CA2978100C - Compounds and methods for modulating tmprss6 expression - Google Patents

Compounds and methods for modulating tmprss6 expression Download PDF

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Publication number
CA2978100C
CA2978100C CA2978100A CA2978100A CA2978100C CA 2978100 C CA2978100 C CA 2978100C CA 2978100 A CA2978100 A CA 2978100A CA 2978100 A CA2978100 A CA 2978100A CA 2978100 C CA2978100 C CA 2978100C
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certain embodiments
compound
antisense
thalassemia
composition
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French (fr)
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CA2978100A1 (en
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Shuling Guo
Mariam Aghajan
Eric E. Swayze
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • 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
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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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
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    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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    • 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
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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CA2978100A 2015-04-03 2016-04-04 Compounds and methods for modulating tmprss6 expression Active CA2978100C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562142986P 2015-04-03 2015-04-03
US62/142,986 2015-04-03
PCT/US2016/025883 WO2016161429A1 (en) 2015-04-03 2016-04-04 Compounds and methods for modulating tmprss6 expression

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CA2978100A1 CA2978100A1 (en) 2016-10-06
CA2978100C true CA2978100C (en) 2023-10-17

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CA2978100A Active CA2978100C (en) 2015-04-03 2016-04-04 Compounds and methods for modulating tmprss6 expression

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US (4) US10415038B2 (enExample)
EP (1) EP3277817A4 (enExample)
JP (3) JP6833705B2 (enExample)
KR (1) KR102539587B1 (enExample)
CN (1) CN107429250B (enExample)
AU (1) AU2016244106B2 (enExample)
CA (1) CA2978100C (enExample)
HK (1) HK1249140A1 (enExample)
IL (2) IL253509B2 (enExample)
MX (1) MX2017012426A (enExample)
RU (1) RU2017137410A (enExample)
WO (1) WO2016161429A1 (enExample)

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EP3898977A1 (en) * 2018-12-20 2021-10-27 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
CN113748209A (zh) 2019-02-27 2021-12-03 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
JP2023130528A (ja) * 2020-06-24 2023-09-21 田辺三菱製薬株式会社 Ｐｌｐ１発現を調節するための化合物、方法及び医薬組成物
CA3195722A1 (en) 2020-09-17 2022-03-24 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets
US20240240187A1 (en) 2021-04-27 2024-07-18 Silence Therapeutics Gmbh Sirna targeting tmprss6 for the treatment of myeloproliferative disorders
EP4329774A4 (en) * 2021-04-28 2025-11-05 Quiver Holdings Inc THERAPEUTIC COMPOSITIONS FOR THE TREATMENT OF MULTI-TARGET PAIN
CN115247173B (zh) * 2021-11-11 2025-11-11 南京启真基因工程有限公司 构建tmprss6基因突变的缺铁性贫血猪核移植供体细胞的基因编辑系统及其应用
TWI868755B (zh) 2022-06-24 2025-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
WO2024060649A1 (zh) * 2022-09-29 2024-03-28 广州必贝特医药股份有限公司 用于抑制TMPRSS6基因表达的siRNA或其盐、药物及其应用
EP4596693A1 (en) * 2022-09-29 2025-08-06 Bebetter Med Inc. Sirna or salt thereof and medicament for inhibiting expression of tmprss6 gene, and use thereof
EP4687983A1 (en) 2023-03-27 2026-02-11 Silence Therapeutics GmbH Compounds and compositions for use in stem cell transplantation
TW202527968A (zh) * 2024-01-08 2025-07-16 大陸商上海舶望製藥有限公司 用於抑制跨膜絲氨酸蛋白酶6（tmprss6）表達的組合物和方法
WO2025206092A1 (ja) * 2024-03-27 2025-10-02 田辺三菱製薬株式会社 Ｍｙｏｒｅｇｕｌｉｎ発現を調節するための化合物及び医薬組成物

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