JP2017522046A - 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物 - Google Patents

生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物 Download PDF

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JP2017522046A
JP2017522046A JP2017516650A JP2017516650A JP2017522046A JP 2017522046 A JP2017522046 A JP 2017522046A JP 2017516650 A JP2017516650 A JP 2017516650A JP 2017516650 A JP2017516650 A JP 2017516650A JP 2017522046 A JP2017522046 A JP 2017522046A
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alkyl
optionally substituted
aryl
polynucleotide construct
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JP2017522046A5 (OSRAM
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ブラッドショー，コート，ダブリュ．
サカムリ，スクマー
エルテプ，ラクスマン
ラム，ソン
リウ，ディングオ
ミード，ブライアン
イアコノ，ギウセッペ，デッロ
ストック，ジョセフ
リウ，ビン
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ソルスティス バイオロジクス，リミティッド
ソルスティス バイオロジクス，リミティッド
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JP2017516650A 2014-06-06 2015-06-08 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物 Pending JP2017522046A (ja)

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US201462009123P 2014-06-06 2014-06-06
US62/009,123 2014-06-06
PCT/US2015/034749 WO2015188197A2 (en) 2014-06-06 2015-06-08 Polynucleotide constructs having bioreversible and non-bioreversible groups

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JP2017522046A true JP2017522046A (ja) 2017-08-10
JP2017522046A5 JP2017522046A5 (OSRAM) 2018-07-19

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US (2) US20170114341A1 (OSRAM)
EP (1) EP3152308A4 (OSRAM)
JP (1) JP2017522046A (OSRAM)
CN (1) CN107109405A (OSRAM)
AU (1) AU2015269053A1 (OSRAM)
CA (1) CA2950960A1 (OSRAM)
WO (1) WO2015188197A2 (OSRAM)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPWO2019039403A1 (ja) * 2017-08-22 2020-11-26 国立大学法人東海国立大学機構 修飾ポリヌクレオチド
JP2021509402A (ja) * 2017-12-29 2021-03-25 スーチョウ リボ ライフ サイエンス カンパニー、リミテッドＳｕｚｈｏｕ Ｒｉｂｏ Ｌｉｆｅ Ｓｃｉｅｎｃｅ Ｃｏ．， Ｌｔｄ． 複合体及びその調製方法と使用
JP2022515503A (ja) * 2018-12-28 2022-02-18 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
JP2022546055A (ja) * 2019-08-29 2022-11-02 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 化合物、薬物複合体及びその調製方法と使用
WO2023113038A1 (ja) * 2021-12-17 2023-06-22 リードファーマ株式会社 オリゴヌクレオチドの製造方法

Families Citing this family (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
US20180133327A1 (en) * 2015-03-16 2018-05-17 Amal Therapeutics Sa Cell Penetrating Peptides and Complexes Comprising the Same
CN108027335B (zh) 2015-06-25 2021-05-04 罗斯韦尔生物技术股份有限公司 生物分子传感器和方法
EP3386517A4 (en) * 2015-12-08 2019-08-07 Solstice Biologics, Ltd. POLYNUCLEOTIDE CONSTRUCTS COMPRISING AN AUXILIARY FRACTION RELATED NON-BIOREVERSIBLE TO AN INTERNETOSIDIC PHOSPHATE OR PHOSPHOROTHIOATE
EP3408219B1 (en) 2016-01-28 2022-08-17 Roswell Biotechnologies, Inc Massively parallel dna sequencing apparatus
US11624725B2 (en) 2016-01-28 2023-04-11 Roswell Blotechnologies, Inc. Methods and apparatus for measuring analytes using polymerase in large scale molecular electronics sensor arrays
EP3882616A1 (en) 2016-02-09 2021-09-22 Roswell Biotechnologies, Inc Electronic label-free dna and genome sequencing
US10597767B2 (en) 2016-02-22 2020-03-24 Roswell Biotechnologies, Inc. Nanoparticle fabrication
LT3429618T (lt) 2016-03-16 2024-05-10 Amal Therapeutics Sa Imuninės sistemos patikros taško moduliatoriaus ir komplekso, apimančio į ląsteles prasiskverbiantį peptidą, krovinį ir tlr peptido agonistą, derinys, skirtas naudoti medicinoje
US9829456B1 (en) 2016-07-26 2017-11-28 Roswell Biotechnologies, Inc. Method of making a multi-electrode structure usable in molecular sensing devices
US20210139959A1 (en) * 2016-08-01 2021-05-13 Roswell Biotechnologies, Inc. Modified nucleotide triphosphates for molecular electronic sensors
WO2018035380A1 (en) * 2016-08-17 2018-02-22 Solstice Biologics, Ltd. Polynucleotide constructs
AU2017315670B2 (en) 2016-08-23 2022-03-17 Dicerna Pharmaceuticals, Inc. Compositions comprising reversibly modified oligonucleotides and uses thereof
KR20190057345A (ko) 2016-09-21 2019-05-28 아말 테라퓨틱스 에스에이 암 치료를 위한, 세포 투과 펩타이드, 멀티 에피토프 및 tlr 펩타이드 작용제를 포함하는 융합체
US10902939B2 (en) 2017-01-10 2021-01-26 Roswell Biotechnologies, Inc. Methods and systems for DNA data storage
TWI826365B (zh) * 2017-01-10 2023-12-21 美商愛羅海德製藥公司 α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法
WO2018136148A1 (en) 2017-01-19 2018-07-26 Roswell Biotechnologies, Inc. Solid state sequencing devices comprising two dimensional layer materials
CN110770345A (zh) * 2017-04-14 2020-02-07 托尔奈公司 免疫调节多核苷酸、抗体缀合物及其使用方法
US10508296B2 (en) 2017-04-25 2019-12-17 Roswell Biotechnologies, Inc. Enzymatic circuits for molecular sensors
EP3615685B1 (en) 2017-04-25 2025-02-19 Roswell Biotechnologies, Inc Enzymatic circuits for molecular sensors
EP3622086A4 (en) 2017-05-09 2021-04-21 Roswell Biotechnologies, Inc LINK PROBE CIRCUITS FOR MOLECULAR SENSORS
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
CN111373049A (zh) 2017-08-30 2020-07-03 罗斯威尔生命技术公司 用于dna数据存储的进行性酶分子电子传感器
CN111373051A (zh) 2017-10-10 2020-07-03 罗斯威尔生命技术公司 用于无扩增dna数据存储的方法、装置和系统
IL282881B2 (en) 2017-10-20 2024-10-01 Dicerna Pharmaceuticals Inc Methods for treating hepatitis b infection
AU2018374219C1 (en) 2017-12-01 2023-05-11 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof
KR102834361B1 (ko) 2017-12-01 2025-07-17 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
EP3718572B1 (en) 2017-12-01 2024-07-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method and use
JP7365052B2 (ja) 2017-12-01 2023-10-19 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
CA3086409A1 (en) 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression
AU2019222767A1 (en) 2018-02-14 2020-08-27 Deep Genomics Incorporated Oligonucleotide therapy for Wilson disease
SG11202010910QA (en) 2018-05-07 2020-12-30 Alnylam Pharmaceuticals Inc Extrahepatic delivery
AU2019300324A1 (en) 2018-07-13 2021-01-21 F. Hoffmann-La Roche Ag Oligonucleotides for modulating RTEL1 expression
EP3842534A4 (en) 2018-08-21 2022-07-06 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING NUCLEIC ACID AND METHOD OF USE THEREOF
CN111655297A (zh) 2018-09-30 2020-09-11 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
AU2019360216A1 (en) * 2018-10-17 2021-05-13 Tallac Therapeutics, Inc. Immunomodulating polynucleotide conjugates and methods of use
US11249941B2 (en) * 2018-12-21 2022-02-15 Palo Alto Research Center Incorporated Exabyte-scale data storage using sequence-controlled polymers
WO2020135581A1 (zh) * 2018-12-28 2020-07-02 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
KR20210132661A (ko) 2019-02-12 2021-11-04 다이서나 파마수이티컬, 인크. Cyp27a1의 발현을 억제하기 위한 방법 및 조성물
KR20210148264A (ko) 2019-04-04 2021-12-07 다이서나 파마수이티컬, 인크. 중추신경계에서 유전자 발현을 억제하는 조성물 및 방법
CA3139195A1 (en) 2019-05-22 2020-11-26 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
JP7613751B2 (ja) * 2019-05-24 2025-01-15 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
CN112390835A (zh) * 2019-08-14 2021-02-23 苏州瑞博生物技术股份有限公司 肝靶向化合物及缀合物
AU2020358016A1 (en) 2019-10-02 2022-04-21 Dicerna Pharmaceuticals, Inc. Chemical modifications of small interfering RNA with minimal fluorine content
CN119607006A (zh) 2019-12-09 2025-03-14 艾姆皮瑞克公司 用于治疗血管生成素样4(angptl4)相关疾病的寡核苷酸
EP4081217A1 (en) 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
WO2021130266A1 (en) 2019-12-24 2021-07-01 F. Hoffmann-La Roche Ag Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
WO2021174091A1 (en) 2020-02-28 2021-09-02 Tallac Therapeutics, Inc. Transglutaminase-mediated conjugation
BR112022018667A2 (pt) 2020-03-18 2022-11-29 Dicerna Pharmaceuticals Inc Composições e métodos para inibir a expressão de ang-ptl3
US20230277675A1 (en) 2020-08-04 2023-09-07 Dicerna Pharmaceuticals, Inc. Systemic delivery of oligonucleotides
TW202221120A (zh) 2020-08-04 2022-06-01 美商黛瑟納製藥公司 用於治療代謝症候群之組成物及方法
EP4192954A1 (en) 2020-08-05 2023-06-14 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting lpa expression
CA3190794A1 (en) 2020-08-05 2022-02-10 Soren Ottosen Oligonucleotide treatment of hepatitis b patients
IL301940A (en) 2020-10-08 2023-06-01 Dicerna Pharmaceuticals Inc Selective delivery of oligonucleotides to glial cells
US20230390403A1 (en) * 2020-10-14 2023-12-07 North Carolina State University Compositions and methods for drug delivery
JP2024504475A (ja) * 2021-01-28 2024-01-31 南京▲樺▼冠生物技▲術▼有限公司 複合体およびその使用
JP2024514880A (ja) 2021-04-14 2024-04-03 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 Ｐｎｐｌａ３発現を調節するための組成物及び方法
AU2022384619A1 (en) 2021-11-11 2024-04-11 F. Hoffmann-La Roche Ag Pharmaceutical combinations for treatment of hbv
EP4441218A2 (en) 2021-12-01 2024-10-09 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating apoc3 expression
IL315546A (en) 2022-03-16 2024-11-01 Empirico Inc GALNAC compositions to improve siRNA bioavailability
CA3245064A1 (en) 2022-04-15 2023-10-19 Dicerna Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF MODULATING THE ACTIVITY OF STRUCTURAL AND CYTOSKELETON-ASSOCIATED PROTEINS
CN120112641A (zh) * 2022-10-31 2025-06-06 大睿生物医药科技(上海)有限公司 向细胞内递送siRNA的前药
CN115925586A (zh) * 2022-11-01 2023-04-07 青岛蓝谷多肽生物医药科技有限公司 一种靶向psma的母体及其衍生物的制备方法
WO2024097310A2 (en) * 2022-11-01 2024-05-10 Impilo Therapeutics, Inc. Targeted non-charged-nucleic acid (ncna) delivery and related tumor penetrating nanocomplexes
TW202430637A (zh) 2022-11-16 2024-08-01 美商戴瑟納製藥股份有限公司 Stat3靶向性寡核苷酸及其用途
CN118852442A (zh) 2023-04-26 2024-10-29 上海麦科思生物医药有限公司 抗ptk7抗体及其用途
CN116925160B (zh) * 2023-09-15 2023-12-08 天津全和诚科技有限责任公司 一种GalNAc含糖环中间体及其制备方法

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120040459A1 (en) * 2008-09-22 2012-02-16 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
US20120142763A1 (en) * 2009-06-01 2012-06-07 The Regents Of The University Of California Nucleic acid delivery compositions and methods of use thereof
WO2014031575A1 (en) * 2012-08-20 2014-02-27 The Regents Of The University Of California Polynucleotides having bioreversible groups

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7547684B2 (en) * 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
WO2015069932A1 (en) * 2013-11-06 2015-05-14 Solstice Biologics, Ltd. Polynucleotide constructs having disulfide groups

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120040459A1 (en) * 2008-09-22 2012-02-16 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
US20120142763A1 (en) * 2009-06-01 2012-06-07 The Regents Of The University Of California Nucleic acid delivery compositions and methods of use thereof
WO2014031575A1 (en) * 2012-08-20 2014-02-27 The Regents Of The University Of California Polynucleotides having bioreversible groups

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
CURR. PROTOC. NUCLEIC ACID CHEM., 2008, VOL.35, 4.33.1-4.33.21, JPN6019028553, ISSN: 0004082367 *

Cited By (8)

* Cited by examiner, † Cited by third party
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JPWO2019039403A1 (ja) * 2017-08-22 2020-11-26 国立大学法人東海国立大学機構 修飾ポリヌクレオチド
JP7282379B2 (ja) 2017-08-22 2023-05-29 国立大学法人東海国立大学機構 修飾ポリヌクレオチド
JP2021509402A (ja) * 2017-12-29 2021-03-25 スーチョウ リボ ライフ サイエンス カンパニー、リミテッドＳｕｚｈｏｕ Ｒｉｂｏ Ｌｉｆｅ Ｓｃｉｅｎｃｅ Ｃｏ．， Ｌｔｄ． 複合体及びその調製方法と使用
JP7436030B2 (ja) 2017-12-29 2024-02-21 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 複合体及びその調製方法と使用
JP2022515503A (ja) * 2018-12-28 2022-02-18 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
JP7507495B2 (ja) 2018-12-28 2024-06-28 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
JP2022546055A (ja) * 2019-08-29 2022-11-02 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 化合物、薬物複合体及びその調製方法と使用
WO2023113038A1 (ja) * 2021-12-17 2023-06-22 リードファーマ株式会社 オリゴヌクレオチドの製造方法

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