JP2018515096A5 - - Google Patents
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- JP2018515096A5 JP2018515096A5 JP2017558710A JP2017558710A JP2018515096A5 JP 2018515096 A5 JP2018515096 A5 JP 2018515096A5 JP 2017558710 A JP2017558710 A JP 2017558710A JP 2017558710 A JP2017558710 A JP 2017558710A JP 2018515096 A5 JP2018515096 A5 JP 2018515096A5
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- JP
- Japan
- Prior art keywords
- vector
- nucleic acid
- acid sequence
- seq
- polypeptide
- 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.)
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- 150000007523 nucleic acids Chemical class 0.000 claims 16
- 229920001850 Nucleic acid sequence Polymers 0.000 claims 10
- 229920001184 polypeptide Polymers 0.000 claims 7
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 125000003275 alpha amino acid group Chemical group 0.000 claims 5
- 229940053128 Nerve Growth Factor Drugs 0.000 claims 4
- 102000015336 Nerve Growth Factor Human genes 0.000 claims 4
- 108010025020 Nerve Growth Factor Proteins 0.000 claims 4
- 102000003982 Parathyroid hormone Human genes 0.000 claims 4
- 108090000445 Parathyroid hormone Proteins 0.000 claims 4
- 239000000199 parathyroid hormone Substances 0.000 claims 4
- 229960001319 parathyroid hormone Drugs 0.000 claims 4
- 230000003612 virological Effects 0.000 claims 4
- 102000004040 Capsid Proteins Human genes 0.000 claims 3
- 108090000565 Capsid Proteins Proteins 0.000 claims 3
- 241000124008 Mammalia Species 0.000 claims 3
- 241000432074 Adeno-associated virus Species 0.000 claims 2
- 210000000234 Capsid Anatomy 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 239000003937 drug carrier Substances 0.000 claims 2
- 201000003694 methylmalonic acidemia Diseases 0.000 claims 2
- 239000000813 peptide hormone Substances 0.000 claims 2
- 230000001105 regulatory Effects 0.000 claims 2
- 102000019010 EC 5.4.99.2 Human genes 0.000 claims 1
- 108010051862 EC 5.4.99.2 Proteins 0.000 claims 1
- 208000000038 Hypoparathyroidism Diseases 0.000 claims 1
- 210000003079 Salivary Glands Anatomy 0.000 claims 1
Claims (22)
- 配列番号４又は配列番号９のアミノ酸配列を含むキャプシドをコードする核酸を含む、アデノ随伴ウイルス（ＡＡＶ）ベクターであって、異種核酸配列を更に含む、ＡＡＶベクター。
- 異種核酸配列が、宿主細胞におけるその発現を指令する調節配列に作動可能に連結される、請求項１のＡＡＶベクター。
- 異種核酸配列に、１つ以上の逆位末端反復（ＩＴＲ）配列が隣接する、請求項１又は２のＡＡＶベクター。
- キャプシドが、配列番号５の核酸配列によってコードされる、請求項１〜３のいずれか１項のＡＡＶベクター。
- 異種核酸配列が、メチルマロニルＣｏＡムターゼ酵素をコードする、請求項１〜４のいずれか１項のＡＡＶベクター。
- 異種核酸配列が、神経成長因子（ＮＧＦ）シグナルペプチド及び副甲状腺ホルモン（ＰＴＨ）を含むポリペプチドをコードする、請求項１〜４のいずれか１項のＡＡＶベクター。
- ポリペプチドが、配列番号１及び配列番号２のアミノ酸配列を含む、請求項６のＡＡＶベクター。
- 請求項１〜７のいずれか１項のＡＡＶベクター及び医薬的に許容可能な担体を含む、組成物。
- 神経成長因子（ＮＧＦ）シグナルペプチド及び副甲状腺ホルモン（ＰＴＨ）を含むポリペプチド。
- 配列番号１及び配列番号２のアミノ酸配列を含む、請求項９のポリペプチド。
- 請求項９又は１０のポリペプチドをコードする核酸。
- 請求項１１の核酸を含むベクター。
- 核酸配列が、宿主細胞におけるその発現を指令する調節配列に作動可能に連結される、請求項１２のベクター。
- ベクターがウイルスベクターである、請求項１２又は１３のベクター。
- ウイルスベクターがアデノ随伴ウイルスベクターである、請求項１４のベクター。
- アデノ随伴ウイルスベクターが、
（ｉ）配列番号４のアミノ酸配列を含むキャプシドタンパク質か、又は
（ｉｉ）配列番号５の核酸配列によってコードされるキャプシドタンパク質
をコードする核酸を含む、請求項１５のベクター。 - アデノ随伴ウイルスベクターが、配列番号９のアミノ酸配列を含むキャプシドタンパク質をコードする核酸を含む、請求項１５のベクター。
- 請求項９又は１０のポリペプチドをコードする核酸配列に、１つ以上の逆位末端反復（ＩＴＲ）配列が隣接する、請求項１２〜１７のいずれか１項のベクター。
- 配列番号３の核酸配列を含む、請求項１２のベクター。
- （ａ）請求項９若しくは１０のポリペプチド、（ｂ）請求項１１の核酸、又は（ｃ）請求項１２〜１９のいずれか１項のベクター、及び医薬的に許容可能な担体を含む、組成物。
- 哺乳動物の唾液腺への投与用に製剤化された、請求項１〜７及び１２〜１９のいずれか１項のベクターを含む、哺乳動物における副甲状腺機能低下症の治療剤。
- 請求項５のベクターを含む、哺乳動物におけるメチルマロン酸血症（ＭＭＡ）の治療剤。
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562160552P | 2015-05-12 | 2015-05-12 | |
| US62/160,552 | 2015-05-12 | ||
| US201662331699P | 2016-05-04 | 2016-05-04 | |
| US62/331,699 | 2016-05-04 | ||
| PCT/US2016/032047 WO2016183297A1 (en) | 2015-05-12 | 2016-05-12 | Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018515096A JP2018515096A (ja) | 2018-06-14 |
| JP2018515096A5 true JP2018515096A5 (ja) | 2019-06-20 |
| JP6805174B2 JP6805174B2 (ja) | 2020-12-23 |
Family
ID=56101787
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017558710A Active JP6805174B2 (ja) | 2015-05-12 | 2016-05-12 | 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US11447797B2 (ja) |
| EP (1) | EP3294894B8 (ja) |
| JP (1) | JP6805174B2 (ja) |
| CA (1) | CA2985786A1 (ja) |
| WO (1) | WO2016183297A1 (ja) |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Preparations and methods for the treatment of amyotrophic lateral sclerosis |
| JP6863891B2 (ja) | 2014-11-14 | 2021-04-21 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 調節性ポリヌクレオチド |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| WO2016183297A1 (en) | 2015-05-12 | 2016-11-17 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone |
| IL257628B2 (en) | 2015-09-28 | 2023-04-01 | Univ North Carolina Chapel Hill | Methods and compositions for stealth virus antibody vectors |
| CA3006569A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics Inc | composições e métodos de tratamento da doença de huntington |
| IL302748A (en) | 2016-05-18 | 2023-07-01 | Voyager Therapeutics Inc | modulatory polynucleotides |
| CA3035522A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| GB201706781D0 (en) | 2017-04-28 | 2017-06-14 | Univ Sheffield | Parathyroid hormone fusion polypeptide |
| AU2018261790A1 (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| US11497576B2 (en) | 2017-07-17 | 2022-11-15 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖを送達するための組成物および方法 |
| WO2019079240A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CN108610398B (zh) * | 2018-01-15 | 2020-08-11 | 武汉海特生物制药股份有限公司 | 一段功能序列及在分泌蛋白表达中的应用 |
| AU2019237514A1 (en) * | 2018-03-23 | 2020-10-08 | University Of Massachusetts | Gene therapeutics for treating bone disorders |
| SG11202009452WA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| US20220133909A1 (en) * | 2019-01-23 | 2022-05-05 | University Of Florida Research Foundation, Incorporated | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease |
| WO2021222682A1 (en) | 2020-04-30 | 2021-11-04 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Application of aav44.9 vector in gene therapy for the inner ear |
| KR20240049295A (ko) | 2021-08-04 | 2024-04-16 | 더 유나이티드 스테이츠 오브 어메리카, 애즈 리프리젠티드 바이 더 세크러테리, 디파트먼트 오브 헬쓰 앤드 휴먼 서비씨즈 | 방사선 유발 타액 기능저하를 예방하기 위한 aqp1 유전자 요법 |
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| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4863457A (en) | 1986-11-24 | 1989-09-05 | Lee David A | Drug delivery device |
| US5378475A (en) | 1991-02-21 | 1995-01-03 | University Of Kentucky Research Foundation | Sustained release drug delivery devices |
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| US6342390B1 (en) | 1994-11-23 | 2002-01-29 | The United States Of America As Represented By The Secretary Of Health And Human Services | Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy |
| JP2002538770A (ja) | 1998-11-10 | 2002-11-19 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | ウイルスベクターとその製造及び投与の方法 |
| FR2814642B1 (fr) | 2000-10-03 | 2005-07-01 | Ass Pour Le Dev De La Rech En | Souris transgenique pour la recombinaison ciblee mediee par la cre-er modifiee |
| 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 |
| ES2411479T3 (es) * | 2003-09-30 | 2013-07-05 | The Trustees Of The University Of Pennsylvania | Clados de virus adeno-asociados (AAV), secuencias, vectores que los contienen, y usos de los mismos |
| AU2005316476A1 (en) | 2004-12-15 | 2006-06-22 | University Of Florida Research Foundation, Inc. | Chimeric vectors |
| CN101528916B (zh) * | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
| US8048871B2 (en) | 2006-08-18 | 2011-11-01 | Sequoia Pharmaceuticals, Inc. | Compositions and methods for inhibiting cytochrome P450 |
| EP2709653B1 (en) * | 2011-04-20 | 2017-11-22 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Aav mediated exendin-4 gene transfer to salivary glands to protect subjects from diabetes or obesity |
| ES2644468T3 (es) * | 2013-03-15 | 2017-11-29 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT |
| JP6985795B2 (ja) | 2013-09-26 | 2021-12-22 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー |
| WO2016183297A1 (en) | 2015-05-12 | 2016-11-17 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone |
-
2016
- 2016-05-12 WO PCT/US2016/032047 patent/WO2016183297A1/en unknown
- 2016-05-12 US US15/573,214 patent/US11447797B2/en active Active
- 2016-05-12 EP EP16727258.2A patent/EP3294894B8/en active Active
- 2016-05-12 JP JP2017558710A patent/JP6805174B2/ja active Active
- 2016-05-12 CA CA2985786A patent/CA2985786A1/en not_active Abandoned
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