JPWO2022082017A5 - - Google Patents
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- Publication number
- JPWO2022082017A5 JPWO2022082017A5 JP2023523244A JP2023523244A JPWO2022082017A5 JP WO2022082017 A5 JPWO2022082017 A5 JP WO2022082017A5 JP 2023523244 A JP2023523244 A JP 2023523244A JP 2023523244 A JP2023523244 A JP 2023523244A JP WO2022082017 A5 JPWO2022082017 A5 JP WO2022082017A5
- Authority
- JP
- Japan
- Prior art keywords
- therapeutic composition
- raav
- insect cells
- particles
- eluate
- 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.)
- Pending
Links
- 239000002245 particle Substances 0.000 claims description 19
- 101001027324 Homo sapiens Progranulin Proteins 0.000 claims description 4
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 claims description 4
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 4
- 102000054121 human GRN Human genes 0.000 claims description 4
- 239000002773 nucleotide Substances 0.000 claims description 3
- 125000003729 nucleotide group Chemical group 0.000 claims description 3
- CTKXFMQHOOWWEB-UHFFFAOYSA-N Ethylene oxide/propylene oxide copolymer Chemical compound CCCOC(C)COCCO CTKXFMQHOOWWEB-UHFFFAOYSA-N 0.000 claims description 2
- 239000007983 Tris buffer Substances 0.000 claims description 2
- 229910001629 magnesium chloride Inorganic materials 0.000 claims description 2
- 229920001993 poloxamer 188 Polymers 0.000 claims description 2
- 229940044519 poloxamer 188 Drugs 0.000 claims description 2
- 239000011780 sodium chloride Substances 0.000 claims description 2
- LENZDBCJOHFCAS-UHFFFAOYSA-N tris Chemical compound OCC(N)(CO)CO LENZDBCJOHFCAS-UHFFFAOYSA-N 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 36
- 230000001225 therapeutic effect Effects 0.000 claims 35
- 241000238631 Hexapoda Species 0.000 claims 12
- 108090000623 proteins and genes Proteins 0.000 claims 10
- 239000013592 cell lysate Substances 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 102000004547 Glucosylceramidase Human genes 0.000 claims 4
- 108010017544 Glucosylceramidase Proteins 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 241000701447 unidentified baculovirus Species 0.000 claims 4
- 239000013598 vector Substances 0.000 claims 4
- 108090000565 Capsid Proteins Proteins 0.000 claims 2
- 102100023321 Ceruloplasmin Human genes 0.000 claims 2
- 238000001042 affinity chromatography Methods 0.000 claims 2
- 208000015181 infectious disease Diseases 0.000 claims 2
- 230000002934 lysing effect Effects 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 102000019204 Progranulins Human genes 0.000 claims 1
- 108010012809 Progranulins Proteins 0.000 claims 1
- 238000005571 anion exchange chromatography Methods 0.000 claims 1
- 239000011230 binding agent Substances 0.000 claims 1
- 239000000872 buffer Substances 0.000 claims 1
- 239000006143 cell culture medium Substances 0.000 claims 1
- 238000009295 crossflow filtration Methods 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 239000002158 endotoxin Substances 0.000 claims 1
- 239000012535 impurity Substances 0.000 claims 1
- 230000002458 infectious effect Effects 0.000 claims 1
- 230000000813 microbial effect Effects 0.000 claims 1
- 229920001983 poloxamer Polymers 0.000 claims 1
- 239000012905 visible particle Substances 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 description 4
- 108020004414 DNA Proteins 0.000 description 3
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- 150000007523 nucleic acids Chemical group 0.000 description 2
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 1
- 238000011002 quantification Methods 0.000 description 1
- 239000013608 rAAV vector Substances 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063092179P | 2020-10-15 | 2020-10-15 | |
| US63/092,179 | 2020-10-15 | ||
| PCT/US2021/055243 WO2022082017A2 (en) | 2020-10-15 | 2021-10-15 | Recombinant adeno-associated virus compositions and methods for producing same |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023545830A JP2023545830A (ja) | 2023-10-31 |
| JPWO2022082017A5 true JPWO2022082017A5 (https=) | 2024-10-24 |
| JP2023545830A5 JP2023545830A5 (https=) | 2024-10-24 |
Family
ID=78819612
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023523244A Pending JP2023545830A (ja) | 2020-10-15 | 2021-10-15 | 組換えアデノ随伴ウイルス組成物及びその生産方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20220119843A1 (https=) |
| EP (1) | EP4229206A2 (https=) |
| JP (1) | JP2023545830A (https=) |
| KR (1) | KR20230088393A (https=) |
| CN (1) | CN116710567A (https=) |
| AU (1) | AU2021359852A1 (https=) |
| CA (1) | CA3198675A1 (https=) |
| IL (1) | IL302109A (https=) |
| MX (1) | MX2023004420A (https=) |
| WO (1) | WO2022082017A2 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4598940A1 (en) | 2022-10-04 | 2025-08-13 | Eli Lilly and Company | Gene therapy for trem2-associated diseases and disorders |
| EP4615981A1 (en) | 2022-11-11 | 2025-09-17 | Eli Lilly and Company | Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same |
| CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| TW202432839A (zh) * | 2022-12-06 | 2024-08-16 | 美商再生元醫藥公司 | 用於確定病毒載體基因體滴度之液滴式數位ｐｃｒ分析 |
| WO2025190864A1 (en) * | 2024-03-11 | 2025-09-18 | Sartorius Stedim Biotech Gmbh | Improved cell lysis methods, reagents, and kits for viral vector release and production |
| WO2026043765A1 (en) | 2024-08-21 | 2026-02-26 | Eli Lilly And Company | Engineered regulatory splice elements for gene therapies |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2002359284A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| WO2007084773A2 (en) * | 2006-01-20 | 2007-07-26 | University Of North Carolina At Chapel Hill | Enhanced production of infectious parvovirus vectors in insect cells |
| EP3387138B1 (en) * | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| CN106916793B (zh) * | 2015-12-24 | 2019-01-15 | 中国科学院武汉物理与数学研究所 | 一种重组腺相关病毒的制备方法及重组杆状病毒 |
| US20200316221A1 (en) * | 2016-10-13 | 2020-10-08 | University Of Massachusetts | Aav capsid designs |
| EP3692075A4 (en) * | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| KR102709597B1 (ko) * | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| AU2018354195A1 (en) | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| MX2020004830A (es) * | 2017-11-08 | 2020-11-11 | Avexis Inc | Medios y metodo para preparar vectores virales y usos de los mismos. |
-
2021
- 2021-10-15 KR KR1020237015788A patent/KR20230088393A/ko active Pending
- 2021-10-15 US US17/502,979 patent/US20220119843A1/en active Pending
- 2021-10-15 IL IL302109A patent/IL302109A/en unknown
- 2021-10-15 MX MX2023004420A patent/MX2023004420A/es unknown
- 2021-10-15 CA CA3198675A patent/CA3198675A1/en active Pending
- 2021-10-15 CN CN202180078052.9A patent/CN116710567A/zh active Pending
- 2021-10-15 AU AU2021359852A patent/AU2021359852A1/en active Pending
- 2021-10-15 JP JP2023523244A patent/JP2023545830A/ja active Pending
- 2021-10-15 WO PCT/US2021/055243 patent/WO2022082017A2/en not_active Ceased
- 2021-10-15 EP EP21816205.5A patent/EP4229206A2/en active Pending
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