JP2010505741A - カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ - Google Patents

カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ Download PDF

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JP2010505741A
JP2010505741A JP2009509880A JP2009509880A JP2010505741A JP 2010505741 A JP2010505741 A JP 2010505741A JP 2009509880 A JP2009509880 A JP 2009509880A JP 2009509880 A JP2009509880 A JP 2009509880A JP 2010505741 A JP2010505741 A JP 2010505741A
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oligomer
cationic
seq
bond
sequence
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JP2010505741A5 (enExample
Inventor
ドワイト ディー． ウェラー，
ジェド エヌ． ハッシンガー，
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エイブイアイ バイオファーマ， インコーポレイテッド
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Priority claimed from US11/431,968 external-priority patent/US8785407B2/en
Priority claimed from US11/432,216 external-priority patent/US7625873B2/en
Priority claimed from US11/432,155 external-priority patent/US20070037763A1/en
Priority claimed from US11/432,031 external-priority patent/US8084433B2/en
Priority claimed from US11/433,214 external-priority patent/US8067569B2/en
Priority claimed from US11/433,257 external-priority patent/US20070037764A1/en
Priority claimed from US11/433,213 external-priority patent/US20070004661A1/en
Priority claimed from US11/433,840 external-priority patent/US7507196B2/en
Priority claimed from US11/433,033 external-priority patent/US8415313B2/en
Priority claimed from US11/433,724 external-priority patent/US7888012B2/en
Priority claimed from US11/518,058 external-priority patent/US8329668B2/en
Priority claimed from US11/517,757 external-priority patent/US8524676B2/en
Priority claimed from US11/595,161 external-priority patent/US20070111962A1/en
Priority claimed from US11/715,572 external-priority patent/US7582615B2/en
Application filed by エイブイアイ バイオファーマ， インコーポレイテッド filed Critical エイブイアイ バイオファーマ， インコーポレイテッド
Publication of JP2010505741A publication Critical patent/JP2010505741A/ja
Publication of JP2010505741A5 publication Critical patent/JP2010505741A5/ja
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JP2009509880A 2006-05-10 2007-05-10 カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ Pending JP2010505741A (ja)

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
US11/432,031 US8084433B2 (en) 2004-09-16 2006-05-10 Antisense antiviral compound and method for treating ssRNA viral infection
US11/431,968 US8785407B2 (en) 2006-05-10 2006-05-10 Antisense antiviral agent and method for treating ssRNA viral infection
US11/432,155 US20070037763A1 (en) 2003-12-24 2006-05-10 Oligonucleotide compound and method for treating nidovirus infections
US11/432,216 US7625873B2 (en) 2004-07-02 2006-05-10 Antisense antibacterial method and compound
US80012006P 2006-05-11 2006-05-11
US80007606P 2006-05-11 2006-05-11
US80014506P 2006-05-11 2006-05-11
US79997606P 2006-05-11 2006-05-11
US11/433,840 US7507196B2 (en) 2004-11-01 2006-05-11 Antisense antiviral compounds and methods for treating a filovirus infection
US11/433,214 US8067569B2 (en) 2000-05-04 2006-05-11 Splice-region antisense composition and method
US11/433,724 US7888012B2 (en) 2005-02-09 2006-05-11 Antisense composition and method for treating muscle atrophy
US11/433,257 US20070037764A1 (en) 2003-10-23 2006-05-11 Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US11/433,033 US8415313B2 (en) 2004-01-23 2006-05-11 Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US11/433,213 US20070004661A1 (en) 2004-10-26 2006-05-11 Antisense antiviral compound and method for treating influenza viral infection
US11/517,757 US8524676B2 (en) 2005-09-08 2006-09-08 Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
US11/518,058 US8329668B2 (en) 2005-09-08 2006-09-08 Antisense antiviral compound and method for treating picornavirus infection
US11/595,161 US20070111962A1 (en) 2005-11-08 2006-11-08 Immunosuppression compound and treatment method
US11/715,572 US7582615B2 (en) 2006-03-07 2007-03-07 Antisense antiviral compound and method for treating arenavirus infection
PCT/US2007/011435 WO2008036127A2 (en) 2006-05-10 2007-05-10 Oligonucleotide analogs having cationic intersubunit linkages

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JP2010505741A true JP2010505741A (ja) 2010-02-25
JP2010505741A5 JP2010505741A5 (enExample) 2010-07-01

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US (1) US7943762B2 (enExample)
EP (2) EP2735568B8 (enExample)
JP (1) JP2010505741A (enExample)
AU (1) AU2007297861A1 (enExample)
CA (1) CA2651881A1 (enExample)
CY (2) CY1120409T1 (enExample)
DK (2) DK2024499T3 (enExample)
ES (2) ES2645410T3 (enExample)
HU (2) HUE036995T2 (enExample)
LT (2) LT2735568T (enExample)
PL (1) PL2735568T3 (enExample)
PT (1) PT2735568T (enExample)
SI (2) SI2735568T1 (enExample)
WO (1) WO2008036127A2 (enExample)

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JP2011503184A (ja) * 2007-11-15 2011-01-27 エイブイアイ バイオファーマ， インコーポレイテッド モルホリノオリゴマーの合成方法
JP2013530154A (ja) * 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
JP2015500204A (ja) * 2011-11-18 2015-01-05 サレプタ セラピューティクス， インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
JP2015504650A (ja) * 2011-11-30 2015-02-16 サレプタ セラピューティクス， インコーポレイテッド 脊髄性筋萎縮症における誘発されたエクソン包含
JP2015506669A (ja) * 2011-11-30 2015-03-05 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
WO2016060135A1 (ja) * 2014-10-14 2016-04-21 味の素株式会社 モルフォリノオリゴヌクレオチドの製造方法
US9920085B2 (en) 2012-03-20 2018-03-20 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
JP2018518148A (ja) * 2015-03-18 2018-07-12 サレプタ セラピューティクス，インコーポレイテッド ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
WO2019216433A1 (ja) * 2018-05-10 2019-11-14 日本新薬株式会社 オリゴ核酸化合物の製造方法
JP2020530981A (ja) * 2017-06-27 2020-11-05 エージェンシー フォー サイエンス，テクノロジー アンド リサーチ Ｔ細胞の機能を調節するためのアンチセンスオリゴヌクレオチド
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
US10947536B2 (en) 2011-10-04 2021-03-16 Royal Holloway And Bedford New College Oligomers
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
JP2021518361A (ja) * 2018-03-16 2021-08-02 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達用キメラペプチド

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* Cited by examiner, † Cited by third party
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AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
AU2004235396B2 (en) 2003-04-29 2010-11-25 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US20050171044A1 (en) * 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
WO2005072527A2 (en) * 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
ES2627917T3 (es) 2004-06-28 2017-08-01 The University Of Western Australia Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos
AU2005327188C9 (en) 2004-07-02 2011-09-22 Avi Biopharma, Inc. Antisense antibacterial method and compound
US8129352B2 (en) 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
WO2006086667A2 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007030691A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US20070265215A1 (en) * 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CA2691673A1 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
AU2015200600B2 (en) * 2007-06-29 2017-04-20 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
WO2009014177A1 (ja) * 2007-07-25 2009-01-29 Ajinomoto Co., Inc. ジベンゾフルベン誘導体の淘汰方法
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
US8076476B2 (en) * 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
CA2710013A1 (en) * 2007-12-28 2009-07-09 Avi Biopharma, Inc. Immunomodulatory agents and methods of use
EP3133160B1 (en) * 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Exon skipping compositions for dmd
US8592386B2 (en) * 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2011018798A2 (en) * 2009-08-14 2011-02-17 Indian Association For The Cultivation Of Science Morpholino-based antisense agent
AU2010286368B2 (en) * 2009-08-31 2015-09-03 Gen-Probe Incorporated Dengue virus assay
HUE040445T2 (hu) 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
AU2010319314C1 (en) 2009-11-13 2016-09-01 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating influenza viral infection
EP3214174B1 (en) 2010-03-04 2019-10-16 InteRNA Technologies B.V. A mirna molecule defined by its source and its diagnostic and therapeutic uses in diseases or conditions associated with emt
US9068185B2 (en) 2010-03-12 2015-06-30 Sarepta Therapeutics, Inc. Antisense modulation of nuclear hormone receptors
EP3023496B1 (en) * 2010-05-13 2020-07-29 Sarepta Therapeutics, Inc. Compounds which modulate interleukins 17 and 23 signaling activity
AU2013202731B2 (en) * 2010-05-28 2016-04-21 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
EP2591106A1 (en) 2010-07-06 2013-05-15 InteRNA Technologies B.V. Mirna and its diagnostic and therapeutic uses in diseases or conditions associated with melanoma, or in diseases or conditions associated with activated braf pathway
US8198429B2 (en) 2010-08-09 2012-06-12 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CN103154009B (zh) * 2010-09-30 2015-06-10 日本新药株式会社 吗啉基核酸衍生物
EP2474617A1 (en) 2011-01-11 2012-07-11 InteRNA Technologies BV Mir for treating neo-angiogenesis
WO2012100156A1 (en) 2011-01-20 2012-07-26 Hercules Incorporated Enhanced dry strength and drainage performance by combining glyoxalated acrylamide-containing polymers with cationic aqueous dispersion polymers
AU2011367230B2 (en) 2011-05-05 2017-08-10 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CA2839593A1 (en) 2011-07-15 2013-01-24 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
WO2013086444A2 (en) 2011-12-08 2013-06-13 Sarepta Therapeutics, Inc. Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human lmna
EP3369818B1 (en) 2011-12-22 2021-06-09 InteRNA Technologies B.V. Mirna for treating head and neck cancer
US10111962B2 (en) 2012-09-25 2018-10-30 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
US10201556B2 (en) 2012-11-06 2019-02-12 Interna Technologies B.V. Combination for use in treating diseases or conditions associated with melanoma, or treating diseases or conditions associated with activated B-raf pathway
CN106414739A (zh) 2012-12-20 2017-02-15 萨雷普塔医疗公司 用于治疗肌营养不良症的改良的外显子跳跃组合物
CN103060325B (zh) * 2013-01-14 2015-01-07 韩健宝 抗猪流行性腹泻病毒pedv的反义核酸及肽核酸pna
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3760720A1 (en) 2013-03-14 2021-01-06 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
AU2014317961B2 (en) 2013-09-05 2020-07-30 Murdoch University Antisense-induced exon2 inclusion in acid alpha-glucosidase
DK3044315T3 (da) 2013-09-11 2019-05-20 Synthena Ag Nukleinsyrer og fremgangsmåder til behandling af pompes sygdom
AR100560A1 (es) 2014-05-23 2016-10-12 Genzyme Corp Inhibición o regulación por disminución de la glucógeno sintasa mediante la creación de codones de terminación prematuros utilizando oligonucléotidos antisentido
US10849917B2 (en) 2015-06-01 2020-12-01 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type VII collagen
MX2018002090A (es) 2015-08-24 2018-09-12 Halo Bio Rnai Therapeutics Inc Nanoparticulas de polinucleótido para modulación de expresión génica y sus usos.
US20190054113A1 (en) 2015-09-30 2019-02-21 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
JP2018530560A (ja) 2015-10-09 2018-10-18 サレプタ セラピューティクス， インコーポレイテッド デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
US11060089B2 (en) 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
JP7042255B2 (ja) 2016-04-29 2022-03-25 サレプタ セラピューティクス， インコーポレイテッド ヒトｌｍｎａを標的にするオリゴヌクレオチド類縁体
MX384727B (es) * 2016-05-24 2025-03-14 Sarepta Therapeutics Inc Procesos para preparar oligómeros
AU2017330276B2 (en) * 2016-09-20 2022-01-20 The Regents Of The University Of Colorado, A Body Corporate Synthesis of backbone modified morpholino oligonucleotides and chimeras using phosphoramidite chemistry
NZ755416A (en) 2016-12-19 2023-05-26 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
US11693013B2 (en) 2017-03-10 2023-07-04 UCL Business Ltd. Method relating to myostatin pathway inhibition
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
SG11202002517RA (en) 2017-09-22 2020-04-29 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
TWI812647B (zh) 2017-09-25 2023-08-21 美商薩羅塔治療公司 經由速流合成以製備磷醯二胺嗎啉代寡聚物之製程
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
US12502432B2 (en) 2017-10-17 2025-12-23 Sarepta Therapeutics, Inc. Bicyclic peptide oligonucleotide conjugates
WO2019079386A1 (en) * 2017-10-17 2019-04-25 Sarepta Therapeutics, Inc. CELL PENETRATION PEPTIDES FOR ANTISENSE ADMINISTRATION
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
AU2018358016A1 (en) 2017-11-03 2020-05-07 Interna Technologies B.V. MiRNA molecule, equivalent, antagomir, or source thereof for treating and/or diagnosing a condition and/or a disease associated with neuronal deficiency or for neuronal (re)generation
CN118638787A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
WO2019155094A1 (en) 2018-02-12 2019-08-15 Interna Technologies B.V. Anticancer microrna and lipid formulations thereof
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
US20220098578A1 (en) 2019-01-31 2022-03-31 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
JP7581252B2 (ja) 2019-06-06 2024-11-12 アビディティー バイオサイエンシーズ，インク． Ｕｎａアミダイトおよびその使用
WO2020247782A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
EP3987029A1 (en) 2019-06-19 2022-04-27 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
IL294271A (en) 2019-12-26 2022-08-01 Nippon Shinyaku Co Ltd Antisense nucleic acid that induces skipping of exon 50
PE20230237A1 (es) 2020-02-28 2023-02-07 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido que inducen la omision del exon 51
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
KR102927945B1 (ko) 2020-03-27 2026-02-23 어비디티 바이오사이언시스 인크. 근이영양증의 치료용 조성물 및 방법
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
CN111575409B (zh) * 2020-05-23 2023-04-18 黑龙江八一农垦大学 基于整合素配体识别肽抑制猪流行性腹泻病毒感染的研究方法
AU2021382146A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
JPWO2022230974A1 (enExample) * 2021-04-28 2022-11-03
CN117279926A (zh) * 2021-04-28 2023-12-22 日本新药株式会社 低聚核酸化合物的制造方法
US12378267B2 (en) 2021-06-17 2025-08-05 Entrada Therapeutics, Inc. Synthesis of FMOC-protected morpholino monomers and oligomers
JPWO2022270585A1 (enExample) 2021-06-23 2022-12-29
TW202308664A (zh) 2021-07-08 2023-03-01 日商日本新藥股份有限公司 腎毒性減輕劑
TW202317147A (zh) 2021-07-08 2023-05-01 日商日本新藥股份有限公司 析出抑制劑
US20240316093A1 (en) 2021-07-08 2024-09-26 Nippon Shinyaku Co., Ltd. Nephrotoxicity reducing agent
WO2023034515A2 (en) 2021-09-03 2023-03-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
US20240425864A1 (en) 2021-09-30 2024-12-26 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
WO2023070086A1 (en) 2021-10-22 2023-04-27 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
US20250289851A1 (en) 2022-04-22 2025-09-18 Entrada Therapeutics, Inc. Cyclic peptides for delivering therapeutics
EP4590311A2 (en) 2022-09-21 2025-07-30 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
IL323663A (en) 2023-04-06 2025-11-01 Shanghai Argo Biopharmaceutical Co Ltd Nucleoside analog for 5'-phosphonate modification and oligonucleotide prepared therefrom
EP4702144A1 (en) 2023-04-27 2026-03-04 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of chronic kidney disease
JP2026526969A (ja) 2023-08-02 2026-08-10 サレプタ セラピューティクス， インコーポレイテッド アンチセンスオリゴマー送達のための非標準細胞透過性ペプチド
WO2025076006A2 (en) 2023-10-02 2025-04-10 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 45 skipping
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies
WO2026133078A1 (en) 2024-12-17 2026-06-25 Fondazione Telethon Ets Therapeutic strategy for treating copper-mediated pathologies
CN120058705A (zh) * 2025-04-29 2025-05-30 苏州诺维康生物科技有限公司 一种医药中间体PMO-G(N2-iBu)的合成方法

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5185444A (en) * 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
JPH05504563A (ja) * 1989-12-20 1993-07-15 アンチビラルス・インコーポレイテツド リン含有キラルインターサブユニットリンケージを有する非電荷モルホリノ―基体ポリマー
WO2002092617A1 (en) * 2001-05-17 2002-11-21 Avi Biopharma, Inc. Combined approach to treatment of cancer using a c-myc antisense oligomer
WO2005115479A2 (en) * 2004-05-24 2005-12-08 Avi Biopharma, Inc. Peptide conjugated, inosine-substituted antisense oligomer compound and method
WO2006033933A2 (en) * 2004-09-16 2006-03-30 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssrna viral infection
WO2006047683A2 (en) * 2004-10-26 2006-05-04 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection

Family Cites Families (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン，ジエ−ムス ポリヌクレオチド測定試薬と方法
US5217866A (en) * 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5852188A (en) * 1990-01-11 1998-12-22 Isis Pharmaceuticals, Inc. Oligonucleotides having chiral phosphorus linkages
JPH06501843A (ja) * 1990-08-14 1994-03-03 アイシス・ファーマシューティカルス・インコーポレーテッド アンチセンスオリゴヌクレオチドによるインフルエンザａ型ウイルス，アン・アーバｈ２ｎ２株の阻害
AU659482B2 (en) 1991-06-28 1995-05-18 Massachusetts Institute Of Technology Localized oligonucleotide therapy
US5576302A (en) * 1991-10-15 1996-11-19 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating hepatitis C virus having phosphorothioate linkages of high chiral purity
CA2125269A1 (en) * 1991-12-23 1993-07-08 Chiron Diagnostics Corporation Hav probes for use in solution phase sandwich hybridization assays
US6174868B1 (en) * 1992-09-10 2001-01-16 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of hepatitis C virus-associated diseases
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US6060456A (en) * 1993-11-16 2000-05-09 Genta Incorporated Chimeric oligonucleoside compounds
US5734039A (en) * 1994-09-15 1998-03-31 Thomas Jefferson University Antisense oligonucleotides targeting cooperating oncogenes
GB9510718D0 (en) 1995-05-26 1995-07-19 Sod Conseils Rech Applic Antisense oligonucleotides
US5955318A (en) * 1995-08-14 1999-09-21 Abbott Laboratories Reagents and methods useful for controlling the translation of hepatitis GBV proteins
US6686338B1 (en) 1996-02-23 2004-02-03 The Board Of Regents Of The University Of Nebraska Enzyme inhibitors for metabolic redirection
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
ES2210761T3 (es) * 1997-05-21 2004-07-01 The Board Of Trustees Of The Leland Stanford Junior University Composicion y procedimiento para mejorar el transporte a traves de las membranas biologicas.
AUPP398498A0 (en) 1998-06-09 1998-07-02 Silverbrook Research Pty Ltd A method of manufacture of an image creation apparatus (ijm44)
US6133246A (en) * 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
US6228579B1 (en) * 1997-11-14 2001-05-08 San Diego State University Foundation Method for identifying microbial proliferation genes
ATE250940T1 (de) 1998-07-13 2003-10-15 Univ Nebraska Zielgerichte ortsspezifische arzneimittelzusammensetzungen und verwendungen
US7094765B1 (en) 1999-01-29 2006-08-22 Avi Biopharma, Inc. Antisense restenosis composition and method
KR20010102992A (ko) * 1999-01-29 2001-11-17 추후보정 표적 ｒｎａ를 검출하는 비-침입적 방법
US20030095953A1 (en) 1999-11-12 2003-05-22 Myles C. Cabot Methods of reversing drug resistance in cancer cells
EP1238071B1 (en) * 1999-11-29 2008-04-23 Avi Biopharma, Inc. Uncharged antisense oligonucleotides targeted to bacterial 16s and 23s rrnas and their uses
WO2001049775A2 (en) * 2000-01-04 2001-07-12 Avi Biopharma, Inc. Antisense antibacterial cell division composition and method
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
AU6319701A (en) 2000-05-17 2001-11-26 Avi Biopharma Inc Antisense enzyme inhibitors for metabolic redirection
EP1301525B1 (en) * 2000-07-06 2015-09-02 Sarepta Therapeutics, Inc. Transforming growth factor beta (tgf-beta) blocking agent-treated stem cell composition and method
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
US6828105B2 (en) * 2001-10-16 2004-12-07 Avi Biopharma, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US20030207907A1 (en) 2002-05-03 2003-11-06 Iversen Patrick L. Delivery of microparticle-conjugated drugs for inhibition of stenosis
EA008940B1 (ru) * 2002-09-13 2007-10-26 Репликор, Инк. Антивирусные олигонуклеотиды, не связанные с комплементарностью последовательностей
AU2003298605B2 (en) * 2002-10-16 2007-10-18 Gen-Probe Incorporated Compositions and methods for detecting west nile virus
EP1585755B1 (en) 2002-12-31 2015-08-05 Sigma-Aldrich Co. LLC Methods and compositions for the tandem synthesis of two or more oligonuleotides on the same solid support
KR101032853B1 (ko) 2003-08-05 2011-05-06 에이브이아이 바이오파마 인코포레이티드 웨스트 나일 바이러스 감염을 치료하기 위한 약학적 조성물
US20050171044A1 (en) * 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
WO2005072527A2 (en) 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
AU2005327188C9 (en) * 2004-07-02 2011-09-22 Avi Biopharma, Inc. Antisense antibacterial method and compound
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
TW200622777A (en) * 2004-12-31 2006-07-01 Inventec Corp Manufacturing-order giving system and method thereof
WO2007030691A2 (en) 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
AU2006287530A1 (en) 2005-09-08 2007-03-15 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5185444A (en) * 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
JPH05504563A (ja) * 1989-12-20 1993-07-15 アンチビラルス・インコーポレイテツド リン含有キラルインターサブユニットリンケージを有する非電荷モルホリノ―基体ポリマー
WO2002092617A1 (en) * 2001-05-17 2002-11-21 Avi Biopharma, Inc. Combined approach to treatment of cancer using a c-myc antisense oligomer
JP2004537517A (ja) * 2001-05-17 2004-12-16 エイブイアイ バイオファーマ， インコーポレイテッド ｃ−ｍｙｃアンチセンスオリゴマーを使用した、癌を処置するための併用アプローチ
WO2005115479A2 (en) * 2004-05-24 2005-12-08 Avi Biopharma, Inc. Peptide conjugated, inosine-substituted antisense oligomer compound and method
JP2008509701A (ja) * 2004-05-24 2008-04-03 アビ バイオファーマ， インコーポレイテッド ペプチドが結合された、イノシン置換アンチセンスオリゴマー化合物および方法
WO2006033933A2 (en) * 2004-09-16 2006-03-30 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssrna viral infection
JP2008513012A (ja) * 2004-09-16 2008-05-01 アヴィ バイオファーマ、インコーポレイテッド アンチセンス抗ウイルス化合物及びｓｓＲＮＡウイルス感染を治療する方法
WO2006047683A2 (en) * 2004-10-26 2006-05-04 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection

Cited By (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011503184A (ja) * 2007-11-15 2011-01-27 エイブイアイ バイオファーマ， インコーポレイテッド モルホリノオリゴマーの合成方法
KR101829644B1 (ko) 2010-05-28 2018-02-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
JP2013530154A (ja) * 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
US10760078B2 (en) 2010-05-28 2020-09-01 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP2020018299A (ja) * 2010-05-28 2020-02-06 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
JP2021048886A (ja) * 2010-05-28 2021-04-01 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
KR101981705B1 (ko) 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
US9469664B2 (en) 2010-05-28 2016-10-18 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP7008056B2 (ja) 2010-05-28 2022-01-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
US10202602B2 (en) 2010-05-28 2019-02-12 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
KR20180019751A (ko) * 2010-05-28 2018-02-26 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
JP2018002728A (ja) * 2010-05-28 2018-01-11 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
US11072793B2 (en) 2010-09-03 2021-07-27 Sarepta Therapeutics, Inc. DsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
US10947536B2 (en) 2011-10-04 2021-03-16 Royal Holloway And Bedford New College Oligomers
US9278987B2 (en) 2011-11-18 2016-03-08 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
JP2018048208A (ja) * 2011-11-18 2018-03-29 サレプタ セラピューティクス， インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
US11208655B2 (en) 2011-11-18 2021-12-28 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
US10344281B2 (en) 2011-11-18 2019-07-09 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
US9790499B2 (en) 2011-11-18 2017-10-17 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
JP2015500204A (ja) * 2011-11-18 2015-01-05 サレプタ セラピューティクス， インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
JP2018082717A (ja) * 2011-11-30 2018-05-31 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
JP7057385B2 (ja) 2011-11-30 2022-04-19 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
JP2015506669A (ja) * 2011-11-30 2015-03-05 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
JP2015504650A (ja) * 2011-11-30 2015-02-16 サレプタ セラピューティクス， インコーポレイテッド 脊髄性筋萎縮症における誘発されたエクソン包含
JP2020078347A (ja) * 2011-11-30 2020-05-28 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
US9920085B2 (en) 2012-03-20 2018-03-20 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
US10472624B2 (en) 2014-10-14 2019-11-12 Ajinomoto Co., Inc. Morpholino oligonucleotide manufacturing method
CN106795186B (zh) * 2014-10-14 2021-03-02 味之素株式会社 吗啉代寡核苷酸的制备方法
US11028386B2 (en) 2014-10-14 2021-06-08 Ajinomoto Co., Inc. Morpholino oligonucleotide manufacturing method
JPWO2016060135A1 (ja) * 2014-10-14 2017-07-27 味の素株式会社 モルフォリノオリゴヌクレオチドの製造方法
CN106795186A (zh) * 2014-10-14 2017-05-31 味之素株式会社 吗啉代寡核苷酸的制备方法
WO2016060135A1 (ja) * 2014-10-14 2016-04-21 味の素株式会社 モルフォリノオリゴヌクレオチドの製造方法
JP2021090458A (ja) * 2015-03-18 2021-06-17 サレプタ セラピューティクス，インコーポレイテッド ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
JP2018518148A (ja) * 2015-03-18 2018-07-12 サレプタ セラピューティクス，インコーポレイテッド ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
US12121532B2 (en) 2015-08-28 2024-10-22 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
JP7284718B2 (ja) 2017-06-27 2023-05-31 エージェンシー フォー サイエンス，テクノロジー アンド リサーチ Ｔ細胞の機能を調節するためのアンチセンスオリゴヌクレオチド
JP2020530981A (ja) * 2017-06-27 2020-11-05 エージェンシー フォー サイエンス，テクノロジー アンド リサーチ Ｔ細胞の機能を調節するためのアンチセンスオリゴヌクレオチド
JP2021518361A (ja) * 2018-03-16 2021-08-02 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達用キメラペプチド
JP2024163336A (ja) * 2018-03-16 2024-11-21 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達用キメラペプチド
JP7596148B2 (ja) 2018-03-16 2024-12-09 サレプタ セラピューティクス， インコーポレイテッド アンチセンス送達用キメラペプチド
US11560401B2 (en) 2018-05-10 2023-01-24 Nippon Shinyaku Co., Ltd. Method for preparing oligonucleic acid compound
WO2019216433A1 (ja) * 2018-05-10 2019-11-14 日本新薬株式会社 オリゴ核酸化合物の製造方法

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