JPWO2022115535A5 - - Google Patents
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- Publication number
- JPWO2022115535A5 JPWO2022115535A5 JP2023532127A JP2023532127A JPWO2022115535A5 JP WO2022115535 A5 JPWO2022115535 A5 JP WO2022115535A5 JP 2023532127 A JP2023532127 A JP 2023532127A JP 2023532127 A JP2023532127 A JP 2023532127A JP WO2022115535 A5 JPWO2022115535 A5 JP WO2022115535A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- isolated nucleic
- pharmaceutical composition
- protein
- christchurch
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims 53
- 108020004707 nucleic acids Proteins 0.000 claims 41
- 102000039446 nucleic acids Human genes 0.000 claims 41
- 239000008194 pharmaceutical composition Substances 0.000 claims 15
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 12
- 108090000623 proteins and genes Proteins 0.000 claims 12
- 102000004169 proteins and genes Human genes 0.000 claims 12
- 230000002401 inhibitory effect Effects 0.000 claims 11
- 239000013598 vector Substances 0.000 claims 10
- 238000002347 injection Methods 0.000 claims 8
- 239000007924 injection Substances 0.000 claims 8
- 230000035772 mutation Effects 0.000 claims 7
- 101150037123 APOE gene Proteins 0.000 claims 6
- 239000004055 small Interfering RNA Substances 0.000 claims 6
- 102100029470 Apolipoprotein E Human genes 0.000 claims 5
- 108090000565 Capsid Proteins Proteins 0.000 claims 5
- 102100023321 Ceruloplasmin Human genes 0.000 claims 5
- 210000003169 central nervous system Anatomy 0.000 claims 5
- 108091027967 Small hairpin RNA Proteins 0.000 claims 4
- 230000000694 effects Effects 0.000 claims 4
- 208000024827 Alzheimer disease Diseases 0.000 claims 3
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 3
- 108700011259 MicroRNAs Proteins 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 3
- 239000002679 microRNA Substances 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 101150035190 PSEN1 gene Proteins 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- 208000010877 cognitive disease Diseases 0.000 claims 2
- 208000027061 mild cognitive impairment Diseases 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 230000002093 peripheral effect Effects 0.000 claims 2
- 102200061553 rs63750231 Human genes 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000701022 Cytomegalovirus Species 0.000 claims 1
- 101000934372 Homo sapiens Macrosialin Proteins 0.000 claims 1
- 102100025136 Macrosialin Human genes 0.000 claims 1
- 102100022033 Presenilin-1 Human genes 0.000 claims 1
- 108010036933 Presenilin-1 Proteins 0.000 claims 1
- 230000008499 blood brain barrier function Effects 0.000 claims 1
- 210000001218 blood-brain barrier Anatomy 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 230000003111 delayed effect Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 229920002477 rna polymer Polymers 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063118060P | 2020-11-25 | 2020-11-25 | |
| US63/118,060 | 2020-11-25 | ||
| PCT/US2021/060731 WO2022115535A1 (en) | 2020-11-25 | 2021-11-24 | Gene therapies for neurodegenerative disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023551254A JP2023551254A (ja) | 2023-12-07 |
| JPWO2022115535A5 true JPWO2022115535A5 (https=) | 2024-12-04 |
| JP2023551254A5 JP2023551254A5 (https=) | 2024-12-04 |
Family
ID=81754891
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023532127A Pending JP2023551254A (ja) | 2020-11-25 | 2021-11-24 | 神経変性疾患のための遺伝子治療 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20230405149A1 (https=) |
| EP (1) | EP4251276A4 (https=) |
| JP (1) | JP2023551254A (https=) |
| KR (1) | KR20230112672A (https=) |
| CN (1) | CN116670291A (https=) |
| AU (1) | AU2021386390A1 (https=) |
| CA (1) | CA3203006A1 (https=) |
| IL (1) | IL303156A (https=) |
| MX (1) | MX2023006153A (https=) |
| WO (1) | WO2022115535A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2024258754A1 (en) * | 2023-04-18 | 2025-11-13 | Cornell University | Methods and apoe pharmaceutical compositions for the treatment and the prevention of alzheimers disease |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2000023587A2 (en) * | 1998-10-16 | 2000-04-27 | Introgene B.V. | Gene therapy of alzheimer's disease by delivery of an encoded apolipoprotein e |
| AU2001253233B2 (en) * | 2000-04-06 | 2006-12-07 | Kos Pharmaceuticals, Inc. | Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia |
| JP6469000B2 (ja) * | 2012-05-18 | 2019-02-13 | ユニバーシティー オブ アイオワ リサーチ ファウンデーション | アミロイドの沈着を処置するための方法および組成物 |
| JP7616995B2 (ja) * | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス，インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
| EP4065600A1 (en) * | 2019-11-25 | 2022-10-05 | Cornell University | Apoe gene therapy |
-
2021
- 2021-11-24 KR KR1020237020920A patent/KR20230112672A/ko active Pending
- 2021-11-24 EP EP21899082.8A patent/EP4251276A4/en active Pending
- 2021-11-24 AU AU2021386390A patent/AU2021386390A1/en not_active Abandoned
- 2021-11-24 WO PCT/US2021/060731 patent/WO2022115535A1/en not_active Ceased
- 2021-11-24 JP JP2023532127A patent/JP2023551254A/ja active Pending
- 2021-11-24 CA CA3203006A patent/CA3203006A1/en active Pending
- 2021-11-24 US US18/038,247 patent/US20230405149A1/en active Pending
- 2021-11-24 CN CN202180087728.0A patent/CN116670291A/zh active Pending
- 2021-11-24 MX MX2023006153A patent/MX2023006153A/es unknown
- 2021-11-24 IL IL303156A patent/IL303156A/en unknown
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