JP5524189B2 - 耳障害治療のための組成物および方法 - Google Patents
耳障害治療のための組成物および方法 Download PDFInfo
- Publication number
- JP5524189B2 JP5524189B2 JP2011512270A JP2011512270A JP5524189B2 JP 5524189 B2 JP5524189 B2 JP 5524189B2 JP 2011512270 A JP2011512270 A JP 2011512270A JP 2011512270 A JP2011512270 A JP 2011512270A JP 5524189 B2 JP5524189 B2 JP 5524189B2
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide
- modified
- nucleotides
- ear
- sirna
- 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.)
- Expired - Fee Related
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0046—Ear
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P39/00—General protective or antinoxious agents
-
- 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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Toxicology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13116208P | 2008-06-06 | 2008-06-06 | |
| US61/131,162 | 2008-06-06 | ||
| PCT/IL2009/000570 WO2009147684A2 (en) | 2008-06-06 | 2009-06-07 | Compositions and methods for treatment of ear disorders |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2011522036A JP2011522036A (ja) | 2011-07-28 |
| JP2011522036A5 JP2011522036A5 (https=) | 2012-07-19 |
| JP5524189B2 true JP5524189B2 (ja) | 2014-06-18 |
Family
ID=41398630
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011512270A Expired - Fee Related JP5524189B2 (ja) | 2008-06-06 | 2009-06-07 | 耳障害治療のための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (2) | US8431692B2 (https=) |
| EP (1) | EP2293800B1 (https=) |
| JP (1) | JP5524189B2 (https=) |
| WO (1) | WO2009147684A2 (https=) |
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7825099B2 (en) * | 2006-01-20 | 2010-11-02 | Quark Pharmaceuticals, Inc. | Treatment or prevention of oto-pathologies by inhibition of pro-apoptotic genes |
| WO2009001359A2 (en) | 2007-06-27 | 2008-12-31 | Quark Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of pro-apoptotic genes |
| WO2009147684A2 (en) | 2008-06-06 | 2009-12-10 | Quark Pharmaceuticals, Inc. | Compositions and methods for treatment of ear disorders |
| EP2862929B1 (en) | 2009-12-09 | 2017-09-06 | Quark Pharmaceuticals, Inc. | Compositions and methods for treating diseases, disorders or injury of the CNS |
| CA2801535C (en) | 2010-06-04 | 2017-05-30 | Hough Ear Institute | Composition and method for inner ear sensory hair cell regeneration or replacement |
| JP6194248B2 (ja) | 2010-10-28 | 2017-09-06 | パシラ ファーマシューティカルズ インコーポレーテッド | 非ステロイド性抗炎症薬の徐放性処方物 |
| BR112014002638A2 (pt) * | 2011-08-03 | 2017-02-21 | Quark Pharmaceuticals Inc | compostos de oligonucleo tídeo de fita dupla para tratar desordens de audição e equilíbrio |
| AU2012332517B9 (en) | 2011-11-03 | 2017-08-10 | Quark Pharmaceuticals, Inc. | Methods and compositions for neuroprotection |
| US20140323549A1 (en) | 2011-11-08 | 2014-10-30 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the nervous system |
| BR112014016937A2 (pt) | 2012-01-12 | 2017-06-13 | Quark Pharmaceuticals Inc | terapia de combinação para o tratamento de desordens de audição e do equilíbrio |
| ES2704855T3 (es) | 2012-09-12 | 2019-03-20 | Quark Pharmaceuticals Inc | Moléculas de oligonucleótido de doble cadena para p53 y métodos de uso de las mismas |
| EP2895607B1 (en) | 2012-09-12 | 2021-05-05 | Quark Pharmaceuticals, Inc. | Double-stranded oligonucleotide molecules to ddit4 and methods of use thereof |
| EP3031921B1 (en) | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| MX374532B (es) | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| CN105683379A (zh) | 2013-06-17 | 2016-06-15 | 布罗德研究所有限公司 | 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化 |
| EP3470089A1 (en) | 2013-12-12 | 2019-04-17 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| JP7103750B2 (ja) | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用 |
| EP3119888B1 (en) | 2014-03-19 | 2021-07-28 | Ionis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
| EP3889260A1 (en) | 2014-12-12 | 2021-10-06 | The Broad Institute, Inc. | Protected guide rnas (pgrnas) |
| WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
| CA2970370A1 (en) | 2014-12-24 | 2016-06-30 | Massachusetts Institute Of Technology | Crispr having or associated with destabilization domains |
| WO2016205749A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Novel crispr enzymes and systems |
| AU2016381174A1 (en) | 2015-12-31 | 2018-05-31 | Ionis Pharmaceuticals, Inc. | Methods for reducing Ataxin-2 expression |
| EP3541815A4 (en) | 2016-11-18 | 2020-07-15 | Pacira Pharmaceuticals, Inc. | ZINC MELXICAM COMPLEX MICROPARTICLE MULTIVESICULAR LIPOSOME FORMULATIONS AND METHODS OF MAKING THE SAME |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| WO2018127382A1 (en) * | 2017-01-03 | 2018-07-12 | Ethris Gmbh | Ornithine transcarbamylase coding polyribonucleotides and formulations thereof |
| AR115847A1 (es) | 2018-07-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Compuestos y métodos para reducir la expresión de la atxn2 |
| US12421507B2 (en) | 2018-08-20 | 2025-09-23 | The Broad Institute, Inc. | Methods and compositions for optochemical control of CRISPR-CAS9 |
| CN113544266A (zh) | 2018-12-17 | 2021-10-22 | 博德研究所 | Crispr相关转座酶系统和其使用方法 |
| MA55082A (fr) * | 2019-03-01 | 2022-01-05 | Flagship Pioneering Innovations Vi Llc | Compositions, procédés, et kits pour l'administration de polyribonucléotides |
| CN113811311A (zh) * | 2019-03-15 | 2021-12-17 | 马萨诸塞大学 | 用于组织特异性apoe调节的寡核苷酸 |
| US20220235340A1 (en) | 2019-05-20 | 2022-07-28 | The Broad Institute, Inc. | Novel crispr-cas systems and uses thereof |
| WO2021041922A1 (en) | 2019-08-30 | 2021-03-04 | The Broad Institute, Inc. | Crispr-associated mu transposase systems |
| WO2021146641A1 (en) | 2020-01-17 | 2021-07-22 | The Broad Institute, Inc. | Small type ii-d cas proteins and methods of use thereof |
| WO2025059533A1 (en) | 2023-09-13 | 2025-03-20 | The Broad Institute, Inc. | Crispr enzymes and systems |
| WO2025117544A1 (en) | 2023-11-29 | 2025-06-05 | The Broad Institute, Inc. | Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof |
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| WO2009147684A2 (en) | 2008-06-06 | 2009-12-10 | Quark Pharmaceuticals, Inc. | Compositions and methods for treatment of ear disorders |
| CA2962219C (en) | 2008-10-22 | 2020-08-25 | Quark Pharmaceuticals, Inc. | Methods for treating eye disorders |
-
2009
- 2009-06-07 WO PCT/IL2009/000570 patent/WO2009147684A2/en not_active Ceased
- 2009-06-07 EP EP09758028.6A patent/EP2293800B1/en not_active Not-in-force
- 2009-06-07 US US12/994,725 patent/US8431692B2/en not_active Expired - Fee Related
- 2009-06-07 JP JP2011512270A patent/JP5524189B2/ja not_active Expired - Fee Related
-
2013
- 2013-04-22 US US13/867,837 patent/US9089591B2/en not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| EP2293800A4 (en) | 2012-12-19 |
| EP2293800B1 (en) | 2016-10-05 |
| US20140066493A1 (en) | 2014-03-06 |
| WO2009147684A2 (en) | 2009-12-10 |
| WO2009147684A3 (en) | 2010-03-18 |
| JP2011522036A (ja) | 2011-07-28 |
| EP2293800A2 (en) | 2011-03-16 |
| US9089591B2 (en) | 2015-07-28 |
| US20110142917A1 (en) | 2011-06-16 |
| US8431692B2 (en) | 2013-04-30 |
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