CL2021000203A1 - Oligómeros que omiten exones para la distrofia muscular - Google Patents

Oligómeros que omiten exones para la distrofia muscular

Info

Publication number
CL2021000203A1
CL2021000203A1 CL2021000203A CL2021000203A CL2021000203A1 CL 2021000203 A1 CL2021000203 A1 CL 2021000203A1 CL 2021000203 A CL2021000203 A CL 2021000203A CL 2021000203 A CL2021000203 A CL 2021000203A CL 2021000203 A1 CL2021000203 A1 CL 2021000203A1
Authority
CL
Chile
Prior art keywords
exon
muscular dystrophy
skipping
oligomers
antisense oligomers
Prior art date
Application number
CL2021000203A
Other languages
English (en)
Inventor
Frederick Joseph Schnell
Anat Sommelet
Original Assignee
Sarepta Therapeutics Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of CL2021000203A1 publication Critical patent/CL2021000203A1/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Abstract

Se describen oligómeros antisentido complementarios a un sitio diana seleccionado en el gen de la distrofina humana para inducir la omisión del exón 2. En varios aspectos, los oligómeros antisentido se describen de acuerdo con la Fórmula (I), o una sal farmacéuticamente aceptable de los mismos, en donde T, Nu, n y R100 se definen en la presente descripción.
CL2021000203A 2018-07-27 2021-01-26 Oligómeros que omiten exones para la distrofia muscular CL2021000203A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862711215P 2018-07-27 2018-07-27
US201962868003P 2019-06-28 2019-06-28

Publications (1)

Publication Number Publication Date
CL2021000203A1 true CL2021000203A1 (es) 2021-08-13

Family

ID=67770557

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2021000203A CL2021000203A1 (es) 2018-07-27 2021-01-26 Oligómeros que omiten exones para la distrofia muscular

Country Status (16)

Country Link
US (1) US20220364084A1 (es)
EP (1) EP3830266A1 (es)
JP (2) JP2021531009A (es)
KR (1) KR20210038581A (es)
CN (1) CN112639097A (es)
AU (1) AU2019309394A1 (es)
BR (1) BR112021001575A2 (es)
CA (1) CA3105668A1 (es)
CL (1) CL2021000203A1 (es)
CO (1) CO2021001886A2 (es)
IL (1) IL280299A (es)
MX (1) MX2021000813A (es)
SA (1) SA521421104B1 (es)
SG (1) SG11202012679XA (es)
TW (1) TW202020153A (es)
WO (1) WO2020023688A1 (es)

Family Cites Families (76)

* 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
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
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
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. 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
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
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
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
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
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
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
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
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
KR100782896B1 (ko) 1999-05-04 2007-12-06 엑시콘 에이/에스 Ｌ-리보-ｌｎａ 유사체
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
WO2001047496A1 (en) 1999-12-29 2001-07-05 Mixson A James Histidine copolymer and methods for using same
KR20020097241A (ko) * 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 스플라이스-영역 안티센스 조성물 및 방법
JP4836366B2 (ja) * 2000-08-25 2011-12-14 雅文 松尾 デュシェンヌ型筋ジストロフィー治療剤
EP1446412B1 (en) 2001-09-04 2012-03-07 Exiqon A/S Novel 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
WO2004043977A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations
CA2523672C (en) 2003-04-29 2012-07-17 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
ES2694726T3 (es) 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
JP2010533170A (ja) 2007-07-12 2010-10-21 プロセンサ テクノロジーズ ビー．ブイ． 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子
EP2203173B1 (en) * 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US8076476B2 (en) 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
WO2009064471A1 (en) 2007-11-15 2009-05-22 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
JP2012523225A (ja) 2009-04-10 2012-10-04 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 疾患の処置のためのトリシクロ−ｄｎａアンチセンスオリゴヌクレオチド、組成物及び方法
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
US8470987B2 (en) 2009-09-16 2013-06-25 Chiralgen, Ltd. Protective group for synthesis of RNA and derivative
CN105838714B (zh) * 2009-11-12 2020-07-17 西澳大利亚大学 反义分子和治疗疾病的方法
WO2011078797A2 (en) * 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
KR102339196B1 (ko) 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
DK2581448T3 (en) 2011-10-13 2015-04-27 Ass Inst De Myologie Tricyclo-DNA phosphorothioate
AU2012345638C1 (en) 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
CN104203289B (zh) 2012-01-27 2020-11-03 比奥马林技术公司 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
DE102012101676A1 (de) 2012-02-29 2013-08-29 Klaus-Dieter Rösler Verfahren und Vorrichtung zum Bearbeiten von Formularen mit einer Datenverarbeitungsanlage
EP2872485B1 (en) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Asymmetric auxiliary group
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
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
WO2015108047A1 (ja) 2014-01-15 2015-07-23 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
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
AU2015207773B2 (en) 2014-01-16 2021-06-17 Wave Life Sciences Ltd. Chiral design
EP3143141B1 (en) * 2014-05-16 2019-10-02 Oregon State University Antisense antibacterial compounds and methods
CA2957661A1 (en) * 2014-08-09 2016-02-18 Kevin FLANIGAN Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
WO2016070166A2 (en) 2014-11-02 2016-05-06 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
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
CR20180233A (es) 2015-10-09 2018-05-25 Wave Life Sciences Ltd Composiciones oligonucleotídicas y sus métodos
CN107177593B (zh) * 2016-03-10 2020-10-23 北京大学 利用优化的基因密码子扩展系统通读提前终止密码子疾病中的截短蛋白
KR102329187B1 (ko) * 2016-04-29 2021-11-22 사렙타 쎄러퓨틱스, 인코퍼레이티드 인간 lmna를 표적화하는 올리고뉴클레오타이드 유사체
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs

Also Published As

Publication number Publication date
SA521421104B1 (ar) 2023-03-21
CO2021001886A2 (es) 2021-05-31
KR20210038581A (ko) 2021-04-07
AU2019309394A1 (en) 2021-03-18
JP2024023406A (ja) 2024-02-21
CN112639097A (zh) 2021-04-09
US20220364084A1 (en) 2022-11-17
SG11202012679XA (en) 2021-02-25
IL280299A (en) 2021-03-25
JP2021531009A (ja) 2021-11-18
CA3105668A1 (en) 2020-01-30
EP3830266A1 (en) 2021-06-09
MX2021000813A (es) 2021-06-15
WO2020023688A1 (en) 2020-01-30
TW202020153A (zh) 2020-06-01
BR112021001575A2 (pt) 2021-05-04

Similar Documents

Publication Publication Date Title
CY1122821T1 (el) Παραγωγα 1,3-βενζοδιοξολιου ως αναστολεις εζη1 ή/και εζη2
EA202090744A1 (ru) Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
NZ755438A (en) Exon skipping oligomer conjugates for muscular dystrophy
BR112021022659A2 (pt) Compostos triarílicos para o tratamento de doenças pd-l1
NZ755422A (en) Exon skipping oligomer conjugates for muscular dystrophy
NZ725008A (en) Compounds for treating spinal muscular atrophy
BR112019000006A2 (pt) oligômeros de salto do exon para distrofia muscular
CL2020003030A1 (es) Conjugados de oligómeros de salto de exón para la distrofia muscular
AR103895A1 (es) Lactamas como inhibidores de rock
AR102981A1 (es) Inhibidores de la necrosis celular y métodos de preparación de los mismos
MD4626C1 (ro) Derivaţi de tamoxifen pentru tratamentul afecţiunilor neoplazice, în special cu nivel înalt de proteină HER2
EA201691600A1 (ru) Дигидропиридиноновые ингибиторы mgat2 для применения в лечении метаболических нарушений
CO2021008891A2 (es) Conjugados de oligómeros de salto de exones para distrofia muscular
DOP2020000160A (es) Moduladores de sting (estimulador de genes de interferón) a base de ciclopentano
CO2017009994A2 (es) Nuevos compuestos y derivados de sulfonimidoilpurinona para el tratamiento y profilaxis de infecciones víricas
WO2019241385A3 (en) Exon skipping oligomers for muscular dystropy
MX2016015247A (es) Derivados de benceno-sulfonamida y su uso como moduladores del receptor c huerfano relacionado a acido retinoico (rorc).
CU20160140A7 (es) CROMENE Y 1,1 A,2,7B- tetrahidrociclopropa[C]CROMENE PIRIDOPIRAZINEDIONES COMO MODULADORES DE GAMMA-SECRETASA
EA201691760A1 (ru) Тритерпеноиды с активностью ингибиторов созревания вич, замещенные в положении 3 неароматическим кольцом, имеющим галогеналкильный заместитель
CO2019011967A2 (es) Compuestos antitumorales
SV2017005354A (es) Derivados de quinolizinona como inhibidores de pi3k
CY1121945T1 (el) Προφυλακτικος και θεραπευτικος παραγοντας της ινωσης
ECSP22005485A (es) Compuestos macrocíclicos como agonistas de sting y métodos y usos de los mismos
MX2022005024A (es) Profarmacos de fosforo de estimuladores de guanilato ciclasa soluble (sgc).
CO2017009989A2 (es) Piridopirimidinonas y utilización de las mismas como moduladores de receptores de nmda