JP2020519292A5 - - Google Patents
Download PDFInfo
- 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
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 (https=) | 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 (3) | US11591614B2 (https=) |
| EP (1) | EP3621612A4 (https=) |
| JP (2) | JP7273730B2 (https=) |
| KR (1) | KR102719222B1 (https=) |
| AU (1) | AU2018265531B2 (https=) |
| BR (1) | BR112019023303A2 (https=) |
| CA (1) | CA3061655A1 (https=) |
| IL (2) | IL316926A (https=) |
| RU (1) | RU2019139555A (https=) |
| SG (2) | SG11201910015SA (https=) |
| WO (1) | WO2018209205A1 (https=) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115120746A (zh) | 2013-05-15 | 2022-09-30 | 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 |
| CA3041548A1 (en) * | 2016-11-04 | 2018-05-11 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| BR112021009370A2 (pt) * | 2018-11-14 | 2021-08-17 | Regenxbio Inc. | método de tratamento da doença de batten cln2, composição farmacêutica e kit |
| JP7616995B2 (ja) * | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス，インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
| WO2020206098A1 (en) * | 2019-04-03 | 2020-10-08 | Regenxbio Inc. | Gene therapy for eye pathologies |
| 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 |
| EP3952924A4 (en) * | 2019-04-12 | 2023-05-24 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS |
| MX2022014258A (es) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| AU2021325891A1 (en) * | 2020-08-10 | 2023-04-06 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| AU2021358781A1 (en) * | 2020-10-07 | 2023-05-18 | Tern Therapeutics, Llc | Gene therapy for ocular manifestations of cln2 disease |
| WO2022165313A1 (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 |
| JP2002503954A (ja) | 1997-04-01 | 2002-02-05 | グラクソ、グループ、リミテッド | 核酸増幅法 |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The 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 |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | 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 |
| EP2434420A3 (en) | 2003-08-01 | 2012-07-25 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| 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 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and 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 |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| 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 |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | 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 |
| WO2012135857A1 (en) | 2011-03-31 | 2012-10-04 | 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 |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| EP3024497B1 (en) * | 2013-07-26 | 2021-01-13 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| WO2015164723A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| KR102738860B1 (ko) * | 2015-10-23 | 2024-12-04 | 유니버시티 오브 아이오와 리써치 파운데이션 | 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
-
2018
- 2018-05-11 IL IL316926A patent/IL316926A/en unknown
- 2018-05-11 KR KR1020197036041A patent/KR102719222B1/ko active Active
- 2018-05-11 US US16/611,512 patent/US11591614B2/en active Active
- 2018-05-11 RU RU2019139555A patent/RU2019139555A/ru unknown
- 2018-05-11 AU AU2018265531A patent/AU2018265531B2/en active Active
- 2018-05-11 SG SG11201910015S patent/SG11201910015SA/en unknown
- 2018-05-11 BR BR112019023303-2A patent/BR112019023303A2/pt active Search and Examination
- 2018-05-11 WO PCT/US2018/032278 patent/WO2018209205A1/en not_active Ceased
- 2018-05-11 CA CA3061655A patent/CA3061655A1/en active Pending
- 2018-05-11 IL IL270422A patent/IL270422B2/en unknown
- 2018-05-11 JP JP2019562418A patent/JP7273730B2/ja active Active
- 2018-05-11 EP EP18798476.0A patent/EP3621612A4/en active Pending
- 2018-05-11 SG SG10201912401QA patent/SG10201912401QA/en unknown
-
2022
- 2022-12-29 US US18/148,077 patent/US12428653B2/en active Active
-
2023
- 2023-04-28 JP JP2023073941A patent/JP2023099113A/ja active Pending
-
2025
- 2025-08-26 US US19/309,870 patent/US20260078406A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020519292A5 (https=) | ||
| RU2019139555A (ru) | Генная терапия нейрональных цероидных липофусцинозов | |
| JP2020510428A5 (https=) | ||
| JP2020514286A5 (https=) | ||
| JP2019513794A5 (https=) | ||
| FI3906066T3 (fi) | Geeniterapiakonstrukteja wilsonin taudin hoitamiseksi | |
| FI4206216T3 (fi) | Adenoassosioituneen viruksen kapsidimuunnoksia ja menetelmiä niiden käyttämiseksi | |
| JP2022513034A5 (https=) | ||
| JP2022516283A5 (https=) | ||
| JP2010516252A5 (https=) | ||
| RU2020137429A (ru) | Лечение пигментного ретинита | |
| RU2018129159A (ru) | Рекомбинантные aav векторы, экспрессирующие остеопротективные гены, включая has2 и лубрицин, пригодные при лечении остеоартрита и сходных заболеваний суставов у млекопитающих | |
| JP2021507687A5 (https=) | ||
| KR20210153069A (ko) | 섬유아세포 성장 인자 23 관련 저인산혈증성 질환의 유전자 요법 | |
| JP2024056832A5 (https=) | ||
| JP2023040219A5 (https=) | ||
| JP2024545186A5 (https=) | ||
| JP2025090820A5 (https=) | ||
| JP2018509154A5 (https=) | ||
| JP2020510433A5 (https=) | ||
| JP2024528997A (ja) | 放射線誘発性唾液機能低下を予防するためのａｑｐ１遺伝子療法 | |
| US12544459B2 (en) | Methods and means for the prevention and/or treatment of hemophilic arthropathy in hemophilia | |
| RU2024110309A (ru) | Средства и способ получения вирусных векторов и варианты их применения | |
| RU2021123108A (ru) | Генотерапевтические конструкции для лечения болезни вильсона | |
| RU2021102985A (ru) | Генная терапия нейродегенеративных нарушений |