JP2024112876A5 - - Google Patents
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- JP2024112876A5 JP2024112876A5 JP2024079088A JP2024079088A JP2024112876A5 JP 2024112876 A5 JP2024112876 A5 JP 2024112876A5 JP 2024079088 A JP2024079088 A JP 2024079088A JP 2024079088 A JP2024079088 A JP 2024079088A JP 2024112876 A5 JP2024112876 A5 JP 2024112876A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- human subject
- composition according
- human
- recombinant
- 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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Applications Claiming Priority (12)
| 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) |
| JP2019541264A 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 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019541264A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024112876A JP2024112876A (ja) | 2024-08-21 |
| JP2024112876A5 true JP2024112876A5 (https=) | 2024-11-15 |
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 Before (1)
| 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 |
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 |
|---|---|---|---|---|
| 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 |
| IL280198B2 (en) * | 2018-07-18 | 2026-01-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus) |
| WO2021150570A1 (en) * | 2020-01-22 | 2021-07-29 | Regenxbio Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
| CA3201247A1 (en) * | 2020-12-07 | 2022-06-16 | Gaurav Shah | Treatment of danon disease |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| CA3236188A1 (en) * | 2021-10-27 | 2023-05-04 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of 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 |
| AU649897B2 (en) * | 1991-11-14 | 1994-06-02 | Women's And Children's Hospital | 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 |
| 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 |
| 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 |
| AU2001267337A1 (en) * | 2000-06-30 | 2002-01-14 | Maxygen Aps | Peptide extended glycosylated polypeptides |
| 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 |
| US20050036994A1 (en) * | 2003-07-16 | 2005-02-17 | Koichiro Mihara | Compounds and methods for downregulating the effects of TGF-beta |
| 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 |
| US7714119B2 (en) * | 2004-07-13 | 2010-05-11 | Biosante Pharmaceuticals, Inc. | AAV vector compositions and methods for enhanced expression of immunoglobulins using the same |
| 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 |
| 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 |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| 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 |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| 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 |
| 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 |
| 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 |
| EP2970946A4 (en) | 2013-03-13 | 2016-09-07 | Philadelphia Children Hospital | ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF |
| 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 |
| CA2915296A1 (en) * | 2013-06-11 | 2015-02-26 | Portage Pharmaceuticals Ltd. | Structure, manufacturing and uses of human-derived cell-permeable peptides conjugated with specific biologically active cargo peptides |
| WO2015009961A1 (en) * | 2013-07-19 | 2015-01-22 | Armagen Technologies, Inc. | Compositions and methods related to structures that cross the blood brain barrier |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| 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 |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| MX382488B (es) * | 2014-10-20 | 2025-03-13 | Neuralstem Inc | Celulas madre neurales estables que comprenden un polinucleótido exógeno que codifica un factor de crecimiento y métodos de uso de las mismas. |
| EP3218000A2 (en) * | 2014-11-10 | 2017-09-20 | 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 CA CA3049915A patent/CA3049915A1/en active Pending
- 2018-01-30 BR BR112019015482-5A patent/BR112019015482A2/pt not_active Application Discontinuation
- 2018-01-30 US US16/482,057 patent/US20190358303A1/en not_active Abandoned
- 2018-01-30 EP EP18748381.3A patent/EP3576768A4/en active Pending
- 2018-01-30 JP JP2019541264A patent/JP2020508289A/ja active Pending
- 2018-01-30 IL IL268076A patent/IL268076B2/en unknown
- 2018-01-30 KR KR1020257002646A patent/KR20250022877A/ko active Pending
- 2018-01-30 WO PCT/US2018/015910 patent/WO2018144441A1/en not_active Ceased
- 2018-01-30 SG SG11201906452PA patent/SG11201906452PA/en unknown
- 2018-01-30 AU AU2018216807A patent/AU2018216807A1/en not_active Abandoned
- 2018-01-30 UY UY0001037587A patent/UY37587A/es not_active Application Discontinuation
- 2018-01-30 MA MA047436A patent/MA47436A/fr unknown
- 2018-01-30 KR KR1020197025133A patent/KR102761412B1/ko active Active
-
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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