CA3032165C - Compositions comprising reversibly modified oligonucleotides and uses thereof - Google Patents

Compositions comprising reversibly modified oligonucleotides and uses thereof Download PDF

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Publication number
CA3032165C
CA3032165C CA3032165A CA3032165A CA3032165C CA 3032165 C CA3032165 C CA 3032165C CA 3032165 A CA3032165 A CA 3032165A CA 3032165 A CA3032165 A CA 3032165A CA 3032165 C CA3032165 C CA 3032165C
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substituted
ive
glutathione
formula
unsubstituted
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CA3032165A1 (en
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Weimin Wang
Venkata KRISHNAMURTHY
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
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    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
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    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A61K31/7072—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
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    • 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
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    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
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    • 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
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CA3032165A 2016-08-23 2017-08-23 Compositions comprising reversibly modified oligonucleotides and uses thereof Active CA3032165C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662378635P 2016-08-23 2016-08-23
US62/378,635 2016-08-23
PCT/US2017/048239 WO2018039364A1 (en) 2016-08-23 2017-08-23 Compositions comprising reversibly modified oligonucleotides and uses thereof

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CA3032165A1 CA3032165A1 (en) 2018-03-01
CA3032165C true CA3032165C (en) 2023-05-16

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CA3032165A Active CA3032165C (en) 2016-08-23 2017-08-23 Compositions comprising reversibly modified oligonucleotides and uses thereof

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US (2) US11390642B2 (https=)
EP (1) EP3503730B1 (https=)
JP (2) JP7249080B2 (https=)
KR (1) KR102530513B1 (https=)
CN (2) CN109890207B (https=)
AU (2) AU2017315670B2 (https=)
CA (1) CA3032165C (https=)
IL (2) IL264685B (https=)
MX (2) MX2019002075A (https=)
WO (1) WO2018039364A1 (https=)

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EP3697423A4 (en) 2018-01-05 2021-08-11 Dicerna Pharmaceuticals, Inc. REDUCED EXPRESSION OF BETA-CATENIN AND IDO TO POTENTIALIZE IMMUNOTHERAPY
WO2019143621A1 (en) 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression
CA3135402A1 (en) 2019-04-04 2020-10-08 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression in the central nervous system
EP4116422A4 (en) * 2020-03-03 2024-03-20 Nexmos Co., Ltd. Dna aptamer specifically binding to glutathione and use thereof
IL300283A (en) 2020-08-04 2023-04-01 Dicerna Pharmaceuticals Inc Systemic administration of oligonucleotides
IL301940A (en) 2020-10-08 2023-06-01 Dicerna Pharmaceuticals Inc Selective delivery of oligonucleotides to glial cells
EP4271695A2 (en) * 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
US12084662B2 (en) 2021-04-14 2024-09-10 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating PNPLA3 expression
AU2022324471A1 (en) 2021-08-05 2024-02-15 Sanegene Bio Usa Inc. 1'-alkyl modified ribose derivatives and methods of 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
WO2023047338A1 (en) * 2021-09-24 2023-03-30 Crispr Therapeutics Ag PRODRUG INCORPORATED sgRNA SYNTHESIS
CN118076621A (zh) 2021-10-05 2024-05-24 美国圣因生物股份有限公司 多羟基化的环戊烷衍生物及使用方法
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