir expressão de lpa

Country Status (11)

Country Link
EP (1) EP4192954A1 (pt)
JP (1) JP7476422B2 (pt)
KR (1) KR20230043912A (pt)
CN (1) CN116323943A (pt)
AU (1) AU2021320550A1 (pt)
BR (1) BR112023001957A2 (pt)
CA (1) CA3185348A1 (pt)
IL (1) IL300338A (pt)
MX (1) MX2023001538A (pt)
TW (1) TW202220673A (pt)
WO (1) WO2022032288A1 (pt)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114703184B (zh) * 2022-03-11 2024-06-18 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
US20230374522A1 (en) * 2022-04-15 2023-11-23 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating scap activity
WO2024035898A2 (en) * 2022-08-11 2024-02-15 Sirius Therapeutics, Inc. Polynucleic acid molecules for inhibiting expression of lp(a), pharmaceutical compositions, and uses thereof
CN115851723B (zh) * 2022-10-24 2023-10-03 厦门甘宝利生物医药有限公司 一种抑制lpa基因表达的rna抑制剂及其应用
WO2024112865A1 (en) 2022-11-23 2024-05-30 Eli Lilly And Company Method of synthesizing 4'-phosphate analog nucleotide phosphoramidite
WO2024112877A1 (en) 2022-11-23 2024-05-30 Eli Lilly And Company Methods of synthesizing a targeting ligand-conjugated nucleotide phosphoramidite

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US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
EP0748382B1 (en) 1993-09-02 2002-11-06 Ribozyme Pharmaceuticals, Inc. Non-nucleotide containing enzymatic nucleic acid
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
NZ553687A (en) 2000-03-30 2010-03-26 Whitehead Biomedical Inst RNA sequence-specific mediators of RNA interference
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CN107109405A (zh) 2014-06-06 2017-08-29 索尔斯蒂斯生物有限公司 具有生物可逆性和非生物可逆性基团的多核苷酸构建体
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JP6978099B2 (ja) 2016-09-02 2021-12-08 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 ４’−リン酸アナログ及びそれを含むオリゴヌクレオチド
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Also Published As

Publication number Publication date
AU2021320550A1 (en) 2023-02-16
KR20230043912A (ko) 2023-03-31
JP2023536974A (ja) 2023-08-30
CA3185348A1 (en) 2022-02-10
WO2022032288A1 (en) 2022-02-10
IL300338A (en) 2023-04-01
EP4192954A1 (en) 2023-06-14
CN116323943A (zh) 2023-06-23
JP7476422B2 (ja) 2024-04-30
TW202220673A (zh) 2022-06-01
MX2023001538A (es) 2023-03-08

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