KR20160147974A - 망막 및 cns 유전자 요법을 위한 aav 벡터 - Google Patents
망막 및 cns 유전자 요법을 위한 aav 벡터 Download PDFInfo
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- KR20160147974A KR20160147974A KR1020167033392A KR20167033392A KR20160147974A KR 20160147974 A KR20160147974 A KR 20160147974A KR 1020167033392 A KR1020167033392 A KR 1020167033392A KR 20167033392 A KR20167033392 A KR 20167033392A KR 20160147974 A KR20160147974 A KR 20160147974A
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- amino acid
- raav
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- 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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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- A61P27/06—Antiglaucoma agents or miotics
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- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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- 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
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- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
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- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| KR1020247006248A KR102863726B1 (ko) | 2014-05-02 | 2015-05-02 | 망막 및 cns 유전자 요법을 위한 aav 벡터 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461988131P | 2014-05-02 | 2014-05-02 | |
| US61/988,131 | 2014-05-02 | ||
| US201562114575P | 2015-02-10 | 2015-02-10 | |
| US62/114,575 | 2015-02-10 | ||
| PCT/US2015/028966 WO2015168666A2 (en) | 2014-05-02 | 2015-05-02 | Aav vectors for retinal and cns gene therapy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247006248A Division KR102863726B1 (ko) | 2014-05-02 | 2015-05-02 | 망막 및 cns 유전자 요법을 위한 aav 벡터 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20160147974A true KR20160147974A (ko) | 2016-12-23 |
Family
ID=53189202
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020167033392A Ceased KR20160147974A (ko) | 2014-05-02 | 2015-05-02 | 망막 및 cns 유전자 요법을 위한 aav 벡터 |
| KR1020247006248A Active KR102863726B1 (ko) | 2014-05-02 | 2015-05-02 | 망막 및 cns 유전자 요법을 위한 aav 벡터 |
| KR1020257031397A Pending KR20250141850A (ko) | 2014-05-02 | 2015-05-02 | 망막 및 cns 유전자 요법을 위한 aav 벡터 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247006248A Active KR102863726B1 (ko) | 2014-05-02 | 2015-05-02 | 망막 및 cns 유전자 요법을 위한 aav 벡터 |
| KR1020257031397A Pending KR20250141850A (ko) | 2014-05-02 | 2015-05-02 | 망막 및 cns 유전자 요법을 위한 aav 벡터 |
Country Status (32)
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20200098481A (ko) * | 2017-10-16 | 2020-08-20 | 비게네론 게엠베하 | Aav 벡터 |
Families Citing this family (178)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| PL4342992T3 (pl) | 2009-05-02 | 2025-09-22 | Genzyme Corporation | Terapia genowa dla zaburzeń neurodegeneracyjnych |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| EP2561075B1 (en) | 2010-04-23 | 2018-06-27 | University of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| WO2011133890A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| ES2661680T3 (es) | 2011-04-21 | 2018-04-03 | University Of Massachusetts | Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina |
| HRP20220036T1 (hr) | 2011-04-22 | 2022-04-01 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe |
| KR20240093939A (ko) * | 2013-05-15 | 2024-06-24 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| CA2907799A1 (en) | 2013-05-31 | 2014-12-04 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| EP3561062A1 (en) | 2013-09-13 | 2019-10-30 | California Institute of Technology | Selective recovery |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| KR102288849B1 (ko) | 2014-03-17 | 2021-08-12 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| JP6741591B2 (ja) | 2014-05-02 | 2020-08-19 | ジェンザイム・コーポレーション | 網膜およびcns遺伝子治療用aavベクター |
| RU2718248C2 (ru) * | 2014-05-14 | 2020-03-31 | Эстев Фармасьютикалс, С.А. | Аденоассоциированные вирусные векторы для лечения лизосомных болезней накопления |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| JP6842410B2 (ja) | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるaavベクター |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| AU2015335923B2 (en) | 2014-10-21 | 2021-04-29 | University Of Massachusetts | Recombinant AAV variants and uses thereof |
| HUE057272T2 (hu) * | 2015-02-10 | 2022-04-28 | Genzyme Corp | Vírusrészecskék fokozott bevitele a csíkolt testbe és az agykéregbe |
| EA201791805A1 (ru) | 2015-02-10 | 2018-05-31 | Джензим Корпорейшн | ВАРИАНТ СРЕДСТВА ДЛЯ RNAi |
| US10584321B2 (en) | 2015-02-13 | 2020-03-10 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| CN114480502A (zh) | 2015-03-02 | 2022-05-13 | 阿德夫拉姆生物技术股份有限公司 | 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法 |
| JP6935049B2 (ja) | 2015-03-11 | 2021-09-15 | アメリカ合衆国 | X連鎖性網膜色素変性症の治療のためのrp2及びrpgrベクター |
| JP6836999B2 (ja) | 2015-03-24 | 2021-03-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California | アデノ随伴ウイルス変異体及びその使用方法 |
| CA2981616C (en) | 2015-04-06 | 2025-06-17 | Arcellx, Inc. | De novo binding domain polypeptides and their uses |
| ES2755725T3 (es) | 2015-04-08 | 2020-04-23 | Us Health | Terapia génica viral como tratamiento para una enfermedad o un trastorno por almacenamiento de colesterol |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| WO2016172008A1 (en) | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| US10745447B2 (en) | 2015-09-28 | 2020-08-18 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20200098481A (ko) * | 2017-10-16 | 2020-08-20 | 비게네론 게엠베하 | Aav 벡터 |
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