JP2018527365A5 - - Google Patents
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- JP2018527365A5 JP2018527365A5 JP2018513504A JP2018513504A JP2018527365A5 JP 2018527365 A5 JP2018527365 A5 JP 2018527365A5 JP 2018513504 A JP2018513504 A JP 2018513504A JP 2018513504 A JP2018513504 A JP 2018513504A JP 2018527365 A5 JP2018527365 A5 JP 2018527365A5
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- JP
- Japan
- Prior art keywords
- seq
- raav
- aav
- nucleotides
- administration
- 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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- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 14
- 238000001990 intravenous administration Methods 0.000 claims description 10
- 208000021642 Muscular disease Diseases 0.000 claims description 8
- 201000009623 Myopathy Diseases 0.000 claims description 8
- 238000007918 intramuscular administration Methods 0.000 claims description 8
- 210000003205 muscle Anatomy 0.000 claims description 8
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 7
- 101000729812 Homo sapiens Beta-1,4 N-acetylgalactosaminyltransferase 2 Proteins 0.000 claims description 7
- 201000006815 congenital muscular dystrophy Diseases 0.000 claims description 7
- 238000001802 infusion Methods 0.000 claims description 7
- 102100031504 Beta-1,4 N-acetylgalactosaminyltransferase 2 Human genes 0.000 claims description 6
- 201000006938 muscular dystrophy Diseases 0.000 claims description 6
- 208000018360 neuromuscular disease Diseases 0.000 claims description 6
- 230000010412 perfusion Effects 0.000 claims description 6
- 241000702421 Dependoparvovirus Species 0.000 claims description 5
- 102100022745 Laminin subunit alpha-2 Human genes 0.000 claims description 5
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims description 4
- 201000006793 Walker-Warburg syndrome Diseases 0.000 claims description 4
- 229920001184 polypeptide Polymers 0.000 claims description 4
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 4
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims description 2
- 208000014644 Brain disease Diseases 0.000 claims description 2
- 208000004117 Congenital Myasthenic Syndromes Diseases 0.000 claims description 2
- 201000002169 Mitochondrial myopathy Diseases 0.000 claims description 2
- 201000001087 Miyoshi muscular dystrophy Diseases 0.000 claims description 2
- 208000009376 Miyoshi myopathy Diseases 0.000 claims description 2
- 208000029578 Muscle disease Diseases 0.000 claims description 2
- 206010028372 Muscular weakness Diseases 0.000 claims description 2
- 206010068871 Myotonic dystrophy Diseases 0.000 claims description 2
- 201000001981 dermatomyositis Diseases 0.000 claims description 2
- 201000009338 distal myopathy Diseases 0.000 claims description 2
- 210000001508 eye Anatomy 0.000 claims description 2
- 208000030533 eye disease Diseases 0.000 claims description 2
- 230000001815 facial effect Effects 0.000 claims description 2
- 208000023692 inborn mitochondrial myopathy Diseases 0.000 claims description 2
- 210000003000 inclusion body Anatomy 0.000 claims description 2
- 201000008319 inclusion body myositis Diseases 0.000 claims description 2
- 208000025855 muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A, 4 Diseases 0.000 claims description 2
- 230000036473 myasthenia Effects 0.000 claims description 2
- 206010028417 myasthenia gravis Diseases 0.000 claims description 2
- 208000005987 polymyositis Diseases 0.000 claims description 2
- 208000000943 scapulohumeral muscular dystrophy Diseases 0.000 claims description 2
- 208000011580 syndromic disease Diseases 0.000 claims description 2
- 208000034373 type A muscular dystrophy-dystroglycanopathy Diseases 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims 17
- 125000003729 nucleotide group Chemical group 0.000 claims 17
- 108020004707 nucleic acids Proteins 0.000 claims 11
- 102000039446 nucleic acids Human genes 0.000 claims 11
- 150000007523 nucleic acids Chemical class 0.000 claims 11
- 239000000203 mixture Substances 0.000 claims 5
- 101100111156 Homo sapiens B4GALNT2 gene Proteins 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 1
- 108010059343 MM Form Creatine Kinase Proteins 0.000 claims 1
- 208000034965 Nemaline Myopathies Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 230000002458 infectious effect Effects 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 210000003414 extremity Anatomy 0.000 description 15
- 238000000034 method Methods 0.000 description 9
- 230000009885 systemic effect Effects 0.000 description 6
- 238000001361 intraarterial administration Methods 0.000 description 5
- 210000002966 serum Anatomy 0.000 description 3
- 101150079978 AGRN gene Proteins 0.000 description 2
- 102100040026 Agrin Human genes 0.000 description 2
- 108700019743 Agrin Proteins 0.000 description 2
- 108010047294 Lamins Proteins 0.000 description 2
- 102000006835 Lamins Human genes 0.000 description 2
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 description 2
- 210000005053 lamin Anatomy 0.000 description 2
- 230000004118 muscle contraction Effects 0.000 description 2
- 229910052760 oxygen Inorganic materials 0.000 description 2
- 239000001301 oxygen Substances 0.000 description 2
- 102100030685 Alpha-sarcoglycan Human genes 0.000 description 1
- 102100024108 Dystrophin Human genes 0.000 description 1
- 108010069091 Dystrophin Proteins 0.000 description 1
- 101000703500 Homo sapiens Alpha-sarcoglycan Proteins 0.000 description 1
- 101000990902 Homo sapiens Matrix metalloproteinase-9 Proteins 0.000 description 1
- 102100030412 Matrix metalloproteinase-9 Human genes 0.000 description 1
- 206010028289 Muscle atrophy Diseases 0.000 description 1
- 201000002481 Myositis Diseases 0.000 description 1
- 206010030113 Oedema Diseases 0.000 description 1
- 102100030477 Plectin Human genes 0.000 description 1
- 108010054050 Plectin Proteins 0.000 description 1
- 102100029092 Utrophin Human genes 0.000 description 1
- 108010075653 Utrophin Proteins 0.000 description 1
- 210000004369 blood Anatomy 0.000 description 1
- 239000008280 blood Substances 0.000 description 1
- 230000002308 calcification Effects 0.000 description 1
- 230000000747 cardiac effect Effects 0.000 description 1
- 230000008602 contraction Effects 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 230000004217 heart function Effects 0.000 description 1
- 210000003127 knee Anatomy 0.000 description 1
- 108010088360 laminin alpha5 Proteins 0.000 description 1
- 238000005259 measurement Methods 0.000 description 1
- 206010028320 muscle necrosis Diseases 0.000 description 1
- 230000009756 muscle regeneration Effects 0.000 description 1
- 201000000585 muscular atrophy Diseases 0.000 description 1
- 230000002107 myocardial effect Effects 0.000 description 1
- 210000001087 myotubule Anatomy 0.000 description 1
- 230000006911 nucleation Effects 0.000 description 1
- 238000010899 nucleation Methods 0.000 description 1
- 108090000623 proteins and genes Proteins 0.000 description 1
- 102000004169 proteins and genes Human genes 0.000 description 1
- 230000004202 respiratory function Effects 0.000 description 1
- 230000036387 respiratory rate Effects 0.000 description 1
- 238000004904 shortening Methods 0.000 description 1
- 230000000153 supplemental effect Effects 0.000 description 1
- 238000012360 testing method Methods 0.000 description 1
- 230000002861 ventricular Effects 0.000 description 1
- 230000002747 voluntary effect Effects 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021180893A JP2022010079A (ja) | 2015-09-17 | 2021-11-05 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2024087848A JP2024109934A (ja) | 2015-09-17 | 2024-05-30 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562220107P | 2015-09-17 | 2015-09-17 | |
| US62/220,107 | 2015-09-17 | ||
| US201562221068P | 2015-09-20 | 2015-09-20 | |
| US62/221,068 | 2015-09-20 | ||
| US201662301260P | 2016-02-29 | 2016-02-29 | |
| US62/301,260 | 2016-02-29 | ||
| PCT/US2016/052051 WO2017049031A1 (en) | 2015-09-17 | 2016-09-16 | Methods and materials for galgt2 gene therapy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021180893A Division JP2022010079A (ja) | 2015-09-17 | 2021-11-05 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018527365A JP2018527365A (ja) | 2018-09-20 |
| JP2018527365A5 true JP2018527365A5 (enExample) | 2019-08-29 |
| JP7338970B2 JP7338970B2 (ja) | 2023-09-05 |
Family
ID=58289891
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018513504A Active JP7338970B2 (ja) | 2015-09-17 | 2016-09-16 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2021180893A Pending JP2022010079A (ja) | 2015-09-17 | 2021-11-05 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2024087848A Pending JP2024109934A (ja) | 2015-09-17 | 2024-05-30 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021180893A Pending JP2022010079A (ja) | 2015-09-17 | 2021-11-05 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
| JP2024087848A Pending JP2024109934A (ja) | 2015-09-17 | 2024-05-30 | Ｇａｌｇｔ２遺伝子治療のための方法および物質 |
Country Status (14)
| Country | Link |
|---|---|
| US (3) | US10980897B2 (enExample) |
| EP (1) | EP3350331A4 (enExample) |
| JP (3) | JP7338970B2 (enExample) |
| KR (1) | KR102799163B1 (enExample) |
| CN (1) | CN108136049A (enExample) |
| AU (3) | AU2016323575B2 (enExample) |
| CA (1) | CA2998636A1 (enExample) |
| CO (1) | CO2018003960A2 (enExample) |
| EA (1) | EA201890543A1 (enExample) |
| HK (1) | HK1257519A1 (enExample) |
| IL (2) | IL258005B2 (enExample) |
| MX (2) | MX2018003234A (enExample) |
| SA (1) | SA518391113B1 (enExample) |
| WO (1) | WO2017049031A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
| MA45477A (fr) * | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| JP7162021B2 (ja) | 2017-03-17 | 2022-10-27 | ニューカッスル ユニバーシティ | 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達 |
| WO2018191666A1 (en) * | 2017-04-14 | 2018-10-18 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells |
| US11534501B2 (en) | 2017-10-18 | 2022-12-27 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| EP3697916A4 (en) | 2017-10-20 | 2021-08-11 | Research Institute at Nationwide Children's Hospital | METHODS AND MATERIALS FOR NT-3 GENE THERAPY |
| MY208145A (en) | 2018-06-18 | 2025-04-18 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| EP3807309A1 (en) * | 2018-06-18 | 2021-04-21 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies |
| KR20220105643A (ko) * | 2019-10-25 | 2022-07-27 | 오덴테스 테라퓨틱스, 인크. | 글리코겐 축적 장애를 치료하기 위한 조성물 및 방법 |
| US20220411821A1 (en) * | 2019-10-28 | 2022-12-29 | University Of Florida Research Foundation, Incorporated | Gene therapy vectors |
| CN116323925A (zh) * | 2020-08-07 | 2023-06-23 | 詹森生物科技公司 | 高度纯化的病毒颗粒的制剂 |
| EP4230196A1 (en) | 2022-02-21 | 2023-08-23 | Som Innovation Biotech, S.A. | Compounds for use in the treatment of dystrophinopathies |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| ATE272123T1 (de) | 1993-11-09 | 2004-08-15 | Ohio Med College | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| JPH11511326A (ja) | 1995-08-30 | 1999-10-05 | ジエンザイム コーポレイション | アデノウィルスおよびａａｖの精製 |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| ATE465267T1 (de) | 1996-09-06 | 2010-05-15 | Univ Pennsylvania | Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen |
| WO1999011764A2 (en) | 1997-09-05 | 1999-03-11 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of recombinant aav vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2394976A1 (en) | 1999-12-13 | 2001-06-14 | Lexicon Genetics Incorporated | Novel human transferase proteins and polynucleotides encoding the same |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| EP1390490B1 (en) * | 2001-05-24 | 2009-04-15 | Genzyme Corporation | Muscle-specific expression vectors |
| US20060194265A1 (en) * | 2001-10-23 | 2006-08-31 | Morris David W | Novel therapeutic targets in cancer |
| WO2008048986A2 (en) * | 2006-10-17 | 2008-04-24 | Children's Hospital Medical Center | Gene array technique for predicting response in inflammatory bowel diseases |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| JP2015503924A (ja) * | 2012-01-05 | 2015-02-05 | ハダシット メディカル リサーチ サービシーズ アンド ディベロップメント リミテッド | 遺伝子導入のための方法及び組成物 |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| IL258005B2 (en) | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
-
2016
- 2016-09-16 IL IL258005A patent/IL258005B2/en unknown
- 2016-09-16 JP JP2018513504A patent/JP7338970B2/ja active Active
- 2016-09-16 US US15/759,474 patent/US10980897B2/en active Active
- 2016-09-16 HK HK18116463.4A patent/HK1257519A1/zh unknown
- 2016-09-16 WO PCT/US2016/052051 patent/WO2017049031A1/en not_active Ceased
- 2016-09-16 CN CN201680054261.9A patent/CN108136049A/zh active Pending
- 2016-09-16 EP EP16847351.0A patent/EP3350331A4/en active Pending
- 2016-09-16 IL IL314884A patent/IL314884A/en unknown
- 2016-09-16 CA CA2998636A patent/CA2998636A1/en active Pending
- 2016-09-16 KR KR1020187010628A patent/KR102799163B1/ko active Active
- 2016-09-16 MX MX2018003234A patent/MX2018003234A/es unknown
- 2016-09-16 AU AU2016323575A patent/AU2016323575B2/en active Active
- 2016-09-16 EA EA201890543A patent/EA201890543A1/ru unknown
-
2018
- 2018-03-13 SA SA518391113A patent/SA518391113B1/ar unknown
- 2018-03-14 MX MX2023013509A patent/MX2023013509A/es unknown
- 2018-04-13 CO CONC2018/0003960A patent/CO2018003960A2/es unknown
-
2021
- 2021-03-16 US US17/203,232 patent/US12121595B2/en active Active
- 2021-11-05 JP JP2021180893A patent/JP2022010079A/ja active Pending
-
2023
- 2023-01-31 AU AU2023200499A patent/AU2023200499B2/en active Active
-
2024
- 2024-05-30 JP JP2024087848A patent/JP2024109934A/ja active Pending
- 2024-10-03 US US18/905,388 patent/US20250025578A1/en active Pending
-
2025
- 2025-08-06 AU AU2025213610A patent/AU2025213610A1/en active Pending
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