JP2020508289A5 - - Google Patents
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- Publication number
- JP2020508289A5 JP2020508289A5 JP2019541264A JP2019541264A JP2020508289A5 JP 2020508289 A5 JP2020508289 A5 JP 2020508289A5 JP 2019541264 A JP2019541264 A JP 2019541264A JP 2019541264 A JP2019541264 A JP 2019541264A JP 2020508289 A5 JP2020508289 A5 JP 2020508289A5
- Authority
- JP
- Japan
- Prior art keywords
- human
- idua
- pharmaceutical composition
- subject
- expression vector
- 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.)
- Pending
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024079088A JP2024112876A (ja) | 2017-01-31 | 2024-05-15 | 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762452769P | 2017-01-31 | 2017-01-31 | |
| US62/452,769 | 2017-01-31 | ||
| US201762485655P | 2017-04-14 | 2017-04-14 | |
| US62/485,655 | 2017-04-14 | ||
| US201762529366P | 2017-07-06 | 2017-07-06 | |
| US62/529,366 | 2017-07-06 | ||
| US201762579690P | 2017-10-31 | 2017-10-31 | |
| US62/579,690 | 2017-10-31 | ||
| US201862616234P | 2018-01-11 | 2018-01-11 | |
| US62/616,234 | 2018-01-11 | ||
| PCT/US2018/015910 WO2018144441A1 (en) | 2017-01-31 | 2018-01-30 | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024079088A Division JP2024112876A (ja) | 2017-01-31 | 2024-05-15 | 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020508289A JP2020508289A (ja) | 2020-03-19 |
| JP2020508289A5 true JP2020508289A5 (https=) | 2021-03-18 |
Family
ID=63041038
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019541264A Pending JP2020508289A (ja) | 2017-01-31 | 2018-01-30 | 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 |
| JP2024079088A Pending JP2024112876A (ja) | 2017-01-31 | 2024-05-15 | 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024079088A Pending JP2024112876A (ja) | 2017-01-31 | 2024-05-15 | 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20190358303A1 (https=) |
| EP (1) | EP3576768A4 (https=) |
| JP (2) | JP2020508289A (https=) |
| KR (2) | KR20250022877A (https=) |
| AU (1) | AU2018216807A1 (https=) |
| BR (1) | BR112019015482A2 (https=) |
| CA (1) | CA3049915A1 (https=) |
| IL (1) | IL268076B2 (https=) |
| MA (1) | MA47436A (https=) |
| SG (1) | SG11201906452PA (https=) |
| UY (1) | UY37587A (https=) |
| WO (1) | WO2018144441A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| IL280198B2 (en) * | 2018-07-18 | 2026-01-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus) |
| KR20220131522A (ko) * | 2020-01-22 | 2022-09-28 | 리젠엑스바이오 인크. | 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 |
| CN116887868A (zh) * | 2020-12-07 | 2023-10-13 | 太空飞船七有限责任公司 | 达农病的治疗 |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| EP4423264A4 (en) * | 2021-10-27 | 2025-12-17 | Univ North Carolina Chapel Hill | AAV-IDUA VECTOR FOR TREATING MPS I |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5196315A (en) * | 1990-05-01 | 1993-03-23 | The Johns Hopkins University | Human neuronal cell line |
| CA2099503C (en) * | 1991-11-14 | 2007-10-09 | Hamish Steele Scott | Synthetic alpha-l-iduronidase and genetic sequences encoding same |
| TWI235158B (en) * | 1997-09-01 | 2005-07-01 | Kirin Brewery | Human thrombopoietin produced by human established cell lines, processes foe preparing the same, and pharmaceutical composition comprising the same |
| 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 |
| US6585971B1 (en) * | 1999-11-12 | 2003-07-01 | Harbor-Ucla Research And Education Institute | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof |
| CA2412882A1 (en) * | 2000-06-30 | 2002-01-10 | Maxygen Aps | Peptide extended glycosylated polypeptides |
| 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 |
| DE602004027048D1 (de) * | 2003-07-16 | 2010-06-17 | Resverlogix Inc | Verbindungen und methoden für das downregulating der effekte von tgf-beta |
| 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 |
| US7714119B2 (en) * | 2004-07-13 | 2010-05-11 | Biosante Pharmaceuticals, Inc. | AAV vector compositions and methods for enhanced expression of immunoglobulins using the same |
| 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 |
| US9375440B2 (en) * | 2006-11-03 | 2016-06-28 | Medtronic, Inc. | Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| WO2012057363A1 (ja) | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| 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 |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| CA2905952A1 (en) | 2013-03-13 | 2014-10-02 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
| 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 |
| JP6479777B2 (ja) * | 2013-06-11 | 2019-03-06 | ポルタゲ ファーマシューティカルス エルティーディー． | 生物学的に活性な特異的カーゴペプチドと結合したヒト由来の細胞透過性ペプチドの構造、製造および使用 |
| WO2015009961A1 (en) * | 2013-07-19 | 2015-01-22 | Armagen Technologies, Inc. | Compositions and methods related to structures that cross the blood brain barrier |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015164757A1 (en) | 2014-04-25 | 2015-10-29 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016064737A1 (en) * | 2014-10-20 | 2016-04-28 | Neuralstem, Inc. | Stable neural stem cells comprising an exogenous polynucleotide coding for a growth factor and methods of use thereof |
| US20180185495A1 (en) * | 2014-11-10 | 2018-07-05 | Alexion Pharmaceuticals, Inc. | Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof |
| EA201792500A1 (ru) * | 2015-05-13 | 2018-04-30 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Aav-опосредованная экспрессия антител против гриппа и способы их использования |
-
2018
- 2018-01-30 KR KR1020257002646A patent/KR20250022877A/ko active Pending
- 2018-01-30 KR KR1020197025133A patent/KR102761412B1/ko active Active
- 2018-01-30 UY UY0001037587A patent/UY37587A/es not_active Application Discontinuation
- 2018-01-30 JP JP2019541264A patent/JP2020508289A/ja active Pending
- 2018-01-30 BR BR112019015482-5A patent/BR112019015482A2/pt not_active Application Discontinuation
- 2018-01-30 WO PCT/US2018/015910 patent/WO2018144441A1/en not_active Ceased
- 2018-01-30 CA CA3049915A patent/CA3049915A1/en active Pending
- 2018-01-30 AU AU2018216807A patent/AU2018216807A1/en not_active Abandoned
- 2018-01-30 EP EP18748381.3A patent/EP3576768A4/en active Pending
- 2018-01-30 US US16/482,057 patent/US20190358303A1/en not_active Abandoned
- 2018-01-30 MA MA047436A patent/MA47436A/fr unknown
- 2018-01-30 SG SG11201906452PA patent/SG11201906452PA/en unknown
- 2018-01-30 IL IL268076A patent/IL268076B2/en unknown
-
2023
- 2023-10-03 US US18/480,172 patent/US20240050537A1/en active Pending
-
2024
- 2024-05-15 JP JP2024079088A patent/JP2024112876A/ja active Pending
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