JP2014515762A5 - - Google Patents

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JP2014515762A5
JP2014515762A5 JP2014509281A JP2014509281A JP2014515762A5 JP 2014515762 A5 JP2014515762 A5 JP 2014515762A5 JP 2014509281 A JP2014509281 A JP 2014509281A JP 2014509281 A JP2014509281 A JP 2014509281A JP 2014515762 A5 JP2014515762 A5 JP 2014515762A5
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conjugate
occurrence
independently
amino acid
carrier peptide
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JP2014509281A
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Japanese (ja)
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JP2014515762A (ja
JP6478632B2 (ja
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Priority claimed from US13/101,942 external-priority patent/US20110269665A1/en
Priority claimed from US13/107,528 external-priority patent/US9238042B2/en
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Priority claimed from PCT/US2011/061282 external-priority patent/WO2012150960A1/en
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Publication of JP2014515762A5 publication Critical patent/JP2014515762A5/ja
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JP2014509281A 2011-05-05 2011-11-17 ペプチドオリゴヌクレオチドコンジュゲート Active JP6478632B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US13/101,942 US20110269665A1 (en) 2009-06-26 2011-05-05 Compound and method for treating myotonic dystrophy
US13/101,942 2011-05-05
US13/107,528 2011-05-13
US13/107,528 US9238042B2 (en) 2010-05-13 2011-05-13 Antisense modulation of interleukins 17 and 23 signaling
PCT/US2011/061282 WO2012150960A1 (en) 2011-05-05 2011-11-17 Peptide oligonucleotide conjugates

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2016151179A Division JP2016185991A (ja) 2011-05-05 2016-08-01 ペプチドオリゴヌクレオチドコンジュゲート
JP2018108772A Division JP2018135396A (ja) 2011-05-05 2018-06-06 ペプチドオリゴヌクレオチドコンジュゲート

Publications (3)

Publication Number Publication Date
JP2014515762A JP2014515762A (ja) 2014-07-03
JP2014515762A5 true JP2014515762A5 (ko) 2015-01-29
JP6478632B2 JP6478632B2 (ja) 2019-03-06

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JP2014509281A Active JP6478632B2 (ja) 2011-05-05 2011-11-17 ペプチドオリゴヌクレオチドコンジュゲート
JP2016151179A Pending JP2016185991A (ja) 2011-05-05 2016-08-01 ペプチドオリゴヌクレオチドコンジュゲート
JP2018108772A Withdrawn JP2018135396A (ja) 2011-05-05 2018-06-06 ペプチドオリゴヌクレオチドコンジュゲート
JP2020070566A Active JP6884250B2 (ja) 2011-05-05 2020-04-09 ペプチドオリゴヌクレオチドコンジュゲート
JP2021080358A Pending JP2021113232A (ja) 2011-05-05 2021-05-11 ペプチドオリゴヌクレオチドコンジュゲート
JP2024014898A Pending JP2024032974A (ja) 2011-05-05 2024-02-02 ペプチドオリゴヌクレオチドコンジュゲート

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JP2016151179A Pending JP2016185991A (ja) 2011-05-05 2016-08-01 ペプチドオリゴヌクレオチドコンジュゲート
JP2018108772A Withdrawn JP2018135396A (ja) 2011-05-05 2018-06-06 ペプチドオリゴヌクレオチドコンジュゲート
JP2020070566A Active JP6884250B2 (ja) 2011-05-05 2020-04-09 ペプチドオリゴヌクレオチドコンジュゲート
JP2021080358A Pending JP2021113232A (ja) 2011-05-05 2021-05-11 ペプチドオリゴヌクレオチドコンジュゲート
JP2024014898A Pending JP2024032974A (ja) 2011-05-05 2024-02-02 ペプチドオリゴヌクレオチドコンジュゲート

Country Status (8)

Country Link
EP (1) EP2704749A1 (ko)
JP (6) JP6478632B2 (ko)
KR (3) KR102339196B1 (ko)
CN (2) CN103619356B (ko)
AU (5) AU2011367230B2 (ko)
CA (2) CA3092114A1 (ko)
IL (2) IL273838B (ko)
WO (1) WO2012150960A1 (ko)

Families Citing this family (86)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK1766010T3 (da) 2004-06-28 2011-06-06 Univ Western Australia Antisense-oligonukleotider til induktion af exon-skipping og fremgangsmåder til anvendelse deraf
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
CA2660523C (en) 2006-08-11 2019-03-19 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
KR102366851B1 (ko) 2009-11-12 2022-02-23 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
KR20140097398A (ko) * 2011-11-18 2014-08-06 사렙타 쎄러퓨틱스, 인코퍼레이티드 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트
US10066228B2 (en) 2011-11-30 2018-09-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
EP2785840B8 (en) 2011-11-30 2019-05-22 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
LT2788487T (lt) 2011-12-08 2018-09-10 Sarepta Therapeutics, Inc. Oligonukleotido analogai, nukreipti į žmogaus lmna
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
SI2841578T1 (sl) 2012-04-23 2017-12-29 Biomarin Technologies B.V. Rna modulirajoči oligonukleotidi z izboljšanimi karakteristikami za zdravljenje nevromuskularnih motenj
JP2016502858A (ja) 2012-12-20 2016-02-01 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物
WO2014113540A1 (en) 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
WO2014153220A2 (en) * 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
LT2970964T (lt) 2013-03-14 2019-04-25 Sarepta Therapeutics, Inc. Kompozicijos su egzono praleidimu, skirtos raumenų distrofijos gydymui
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
EP3015467A4 (en) 2013-05-24 2016-11-02 Ajinomoto Kk PROCESS FOR THE PRODUCTION OF MORPHOLINO-OLIGONUCLEOTIDE
AU2014317961B2 (en) * 2013-09-05 2020-07-30 Murdoch University Antisense-induced exon2 inclusion in acid alpha-glucosidase
ES2818073T3 (es) 2013-12-24 2021-04-09 Sentiss Pharma Private Ltd Solución oftálmica de tartrato de brimonidina tópica
RU2702424C2 (ru) * 2014-03-12 2019-10-08 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
KR101661277B1 (ko) * 2014-03-17 2016-09-30 제주광어주식회사 한 개의 뉴크레오타이드 염기 변화를 통한 약독화 바이러스성 출혈패혈증 바이러스
CA2948373A1 (en) 2014-05-16 2015-11-19 Oregon State University Antisense antibacterial compounds and methods
CA2948568A1 (en) 2014-05-19 2015-11-26 David Greenberg Antisense antibacterial compounds and methods
AU2015372560B2 (en) 2014-12-31 2021-12-02 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
MX2017011004A (es) * 2015-02-27 2018-02-09 Sarepta Therapeutics Inc Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida.
MA41795A (fr) * 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
KR20240035901A (ko) * 2015-05-19 2024-03-18 사렙타 쎄러퓨틱스 인코퍼레이티드 펩티드 올리고뉴클레오티드 콘주게이트
EP3302497A4 (en) 2015-06-01 2019-01-16 Sarepta Therapeutics, Inc. EXCLUSION OF EXON INDUCED PAT TECHNOLOGY ANTISENSE IN COLLAGEN TYPE VII
EP3302489A4 (en) 2015-06-04 2019-02-06 Sarepta Therapeutics, Inc. METHOD AND COMPOUNDS FOR THE TREATMENT OF DISEASES AND SUFFERING IN CONNECTION WITH LYMPHOCYTES
UA123995C2 (uk) * 2015-08-05 2021-07-07 ЕЙСАЙ Ар ЕНД Ді МЕНЕДЖМЕНТ КО., ЛТД. Хіральні реагенти для одержання гомогенних олігомерів
US10905709B2 (en) * 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
KR101841241B1 (ko) * 2015-09-10 2018-03-22 한양대학교 산학협력단 피부 질환에 대한 예방 또는 치료효과를 갖는 펩티드 융합체 및 이를 유효성분으로 하는 약학 조성물
EP3359668A4 (en) 2015-10-09 2019-06-05 Sarepta Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF
GB2545898B (en) * 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
JP7393121B2 (ja) 2015-12-23 2023-12-06 オレゴン ステイト ユニバーシティー アンチセンス抗菌化合物および方法
CA3010084A1 (en) 2015-12-23 2017-06-29 Oregon State University Antisense antibacterial compounds and methods
EP3445405A4 (en) * 2016-04-18 2019-12-18 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERS AND METHOD FOR USE THEREOF TO TREAT DISEASES RELATING TO THE ACID ALPHA GLUCOSIDASE GENE
BR112018072279A2 (pt) * 2016-04-29 2019-02-12 Sarepta Therapeutics, Inc. análogos de oligonucleotídeo tendo como alvo lmna humana
CA3024153A1 (en) * 2016-06-30 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
CA3025575A1 (en) * 2016-06-30 2018-01-04 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
IL267246B2 (en) 2016-12-19 2023-03-01 Sarepta Therapeutics Inc Exon-skipping oligomer conjugates for muscular dystrophy
KR102639633B1 (ko) * 2016-12-19 2024-02-26 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
NZ755416A (en) 2016-12-19 2023-05-26 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
TW201919682A (zh) 2017-08-08 2019-06-01 西班牙商阿爾米雷爾有限公司 活化Nrf2路徑的新穎化合物
EA201991450A1 (ru) * 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
EP3684933A4 (en) * 2017-09-22 2021-06-23 The Regents of the University of Colorado, A Body Corporate OLIGONUCLEOTIDES THIOMORPHOLINO FOR THE TREATMENT OF MUSCLE DYSTROPHY
WO2019067975A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
WO2019067981A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
EP3687519A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
EP3697422A4 (en) * 2017-10-17 2021-08-11 Sarepta Therapeutics, Inc. CELL-PENETANT PEPTIDES FOR ANTISENSE RELEASE
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
EP3784248A4 (en) * 2018-04-26 2022-08-10 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATE FOR MUSCLE DYSTROPHY
EP3806868A4 (en) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
US20220251551A1 (en) * 2018-06-13 2022-08-11 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
TW202020153A (zh) 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
EP3829597A4 (en) * 2018-07-30 2022-05-18 Sarepta Therapeutics, Inc. TRIMER PEPTIDES FOR ANTISENSE ADMINISTRATION
GB201812980D0 (en) * 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
WO2020115494A1 (en) * 2018-12-07 2020-06-11 Oxford University Innovation Limited Linkers
EP3894558A1 (en) 2018-12-13 2021-10-20 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
US20220193246A1 (en) 2019-04-18 2022-06-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
CN110724180B (zh) * 2019-10-17 2021-08-20 山东大学 一种抑制新生血管生成的多肽及其应用
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
AU2021224412A1 (en) 2020-02-22 2022-09-15 Jcr Pharmaceuticals Co., Ltd. Human transferrin receptor binding peptide
KR20220145865A (ko) 2020-02-28 2022-10-31 니뽄 신야쿠 가부시키가이샤 엑손 51의 스키핑을 유도하는 안티센스 핵산
KR20220148230A (ko) 2020-02-28 2022-11-04 아이오니스 파마수티컬즈, 인코포레이티드 Smn2를 조절하기 위한 화합물 및 방법
US20240024414A1 (en) * 2020-09-21 2024-01-25 Icahn School Of Medicine At Mount Sinai Archaea l30 proteins as universal influenza virus therapeutics
WO2022140535A1 (en) 2020-12-23 2022-06-30 Sarepta Therapeutics, Inc. Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
TW202307208A (zh) 2021-06-23 2023-02-16 日商日本新藥股份有限公司 反義寡聚物之組合
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
WO2023282345A1 (ja) 2021-07-08 2023-01-12 日本新薬株式会社 腎毒性軽減剤
AU2022306542A1 (en) 2021-07-08 2024-01-18 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
WO2023026994A1 (ja) 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド-薬物コンジュゲート
CA3229962A1 (en) 2021-08-24 2023-03-02 Peptidream Inc. Human transferrin receptor-binding antibody-peptide conjugate
WO2023055774A1 (en) 2021-09-30 2023-04-06 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
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
WO2024097822A1 (en) 2022-11-02 2024-05-10 Sarepta Therapeutics, Inc. Formulation of an antisense oligomer conjugate

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
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
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
ATE171185T1 (de) 1985-03-15 1998-10-15 Antivirals Inc Immunotestmittel für polynukleotid und verfahren
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
WO1993001286A2 (en) 1991-06-28 1993-01-21 Massachusetts Institute Of Technology Localized oligonucleotide therapy
DK0656950T3 (da) 1992-08-21 1999-07-19 Biogen Inc TAT-afledte transportpolypeptider
JPH0915828A (ja) 1995-07-03 1997-01-17 Fuji Photo Film Co Ltd 写真処理システム用ペーパーカッター
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
BR9809138A (pt) 1997-05-21 2001-08-28 Trustees For The Leland Stanfo Conjugado e método para aumentar o transporte de um composto selecionado atravessando uma membrana biológica
WO1999005302A1 (en) * 1997-07-24 1999-02-04 The Perkin-Elmer Corporation Conjugates of transporter peptides and nucleic acid analogs, and their use
DE19933492B4 (de) * 1999-07-16 2008-01-10 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Konjugat zur Vermittlung eines zell-, kompartiment- oder membranspezifischen Transports von Wirksubstanzen, Verfahren zu dessen Herstellung und dessen Verwendung
US20020009491A1 (en) 2000-02-14 2002-01-24 Rothbard Jonathan B. Compositions and methods for enhancing drug delivery across biological membranes and tissues
DE60233137D1 (de) 2001-02-16 2009-09-10 Univ R Transporter mit beabstandeten arginin-teilchen
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
WO2004097017A2 (en) 2003-04-29 2004-11-11 Avi Biopharma, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
WO2005030800A2 (en) 2003-08-05 2005-04-07 Avi Biopharma, Inc. Oligonucleotide analog and method for treating flavivirus infections
US20050171044A1 (en) 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
MXPA06012605A (es) * 2004-05-04 2006-12-15 Nastech Pharm Co Composiciones y metodos para mejorar el suministro de acidos nucleicos en celulas y para modificar la expresion de genes objetivo en celulas.
US20050288246A1 (en) * 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
DK1766010T3 (da) 2004-06-28 2011-06-06 Univ Western Australia Antisense-oligonukleotider til induktion af exon-skipping og fremgangsmåder til anvendelse deraf
US8129352B2 (en) 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US8357664B2 (en) 2004-10-26 2013-01-22 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
US7524829B2 (en) 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
AU2006287530A1 (en) 2005-09-08 2007-03-15 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating picornavirus infection
WO2007030691A2 (en) 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US7582615B2 (en) 2006-03-07 2009-09-01 Avi Biopharma, Inc. Antisense antiviral compound and method for treating arenavirus infection
SI2735568T1 (en) 2006-05-10 2018-01-31 Sarepta Therapeutics, Inc. Analogues of the oligonucleotide, with cationic links between subunits
CA2660523C (en) 2006-08-11 2019-03-19 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
EP2560001B1 (en) 2006-09-21 2016-04-13 University of Rochester Compositions and methods related to protein displacement therapy for myotonic distrophy
JP5227803B2 (ja) 2006-11-24 2013-07-03 ハイクス ラボラトリーズ合同会社 スピロキノン化合物及び医薬組成物
WO2009005783A1 (en) * 2007-06-28 2009-01-08 Blanchette Rockefeller Neurosciences Institute Peptides, compositions and methods for reducing beta-amyloid-mediated apoptosis
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
AU2008271050B2 (en) * 2007-06-29 2014-11-06 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
CN101861318A (zh) 2007-11-15 2010-10-13 Avi生物制药公司 合成吗啉代低聚物的方法
RU2010129761A (ru) * 2007-12-20 2012-01-27 Ангиочем Инк. (Ca) Конъюгаты полипептидов с нуклеиновыми кислотами и их применение
WO2009144481A2 (en) * 2008-05-30 2009-12-03 Isis Innovation Limited Conjugates for delivery of biologically active compounds
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
WO2010072228A1 (en) * 2008-12-22 2010-07-01 Xigen S.A. Novel transporter constructs and transporter cargo conjugate molecules
WO2010120820A1 (en) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
KR20190037378A (ko) 2009-06-17 2019-04-05 바이오젠 엠에이 인코포레이티드 대상에게서 smn2 스플라이싱을 조정하기 위한 조성물 및 방법
ES2816898T3 (es) * 2010-05-13 2021-04-06 Sarepta Therapeutics Inc Compuestos que modulan la actividad de señalización de las interleucinas 17 y 23

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