JP2019512005A5 - - Google Patents
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- JP2019512005A5 JP2019512005A5 JP2018540838A JP2018540838A JP2019512005A5 JP 2019512005 A5 JP2019512005 A5 JP 2019512005A5 JP 2018540838 A JP2018540838 A JP 2018540838A JP 2018540838 A JP2018540838 A JP 2018540838A JP 2019512005 A5 JP2019512005 A5 JP 2019512005A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- hidua
- pharmaceutical composition
- patient
- suspension
- 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 |
|---|---|---|---|
| JP2023023041A JP2023062088A (ja) | 2016-02-03 | 2023-02-17 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662290547P | 2016-02-03 | 2016-02-03 | |
| US62/290,547 | 2016-02-03 | ||
| US201662323271P | 2016-04-15 | 2016-04-15 | |
| US62/323,271 | 2016-04-15 | ||
| US201662337178P | 2016-05-16 | 2016-05-16 | |
| US62/337,178 | 2016-05-16 | ||
| US201662367798P | 2016-07-28 | 2016-07-28 | |
| US62/367,798 | 2016-07-28 | ||
| US201762452560P | 2017-01-31 | 2017-01-31 | |
| US62/452,560 | 2017-01-31 | ||
| PCT/US2017/016133 WO2017136500A1 (en) | 2016-02-03 | 2017-02-02 | Gene therapy for treating mucopolysaccharidosis type i |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023023041A Division JP2023062088A (ja) | 2016-02-03 | 2023-02-17 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019512005A JP2019512005A (ja) | 2019-05-09 |
| JP2019512005A5 true JP2019512005A5 (https=) | 2020-03-12 |
| JP7360241B2 JP7360241B2 (ja) | 2023-10-12 |
Family
ID=58108727
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018540838A Active JP7360241B2 (ja) | 2016-02-03 | 2017-02-02 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
| JP2023023041A Pending JP2023062088A (ja) | 2016-02-03 | 2023-02-17 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023023041A Pending JP2023062088A (ja) | 2016-02-03 | 2023-02-17 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US10973929B2 (https=) |
| EP (1) | EP3411488A1 (https=) |
| JP (2) | JP7360241B2 (https=) |
| KR (2) | KR20250171426A (https=) |
| AU (3) | AU2017215211C1 (https=) |
| BR (1) | BR112018015751A2 (https=) |
| CA (1) | CA3012195A1 (https=) |
| IL (1) | IL260959B2 (https=) |
| MA (1) | MA43968A (https=) |
| SG (2) | SG10201912758TA (https=) |
| WO (1) | WO2017136500A1 (https=) |
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI2984166T1 (sl) * | 2013-03-15 | 2020-09-30 | The Trustees Of The University Of Pennsylvania | Sestavki za zdravljenje MPSI |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 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 |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| JP7455579B2 (ja) | 2017-02-28 | 2024-03-26 | 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 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| KR102719222B1 (ko) | 2017-05-11 | 2024-10-21 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 신경 세로이드 지질갈색소증에 대한 유전자 요법 |
| MA50016A (fr) | 2017-07-06 | 2020-07-08 | Univ Pennsylvania | Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| CN107699590A (zh) * | 2017-10-13 | 2018-02-16 | 成都中医药大学 | 一种制备重组人α‑L‑艾杜糖醛酸酶的方法 |
| KR102823833B1 (ko) | 2017-11-30 | 2025-06-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 뮤코다당류증 iiia형에 대한 유전자 요법 |
| KR20200104864A (ko) | 2017-11-30 | 2020-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 뮤코다당류증 iiib형에 대한 유전자 요법 |
| IL280198B2 (en) * | 2018-07-18 | 2026-01-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus) |
| CN113438954A (zh) | 2018-10-01 | 2021-09-24 | 宾夕法尼亚州大学信托人 | 可用于治疗gm1神经节苷脂病的组合物 |
| US11345929B2 (en) | 2018-10-25 | 2022-05-31 | Regeneron Pharmaceuticals, Inc. | Methods for analysis of viral capsid protein composition |
| EP3894864A2 (en) * | 2018-12-10 | 2021-10-20 | Denali Therapeutics Inc. | Lysosomal storage disorder biomarkers and methods of use thereof |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| EP3826664A4 (en) * | 2019-02-06 | 2022-10-26 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type i |
| BR112021015817A2 (pt) | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
| WO2021081217A1 (en) * | 2019-10-23 | 2021-04-29 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2021150570A1 (en) * | 2020-01-22 | 2021-07-29 | Regenxbio Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
| MX2022009462A (es) * | 2020-02-02 | 2022-11-10 | Univ Pennsylvania | Composiciones útiles para tratar la gangliosidosis gm1. |
| US20230304034A1 (en) | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| EP4204014A1 (en) * | 2020-08-26 | 2023-07-05 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| AU2021356684A1 (en) | 2020-10-09 | 2023-05-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| EP4236968A4 (en) * | 2020-10-30 | 2024-03-27 | Immusoft Corporation | Methods of administering genetically modified b cells for in vivo delivery of therapeutic agents |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2023049846A1 (en) * | 2021-09-24 | 2023-03-30 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| CA3236188A1 (en) * | 2021-10-27 | 2023-05-04 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of mps i |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023102517A1 (en) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| WO2023172975A1 (en) * | 2022-03-08 | 2023-09-14 | University Of Massachusetts | Methods of raav packaging |
| US20250250326A1 (en) | 2022-04-06 | 2025-08-07 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| JP2025512969A (ja) | 2022-04-06 | 2025-04-22 | 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 | Ｈｅｒ２陽性転移性乳がん及び他のがんを治療するための組成物及び方法 |
| CN115838709A (zh) * | 2022-11-29 | 2023-03-24 | 武汉禾元生物科技股份有限公司 | 以基因工程水稻表达和制备重组人艾杜糖苷酸酶的方法 |
| EP4724085A1 (en) | 2023-06-12 | 2026-04-15 | The Trustees of The University of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| AR133098A1 (es) | 2023-06-29 | 2025-08-27 | Univ Pennsylvania | Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen |
| IL326390A (en) | 2023-08-10 | 2026-04-01 | Univ Pennsylvania | Compositions and methods for treating spinal muscular atrophy |
Family Cites Families (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6149909A (en) | 1995-06-23 | 2000-11-21 | Women's And Children's Hospital | Synthetic α-L-iduronidase and genetic sequences encoding same |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| AU722624B2 (en) | 1996-09-06 | 2000-08-10 | Trustees Of The University Of Pennsylvania, The | An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase |
| CA2328518A1 (en) | 1998-05-13 | 1999-11-18 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
| US6426208B1 (en) | 1999-11-12 | 2002-07-30 | Harbor-Ucla Research And Education Institute | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| RU2196988C2 (ru) | 2000-04-19 | 2003-01-20 | Государственный новосибирский областной клинический диагностический центр | Способ диагностики мукополисахаридозов |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| AU2003302724A1 (en) | 2002-08-13 | 2004-07-09 | Mcivor, Scott, R. | Methods of using vectors to treat metabolic disorders |
| US7155804B2 (en) | 2003-09-17 | 2007-01-02 | Moog Inc. | Method of forming an electric motor |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20090062144A1 (en) | 2007-04-03 | 2009-03-05 | Nancy Lan Guo | Gene signature for prognosis and diagnosis of lung cancer |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| DK3444342T3 (da) | 2012-07-11 | 2020-08-24 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til behandlingen af lysosomale aflejringssygdomme |
| WO2014125647A1 (ja) | 2013-02-18 | 2014-08-21 | 富士通オプティカルコンポーネンツ株式会社 | 光受信装置 |
| SI2984166T1 (sl) | 2013-03-15 | 2020-09-30 | The Trustees Of The University Of Pennsylvania | Sestavki za zdravljenje MPSI |
| CN105377039A (zh) | 2013-05-15 | 2016-03-02 | 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 |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| BR112017005892A2 (pt) | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
| CN107002096A (zh) | 2014-10-06 | 2017-08-01 | 阿罗根有限公司 | 基于aav的基因疗法 |
| HK1256341A1 (zh) | 2015-05-15 | 2019-09-20 | 明尼苏达大学董事会 | 用於治疗性递送到中枢神经系统的腺相关物 |
| US10604747B2 (en) | 2015-08-06 | 2020-03-31 | Academia Sinica | Engineered enzyme for enzyme replacement therapy |
| US20180236105A1 (en) | 2015-10-23 | 2018-08-23 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| CA3010738A1 (en) | 2016-01-15 | 2017-07-20 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| MA50016A (fr) | 2017-07-06 | 2020-07-08 | Univ Pennsylvania | Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i |
-
2017
- 2017-02-02 EP EP17706915.0A patent/EP3411488A1/en active Pending
- 2017-02-02 IL IL260959A patent/IL260959B2/en unknown
- 2017-02-02 SG SG10201912758TA patent/SG10201912758TA/en unknown
- 2017-02-02 KR KR1020257038989A patent/KR20250171426A/ko active Pending
- 2017-02-02 WO PCT/US2017/016133 patent/WO2017136500A1/en not_active Ceased
- 2017-02-02 JP JP2018540838A patent/JP7360241B2/ja active Active
- 2017-02-02 SG SG11201806270XA patent/SG11201806270XA/en unknown
- 2017-02-02 AU AU2017215211A patent/AU2017215211C1/en active Active
- 2017-02-02 KR KR1020187025463A patent/KR20180121899A/ko not_active Ceased
- 2017-02-02 CA CA3012195A patent/CA3012195A1/en active Pending
- 2017-02-02 MA MA043968A patent/MA43968A/fr unknown
- 2017-02-02 BR BR112018015751-1A patent/BR112018015751A2/pt not_active Application Discontinuation
- 2017-02-02 US US16/075,056 patent/US10973929B2/en active Active
-
2021
- 2021-03-15 US US17/201,301 patent/US20210228740A1/en not_active Abandoned
-
2023
- 2023-02-17 JP JP2023023041A patent/JP2023062088A/ja active Pending
- 2023-05-02 AU AU2023202680A patent/AU2023202680A1/en not_active Abandoned
- 2023-09-27 US US18/475,999 patent/US20240024510A1/en active Pending
-
2026
- 2026-03-03 AU AU2026201588A patent/AU2026201588A1/en active Pending
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