JP2017515819A - ハンチントン病を処置するための方法および組成物 - Google Patents
ハンチントン病を処置するための方法および組成物 Download PDFInfo
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- JP2017515819A JP2017515819A JP2016566652A JP2016566652A JP2017515819A JP 2017515819 A JP2017515819 A JP 2017515819A JP 2016566652 A JP2016566652 A JP 2016566652A JP 2016566652 A JP2016566652 A JP 2016566652A JP 2017515819 A JP2017515819 A JP 2017515819A
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/54—Mixtures of enzymes or proenzymes covered by more than a single one of groups A61K38/44 - A61K38/46 or A61K38/51 - A61K38/53
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
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- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
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- 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
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- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- A61K39/0005—Vertebrate antigens
- A61K39/0011—Cancer antigens
- A61K39/001152—Transcription factors, e.g. SOX or c-MYC
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- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- C07K2319/00—Fusion polypeptide
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
- C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Gastroenterology & Hepatology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Marine Sciences & Fisheries (AREA)
- Psychology (AREA)
- Virology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461990521P | 2014-05-08 | 2014-05-08 | |
| US61/990,521 | 2014-05-08 | ||
| US201462051724P | 2014-09-17 | 2014-09-17 | |
| US62/051,724 | 2014-09-17 | ||
| PCT/US2015/029748 WO2015171932A1 (en) | 2014-05-08 | 2015-05-07 | Methods and compositions for treating huntington's disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017515819A true JP2017515819A (ja) | 2017-06-15 |
| JP2017515819A5 JP2017515819A5 (enExample) | 2018-04-05 |
Family
ID=54393014
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016566652A Pending JP2017515819A (ja) | 2014-05-08 | 2015-05-07 | ハンチントン病を処置するための方法および組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US11110154B2 (enExample) |
| EP (1) | EP3139966B1 (enExample) |
| JP (1) | JP2017515819A (enExample) |
| KR (1) | KR102380324B1 (enExample) |
| CN (1) | CN106413760B (enExample) |
| AU (1) | AU2015255877B2 (enExample) |
| BR (1) | BR112016025849A2 (enExample) |
| CA (1) | CA2947035A1 (enExample) |
| MX (1) | MX378789B (enExample) |
| RU (1) | RU2691102C2 (enExample) |
| WO (1) | WO2015171932A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2021521775A (ja) * | 2018-04-18 | 2021-08-30 | サンガモ セラピューティクス, インコーポレイテッド | ハンチンチン(htt)の調節のためのジンクフィンガータンパク質組成物 |
Families Citing this family (97)
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| BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| SG11201500243WA (en) | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
| DK2872147T3 (da) | 2012-07-13 | 2023-02-20 | Wave Life Sciences Ltd | Fremgangsmåde til fremstilling af kirale oligonukleotider |
| WO2014010250A1 (en) | 2012-07-13 | 2014-01-16 | Chiralgen, Ltd. | Asymmetric auxiliary group |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US20150166984A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting alpha-antitrypsin point mutations |
| EP3095459A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| WO2015108046A1 (ja) | 2014-01-15 | 2015-07-23 | 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 |
| EP3094728B1 (en) | 2014-01-16 | 2022-03-09 | Wave Life Sciences Ltd. | Chiral design |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| ES2730378T3 (es) | 2014-08-27 | 2019-11-11 | Caribou Biosciences Inc | Procedimientos para incrementar la eficiencia de la modificación mediada por Cas9 |
| CN113699113A (zh) | 2014-09-16 | 2021-11-26 | 桑格摩治疗股份有限公司 | 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物 |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| SG10202112057QA (en) | 2015-05-12 | 2021-12-30 | Sangamo Therapeutics Inc | Nuclease-mediated regulation of gene expression |
| US9957501B2 (en) | 2015-06-18 | 2018-05-01 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| CA2991301A1 (en) | 2015-07-13 | 2017-01-19 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| CN117965536A (zh) | 2015-07-22 | 2024-05-03 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| DK3352776T3 (da) | 2015-09-23 | 2025-06-23 | Sangamo Therapeutics Inc | Htt-repressorer og deres anvendelse |
| WO2017070632A2 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
| EP3380622A4 (en) | 2015-11-23 | 2019-08-07 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODIFYING IMMUNITY |
| EP3402533B1 (en) | 2016-01-15 | 2021-08-04 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| EP3416663B1 (en) | 2016-02-18 | 2021-04-14 | The Penn State Research Foundation | Generating gabaergic neurons in brains |
| EP3452596A4 (en) | 2016-05-04 | 2020-03-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| US10584337B2 (en) | 2016-05-18 | 2020-03-10 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
| AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
| MA46018A (fr) * | 2016-08-19 | 2019-06-26 | Bluebird Bio Inc | Activateurs d'édition du génome |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| WO2018039440A1 (en) | 2016-08-24 | 2018-03-01 | Sangamo Therapeutics, Inc. | Regulation of gene expression using engineered nucleases |
| IL264792B2 (en) | 2016-08-24 | 2023-10-01 | Sangamo Therapeutics Inc | Engineered target-specific zinc-finger nucleases |
| JP7256739B2 (ja) | 2016-09-07 | 2023-04-12 | サンガモ セラピューティクス, インコーポレイテッド | 肝臓遺伝子のモジュレーション |
| KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
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| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| KR20190123328A (ko) | 2017-03-09 | 2019-10-31 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 암 백신 |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
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| WO2015171932A1 (en) | 2015-11-12 |
| MX2016014565A (es) | 2017-05-23 |
| AU2015255877A1 (en) | 2016-11-10 |
| HK1232141A1 (zh) | 2018-01-05 |
| RU2691102C2 (ru) | 2019-06-11 |
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