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) | KR20180121899A (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 |
|---|---|---|---|---|
| AU2014235096B2 (en) | 2013-03-15 | 2020-03-19 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| 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 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| IL316926A (en) | 2017-05-11 | 2025-01-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
| CA3068328A1 (en) * | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| 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 |
| CN111989396A (zh) | 2017-11-30 | 2020-11-24 | 宾夕法尼亚州大学信托人 | 用于iiib型粘多糖贮积病的基因疗法 |
| AU2018375164B2 (en) | 2017-11-30 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for Mucopolysaccharidosis IIIA |
| IL280198B2 (en) * | 2018-07-18 | 2026-01-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus) |
| CA3114175A1 (en) | 2018-10-01 | 2020-04-09 | The Trustees Of The University Of Pennsylvania | Beta-galactosidase based aav for treating gm1 gangliosidosis |
| IL282411B2 (en) | 2018-10-25 | 2025-09-01 | Regeneron Pharma | Methods for analyzing viral capsid protein composition |
| US20220025065A1 (en) * | 2018-12-10 | 2022-01-27 | 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 |
| AU2019428629A1 (en) * | 2019-02-06 | 2021-01-28 | 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 |
| KR20220131522A (ko) * | 2020-01-22 | 2022-09-28 | 리젠엑스바이오 인크. | 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 |
| KR20220145838A (ko) * | 2020-02-02 | 2022-10-31 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Gm1 강글리오사이드증을 치료하는 데 유용한 조성물 |
| MX2022014258A (es) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| 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 |
| WO2022094284A1 (en) * | 2020-10-30 | 2022-05-05 | 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 |
| EP4423264A4 (en) * | 2021-10-27 | 2025-12-17 | Univ North Carolina Chapel Hill | AAV-IDUA VECTOR FOR TREATING MPS I |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20240408157A1 (en) | 2021-12-02 | 2024-12-12 | 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 |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| EP4504797A1 (en) | 2022-04-06 | 2025-02-12 | The Trustees of The University of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
| 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 |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| 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 |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| AU4072499A (en) | 1998-05-13 | 1999-11-29 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | 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 |
| TR201908020T4 (tr) | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| CN105755044A (zh) | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| EP3196301B1 (en) | 2012-07-11 | 2018-10-17 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of monogenic diseases |
| 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 |
| AU2014235096B2 (en) | 2013-03-15 | 2020-03-19 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| 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 |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3204503A1 (en) | 2014-10-06 | 2017-08-16 | Arthrogen BV | Aav-based gene therapy |
| 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 |
| TWI617573B (zh) | 2015-08-06 | 2018-03-11 | 中央研究院 | 用於酵素置換療法的改造酵素 |
| 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 |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| WO2017123757A1 (en) | 2016-01-15 | 2017-07-20 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| CA3068328A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
-
2017
- 2017-02-02 CA CA3012195A patent/CA3012195A1/en active Pending
- 2017-02-02 JP JP2018540838A patent/JP7360241B2/ja active Active
- 2017-02-02 WO PCT/US2017/016133 patent/WO2017136500A1/en not_active Ceased
- 2017-02-02 KR KR1020187025463A patent/KR20180121899A/ko not_active Ceased
- 2017-02-02 KR KR1020257038989A patent/KR20250171426A/ko active Pending
- 2017-02-02 EP EP17706915.0A patent/EP3411488A1/en active Pending
- 2017-02-02 US US16/075,056 patent/US10973929B2/en active Active
- 2017-02-02 AU AU2017215211A patent/AU2017215211C1/en active Active
- 2017-02-02 IL IL260959A patent/IL260959B2/en unknown
- 2017-02-02 MA MA043968A patent/MA43968A/fr unknown
- 2017-02-02 SG SG10201912758TA patent/SG10201912758TA/en unknown
- 2017-02-02 BR BR112018015751-1A patent/BR112018015751A2/pt not_active Application Discontinuation
- 2017-02-02 SG SG11201806270XA patent/SG11201806270XA/en unknown
-
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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