JP2020010684A5 - - Google Patents
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- JP2020010684A5 JP2020010684A5 JP2019135745A JP2019135745A JP2020010684A5 JP 2020010684 A5 JP2020010684 A5 JP 2020010684A5 JP 2019135745 A JP2019135745 A JP 2019135745A JP 2019135745 A JP2019135745 A JP 2019135745A JP 2020010684 A5 JP2020010684 A5 JP 2020010684A5
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- amino acid
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- cells
- capsid protein
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- 125000003275 alpha amino acid group Chemical group 0.000 claims 30
- 210000002845 Virion Anatomy 0.000 claims 17
- 102000004040 Capsid Proteins Human genes 0.000 claims 16
- 108090000565 Capsid Proteins Proteins 0.000 claims 16
- 230000002458 infectious Effects 0.000 claims 14
- 108020004707 nucleic acids Proteins 0.000 claims 12
- 150000007523 nucleic acids Chemical class 0.000 claims 12
- 210000004027 cells Anatomy 0.000 claims 11
- 150000001413 amino acids Chemical class 0.000 claims 9
- 210000000130 stem cell Anatomy 0.000 claims 9
- 241000432074 Adeno-associated virus Species 0.000 claims 8
- 108090001123 antibodies Proteins 0.000 claims 8
- 102000004965 antibodies Human genes 0.000 claims 8
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 4
- 230000005591 charge neutralization Effects 0.000 claims 4
- 210000004962 mammalian cells Anatomy 0.000 claims 4
- 230000001264 neutralization Effects 0.000 claims 4
- 238000006386 neutralization reaction Methods 0.000 claims 4
- 239000003795 chemical substances by application Substances 0.000 claims 3
- 230000026683 transduction Effects 0.000 claims 3
- 238000010361 transduction Methods 0.000 claims 3
- 210000003958 Hematopoietic Stem Cells Anatomy 0.000 claims 2
- 210000004185 Liver Anatomy 0.000 claims 2
- 108010061543 Neutralizing Antibodies Proteins 0.000 claims 2
- 229930002945 all-trans-retinaldehyde Natural products 0.000 claims 2
- 229920001184 polypeptide Polymers 0.000 claims 2
- 230000002207 retinal Effects 0.000 claims 2
- 235000020945 retinal Nutrition 0.000 claims 2
- 239000011604 retinal Substances 0.000 claims 2
- 210000001671 Embryonic Stem Cells Anatomy 0.000 claims 1
- 210000002889 Endothelial Cells Anatomy 0.000 claims 1
- 210000004263 Induced Pluripotent Stem Cells Anatomy 0.000 claims 1
- 210000004072 Lung Anatomy 0.000 claims 1
- 210000002901 Mesenchymal Stem Cells Anatomy 0.000 claims 1
- 210000004413 Myocytes, Cardiac Anatomy 0.000 claims 1
- 210000000933 Neural Crest Anatomy 0.000 claims 1
- 210000001178 Neural Stem Cells Anatomy 0.000 claims 1
- 210000000496 Pancreas Anatomy 0.000 claims 1
- 210000001744 T-Lymphocytes Anatomy 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 210000002317 cardiac myocyte Anatomy 0.000 claims 1
- 201000009910 diseases by infectious agent Diseases 0.000 claims 1
- 210000002950 fibroblast Anatomy 0.000 claims 1
- 210000001665 muscle stem cell Anatomy 0.000 claims 1
- 230000001537 neural Effects 0.000 claims 1
- 210000004255 neuroglia Anatomy 0.000 claims 1
- 210000002569 neurons Anatomy 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001568 sexual Effects 0.000 claims 1
- 210000002363 skeletal muscle cell Anatomy 0.000 claims 1
Claims (29)
- （ａ）配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９０％のアミノ酸配列同一性を有するアミノ酸配列を含む、変異体アデノ随伴ウイルス（ＡＡＶ）カプシドタンパク質、ならびに
（ｂ）異種核酸
を含み、ＡＡＶ２によって示されるヒト中和抗体への耐性と比較して、少なくとも５倍高い該抗体への耐性を示す、感染性組換えアデノ随伴ウイルス（ｒＡＡＶ）ビリオン。 - 前記変異体ＡＡＶカプシドタンパク質が、配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９５％のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項１に記載の前記感染性ｒＡＡＶビリオン。
- 前記変異体ＡＡＶカプシドタンパク質が、配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９８％のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項１に記載の前記感染性ｒＡＡＶビリオン。
- 前記変異体ＡＡＶカプシドタンパク質が、配列番号１１または配列番号１２に記載されるアミノ酸配列を含む、請求項１に記載の前記感染性ｒＡＡＶビリオン。
- 前記ｒＡＡＶビリオンが、ＡＡＶ２によって示される耐性よりも少なくとも１０倍高いヒト中和抗体への耐性を示す、請求項１〜４のいずれか一項に記載の前記感染性ｒＡＡＶビリオン。
- 前記ｒＡＡＶビリオンが、ＡＡＶ２によって示される耐性よりも少なくとも３０倍高いヒト中和抗体への耐性を示す、請求項１〜４のいずれか一項に記載の前記感染性ｒＡＡＶビリオン。
- 野生型ＡＡＶ血清型２（ＡＡＶ２）によって示される哺乳類細胞への形質導入と比較して、ヒト中和抗体の存在下における哺乳類細胞への増加した形質導入を示す、請求項１〜６のいずれか一項に記載の前記感染性ｒＡＡＶビリオン。
- 前記哺乳類細胞が、肝臓細胞、膵臓細胞、骨格筋細胞、心筋細胞、線維芽細胞、網膜細胞、滑膜関節細胞、肺細胞、Ｔ細胞、ニューロン、グリア細胞、幹細胞、内皮細胞、または癌細胞である、請求項７に記載の前記感染性ｒＡＡＶビリオン。
- 前記幹細胞が、造血幹細胞、造血前駆細胞、神経幹細胞、神経前駆細胞、神経堤幹細胞、胚幹細胞、誘導多能性幹細胞（ｉＰＳ細胞）、間葉幹細胞、中胚葉幹細胞、肝臓幹細胞、膵臓幹細胞、膵臓前駆細胞、筋幹細胞、または網膜幹細胞である、請求項８に記載の前記感染性ｒＡＡＶビリオン。
- 前記異種核酸が干渉ＲＮＡを含む、請求項１〜９のいずれか一項に記載の前記感染性ｒＡＡＶビリオン。
- 前記異種核酸が、ポリペプチドをコードするヌクレオチド配列を含む、請求項１〜９のいずれか一項に記載の前記感染性ｒＡＡＶビリオン。
- 配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９０％のアミノ酸配列同一性を有するアミノ酸配列を含む変異体アデノ随伴ウイルス（ＡＡＶ）カプシドタンパク質をコードするヌクレオチド配列を含み、該コードされた変異体ＡＡＶカプシドタンパク質が、ＡＡＶ２によって示されるヒト抗体による中和への耐性と比較して、少なくとも５倍上昇した、該抗体による中和への耐性を、感染性組換えアデノ随伴ウイルス（ｒＡＡＶ）ビリオンに与える、単離核酸。
- 前記コードされた変異体ＡＡＶカプシドタンパク質が、配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９５％のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項１２に記載の前記単離核酸。
- 前記コードされた変異体ＡＡＶカプシドタンパク質が、配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９８％のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項１２に記載の前記単離核酸。
- 前記コードされた変異体ＡＡＶカプシドタンパク質が、配列番号１１または配列番号１２に記載されるアミノ酸配列を含む、請求項１２に記載の前記単離核酸。
- 請求項１２〜１５のいずれか一項に記載の前記核酸を含む、単離宿主細胞。
- 前記核酸で安定的にトランスフェクトされる、請求項１６に記載の前記単離宿主細胞。
- ＡＡＶｒｅｐタンパク質をコードするヌクレオチド配列を含む核酸をさらに含む、請求項１６または１７に記載の前記単離宿主細胞。
- 組換えＡＡＶベクターをさらに含む、請求項１６〜１８のいずれか一項に記載の前記単離宿主細胞。
- 請求項１〜１１のいずれか一項に記載の感染性組換えアデノ随伴ウイルス（ｒＡＡＶ）ビリオンを含む、遺伝子産物の送達剤。
- 前記異種核酸が干渉ＲＮＡを含む、請求項２０に記載の前記送達剤。
- 前記異種核酸がポリペプチドをコードするヌクレオチド配列を含む、請求項２０に記載の前記送達剤。
- 配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９０％のアミノ酸配列同一性を有するアミノ酸配列を含み、ＡＡＶ２によって示されるヒト抗体による中和への耐性と比較して、少なくとも５倍上昇した、該抗体による中和への耐性を、感染性組換えアデノ随伴ウイルスビリオンに与える、変異体アデノ随伴ウイルス（ＡＡＶ）カプシドタンパク質。
- 配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９５％のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項２３に記載の前記変異体ＡＡＶカプシドタンパク質。
- 配列番号１１または配列番号１２に記載されるアミノ酸配列のアミノ酸２０３〜７３６と少なくとも９８％のアミノ酸配列同一性を有するアミノ酸配列を含む、請求項２３に記載の前記変異体ＡＡＶカプシドタンパク質。
- 配列番号１１または配列番号１２に記載されるアミノ酸配列を含む、請求項２３に記載の前記変異体ＡＡＶカプシドタンパク質。
- 前記上昇した耐性が、ＡＡＶ２によって示される耐性よりも少なくとも１０倍高い、請求項２３〜２６のいずれか一項に記載の前記変異体ＡＡＶカプシドタンパク質。
- 前記上昇した耐性が、ＡＡＶ２によって示される耐性よりも少なくとも３０倍高い、請求項２３〜２６のいずれか一項に記載の前記変異体ＡＡＶカプシドタンパク質。
- ＡＡＶ２によって示される形質導入と比較して、ヒト中和抗体の存在下における哺乳類細胞への増加した形質導入を、感染性組換えアデノ随伴ウイルス（ｒＡＡＶ）ビリオンに与える、請求項２３〜２８のいずれか一項に記載の前記変異体ＡＡＶカプシドタンパク質。
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022052809A JP2022097480A (ja) | 2013-05-31 | 2022-03-29 | アデノ随伴ウイルス変異体及びその使用方法 |
| JP2024006465A JP2024026872A (ja) | 2013-05-31 | 2024-01-19 | アデノ随伴ウイルス変異体及びその使用方法 |
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361829735P | 2013-05-31 | 2013-05-31 | |
| US61/829,735 | 2013-05-31 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016517017A Division JP6600624B2 (ja) | 2013-05-31 | 2014-05-29 | アデノ随伴ウイルス変異体及びその使用方法 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022052809A Division JP2022097480A (ja) | 2013-05-31 | 2022-03-29 | アデノ随伴ウイルス変異体及びその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020010684A JP2020010684A (ja) | 2020-01-23 |
| JP2020010684A5 true JP2020010684A5 (ja) | 2020-05-21 |
| JP7069088B2 JP7069088B2 (ja) | 2022-05-17 |
Family
ID=51989402
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016517017A Active JP6600624B2 (ja) | 2013-05-31 | 2014-05-29 | アデノ随伴ウイルス変異体及びその使用方法 |
| JP2019135745A Active JP7069088B2 (ja) | 2013-05-31 | 2019-07-24 | アデノ随伴ウイルス変異体及びその使用方法 |
| JP2022052809A Pending JP2022097480A (ja) | 2013-05-31 | 2022-03-29 | アデノ随伴ウイルス変異体及びその使用方法 |
| JP2024006465A Pending JP2024026872A (ja) | 2013-05-31 | 2024-01-19 | アデノ随伴ウイルス変異体及びその使用方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
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| JP2016517017A Active JP6600624B2 (ja) | 2013-05-31 | 2014-05-29 | アデノ随伴ウイルス変異体及びその使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
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| JP2022052809A Pending JP2022097480A (ja) | 2013-05-31 | 2022-03-29 | アデノ随伴ウイルス変異体及びその使用方法 |
| JP2024006465A Pending JP2024026872A (ja) | 2013-05-31 | 2024-01-19 | アデノ随伴ウイルス変異体及びその使用方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (4) | US11136557B2 (ja) |
| EP (1) | EP3003391B1 (ja) |
| JP (4) | JP6600624B2 (ja) |
| CN (1) | CN105247044B (ja) |
| CA (1) | CA2907799A1 (ja) |
| ES (1) | ES2897508T3 (ja) |
| HK (1) | HK1223016A1 (ja) |
| WO (1) | WO2014194132A1 (ja) |
Families Citing this family (100)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| 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 |
| EP2699270B1 (en) | 2011-04-22 | 2017-06-21 | The Regents of The University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| AU2015231439B2 (en) | 2014-03-17 | 2019-11-14 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| US10746742B2 (en) * | 2014-04-25 | 2020-08-18 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| MX2017005834A (es) | 2014-11-05 | 2017-11-17 | Voyager Therapeutics Inc | Polinucleotidos aad para el tratamiento de la enfermedad de parkinson. |
| KR102599909B1 (ko) | 2014-11-14 | 2023-11-09 | 보이저 테라퓨틱스, 인크. | 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법 |
| RU2749882C2 (ru) | 2014-11-14 | 2021-06-18 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| KR20170137730A (ko) | 2015-03-02 | 2017-12-13 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 |
| WO2016154344A1 (en) | 2015-03-24 | 2016-09-29 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| WO2017075335A1 (en) | 2015-10-28 | 2017-05-04 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| EP3370778A1 (en) | 2015-11-05 | 2018-09-12 | Bamboo Therapeutics, Inc. | Modified friedreich ataxia genes and vectors for gene therapy |
| CA3006569A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
| CN109415704B (zh) * | 2016-02-16 | 2022-02-25 | 利兰斯坦福初级大学董事会 | 对先已存在的人中和抗体有抗性的新型重组腺相关病毒衣壳 |
| US11185555B2 (en) | 2016-04-11 | 2021-11-30 | Noah James Harrison | Method to kill pathogenic microbes in a patient |
| IL262583B2 (en) * | 2016-04-28 | 2023-10-01 | Spark Therapeutics Inc | Relative potency assay for a vector encoding isomerohydrolases |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| CN116333057A (zh) | 2016-05-13 | 2023-06-27 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和其使用方法 |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| IL302748A (en) | 2016-05-18 | 2023-07-01 | Voyager Therapeutics Inc | modulatory polynucleotides |
| CA2971303A1 (en) | 2016-06-21 | 2017-12-21 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| CN109715650B (zh) * | 2016-07-26 | 2024-03-08 | 生物马林药物股份有限公司 | 新颖腺相关病毒衣壳蛋白 |
| KR102508820B1 (ko) | 2016-07-29 | 2023-03-13 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법 |
| JP2019524162A (ja) * | 2016-08-18 | 2019-09-05 | 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 | モジュラーＡＡＶ送達システムによるＣＲＩＳＰＲ−Ｃａｓゲノム編集 |
| AU2017321488B2 (en) | 2016-08-30 | 2022-10-20 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| US11028131B2 (en) | 2017-01-30 | 2021-06-08 | Nippon Medical School Foundation | Mutant of adeno-associated virus (AAV) capsid protein |
| EP3595694A4 (en) * | 2017-03-14 | 2021-06-09 | The Regents of The University of California | CONSTRUCTION OF CAS9 CRISPR IMMUNE FURTIF |
| WO2018170473A1 (en) | 2017-03-17 | 2018-09-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression |
| WO2018204786A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| WO2018209113A1 (en) * | 2017-05-10 | 2018-11-15 | University Of Utah Research Foundation | Compositions and methods of use of arc capsids |
| CN110831611A (zh) * | 2017-05-10 | 2020-02-21 | 马萨诸塞眼科耳科诊所 | 用于修饰病毒的组装活化蛋白(aap)依赖性的方法和组合物 |
| CA3061968A1 (en) | 2017-05-10 | 2018-11-15 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses |
| WO2018222503A1 (en) * | 2017-05-31 | 2018-12-06 | The Regents Of The University Of California | Adeno-associated virus with variant capsid and methods of use thereof |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| CA3059995A1 (en) | 2017-08-28 | 2019-03-07 | The Regents Of The University Of California | Adeno-associated virus capsid variants and methods of use thereof |
| KR20220070075A (ko) | 2017-09-20 | 2022-05-27 | 4디 몰레큘러 테라퓨틱스 아이엔씨. | 아데노-관련 바이러스 변이체 캡시드 및 그 용도 |
| TWI804518B (zh) | 2017-10-16 | 2023-06-11 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CN111770999A (zh) | 2017-11-27 | 2020-10-13 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和用于抑制血管生成的应用 |
| US20210030823A1 (en) * | 2018-01-26 | 2021-02-04 | Tokushima University | Novel adeno-associated virus virion for treatment of tay-sachs disease and sandhoff disease |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| WO2019161365A1 (en) | 2018-02-19 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof |
| US11608510B2 (en) * | 2018-03-30 | 2023-03-21 | The Board Of Trustees Of The Leland Stanford Junior University | Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism |
| CN112272672A (zh) * | 2018-04-03 | 2021-01-26 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| JP2021523702A (ja) * | 2018-05-04 | 2021-09-09 | オレゴン ヘルス アンド サイエンス ユニバーシティ | ヒト抗ａａｖ２カプシドポリクローナル抗体エピトープ |
| WO2019217911A1 (en) * | 2018-05-11 | 2019-11-14 | Massachusetts Eye And Ear Infirmary | Liver-specific tropism of adeno-associated viruses |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| MA52631A (fr) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
| US20210230632A1 (en) | 2018-05-15 | 2021-07-29 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| CN113301924A (zh) * | 2018-08-18 | 2021-08-24 | 哈佛学院校长同事会 | 原位基因编辑 |
| AU2019346655A1 (en) | 2018-09-28 | 2021-05-06 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
| AU2020208467A1 (en) | 2019-01-18 | 2021-08-05 | Voyager Therapeutics, Inc. | Methods and systems for producing AAV particles |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| TW202039856A (zh) * | 2019-03-28 | 2020-11-01 | 西班牙商艾斯提夫製藥股份有限公司 | 製造重組病毒載體之方法 |
| US20240124889A1 (en) | 2019-05-07 | 2024-04-18 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| WO2020261178A1 (en) | 2019-06-27 | 2020-12-30 | Pfizer Inc. | Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy |
| EP4006160A4 (en) * | 2019-07-12 | 2023-06-28 | Gene Therapy Research Institution Co., Ltd. | Adeno-associated virus virion for gene transfer to human liver |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| EP4045664A1 (en) * | 2019-10-16 | 2022-08-24 | Wuxi Apptec (Shanghai) Co., Ltd. | A novel aav variant |
| IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| IL297200A (en) | 2020-04-15 | 2022-12-01 | Voyager Therapeutics Inc | tau binding compounds |
| WO2021222094A1 (en) | 2020-04-27 | 2021-11-04 | 4D Molecular Therapeutics Inc. | Codon optimized gla genes and uses thereof |
| CN115552021A (zh) * | 2020-04-27 | 2022-12-30 | 4D分子治疗有限公司 | 用于肺递送的腺相关变体、制剂和方法 |
| BR112022023106A2 (pt) | 2020-05-13 | 2023-01-17 | Voyager Therapeutics Inc | Redirecionamento de tropismo de capsídeos de aav |
| CN116063403A (zh) * | 2020-05-22 | 2023-05-05 | 中国医学科学院血液病医院(中国医学科学院血液学研究所) | 腺相关病毒突变体及其应用 |
| WO2021242909A1 (en) * | 2020-05-26 | 2021-12-02 | Shape Therapeutics Inc. | High throughput engineering of functional aav capsids |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
| WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| CN111876432B (zh) * | 2020-07-29 | 2022-10-28 | 舒泰神（北京）生物制药股份有限公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| CN112226461B (zh) * | 2020-08-21 | 2022-04-22 | 华侨大学 | Cd4阳性细胞特异性基因传递载体及其应用 |
| WO2022097008A1 (en) | 2020-11-03 | 2022-05-12 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| US20240052322A1 (en) | 2020-12-15 | 2024-02-15 | Pfizer Inc. | HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins |
| MX2023007612A (es) | 2020-12-23 | 2023-07-12 | Pfizer | Metodos para purificacion de vectores aav por cromatografia de afinidad. |
| WO2022208342A1 (en) | 2021-04-01 | 2022-10-06 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| EP4329820A1 (en) | 2021-04-26 | 2024-03-06 | Alexion Pharma International Operations Limited | Adeno-associated viral vector capsids with improved tissue tropism |
| WO2022269466A1 (en) | 2021-06-22 | 2022-12-29 | Pfizer Inc. | Production of adeno-associated virus vector in insect cells |
| US20230241118A1 (en) | 2021-10-20 | 2023-08-03 | University Of Rochester | Rejuvenation treatment of age-related white matter loss |
| US20230270818A1 (en) | 2021-11-02 | 2023-08-31 | University Of Rochester | Tcf7l2 mediated remyelination in the brain |
| WO2023114816A1 (en) | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Recombinant optimized galc constructs and methods for treating galc-associated disorders |
| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| GB202201242D0 (en) | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| WO2023207918A1 (en) * | 2022-04-25 | 2023-11-02 | Beijing Hanmoluojie Technology Co., Ltd. | Aav capsid 3d molecular surface feature mapping |
| GB202206336D0 (en) | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| WO2023214346A1 (en) | 2022-05-06 | 2023-11-09 | Novartis Ag | Novel recombinant aav vp2 fusion polypeptides |
| WO2023240062A1 (en) | 2022-06-07 | 2023-12-14 | Adverum Biotechnologies, Inc. | Melanopsin variants for vision restoration |
| WO2024003687A1 (en) | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| WO2024038365A1 (en) | 2022-08-16 | 2024-02-22 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| WO2024120528A1 (en) * | 2022-12-08 | 2024-06-13 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Improved system for producing rna-packaged aav particles |
Family Cites Families (87)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9206016D0 (en) * | 1992-03-19 | 1992-04-29 | Sandoz Ltd | Improvements in or relating to organic compounds |
| US6440425B1 (en) | 1995-05-01 | 2002-08-27 | Aventis Pasteur Limited | High molecular weight major outer membrane protein of moraxella |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| US6096548A (en) | 1996-03-25 | 2000-08-01 | Maxygen, Inc. | Method for directing evolution of a virus |
| EP0927044A4 (en) | 1996-04-16 | 1999-09-08 | Immusol Inc | DEFINED TARGET VIRAL VECTORS |
| US6710036B2 (en) | 1997-07-25 | 2004-03-23 | Avigen, Inc. | Induction of immune response to antigens expressed by recombinant adeno-associated virus |
| AU9319198A (en) | 1997-09-19 | 1999-04-05 | Trustees Of The University Of Pennsylvania, The | Methods and vector constructs useful for production of recombinant aav |
| AU1449499A (en) | 1997-10-31 | 1999-05-24 | Maxygen, Incorporated | Modification of virus tropism and host range by viral genome shuffling |
| US6410300B1 (en) | 1998-01-12 | 2002-06-25 | The University Of North Carolina At Chapel Hill | Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV |
| US6551795B1 (en) | 1998-02-18 | 2003-04-22 | Genome Therapeutics Corporation | Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics |
| DE19827457C1 (de) | 1998-06-19 | 2000-03-02 | Medigene Ag | Strukturprotein von AAV, seine Herstellung und Verwendung |
| DE69925134T2 (de) | 1998-09-11 | 2006-01-19 | The Regents Of The University Of California, La Jolla | Rekombinante adenovirus für gewebespezifische genexpression ins herz |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| US6943153B1 (en) | 1999-03-15 | 2005-09-13 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| US6498244B1 (en) | 1999-05-28 | 2002-12-24 | Cell Genesys, Inc. | Adeno-associated virus capsid immunologic determinants |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| DE19933288A1 (de) | 1999-07-15 | 2001-01-18 | Medigene Ag | Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung |
| DE19933719A1 (de) | 1999-07-19 | 2001-01-25 | Medigene Ag | Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung |
| US8232081B2 (en) | 1999-09-21 | 2012-07-31 | Basf Se | Methods and microorganisms for production of panto-compounds |
| US20070026394A1 (en) | 2000-02-11 | 2007-02-01 | Lawrence Blatt | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies |
| US6855314B1 (en) | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
| US7647184B2 (en) | 2001-08-27 | 2010-01-12 | Hanall Pharmaceuticals, Co. Ltd | High throughput directed evolution by rational mutagenesis |
| US20030129203A1 (en) | 2001-08-27 | 2003-07-10 | Nautilus Biotech S.A. | Mutant recombinant adeno-associated viruses |
| NZ600121A (en) | 2001-11-13 | 2013-12-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| WO2003054197A2 (en) | 2001-12-21 | 2003-07-03 | Medigene Ag | A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism |
| AU2003221733A1 (en) | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
| WO2003093436A2 (en) | 2002-04-30 | 2003-11-13 | University Of Florida | Treatment for phenylketonuria |
| US7071172B2 (en) | 2002-04-30 | 2006-07-04 | The University Of North Carolina At Chapel Hill | Secretion signal vectors |
| US7858367B2 (en) | 2002-04-30 | 2010-12-28 | Duke University | Viral vectors and methods for producing and using the same |
| US7254489B2 (en) | 2002-05-31 | 2007-08-07 | Microsoft Corporation | Systems, methods and apparatus for reconstructing phylogentic trees |
| US7220577B2 (en) | 2002-08-28 | 2007-05-22 | University Of Florida Research Foundation, Inc. | Modified AAV |
| WO2004083441A2 (en) | 2003-03-19 | 2004-09-30 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Random peptide library displayed on aav vectors |
| WO2004108922A2 (en) * | 2003-04-25 | 2004-12-16 | The Trustees Of The University Of Pennsylvania | Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| ES2629380T3 (es) | 2003-06-19 | 2017-08-09 | Genzyme Corporation | Viriones de AAV con inmunorreactividad reducida y usos de los mismos |
| CA2530582C (en) | 2003-06-23 | 2014-09-09 | A&G Pharmaceutical, Inc. | Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US20050019927A1 (en) | 2003-07-13 | 2005-01-27 | Markus Hildinger | DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER |
| EP1670897A4 (en) | 2003-09-24 | 2008-02-27 | Univ Leland Stanford Junior | IGF-1 MULTIPOTENTE ADULTS HAS NEW NEURAL CNS STEM CELLS INTO AN OLIGOD DROGLIA CELLINE |
| SI2292780T1 (sl) * | 2003-09-30 | 2017-12-29 | The Trustees Of The University Of Pennsylvania | Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba |
| US7427396B2 (en) | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| WO2006066066A2 (en) | 2004-12-15 | 2006-06-22 | University Of North Carolina At Chapel Hill | Chimeric vectors |
| US20100172871A1 (en) | 2005-02-17 | 2010-07-08 | Flannery John G | Muller Cell Specific Gene Therapy |
| ES2525143T3 (es) * | 2005-04-07 | 2014-12-18 | The Trustees Of The University Of Pennsylvania | Cápsides de AAVrh.64 modificado, composiciones que las contienen y usos de las mismas |
| WO2007089632A2 (en) | 2006-01-27 | 2007-08-09 | The University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| AU2007247929A1 (en) | 2006-05-04 | 2007-11-15 | Pennsylvania College Of Optometry | Restoration of visual responses by In Vivo delivery of rhodopsin nucleic acids |
| CN1966082B (zh) | 2006-11-03 | 2010-06-30 | 许瑞安 | 一种防治结直肠癌的基因药物及其制备方法和用途 |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| NZ579985A (en) | 2007-04-13 | 2012-02-24 | Catalyst Biosciences Inc | Modified factor vii polypetides and uses thereof |
| EP1985708B1 (en) | 2007-04-27 | 2015-04-15 | Universität Rostock | Selective targeting of viruses to neural precursor cells |
| WO2009137006A2 (en) | 2008-04-30 | 2009-11-12 | The University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| EP2910637A1 (en) | 2008-05-20 | 2015-08-26 | University of Florida Research Foundation, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| US20110171262A1 (en) | 2008-06-17 | 2011-07-14 | Andrew Christian Bakker | Parvoviral capsid with incorporated gly-ala repeat region |
| WO2010093784A2 (en) | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| CN101532024A (zh) | 2009-04-30 | 2009-09-16 | 许瑞安 | 一种用于基因治疗的新型细胞特异性内含microRNA结合序列的基因HAAVmir |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| WO2011117258A2 (en) | 2010-03-22 | 2011-09-29 | Association Institut De Myologie | Methods of increasing efficiency of vector penetration of target tissue |
| US8263396B2 (en) * | 2010-04-01 | 2012-09-11 | Weidong Xiao | Methods and compositions for the production of recombinant virus vectors |
| 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 |
| CN103189507A (zh) * | 2010-10-27 | 2013-07-03 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
| EP2699270B1 (en) | 2011-04-22 | 2017-06-21 | The Regents of The University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| PL2839014T3 (pl) * | 2012-04-18 | 2021-08-23 | The Children's Hospital Of Philadelphia | Kompozycja i sposoby na wysoko wydajny transfer genu za pomocą kapsydu wariantów aav |
| US9677088B2 (en) | 2012-05-09 | 2017-06-13 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| TWI702955B (zh) | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ） |
| WO2014103957A1 (ja) | 2012-12-25 | 2014-07-03 | タカラバイオ株式会社 | Ａａｖ変異体 |
| WO2014124282A1 (en) | 2013-02-08 | 2014-08-14 | The Trustees Of The University Of Pennsylvania | Enhanced aav-mediated gene transfer for retinal therapies |
| AU2014273085B2 (en) | 2013-05-29 | 2020-10-22 | Cellectis | New compact scaffold of Cas9 in the type II CRISPR system |
| US11136557B2 (en) * | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| US9597415B2 (en) | 2013-06-28 | 2017-03-21 | Inserm (Institute National De La Sante Et De La Recherche Medicale) | Methods for expressing a polynucleotide of interest in the retina of a subject |
| AU2014324717B2 (en) | 2013-09-26 | 2020-10-08 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial AAV capsid library for targeted gene therapy |
| PT3459965T (pt) | 2013-10-11 | 2021-02-25 | Massachusetts Eye & Ear Infirmary | Métodos de previsão de sequências de vírus ancestrais e suas utilizações |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| AU2015231439B2 (en) | 2014-03-17 | 2019-11-14 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| PT3137497T (pt) | 2014-05-02 | 2021-07-12 | Genzyme Corp | Vetores de aav para terapia genética na retina e snc |
| EP3155116A4 (en) | 2014-06-10 | 2017-12-27 | Massachusetts Institute Of Technology | Method for gene editing |
| KR102655319B1 (ko) | 2015-02-20 | 2024-04-04 | 유니버시티 오브 아이오와 리써치 파운데이션 | 유전적 안 질환 치료용 방법 및 조성물 |
| KR20170137730A (ko) | 2015-03-02 | 2017-12-13 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 |
| PT3265568T (pt) | 2015-03-06 | 2020-08-20 | Massachusetts Eye & Ear Infirmary | Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31 |
| US20190000940A1 (en) | 2015-07-31 | 2019-01-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
| CN116333057A (zh) | 2016-05-13 | 2023-06-27 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和其使用方法 |
| MA44546B1 (fr) | 2016-06-15 | 2021-03-31 | Univ California | Virus adéno-associés variants et procédés d'utilisation |
| CA3059995A1 (en) | 2017-08-28 | 2019-03-07 | The Regents Of The University Of California | Adeno-associated virus capsid variants and methods of use thereof |
-
2014
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- 2014-05-29 WO PCT/US2014/040083 patent/WO2014194132A1/en active Application Filing
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2016
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2021
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2022
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2023
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2024
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