JP2020519629A5 - - Google Patents
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- Publication number
- JP2020519629A5 JP2020519629A5 JP2019562293A JP2019562293A JP2020519629A5 JP 2020519629 A5 JP2020519629 A5 JP 2020519629A5 JP 2019562293 A JP2019562293 A JP 2019562293A JP 2019562293 A JP2019562293 A JP 2019562293A JP 2020519629 A5 JP2020519629 A5 JP 2020519629A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- sgsh
- particle according
- variant
- raav particle
- 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
- 239000002245 particle Substances 0.000 claims 72
- 102100027661 N-sulphoglucosamine sulphohydrolase Human genes 0.000 claims 54
- 101000651201 Homo sapiens N-sulphoglucosamine sulphohydrolase Proteins 0.000 claims 45
- 241000124008 Mammalia Species 0.000 claims 18
- 210000004027 cell Anatomy 0.000 claims 16
- 230000007812 deficiency Effects 0.000 claims 14
- 210000003169 central nervous system Anatomy 0.000 claims 10
- 108010006140 N-sulfoglucosamine sulfohydrolase Proteins 0.000 claims 9
- 241000702421 Dependoparvovirus Species 0.000 claims 8
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 7
- 108020004707 nucleic acids Proteins 0.000 claims 7
- 102000039446 nucleic acids Human genes 0.000 claims 7
- 150000007523 nucleic acids Chemical class 0.000 claims 7
- 241000288906 Primates Species 0.000 claims 6
- 241000282414 Homo sapiens Species 0.000 claims 5
- 201000010099 disease Diseases 0.000 claims 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 5
- 230000006870 function Effects 0.000 claims 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 4
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 4
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 4
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 4
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 4
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 4
- 210000004556 brain Anatomy 0.000 claims 4
- 210000003140 lateral ventricle Anatomy 0.000 claims 4
- 210000002569 neuron Anatomy 0.000 claims 4
- 238000006467 substitution reaction Methods 0.000 claims 4
- 230000002861 ventricular Effects 0.000 claims 4
- 108090000565 Capsid Proteins Proteins 0.000 claims 3
- 102100023321 Ceruloplasmin Human genes 0.000 claims 3
- 241000283984 Rodentia Species 0.000 claims 3
- 210000003038 endothelium Anatomy 0.000 claims 3
- 210000000981 epithelium Anatomy 0.000 claims 3
- 230000002401 inhibitory effect Effects 0.000 claims 3
- 210000004498 neuroglial cell Anatomy 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- 210000002330 subarachnoid space Anatomy 0.000 claims 3
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 claims 2
- 241000283707 Capra Species 0.000 claims 2
- 241000283073 Equus caballus Species 0.000 claims 2
- 241001494479 Pecora Species 0.000 claims 2
- 241000009328 Perro Species 0.000 claims 2
- 241000282898 Sus scrofa Species 0.000 claims 2
- 235000001014 amino acid Nutrition 0.000 claims 2
- 150000001413 amino acids Chemical group 0.000 claims 2
- 235000009582 asparagine Nutrition 0.000 claims 2
- 229960001230 asparagine Drugs 0.000 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 239000003623 enhancer Substances 0.000 claims 2
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 210000005240 left ventricle Anatomy 0.000 claims 2
- 210000002418 meninge Anatomy 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 208000028698 Cognitive impairment Diseases 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims 1
- 108010031792 IGF Type 2 Receptor Proteins 0.000 claims 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 1
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical compound OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 claims 1
- 102000019218 Mannose-6-phosphate receptors Human genes 0.000 claims 1
- 208000026139 Memory disease Diseases 0.000 claims 1
- 108091081062 Repeated sequence (DNA) Proteins 0.000 claims 1
- 238000009825 accumulation Methods 0.000 claims 1
- 230000002411 adverse Effects 0.000 claims 1
- 150000001508 asparagines Chemical class 0.000 claims 1
- 230000004700 cellular uptake Effects 0.000 claims 1
- 208000010877 cognitive disease Diseases 0.000 claims 1
- 230000001149 cognitive effect Effects 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 239000000945 filler Substances 0.000 claims 1
- 229940125721 immunosuppressive agent Drugs 0.000 claims 1
- 239000003018 immunosuppressive agent Substances 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000007914 intraventricular administration Methods 0.000 claims 1
- 210000005171 mammalian brain Anatomy 0.000 claims 1
- 210000003446 pia mater Anatomy 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 210000005241 right ventricle Anatomy 0.000 claims 1
- 230000028327 secretion Effects 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 230000002463 transducing effect Effects 0.000 claims 1
- 230000031836 visual learning Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762505423P | 2017-05-12 | 2017-05-12 | |
| US62/505,423 | 2017-05-12 | ||
| PCT/US2018/032454 WO2018209317A1 (en) | 2017-05-12 | 2018-05-11 | Sulfamidase (sgsh) variants, vectors, compositions and methods and uses for treating mucopolysaccharidosis type iiia (mps iiia) |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020519629A JP2020519629A (ja) | 2020-07-02 |
| JP2020519629A5 true JP2020519629A5 (enExample) | 2021-07-26 |
| JP7196099B2 JP7196099B2 (ja) | 2022-12-26 |
Family
ID=64104969
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019562293A Active JP7196099B2 (ja) | 2017-05-12 | 2018-05-11 | ムコ多糖症ｉｉｉａ型（ｍｐｓ ｉｉｉａ）を処置するための、スルファミダーゼ（ｓｇｓｈ）バリアント、ベクター、組成物並びに方法及び使用 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12221635B2 (enExample) |
| EP (1) | EP3622060A4 (enExample) |
| JP (1) | JP7196099B2 (enExample) |
| CN (1) | CN110809626A (enExample) |
| AU (1) | AU2018265869C1 (enExample) |
| BR (1) | BR112019023869A2 (enExample) |
| CA (1) | CA3061925A1 (enExample) |
| MX (1) | MX2019013528A (enExample) |
| RU (1) | RU2019140862A (enExample) |
| WO (1) | WO2018209317A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN108728495A (zh) * | 2018-05-31 | 2018-11-02 | 深圳市免疫基因治疗研究院 | 一种Sanfilippo A综合症慢病毒载体、慢病毒及其制备方法和应用 |
| EP3897695B1 (en) * | 2018-12-20 | 2025-06-25 | Esteve Pharmaceuticals, S.A. | Recombinant vectors for the long term treatment of mucchopolysacharidosis |
| AU2020314866A1 (en) * | 2019-07-18 | 2022-02-03 | Lysogene | Compositions and methods for the treatment of Sanfilippo disease and other disorders |
| MX2022006188A (es) * | 2019-11-22 | 2022-08-22 | Childrens Hospital Philadelphia | Variantes del vector viral adenoasociado. |
| US20230133201A1 (en) * | 2020-03-11 | 2023-05-04 | Remotor Therapeutics, Inc. | Methods and materials for disseminating a protein throughout the central nervous system |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
| US20250034533A1 (en) * | 2021-11-12 | 2025-01-30 | Amicus Therapeutics, Inc. | Compositions And Methods For Treating Mucopolysaccharidosis IIIA |
| JP2023159049A (ja) * | 2022-04-19 | 2023-10-31 | Ｊｃｒファーマ株式会社 | マンノース－６－リン酸の数を減少させた糖蛋白質 |
| 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 (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5863782A (en) * | 1995-04-19 | 1999-01-26 | Women's And Children's Hospital | Synthetic mammalian sulphamidase and genetic sequences encoding same |
| JP2013527753A (ja) * | 2010-03-23 | 2013-07-04 | イントレキソン コーポレーション | 治療タンパク質を条件的に発現するベクター、該ベクターを含む宿主細胞およびそれらの使用 |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| WO2012135857A1 (en) * | 2011-03-31 | 2012-10-04 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP3166626B1 (en) * | 2014-07-11 | 2021-04-28 | Biostrategies LC | Materials and methods for treating disorders associated with sulfatase enzymes |
-
2018
- 2018-05-11 CN CN201880043043.4A patent/CN110809626A/zh active Pending
- 2018-05-11 AU AU2018265869A patent/AU2018265869C1/en active Active
- 2018-05-11 CA CA3061925A patent/CA3061925A1/en active Pending
- 2018-05-11 WO PCT/US2018/032454 patent/WO2018209317A1/en not_active Ceased
- 2018-05-11 MX MX2019013528A patent/MX2019013528A/es unknown
- 2018-05-11 JP JP2019562293A patent/JP7196099B2/ja active Active
- 2018-05-11 BR BR112019023869A patent/BR112019023869A2/pt unknown
- 2018-05-11 EP EP18798587.4A patent/EP3622060A4/en active Pending
- 2018-05-11 RU RU2019140862A patent/RU2019140862A/ru unknown
- 2018-05-11 US US16/611,458 patent/US12221635B2/en active Active
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