JP2021508241A5 - - Google Patents
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- Publication number
- JP2021508241A5 JP2021508241A5 JP2020529562A JP2020529562A JP2021508241A5 JP 2021508241 A5 JP2021508241 A5 JP 2021508241A5 JP 2020529562 A JP2020529562 A JP 2020529562A JP 2020529562 A JP2020529562 A JP 2020529562A JP 2021508241 A5 JP2021508241 A5 JP 2021508241A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- cells
- suspension
- hsgsh
- subject
- 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
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- 210000004027 cell Anatomy 0.000 claims 10
- 239000000725 suspension Substances 0.000 claims 9
- 239000013598 vector Substances 0.000 claims 7
- 208000036632 Brain mass Diseases 0.000 claims 4
- 208000005340 mucopolysaccharidosis III Diseases 0.000 claims 4
- 208000005392 Spasm Diseases 0.000 claims 3
- 206010044565 Tremor Diseases 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 238000002641 enzyme replacement therapy Methods 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 241000124740 Bocaparvovirus Species 0.000 claims 2
- 108091026890 Coding region Proteins 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 238000011260 co-administration Methods 0.000 claims 2
- 210000005260 human cell Anatomy 0.000 claims 2
- 238000001990 intravenous administration Methods 0.000 claims 2
- 210000004962 mammalian cell Anatomy 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 241000588724 Escherichia coli Species 0.000 claims 1
- 241000238631 Hexapoda Species 0.000 claims 1
- 101000651201 Homo sapiens N-sulphoglucosamine sulphohydrolase Proteins 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 1
- 102100027661 N-sulphoglucosamine sulphohydrolase Human genes 0.000 claims 1
- -1 Polyplexes Polymers 0.000 claims 1
- 241000700584 Simplexvirus Species 0.000 claims 1
- 102000005262 Sulfatase Human genes 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- KZWHEHSUEBTKJM-SLPGGIOYSA-N [(2r,3r,4s,5r)-3,4,5,6-tetrahydroxy-1-oxohexan-2-yl]sulfamic acid Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](C=O)NS(O)(=O)=O KZWHEHSUEBTKJM-SLPGGIOYSA-N 0.000 claims 1
- 239000000090 biomarker Substances 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 239000000562 conjugate Substances 0.000 claims 1
- 210000004748 cultured cell Anatomy 0.000 claims 1
- 239000000412 dendrimer Substances 0.000 claims 1
- 229920000736 dendritic polymer Polymers 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 210000003494 hepatocyte Anatomy 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 239000002122 magnetic nanoparticle Substances 0.000 claims 1
- 239000006249 magnetic particle Substances 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000003607 modifier Substances 0.000 claims 1
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 229920000575 polymersome Polymers 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
- 238000011272 standard treatment Methods 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 210000002330 subarachnoid space Anatomy 0.000 claims 1
- 108060007951 sulfatase Proteins 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 230000004304 visual acuity Effects 0.000 claims 1
- 230000004393 visual impairment Effects 0.000 claims 1
- 210000005253 yeast cell Anatomy 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023133281A JP2023159274A (ja) | 2017-11-30 | 2023-08-18 | ムコ多糖症ｉｉｉａ型のための遺伝子療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762593081P | 2017-11-30 | 2017-11-30 | |
| US62/593,081 | 2017-11-30 | ||
| PCT/US2018/063168 WO2019108857A1 (en) | 2017-11-30 | 2018-11-29 | Gene therapy for mucopolysaccharidosis iiia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023133281A Division JP2023159274A (ja) | 2017-11-30 | 2023-08-18 | ムコ多糖症ｉｉｉａ型のための遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021508241A JP2021508241A (ja) | 2021-03-04 |
| JP2021508241A5 true JP2021508241A5 (https=) | 2022-01-06 |
| JP7389744B2 JP7389744B2 (ja) | 2023-11-30 |
Family
ID=66665823
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020529562A Active JP7389744B2 (ja) | 2017-11-30 | 2018-11-29 | ムコ多糖症ｉｉｉａ型のための遺伝子療法 |
| JP2023133281A Pending JP2023159274A (ja) | 2017-11-30 | 2023-08-18 | ムコ多糖症ｉｉｉａ型のための遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023133281A Pending JP2023159274A (ja) | 2017-11-30 | 2023-08-18 | ムコ多糖症ｉｉｉａ型のための遺伝子療法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US11555206B2 (https=) |
| EP (1) | EP3717653A4 (https=) |
| JP (2) | JP7389744B2 (https=) |
| KR (1) | KR102823833B1 (https=) |
| AU (1) | AU2018375164B2 (https=) |
| BR (1) | BR112020010735A2 (https=) |
| CA (1) | CA3083416A1 (https=) |
| IL (1) | IL274939B2 (https=) |
| MX (1) | MX2020005663A (https=) |
| WO (1) | WO2019108857A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| AU2018375164B2 (en) * | 2017-11-30 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for Mucopolysaccharidosis IIIA |
| ES3035673T3 (en) * | 2018-12-20 | 2025-09-08 | Esteve Pharmaceuticals Sa | Recombinant vectors for the long term treatment of mucchopolysacharidosis |
| EP3999647A1 (en) * | 2019-07-18 | 2022-05-25 | Lysogene | Compositions and methods for the treatment of sanfilippo disease and other disorders |
| MX2022014258A (es) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| KR20240133693A (ko) * | 2021-11-12 | 2024-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 점액다당류증 iiia의 치료를 위한 유전자 요법 |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CN115029360B (zh) * | 2022-05-30 | 2024-08-02 | 上海勉亦生物科技有限公司 | 用于治疗粘多糖贮积症iiia型的转基因表达盒 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
| 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 |
| 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 |
| 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 |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| 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 |
| US20130039888A1 (en) * | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| EP2492347A1 (en) * | 2012-05-22 | 2012-08-29 | Laboratorios Del. Dr. Esteve, S.A. | Methods for the production of vectors |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| 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 |
| 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 |
| 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 |
| WO2017136533A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| AU2018375164B2 (en) * | 2017-11-30 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for Mucopolysaccharidosis IIIA |
-
2018
- 2018-11-29 AU AU2018375164A patent/AU2018375164B2/en active Active
- 2018-11-29 IL IL274939A patent/IL274939B2/en unknown
- 2018-11-29 BR BR112020010735-2A patent/BR112020010735A2/pt unknown
- 2018-11-29 MX MX2020005663A patent/MX2020005663A/es unknown
- 2018-11-29 US US16/768,095 patent/US11555206B2/en active Active
- 2018-11-29 JP JP2020529562A patent/JP7389744B2/ja active Active
- 2018-11-29 KR KR1020207018108A patent/KR102823833B1/ko active Active
- 2018-11-29 EP EP18884183.7A patent/EP3717653A4/en active Pending
- 2018-11-29 CA CA3083416A patent/CA3083416A1/en active Pending
- 2018-11-29 WO PCT/US2018/063168 patent/WO2019108857A1/en not_active Ceased
-
2022
- 2022-11-15 US US18/055,731 patent/US20230279430A1/en not_active Abandoned
-
2023
- 2023-08-18 JP JP2023133281A patent/JP2023159274A/ja active Pending
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