JP2019514426A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2019514426A5 JP2019514426A5 JP2019508165A JP2019508165A JP2019514426A5 JP 2019514426 A5 JP2019514426 A5 JP 2019514426A5 JP 2019508165 A JP2019508165 A JP 2019508165A JP 2019508165 A JP2019508165 A JP 2019508165A JP 2019514426 A5 JP2019514426 A5 JP 2019514426A5
- Authority
- JP
- Japan
- Prior art keywords
- oligomeric compound
- antisense oligomeric
- seq
- antisense
- cpp
- 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
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022000125A JP2022046725A (ja) | 2016-04-29 | 2022-01-04 | ヒトlmnaを標的にするオリゴヌクレオチド類縁体 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662330027P | 2016-04-29 | 2016-04-29 | |
| US62/330,027 | 2016-04-29 | ||
| PCT/US2017/030174 WO2017190041A1 (en) | 2016-04-29 | 2017-04-28 | Oligonucleotide analogues targeting human lmna |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022000125A Division JP2022046725A (ja) | 2016-04-29 | 2022-01-04 | ヒトlmnaを標的にするオリゴヌクレオチド類縁体 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019514426A JP2019514426A (ja) | 2019-06-06 |
| JP2019514426A5 true JP2019514426A5 (enExample) | 2020-05-21 |
| JP7042255B2 JP7042255B2 (ja) | 2022-03-25 |
Family
ID=59285320
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019508165A Active JP7042255B2 (ja) | 2016-04-29 | 2017-04-28 | ヒトlmnaを標的にするオリゴヌクレオチド類縁体 |
| JP2022000125A Pending JP2022046725A (ja) | 2016-04-29 | 2022-01-04 | ヒトlmnaを標的にするオリゴヌクレオチド類縁体 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022000125A Pending JP2022046725A (ja) | 2016-04-29 | 2022-01-04 | ヒトlmnaを標的にするオリゴヌクレオチド類縁体 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US10822608B2 (enExample) |
| EP (1) | EP3449000A1 (enExample) |
| JP (2) | JP7042255B2 (enExample) |
| KR (1) | KR102329187B1 (enExample) |
| CN (1) | CN109477109B (enExample) |
| AU (2) | AU2017258642B2 (enExample) |
| CA (1) | CA3022303A1 (enExample) |
| CO (1) | CO2018012873A2 (enExample) |
| EA (1) | EA201892467A1 (enExample) |
| MA (1) | MA45616A (enExample) |
| MX (1) | MX2018013157A (enExample) |
| NZ (1) | NZ747685A (enExample) |
| SG (1) | SG11201809468XA (enExample) |
| WO (1) | WO2017190041A1 (enExample) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP4434589A3 (en) | 2015-10-23 | 2025-05-14 | President and Fellows of Harvard College | Evolved cas9 proteins for gene editing |
| NZ747685A (en) * | 2016-04-29 | 2023-05-26 | Sarepta Therapeutics Inc | Oligonucleotide analogues targeting human lmna |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| HRP20240705T1 (hr) | 2016-12-19 | 2024-08-30 | Sarepta Therapeutics, Inc. | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| CN110662556A (zh) | 2017-03-09 | 2020-01-07 | 哈佛大学的校长及成员们 | 癌症疫苗 |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | プレジデント アンド フェローズ オブ ハーバード カレッジ | シトシンからグアニンへの塩基編集因子 |
| WO2018176009A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| KR20200121782A (ko) | 2017-10-16 | 2020-10-26 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| TW202449155A (zh) * | 2018-07-27 | 2024-12-16 | 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 |
| WO2020051360A1 (en) * | 2018-09-05 | 2020-03-12 | The Broad Institute, Inc. | Base editing for treating hutchinson-gilford progeria syndrome |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2022098014A1 (ko) * | 2020-11-03 | 2022-05-12 | 한국생명공학연구원 | 유전자 교정을 이용한 조로증 및 자연노화의 예방 또는 치료용 조성물 |
| JP2025537104A (ja) * | 2022-11-02 | 2025-11-14 | 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コンジュゲートの製剤化 |
| CN119101680A (zh) * | 2023-06-09 | 2024-12-10 | 良渚实验室 | 多核苷酸、药物组合物及其用途 |
| WO2025262186A2 (en) * | 2024-06-20 | 2025-12-26 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Therapeutic oligonucleotides for treating diseases being associated with or caused by a missense, nonsense or in-frame indel mutation |
Family Cites Families (74)
| 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 |
| US3426011A (en) | 1967-02-13 | 1969-02-04 | Corn Products Co | Cyclodextrins with anionic properties |
| US3453257A (en) | 1967-02-13 | 1969-07-01 | Corn Products Co | Cyclodextrin with cationic 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 |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| AU5661386A (en) | 1985-03-15 | 1986-10-13 | Stirchak, E. | Stereoregular polynucleotide-binding polymers |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| 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 |
| 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 |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| WO1993001286A2 (en) | 1991-06-28 | 1993-01-21 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| WO1994002595A1 (en) | 1992-07-17 | 1994-02-03 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for treatment of animal diseases |
| 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 |
| US6245747B1 (en) | 1996-03-12 | 2001-06-12 | The Board Of Regents Of The University Of Nebraska | Targeted site specific antisense oligodeoxynucleotide delivery method |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
| WO1999042091A2 (en) | 1998-02-19 | 1999-08-26 | Massachusetts Institute Of Technology | Use of polycations as endosomolytic agents |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| AU776362B2 (en) | 1999-05-04 | 2004-09-09 | Roche Innovation Center Copenhagen A/S | L-ribo-LNA analogues |
| AU2281201A (en) | 1999-12-29 | 2001-07-09 | A. James Mixson | 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 |
| WO2004044140A2 (en) | 2002-11-05 | 2004-05-27 | Isis Pharmaceticals, Inc. | 2’-substituted oligomeric compounds and compositions for use in gene modulations |
| ES2351976T3 (es) | 2003-04-29 | 2011-02-14 | Avi Biopharma, Inc. | Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células. |
| US7211668B2 (en) | 2003-07-28 | 2007-05-01 | Panagene, Inc. | PNA monomer and precursor |
| US8258109B2 (en) | 2005-10-20 | 2012-09-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of LMNA expression |
| ES2657400T3 (es) | 2006-05-10 | 2018-03-05 | Sarepta Therapeutics, Inc. | Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos |
| CN101534643A (zh) * | 2006-09-15 | 2009-09-16 | 安佐制药股份有限公司 | 用于递送寡核苷酸的基于位阻酯的生物可降解连接体 |
| AU2008271050B2 (en) | 2007-06-29 | 2014-11-06 | Sarepta Therapeutics, Inc. | Tissue specific peptide conjugates and methods |
| WO2009008725A2 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
| CA2884340C (en) | 2007-11-15 | 2017-07-25 | Sarepta Therapeutics, Inc. | Method of synthesis of morpholino oligomers |
| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
| CN102625840A (zh) | 2009-04-10 | 2012-08-01 | 肌肉学研究协会 | 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法 |
| US20110269665A1 (en) * | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| EP2479182B8 (en) | 2009-09-16 | 2016-07-13 | Wave Life Sciences Japan, Inc. | Novel protecting group for synthesizing rna and derivative thereof |
| EP3023496B1 (en) * | 2010-05-13 | 2020-07-29 | Sarepta Therapeutics, Inc. | Compounds which modulate interleukins 17 and 23 signaling activity |
| 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 |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| PL2623507T3 (pl) | 2010-09-30 | 2017-03-31 | Nippon Shinyaku Co., Ltd. | Pochodna kwasu morfolinonukleinowego |
| JP5653160B2 (ja) | 2010-10-15 | 2015-01-14 | ダンロップスポーツ株式会社 | ゴルフボール |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| WO2012150960A1 (en) | 2011-05-05 | 2012-11-08 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
| WO2013030569A2 (en) | 2011-08-30 | 2013-03-07 | Michael John Gait | Peptides |
| ES2535654T3 (es) | 2011-10-13 | 2015-05-13 | Association Institut De Myologie | ADN triciclo-fosforotioato |
| BR112014011875B1 (pt) | 2011-11-18 | 2022-01-04 | Sarepta Therapeutics, Inc | Oligonucleotídeos funcionalmente modificados e subunidades dos mesmos |
| PT2788487T (pt) * | 2011-12-08 | 2018-07-03 | Sarepta Therapeutics Inc | Análogos de oligonucleótido visando lmna humano |
| ES2907250T3 (es) | 2012-01-27 | 2022-04-22 | Biomarin Tech Bv | Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker |
| DE102012101676A1 (de) | 2012-02-29 | 2013-08-29 | Klaus-Dieter Rösler | Verfahren und Vorrichtung zum Bearbeiten von Formularen mit einer Datenverarbeitungsanlage |
| KR20240148947A (ko) | 2012-07-13 | 2024-10-11 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| WO2014172507A1 (en) * | 2013-04-16 | 2014-10-23 | Memorial Sloan-Kettering Cancer Center | Age-modified cells and methods for making age-modified cells |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 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 |
| EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| 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 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| WO2015175977A2 (en) | 2014-05-16 | 2015-11-19 | Geller Bruce L | Antisense antibacterial compounds and methods |
| EP3569252B1 (en) * | 2014-05-19 | 2021-12-15 | Oregon State University | Antisense antibacterial compounds and methods |
| WO2015179743A1 (en) * | 2014-05-23 | 2015-11-26 | Genzyme Corporation | Library and screening approach for improved cell penetrating peptides |
| NZ747685A (en) * | 2016-04-29 | 2023-05-26 | Sarepta Therapeutics Inc | Oligonucleotide analogues targeting human lmna |
-
2017
- 2017-04-28 NZ NZ747685A patent/NZ747685A/en unknown
- 2017-04-28 EP EP17735676.3A patent/EP3449000A1/en active Pending
- 2017-04-28 KR KR1020187034615A patent/KR102329187B1/ko active Active
- 2017-04-28 JP JP2019508165A patent/JP7042255B2/ja active Active
- 2017-04-28 WO PCT/US2017/030174 patent/WO2017190041A1/en not_active Ceased
- 2017-04-28 US US16/096,524 patent/US10822608B2/en active Active
- 2017-04-28 SG SG11201809468XA patent/SG11201809468XA/en unknown
- 2017-04-28 EA EA201892467A patent/EA201892467A1/ru unknown
- 2017-04-28 MX MX2018013157A patent/MX2018013157A/es unknown
- 2017-04-28 CN CN201780040785.7A patent/CN109477109B/zh active Active
- 2017-04-28 CA CA3022303A patent/CA3022303A1/en active Pending
- 2017-04-28 AU AU2017258642A patent/AU2017258642B2/en active Active
- 2017-04-28 MA MA045616A patent/MA45616A/fr unknown
-
2018
- 2018-11-27 CO CONC2018/0012873A patent/CO2018012873A2/es unknown
-
2020
- 2020-09-17 US US17/024,100 patent/US11802283B2/en active Active
-
2022
- 2022-01-04 JP JP2022000125A patent/JP2022046725A/ja active Pending
-
2023
- 2023-11-30 AU AU2023274161A patent/AU2023274161A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019514426A5 (enExample) | ||
| JP2010527913A5 (enExample) | ||
| JP2019512478A5 (enExample) | ||
| JP2016153410A5 (enExample) | ||
| MY161450A (en) | Double-acylated glp-1 derivatives | |
| JP2020536091A5 (enExample) | ||
| JP2016527215A5 (enExample) | ||
| JP2020502049A5 (enExample) | ||
| WO2019154859A1 (en) | Fap inhibitor | |
| JP2013511553A5 (enExample) | ||
| JP2009544660A5 (enExample) | ||
| JP2009524696A5 (enExample) | ||
| JP2016505595A5 (enExample) | ||
| JP2019521139A5 (enExample) | ||
| JP2017536344A5 (enExample) | ||
| JP2017538682A5 (enExample) | ||
| JP2020503009A5 (enExample) | ||
| JP2010505741A5 (enExample) | ||
| JP2014515762A5 (enExample) | ||
| AU2019260738B2 (en) | Integrin targeting ligands and uses thereof | |
| JP2020514299A5 (enExample) | ||
| JP2020500916A5 (enExample) | ||
| MX2009010965A (es) | Péptidos de tem8 y vacunas que los contienen. | |
| CN111788191A (zh) | 用于治疗癌症的1-(哌啶基羰基甲基)-2-氧代哌嗪衍生物 | |
| JP2011521902A5 (enExample) |