JP2018501791A5 - - Google Patents
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- Publication number
- JP2018501791A5 JP2018501791A5 JP2017533845A JP2017533845A JP2018501791A5 JP 2018501791 A5 JP2018501791 A5 JP 2018501791A5 JP 2017533845 A JP2017533845 A JP 2017533845A JP 2017533845 A JP2017533845 A JP 2017533845A JP 2018501791 A5 JP2018501791 A5 JP 2018501791A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- composition
- seq
- raav
- nucleic acid
- 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 24
- 102100036264 Glucose-6-phosphatase catalytic subunit 1 Human genes 0.000 claims description 22
- 101000930910 Homo sapiens Glucose-6-phosphatase catalytic subunit 1 Proteins 0.000 claims description 22
- 150000007523 nucleic acids Chemical class 0.000 claims description 16
- 102000039446 nucleic acids Human genes 0.000 claims description 16
- 108020004707 nucleic acids Proteins 0.000 claims description 16
- 239000002773 nucleotide Substances 0.000 claims description 14
- 125000003729 nucleotide group Chemical group 0.000 claims description 14
- 230000002401 inhibitory effect Effects 0.000 claims description 6
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 208000002404 Liver Cell Adenoma Diseases 0.000 claims description 4
- 150000001413 amino acids Chemical class 0.000 claims description 4
- 208000007345 glycogen storage disease Diseases 0.000 claims description 4
- 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 2
- 239000013607 AAV vector Substances 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 208000013016 Hypoglycemia Diseases 0.000 claims description 2
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims description 2
- 235000001014 amino acid Nutrition 0.000 claims description 2
- 235000018417 cysteine Nutrition 0.000 claims description 2
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 claims description 2
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims description 2
- 230000007812 deficiency Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 230000014101 glucose homeostasis Effects 0.000 claims description 2
- 201000004541 glycogen storage disease I Diseases 0.000 claims description 2
- 230000002218 hypoglycaemic effect Effects 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 239000002245 particle Substances 0.000 claims description 2
- 239000013647 rAAV8 vector Substances 0.000 claims description 2
- 230000008085 renal dysfunction Effects 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 230000003612 virological effect Effects 0.000 claims description 2
- 201000009030 Carcinoma Diseases 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000003462 vein Anatomy 0.000 claims 1
- 238000000034 method Methods 0.000 description 7
- 206010018464 Glycogen storage disease type I Diseases 0.000 description 3
- 208000011460 glycogen storage disease due to glucose-6-phosphatase deficiency type IA Diseases 0.000 description 3
- 206010019629 Hepatic adenoma Diseases 0.000 description 2
- 201000002735 hepatocellular adenoma Diseases 0.000 description 2
- 206010073071 hepatocellular carcinoma Diseases 0.000 description 2
- 231100000844 hepatocellular carcinoma Toxicity 0.000 description 2
- 230000001737 promoting effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462096400P | 2014-12-23 | 2014-12-23 | |
| US62/096,400 | 2014-12-23 | ||
| PCT/US2015/067338 WO2016106303A1 (en) | 2014-12-23 | 2015-12-22 | Adeno-associated virus vectors encoding modified g6pc and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019220144A Division JP2020054367A (ja) | 2014-12-23 | 2019-12-05 | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018501791A JP2018501791A (ja) | 2018-01-25 |
| JP2018501791A5 true JP2018501791A5 (https=) | 2018-12-06 |
| JP6824169B2 JP6824169B2 (ja) | 2021-02-03 |
Family
ID=55272587
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017533845A Active JP6824169B2 (ja) | 2014-12-23 | 2015-12-22 | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 |
| JP2019220144A Pending JP2020054367A (ja) | 2014-12-23 | 2019-12-05 | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019220144A Pending JP2020054367A (ja) | 2014-12-23 | 2019-12-05 | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US10415044B2 (https=) |
| EP (1) | EP3236984B1 (https=) |
| JP (2) | JP6824169B2 (https=) |
| CN (1) | CN107636153B (https=) |
| CA (1) | CA2972038C (https=) |
| ES (1) | ES2824829T3 (https=) |
| IL (1) | IL253103B (https=) |
| WO (1) | WO2016106303A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6824169B2 (ja) | 2014-12-23 | 2021-02-03 | ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 |
| AU2018212002A1 (en) * | 2017-01-30 | 2019-08-29 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Recombinant virus vectors for the treatment of glycogen storage disease |
| US11963974B2 (en) | 2017-03-10 | 2024-04-23 | National Center For Child Health And Development | Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia |
| CN111511915A (zh) | 2018-03-09 | 2020-08-07 | 第一三共株式会社 | 糖原病Ia型治疗药 |
| CA3107572A1 (en) * | 2018-08-08 | 2020-02-13 | Genethon | Mini-gde for the treatment of glycogen storage disease iii |
| WO2020056147A2 (en) * | 2018-09-13 | 2020-03-19 | Modernatx, Inc. | Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease |
| ES2969222T3 (es) * | 2018-12-18 | 2024-05-17 | Ultragenyx Pharmaceutical Inc | Métodos y composiciones para el tratamiento de enfermedades del almacenamiento de glucógeno |
| EP3913060A1 (en) * | 2020-05-22 | 2021-11-24 | Genethon | Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy |
| CN111972355A (zh) * | 2020-08-31 | 2020-11-24 | 中国医学科学院北京协和医院 | GSDIa型糖原累积症小鼠模型及其构建方法 |
| EP4504947A4 (en) * | 2022-04-07 | 2026-03-25 | Univ Duke | COMPOSITIONS AND METHODS TO PROMOTE LIVER REGENERATION BY GENE EDITING IN METABOLIC LIVER DISEASE |
| WO2025165616A1 (en) * | 2024-01-31 | 2025-08-07 | The Johns Hopkins University | Compositions and methods for treating and preventing cancer |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| US6627425B1 (en) * | 2000-06-02 | 2003-09-30 | Millennium Pharmaceuticals, Inc. | Human glucose-6-phosphatase molecules and uses thereof |
| DE602004030327D1 (de) | 2003-05-21 | 2011-01-13 | Genzyme Corp | Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind |
| DK2242840T3 (da) | 2008-01-29 | 2019-10-21 | Applied Genetic Tech Corporation | Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension |
| US20120093775A1 (en) | 2009-03-27 | 2012-04-19 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
| 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 |
| DK2529020T3 (en) | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
| WO2015081101A1 (en) | 2013-11-26 | 2015-06-04 | The United States Of America, As Represented By The Secretary Department Of Health And Human Services | Adeno-associated virus vectors for treatment of glycogen storage disease |
| JP6824169B2 (ja) | 2014-12-23 | 2021-02-03 | ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用 |
-
2015
- 2015-12-22 JP JP2017533845A patent/JP6824169B2/ja active Active
- 2015-12-22 US US15/538,852 patent/US10415044B2/en active Active
- 2015-12-22 WO PCT/US2015/067338 patent/WO2016106303A1/en not_active Ceased
- 2015-12-22 CA CA2972038A patent/CA2972038C/en active Active
- 2015-12-22 EP EP15830932.8A patent/EP3236984B1/en active Active
- 2015-12-22 CN CN201580076462.4A patent/CN107636153B/zh active Active
- 2015-12-22 ES ES15830932T patent/ES2824829T3/es active Active
-
2017
- 2017-06-22 IL IL253103A patent/IL253103B/en active IP Right Grant
-
2019
- 2019-07-30 US US16/526,327 patent/US11535870B2/en active Active
- 2019-12-05 JP JP2019220144A patent/JP2020054367A/ja active Pending
-
2022
- 2022-11-23 US US18/058,457 patent/US12440580B2/en active Active
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