JP2020519292A5 - - Google Patents
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- Publication number
- JP2020519292A5 JP2020519292A5 JP2019562418A JP2019562418A JP2020519292A5 JP 2020519292 A5 JP2020519292 A5 JP 2020519292A5 JP 2019562418 A JP2019562418 A JP 2019562418A JP 2019562418 A JP2019562418 A JP 2019562418A JP 2020519292 A5 JP2020519292 A5 JP 2020519292A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- suspension
- aav
- itr
- vector genome
- 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.)
- Granted
Links
- 241000702421 Dependoparvovirus Species 0.000 claims 12
- 239000000725 suspension Substances 0.000 claims 12
- 239000003623 enhancer Substances 0.000 claims 8
- 238000007913 intrathecal administration Methods 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- 239000007900 aqueous suspension Substances 0.000 claims 5
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 4
- 101100261165 Homo sapiens TPP1 gene Proteins 0.000 claims 4
- 108091026890 Coding region Proteins 0.000 claims 3
- 101100426960 Homo sapiens TTPA gene Proteins 0.000 claims 3
- 108010006025 bovine growth hormone Proteins 0.000 claims 3
- 239000007788 liquid Substances 0.000 claims 3
- 108010000521 Human Growth Hormone Proteins 0.000 claims 2
- 102000002265 Human Growth Hormone Human genes 0.000 claims 2
- 239000000854 Human Growth Hormone Substances 0.000 claims 2
- 210000004556 brain Anatomy 0.000 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 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 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 102000002572 Alpha-Globulins Human genes 0.000 claims 1
- 108010068307 Alpha-Globulins Proteins 0.000 claims 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 1
- 102000013918 Apolipoproteins E Human genes 0.000 claims 1
- 108010025628 Apolipoproteins E Proteins 0.000 claims 1
- 101100272395 Arabidopsis thaliana BHLH3 gene Proteins 0.000 claims 1
- 102000006734 Beta-Globulins Human genes 0.000 claims 1
- 108010087504 Beta-Globulins Proteins 0.000 claims 1
- 102000008186 Collagen Human genes 0.000 claims 1
- 108010035532 Collagen Proteins 0.000 claims 1
- 241000701022 Cytomegalovirus Species 0.000 claims 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims 1
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 1
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 1
- 101000721661 Homo sapiens Cellular tumor antigen p53 Proteins 0.000 claims 1
- 101000899111 Homo sapiens Hemoglobin subunit beta Proteins 0.000 claims 1
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 108010058846 Ovalbumin Proteins 0.000 claims 1
- 108010039203 Tripeptidyl-Peptidase 1 Proteins 0.000 claims 1
- 102100034197 Tripeptidyl-peptidase 1 Human genes 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 229920001436 collagen Polymers 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 1
- 229940092253 ovalbumin Drugs 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 239000003755 preservative agent Substances 0.000 claims 1
- 230000002335 preservative effect Effects 0.000 claims 1
- 239000004094 surface-active agent Substances 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023073941A JP2023099113A (ja) | 2017-05-11 | 2023-04-28 | 神経セロイドリポフスチン症のための遺伝子療法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762504817P | 2017-05-11 | 2017-05-11 | |
| US62/504,817 | 2017-05-11 | ||
| US201762599816P | 2017-12-18 | 2017-12-18 | |
| US62/599,816 | 2017-12-18 | ||
| US201862652006P | 2018-04-03 | 2018-04-03 | |
| US62/652,006 | 2018-04-03 | ||
| PCT/US2018/032278 WO2018209205A1 (en) | 2017-05-11 | 2018-05-11 | Gene therapy for neuronal ceroid lipofuscinoses |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023073941A Division JP2023099113A (ja) | 2017-05-11 | 2023-04-28 | 神経セロイドリポフスチン症のための遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020519292A JP2020519292A (ja) | 2020-07-02 |
| JP2020519292A5 true JP2020519292A5 (enExample) | 2021-05-27 |
| JP7273730B2 JP7273730B2 (ja) | 2023-05-15 |
Family
ID=64105028
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019562418A Active JP7273730B2 (ja) | 2017-05-11 | 2018-05-11 | 神経セロイドリポフスチン症のための遺伝子療法 |
| JP2023073941A Pending JP2023099113A (ja) | 2017-05-11 | 2023-04-28 | 神経セロイドリポフスチン症のための遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023073941A Pending JP2023099113A (ja) | 2017-05-11 | 2023-04-28 | 神経セロイドリポフスチン症のための遺伝子療法 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11591614B2 (enExample) |
| EP (1) | EP3621612A4 (enExample) |
| JP (2) | JP7273730B2 (enExample) |
| KR (1) | KR102719222B1 (enExample) |
| AU (1) | AU2018265531B2 (enExample) |
| BR (1) | BR112019023303A2 (enExample) |
| CA (1) | CA3061655A1 (enExample) |
| IL (2) | IL316926A (enExample) |
| RU (1) | RU2019139555A (enExample) |
| SG (2) | SG11201910015SA (enExample) |
| WO (1) | WO2018209205A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HK1222093A1 (zh) | 2013-05-15 | 2017-06-23 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| JP2019537576A (ja) * | 2016-11-04 | 2019-12-26 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用 |
| CA3076036A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| MX2021005517A (es) * | 2018-11-14 | 2021-06-18 | Regenxbio Inc | Terapia genica para lipofuscinosis neuronal ceroidea. |
| CA3121211A1 (en) * | 2018-11-28 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| JP2022527116A (ja) * | 2019-04-08 | 2022-05-30 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | Ｔｐｐ１を発現するａａｖの投与による眼のリソソーム蓄積症の治療 |
| JP2022526425A (ja) * | 2019-04-12 | 2022-05-24 | エンコーデッド セラピューティクス， インコーポレイテッド | 治療剤の投与のための組成物および方法 |
| KR20230010670A (ko) | 2020-05-12 | 2023-01-19 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 이식유전자 발현의 drg-특이적 감소를 위한 조성물 |
| JP2023537904A (ja) * | 2020-08-10 | 2023-09-06 | プリベイル セラピューティクス，インコーポレーテッド | 神経変性障害のための遺伝子療法 |
| CA3209779A1 (en) * | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2025090598A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for treatment of cln2 disease |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| DE69837913T2 (de) | 1997-04-01 | 2008-02-07 | Solexa Ltd., Saffron Walden | Verfahren zur vervielfältigung von nukleinsäure |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| ES2308989T3 (es) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
| EP1286703B1 (en) | 2000-06-01 | 2009-08-05 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CA2447240C (en) | 2001-05-18 | 2013-02-19 | Wisconsin Alumni Research Foundation | Method for the synthesis of dna sequences |
| NZ618298A (en) | 2001-11-13 | 2015-04-24 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| PT2359869T (pt) | 2001-12-17 | 2019-04-16 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| US20080050357A1 (en) | 2003-08-01 | 2008-02-28 | Claes Gustafsson | Systems and Methods for Antibody Engineering |
| EP2292780B1 (en) | 2003-09-30 | 2017-08-23 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2359865B1 (en) | 2005-04-07 | 2013-10-02 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| 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 |
| JP6087504B2 (ja) | 2008-11-07 | 2017-03-01 | マサチューセッツ インスティテュート オブ テクノロジー | アミノアルコールリピドイドおよびその使用 |
| US11219696B2 (en) * | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| WO2010071832A1 (en) * | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| SG183929A1 (en) | 2010-03-29 | 2012-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US9849195B2 (en) | 2011-03-31 | 2017-12-26 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| CA3107288A1 (en) | 2011-06-08 | 2012-12-13 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| HK1206601A1 (en) | 2012-04-02 | 2016-01-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of biologics and proteins associated with human disease |
| BR112014029807A2 (pt) | 2012-06-08 | 2017-06-27 | Ethris Gmbh | administração pulmonar de rna mensageiro |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| DK3024497T3 (da) | 2013-07-26 | 2021-04-12 | Univ Iowa Res Found | Fremgangsmåder og præparater til behandling af hjernesygdomme |
| JP6983511B2 (ja) * | 2014-04-25 | 2021-12-17 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 脳中の転移乳癌および他の癌を処置するための方法および組成物 |
| KR20230067694A (ko) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
| AU2016341428B2 (en) * | 2015-10-23 | 2021-12-02 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| WO2018160582A1 (en) | 2017-02-28 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
-
2018
- 2018-05-11 US US16/611,512 patent/US11591614B2/en active Active
- 2018-05-11 SG SG11201910015S patent/SG11201910015SA/en unknown
- 2018-05-11 SG SG10201912401QA patent/SG10201912401QA/en unknown
- 2018-05-11 WO PCT/US2018/032278 patent/WO2018209205A1/en not_active Ceased
- 2018-05-11 RU RU2019139555A patent/RU2019139555A/ru unknown
- 2018-05-11 IL IL316926A patent/IL316926A/en unknown
- 2018-05-11 BR BR112019023303-2A patent/BR112019023303A2/pt active Search and Examination
- 2018-05-11 IL IL270422A patent/IL270422B2/en unknown
- 2018-05-11 KR KR1020197036041A patent/KR102719222B1/ko active Active
- 2018-05-11 CA CA3061655A patent/CA3061655A1/en active Pending
- 2018-05-11 AU AU2018265531A patent/AU2018265531B2/en active Active
- 2018-05-11 JP JP2019562418A patent/JP7273730B2/ja active Active
- 2018-05-11 EP EP18798476.0A patent/EP3621612A4/en active Pending
-
2022
- 2022-12-29 US US18/148,077 patent/US12428653B2/en active Active
-
2023
- 2023-04-28 JP JP2023073941A patent/JP2023099113A/ja active Pending
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