JPWO2020132115A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020132115A5 JPWO2020132115A5 JP2021534702A JP2021534702A JPWO2020132115A5 JP WO2020132115 A5 JPWO2020132115 A5 JP WO2020132115A5 JP 2021534702 A JP2021534702 A JP 2021534702A JP 2021534702 A JP2021534702 A JP 2021534702A JP WO2020132115 A5 JPWO2020132115 A5 JP WO2020132115A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- raav
- nucleic acid
- composition
- acid molecule
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims description 44
- 108020004707 nucleic acids Proteins 0.000 claims description 38
- 102000039446 nucleic acids Human genes 0.000 claims description 38
- 101000930910 Homo sapiens Glucose-6-phosphatase catalytic subunit 1 Proteins 0.000 claims description 34
- 102100036264 Glucose-6-phosphatase catalytic subunit 1 Human genes 0.000 claims description 32
- 239000013598 vector Substances 0.000 claims description 29
- 238000000034 method Methods 0.000 claims description 24
- 108091023043 Alu Element Proteins 0.000 claims description 21
- 239000002773 nucleotide Substances 0.000 claims description 17
- 125000003729 nucleotide group Chemical group 0.000 claims description 17
- 230000014509 gene expression Effects 0.000 claims description 13
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 12
- 108091026890 Coding region Proteins 0.000 claims description 10
- 206010018464 Glycogen storage disease type I Diseases 0.000 claims description 10
- 241000282414 Homo sapiens Species 0.000 claims description 10
- 208000011460 glycogen storage disease due to glucose-6-phosphatase deficiency type IA Diseases 0.000 claims description 10
- 239000003623 enhancer Substances 0.000 claims description 9
- 239000013607 AAV vector Substances 0.000 claims description 8
- 210000000234 capsid Anatomy 0.000 claims description 8
- 230000008488 polyadenylation Effects 0.000 claims description 8
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 7
- 210000004027 cell Anatomy 0.000 claims description 7
- 241000702421 Dependoparvovirus Species 0.000 claims description 5
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 3
- 238000004113 cell culture Methods 0.000 claims description 3
- 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
- 210000003527 eukaryotic cell Anatomy 0.000 claims description 2
- 238000003306 harvesting Methods 0.000 claims description 2
- 239000013647 rAAV8 vector Substances 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 1
- 241000700605 Viruses Species 0.000 description 4
- 239000013603 viral vector Substances 0.000 description 4
- 238000012217 deletion Methods 0.000 description 3
- 230000037430 deletion Effects 0.000 description 3
- 230000000694 effects Effects 0.000 description 3
- 101100335761 Homo sapiens G6PC1 gene Proteins 0.000 description 2
- 210000004185 liver Anatomy 0.000 description 2
- 108700028146 Genetic Enhancer Elements Proteins 0.000 description 1
- 241000124008 Mammalia Species 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 241000283984 Rodentia Species 0.000 description 1
- 238000001415 gene therapy Methods 0.000 description 1
- 210000003734 kidney Anatomy 0.000 description 1
- 238000004806 packaging method and process Methods 0.000 description 1
- 239000002245 particle Substances 0.000 description 1
- 230000001177 retroviral effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862781380P | 2018-12-18 | 2018-12-18 | |
| US62/781,380 | 2018-12-18 | ||
| PCT/US2019/067247 WO2020132115A1 (en) | 2018-12-18 | 2019-12-18 | Methods and compositions for treating glycogen storage diseases |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022514271A JP2022514271A (ja) | 2022-02-10 |
| JPWO2020132115A5 true JPWO2020132115A5 (https=) | 2022-05-20 |
| JP2022514271A5 JP2022514271A5 (https=) | 2022-05-20 |
| JP7486274B2 JP7486274B2 (ja) | 2024-05-17 |
Family
ID=69400618
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021534702A Active JP7486274B2 (ja) | 2018-12-18 | 2019-12-18 | 糖原病を処置するための方法および組成物 |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20220017922A1 (https=) |
| EP (1) | EP3898981B1 (https=) |
| JP (1) | JP7486274B2 (https=) |
| KR (1) | KR20210105390A (https=) |
| CN (1) | CN113454226B (https=) |
| AU (1) | AU2019405758B2 (https=) |
| BR (1) | BR112021012104A2 (https=) |
| CA (1) | CA3123841A1 (https=) |
| DK (1) | DK3898981T5 (https=) |
| ES (1) | ES2969222T3 (https=) |
| IL (1) | IL283956A (https=) |
| MX (1) | MX2021007379A (https=) |
| PL (1) | PL3898981T3 (https=) |
| PT (1) | PT3898981T (https=) |
| WO (1) | WO2020132115A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20250222134A1 (en) * | 2021-10-19 | 2025-07-10 | University Of Connecticut | Compositions and methods for the treatment of glycogen storage disorders |
Family Cites Families (11)
| 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 |
| JP2002191363A (ja) * | 1999-07-29 | 2002-07-09 | Herikkusu Kenkyusho:Kk | 全長ｃＤＮＡ合成用プライマー、およびその用途 |
| 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 |
| 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 |
| AU2014216160B2 (en) | 2013-02-15 | 2017-03-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | AAV8 retinoschisin expression vector for treating X-linked retinoschisis |
| 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 |
| PL3436593T3 (pl) | 2016-03-28 | 2023-03-27 | Ultragenyx Pharmaceutical Inc. | Sposoby inaktywacji termicznej adenowirusów |
| 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 |
-
2019
- 2019-12-18 ES ES19845789T patent/ES2969222T3/es active Active
- 2019-12-18 MX MX2021007379A patent/MX2021007379A/es unknown
- 2019-12-18 PL PL19845789.7T patent/PL3898981T3/pl unknown
- 2019-12-18 KR KR1020217022096A patent/KR20210105390A/ko active Pending
- 2019-12-18 PT PT198457897T patent/PT3898981T/pt unknown
- 2019-12-18 JP JP2021534702A patent/JP7486274B2/ja active Active
- 2019-12-18 DK DK19845789.7T patent/DK3898981T5/da active
- 2019-12-18 US US17/311,869 patent/US20220017922A1/en active Pending
- 2019-12-18 EP EP19845789.7A patent/EP3898981B1/en active Active
- 2019-12-18 AU AU2019405758A patent/AU2019405758B2/en active Active
- 2019-12-18 CN CN201980083410.8A patent/CN113454226B/zh active Active
- 2019-12-18 BR BR112021012104-8A patent/BR112021012104A2/pt unknown
- 2019-12-18 WO PCT/US2019/067247 patent/WO2020132115A1/en not_active Ceased
- 2019-12-18 CA CA3123841A patent/CA3123841A1/en active Pending
-
2021
- 2021-06-13 IL IL283956A patent/IL283956A/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20230414724A1 (en) | Treatment of hyperbilirubinemia | |
| US20220331409A1 (en) | Factor ix gene therapy | |
| JP2021533805A5 (https=) | ||
| JP2020535804A5 (https=) | ||
| FI3850089T3 (fi) | Polynukleotidejä | |
| JPWO2021226267A5 (https=) | ||
| JPWO2021084133A5 (https=) | ||
| JPWO2020010042A5 (https=) | ||
| JP2022514271A5 (https=) | ||
| JPWO2020132115A5 (https=) | ||
| US20190276856A1 (en) | Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof | |
| JPWO2022015715A5 (https=) | ||
| JP2024528997A (ja) | 放射線誘発性唾液機能低下を予防するためのａｑｐ１遺伝子療法 | |
| JPWO2020038473A5 (https=) | ||
| JPWO2023108159A5 (https=) | ||
| RU2021105703A (ru) | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови IX, и ее применение | |
| JPWO2023108157A5 (https=) | ||
| JP2024529279A (ja) | Ｒｂｍ２０変異のゲノム編集 | |
| WO2024094009A1 (zh) | 用于目的基因的表达盒及其应用 | |
| HK40053434B (en) | Treatment of hyperbilirubinemia | |
| RU2021121087A (ru) | Способы и композиции для лечения болезней накопления гликогена | |
| HK40053434A (en) | Treatment of hyperbilirubinemia | |
| HK40010983A (en) | Nucleic acids comprising a modified intron for use in the treatment of hyperbilirubinemia | |
| JPWO2020028830A5 (https=) | ||
| HK40010983B (en) | Nucleic acids comprising a modified intron for use in the treatment of hyperbilirubinemia |