JP2022511055A5 - - Google Patents

Info

Publication number
JP2022511055A5
JP2022511055A5 JP2021531988A JP2021531988A JP2022511055A5 JP 2022511055 A5 JP2022511055 A5 JP 2022511055A5 JP 2021531988 A JP2021531988 A JP 2021531988A JP 2021531988 A JP2021531988 A JP 2021531988A JP 2022511055 A5 JP2022511055 A5 JP 2022511055A5
Authority
JP
Japan
Prior art keywords
antisense oligomer
seq
oligomer according
pharmaceutically acceptable
cell membrane
Prior art date
Legal status (The legal status 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 status listed.)
Granted
Application number
JP2021531988A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022511055A (ja
JP7658900B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/065581 external-priority patent/WO2020123574A1/en
Publication of JP2022511055A publication Critical patent/JP2022511055A/ja
Publication of JP2022511055A5 publication Critical patent/JP2022511055A5/ja
Priority to JP2024028361A priority Critical patent/JP2024051108A/ja
Application granted granted Critical
Publication of JP7658900B2 publication Critical patent/JP7658900B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021531988A 2018-12-13 2019-12-11 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート Active JP7658900B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024028361A JP2024051108A (ja) 2018-12-13 2024-02-28 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862779028P 2018-12-13 2018-12-13
US62/779,028 2018-12-13
PCT/US2019/065581 WO2020123574A1 (en) 2018-12-13 2019-12-11 Exon skipping oligomer conjugates for muscular dystrophy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024028361A Division JP2024051108A (ja) 2018-12-13 2024-02-28 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

Publications (3)

Publication Number Publication Date
JP2022511055A JP2022511055A (ja) 2022-01-28
JP2022511055A5 true JP2022511055A5 (https=) 2022-12-12
JP7658900B2 JP7658900B2 (ja) 2025-04-08

Family

ID=69570813

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021531988A Active JP7658900B2 (ja) 2018-12-13 2019-12-11 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
JP2024028361A Pending JP2024051108A (ja) 2018-12-13 2024-02-28 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024028361A Pending JP2024051108A (ja) 2018-12-13 2024-02-28 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート

Country Status (17)

Country Link
US (3) US20200190516A1 (https=)
EP (1) EP3894558A1 (https=)
JP (2) JP7658900B2 (https=)
KR (1) KR20210104759A (https=)
CN (1) CN113412330A (https=)
AR (1) AR117320A1 (https=)
AU (2) AU2019397461A1 (https=)
BR (1) BR112021011018A2 (https=)
CA (1) CA3122281A1 (https=)
CL (1) CL2021001523A1 (https=)
CO (1) CO2021008891A2 (https=)
EA (1) EA202191601A1 (https=)
IL (3) IL318474A (https=)
MX (1) MX2021006737A (https=)
SG (1) SG11202104960PA (https=)
TW (1) TW202035695A (https=)
WO (1) WO2020123574A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114901823A (zh) 2019-12-26 2022-08-12 日本新药株式会社 诱导外显子50的跳读的反义核酸
CN119013401A (zh) * 2022-03-17 2024-11-22 萨勒普塔医疗公司 二氨基磷酸酯吗啉代寡聚物缀合物
WO2025212708A2 (en) * 2024-04-03 2025-10-09 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 50 skipping

Family Cites Families (72)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH445129A (fr) 1964-04-29 1967-10-15 Nestle Sa Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé
US3459731A (en) 1966-12-16 1969-08-05 Corn Products Co Cyclodextrin polyethers and their production
US3453257A (en) 1967-02-13 1969-07-01 Corn Products Co Cyclodextrin with cationic properties
US3426011A (en) 1967-02-13 1969-02-04 Corn Products Co Cyclodextrins with anionic properties
US3453259A (en) 1967-03-22 1969-07-01 Corn Products Co Cyclodextrin polyol ethers and their oxidation products
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
WO1986005519A1 (en) 1985-03-15 1986-09-25 James Summerton Polynucleotide assay reagent and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
KR0166088B1 (ko) 1990-01-23 1999-01-15 . 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
WO1999042091A2 (en) 1998-02-19 1999-08-26 Massachusetts Institute Of Technology Use of polycations as endosomolytic agents
US6683173B2 (en) 1998-04-03 2004-01-27 Epoch Biosciences, Inc. Tm leveling methods
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
WO2001047496A1 (en) 1999-12-29 2001-07-05 Mixson A James Histidine copolymer and methods for using same
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
CA2459347C (en) 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
KR100464261B1 (ko) 2002-01-24 2005-01-03 주식회사 파나진 Pna 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
KR20030084444A (ko) 2002-04-26 2003-11-01 주식회사 파나진 Pna 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
AU2003287464A1 (en) 2002-11-05 2004-06-03 Isis Pharmaceuticals, Inc. 2'-fluoro substituted oligomeric compounds and compositions for use in gene modulations
US7468418B2 (en) 2003-04-29 2008-12-23 Avi Biopharma., Inc. Compositions for enhancing transport of molecules into cells
US7211668B2 (en) 2003-07-28 2007-05-01 Panagene, Inc. PNA monomer and precursor
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2009005793A2 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
CA2693742A1 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
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
RU2606627C2 (ru) 2007-11-15 2017-01-10 Серепта Терапьютикс,Инк. Способ синтеза морфолиновых олигомеров
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
BR122020021379B1 (pt) * 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
CN102282155B (zh) 2008-12-02 2017-06-09 日本波涛生命科学公司 磷原子修饰的核酸的合成方法
CA2758189C (en) 2009-04-10 2020-12-29 Association Institut De Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
ES2586683T3 (es) 2009-09-16 2016-10-18 Wave Life Sciences Japan, Inc. Grupo protector novedoso para sintetizar ARN y derivados del mismo
TR201816523T4 (tr) * 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
EP2672977A1 (en) * 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CA3092114A1 (en) 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
PL2581448T3 (pl) 2011-10-13 2015-08-31 Association Inst De Myologie Tricyklo-tiofosforanowy DNA
JP6496549B2 (ja) 2011-11-30 2019-04-03 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
CA2861247C (en) * 2011-12-28 2021-11-16 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
NZ627896A (en) 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
DE102012101676A1 (de) 2012-02-29 2013-08-29 Klaus-Dieter Rösler Verfahren und Vorrichtung zum Bearbeiten von Formularen mit einer Datenverarbeitungsanlage
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
HK1216902A1 (zh) * 2012-12-20 2016-12-09 Sarepta Therapeutics, Inc. 用作治疗肌肉萎缩的改进外显子跳跃组合物
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
AU2015338923B2 (en) 2014-11-02 2021-10-21 Arcturus Therapeutics, Inc. Messenger UNA molecules and uses thereof
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
EP3858993A1 (en) * 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
CN109462995A (zh) 2016-05-04 2019-03-12 波涛生命科学有限公司 生物活性剂的方法和组合物
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS

Similar Documents

Publication Publication Date Title
JP2021526796A5 (https=)
Sugiyama et al. Chiral peptide nucleic acids with a substituent in the N-(2-aminoethy) glycine backbone
ES2857702T3 (es) Oligonucleótidos para la reducción de la expresión de PD-L1
JP2018110601A5 (https=)
JP2022511055A5 (https=)
CN102256999B (zh) 向白细胞的有效转运
JP2012505250A5 (https=)
JP2022036182A (ja) B型肝炎感染症治療用のPAPD5又はPAPD7のmRNA減少用核酸分子
JP2023123491A5 (https=)
JP2021526017A5 (https=)
JP2020511943A5 (https=)
JP2016502858A5 (https=)
Le Grice Targeting the HIV RNA genome: high-hanging fruit only needs a longer ladder
JP2018530560A5 (https=)
JP2014520120A5 (https=)
JP2006506942A5 (https=)
RU2015119411A (ru) Конъюгаты антисмысловых соединений, направленные на аполипопротеин в
RU2007139915A (ru) Haemophilus influenzae типа в
RU2012139432A (ru) Полипептиды
JP2021500016A5 (https=)
JP2010533171A5 (https=)
JP2025508337A5 (https=)
JP2018502956A5 (https=)
JP2008525033A5 (https=)
JP2012505657A5 (https=)