JP2018533941A5 - - Google Patents
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- Publication number
- JP2018533941A5 JP2018533941A5 JP2018520100A JP2018520100A JP2018533941A5 JP 2018533941 A5 JP2018533941 A5 JP 2018533941A5 JP 2018520100 A JP2018520100 A JP 2018520100A JP 2018520100 A JP2018520100 A JP 2018520100A JP 2018533941 A5 JP2018533941 A5 JP 2018533941A5
- Authority
- JP
- Japan
- Prior art keywords
- copper
- vector
- nucleic acid
- composition
- administered
- 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
- 239000013598 vector Substances 0.000 claims description 52
- 150000007523 nucleic acids Chemical class 0.000 claims description 46
- 239000000203 mixture Substances 0.000 claims description 40
- 108020004707 nucleic acids Proteins 0.000 claims description 30
- 102000039446 nucleic acids Human genes 0.000 claims description 30
- RYGMFSIKBFXOCR-UHFFFAOYSA-N Copper Chemical compound [Cu] RYGMFSIKBFXOCR-UHFFFAOYSA-N 0.000 claims description 29
- 241000700605 Viruses Species 0.000 claims description 29
- 229910052802 copper Inorganic materials 0.000 claims description 29
- 239000010949 copper Substances 0.000 claims description 29
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 16
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 12
- 241000649044 Adeno-associated virus 9 Species 0.000 claims description 12
- 108020004705 Codon Proteins 0.000 claims description 10
- 102000003697 P-type ATPases Human genes 0.000 claims description 9
- 108090000069 P-type ATPases Proteins 0.000 claims description 9
- UUYDYUZBCIHUFZ-MDTVQASCSA-L copper;(2s)-2-amino-3-(1h-imidazol-5-yl)propanoate Chemical compound [Cu+2].[O-]C(=O)[C@@H](N)CC1=CN=CN1.[O-]C(=O)[C@@H](N)CC1=CN=CN1 UUYDYUZBCIHUFZ-MDTVQASCSA-L 0.000 claims description 9
- 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 description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 8
- 108090000623 proteins and genes Proteins 0.000 claims description 8
- 239000013607 AAV vector Substances 0.000 claims description 6
- 102000012437 Copper-Transporting ATPases Human genes 0.000 claims description 6
- 108010022637 Copper-Transporting ATPases Proteins 0.000 claims description 6
- 241000702421 Dependoparvovirus Species 0.000 claims description 6
- 208000008948 Menkes Kinky Hair Syndrome Diseases 0.000 claims description 6
- 208000012583 Menkes disease Diseases 0.000 claims description 6
- 102000020856 Copper Transport Proteins Human genes 0.000 claims description 5
- 108091004554 Copper Transport Proteins Proteins 0.000 claims description 5
- OCUCCJIRFHNWBP-IYEMJOQQSA-L Copper gluconate Chemical compound [Cu+2].OC[C@@H](O)[C@@H](O)[C@H](O)[C@@H](O)C([O-])=O.OC[C@@H](O)[C@@H](O)[C@H](O)[C@@H](O)C([O-])=O OCUCCJIRFHNWBP-IYEMJOQQSA-L 0.000 claims description 5
- 229940108925 copper gluconate Drugs 0.000 claims description 5
- 229910000365 copper sulfate Inorganic materials 0.000 claims description 5
- ORTQZVOHEJQUHG-UHFFFAOYSA-L copper(II) chloride Chemical group Cl[Cu]Cl ORTQZVOHEJQUHG-UHFFFAOYSA-L 0.000 claims description 5
- ARUVKPQLZAKDPS-UHFFFAOYSA-L copper(II) sulfate Chemical compound [Cu+2].[O-][S+2]([O-])([O-])[O-] ARUVKPQLZAKDPS-UHFFFAOYSA-L 0.000 claims description 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 5
- 238000002347 injection Methods 0.000 claims description 4
- 239000007924 injection Substances 0.000 claims description 4
- 208000015441 X-linked distal spinal muscular atrophy 3 Diseases 0.000 claims description 3
- 208000013035 X-linked distal spinal muscular atrophy type 3 Diseases 0.000 claims description 3
- 239000003937 drug carrier Substances 0.000 claims description 3
- 238000007913 intrathecal administration Methods 0.000 claims description 3
- 210000004556 brain Anatomy 0.000 claims description 2
- 238000010253 intravenous injection Methods 0.000 claims description 2
- 208000011580 syndromic disease Diseases 0.000 claims description 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 4
- 239000013066 combination product Substances 0.000 claims 3
- 229940127555 combination product Drugs 0.000 claims 3
- 238000000034 method Methods 0.000 description 27
- 150000001413 amino acids Chemical group 0.000 description 4
- 101000936277 Homo sapiens Copper-transporting ATPase 1 Proteins 0.000 description 1
- 210000004027 cell Anatomy 0.000 description 1
- 210000001175 cerebrospinal fluid Anatomy 0.000 description 1
- 102000055255 human ATP7A Human genes 0.000 description 1
- 210000000936 intestine Anatomy 0.000 description 1
- 238000007917 intracranial administration Methods 0.000 description 1
- 238000002560 therapeutic procedure Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562244594P | 2015-10-21 | 2015-10-21 | |
| US62/244,594 | 2015-10-21 | ||
| PCT/US2016/058124 WO2017070472A1 (en) | 2015-10-21 | 2016-10-21 | Codon-optimized reduced-size atp7a cdna and uses for treatment of copper transport disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020218556A Division JP2021052796A (ja) | 2015-10-21 | 2020-12-28 | コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018533941A JP2018533941A (ja) | 2018-11-22 |
| JP2018533941A5 true JP2018533941A5 (enExample) | 2019-11-28 |
| JP6854286B2 JP6854286B2 (ja) | 2021-04-07 |
Family
ID=57256420
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018520100A Active JP6854286B2 (ja) | 2015-10-21 | 2016-10-21 | コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用 |
| JP2020218556A Pending JP2021052796A (ja) | 2015-10-21 | 2020-12-28 | コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020218556A Pending JP2021052796A (ja) | 2015-10-21 | 2020-12-28 | コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用 |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US10988778B2 (enExample) |
| EP (1) | EP3365438B1 (enExample) |
| JP (2) | JP6854286B2 (enExample) |
| CN (1) | CN108431216B (enExample) |
| AU (1) | AU2016341983B8 (enExample) |
| CA (1) | CA3001574A1 (enExample) |
| DK (1) | DK3365438T3 (enExample) |
| ES (1) | ES2879400T3 (enExample) |
| HU (1) | HUE054802T2 (enExample) |
| PL (1) | PL3365438T3 (enExample) |
| PT (1) | PT3365438T (enExample) |
| WO (1) | WO2017070472A1 (enExample) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020014594A1 (en) * | 2018-07-12 | 2020-01-16 | The Taxas A&M University System | Compositions for the treatment of copper deficiency and methods of use |
| AU2022240726A1 (en) * | 2021-03-18 | 2023-09-21 | Cyprium Therapeutics, Inc. | Copper histidinate compositions and uses thereof |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1993017321A1 (en) | 1992-02-25 | 1993-09-02 | Unisearch Limited | Electrothermal atomic absorption and preconcentration device |
| DE606516T1 (de) | 1993-01-13 | 1995-03-16 | Idemitsu Kosan Co | Gegen menschlichen Ceruloplasmin monoklonaler Antikörper. |
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| US6812339B1 (en) * | 2000-09-08 | 2004-11-02 | Applera Corporation | Polymorphisms in known genes associated with human disease, methods of detection and uses thereof |
| KR100459644B1 (ko) | 2001-03-31 | 2004-12-03 | 제노백(주) | 혈액 여과지에서의 효소면역측정법을 이용한셀룰로플라스민 농도 측정 방법 및 이 방법을 이용한윌슨병 스크리닝 검사용 키트와 진단 시약 |
| EP1625210B1 (en) | 2003-05-21 | 2010-12-01 | Genzyme Corporation | Methods for producing preparations of recombinant aav virions substantially free of empty capsids |
| US20070161120A1 (en) | 2006-01-10 | 2007-07-12 | Viorica Lopez-Avila | Ceruloplasmin Assay |
| MX2008012485A (es) * | 2006-03-30 | 2008-10-10 | Novartis Ag | Composiciones y metodos de uso para anticuerpos de c-met. |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| WO2009097129A1 (en) | 2008-01-29 | 2009-08-06 | Applied Genetic Technologies Corporation | Recombinant virus production using mammalian cells in suspension |
| WO2010042102A1 (en) | 2008-10-06 | 2010-04-15 | The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Identification of subjects likely to benefit from copper treatment |
| WO2010071832A1 (en) * | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| US8679837B2 (en) | 2009-04-02 | 2014-03-25 | University Of Florida Research Foundation, Inc. | Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors |
| JP6373763B2 (ja) * | 2012-02-17 | 2018-08-22 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法 |
| AU2013243949A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of biologics and proteins associated with human disease |
| AU2013243954A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of cosmetic proteins and peptides |
-
2016
- 2016-10-21 US US15/769,294 patent/US10988778B2/en active Active
- 2016-10-21 EP EP16794111.1A patent/EP3365438B1/en active Active
- 2016-10-21 ES ES16794111T patent/ES2879400T3/es active Active
- 2016-10-21 PL PL16794111T patent/PL3365438T3/pl unknown
- 2016-10-21 JP JP2018520100A patent/JP6854286B2/ja active Active
- 2016-10-21 PT PT167941111T patent/PT3365438T/pt unknown
- 2016-10-21 WO PCT/US2016/058124 patent/WO2017070472A1/en not_active Ceased
- 2016-10-21 AU AU2016341983A patent/AU2016341983B8/en active Active
- 2016-10-21 CA CA3001574A patent/CA3001574A1/en active Pending
- 2016-10-21 HU HUE16794111A patent/HUE054802T2/hu unknown
- 2016-10-21 CN CN201680074686.6A patent/CN108431216B/zh active Active
- 2016-10-21 DK DK16794111.1T patent/DK3365438T3/da active
-
2020
- 2020-12-28 JP JP2020218556A patent/JP2021052796A/ja active Pending
-
2021
- 2021-03-30 US US17/217,961 patent/US12173306B2/en active Active
-
2024
- 2024-12-16 US US18/982,427 patent/US20250129382A1/en active Pending
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