JPWO2021081201A5 - - Google Patents
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- Publication number
- JPWO2021081201A5 JPWO2021081201A5 JP2022523408A JP2022523408A JPWO2021081201A5 JP WO2021081201 A5 JPWO2021081201 A5 JP WO2021081201A5 JP 2022523408 A JP2022523408 A JP 2022523408A JP 2022523408 A JP2022523408 A JP 2022523408A JP WO2021081201 A5 JPWO2021081201 A5 JP WO2021081201A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- seq
- polynucleotide
- operably linked
- 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.)
- Pending
Links
- 108091033319 polynucleotide Proteins 0.000 claims description 46
- 102000040430 polynucleotide Human genes 0.000 claims description 46
- 239000002157 polynucleotide Substances 0.000 claims description 46
- 150000007523 nucleic acids Chemical group 0.000 claims description 31
- 239000013604 expression vector Substances 0.000 claims description 29
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 19
- 239000013598 vector Substances 0.000 claims description 19
- 238000000034 method Methods 0.000 claims description 18
- 230000001105 regulatory effect Effects 0.000 claims description 18
- 230000008488 polyadenylation Effects 0.000 claims description 17
- 210000004027 cell Anatomy 0.000 claims description 16
- 208000015122 neurodegenerative disease Diseases 0.000 claims description 15
- 108010012809 Progranulins Proteins 0.000 claims description 12
- 239000000203 mixture Substances 0.000 claims description 12
- 102000039446 nucleic acids Human genes 0.000 claims description 12
- 108020004707 nucleic acids Proteins 0.000 claims description 12
- 239000008194 pharmaceutical composition Substances 0.000 claims description 12
- 102000019204 Progranulins Human genes 0.000 claims description 10
- 108700019146 Transgenes Proteins 0.000 claims description 9
- 201000011240 Frontotemporal dementia Diseases 0.000 claims description 8
- 101000821100 Homo sapiens Synapsin-1 Proteins 0.000 claims description 8
- 102000002265 Human Growth Hormone Human genes 0.000 claims description 8
- 108010000521 Human Growth Hormone Proteins 0.000 claims description 8
- 239000000854 Human Growth Hormone Substances 0.000 claims description 8
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims description 8
- 241000700584 Simplexvirus Species 0.000 claims description 8
- 210000002569 neuron Anatomy 0.000 claims description 8
- 201000008051 neuronal ceroid lipofuscinosis Diseases 0.000 claims description 8
- 241001529453 unidentified herpesvirus Species 0.000 claims description 8
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 7
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 7
- 108020005345 3' Untranslated Regions Proteins 0.000 claims description 6
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 6
- 241000649047 Adeno-associated virus 12 Species 0.000 claims description 6
- 241000702421 Dependoparvovirus Species 0.000 claims description 6
- 241000700605 Viruses Species 0.000 claims description 6
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims description 6
- 239000002245 particle Substances 0.000 claims description 6
- 230000001124 posttranscriptional effect Effects 0.000 claims description 6
- 101710154606 Hemagglutinin Proteins 0.000 claims description 5
- 101710093908 Outer capsid protein VP4 Proteins 0.000 claims description 5
- 101710135467 Outer capsid protein sigma-1 Proteins 0.000 claims description 5
- 101710176177 Protein A56 Proteins 0.000 claims description 5
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 5
- 102100032889 Sortilin Human genes 0.000 claims description 5
- 210000004899 c-terminal region Anatomy 0.000 claims description 5
- 239000000185 hemagglutinin Substances 0.000 claims description 5
- 230000005764 inhibitory process Effects 0.000 claims description 5
- 108010014657 sortilin Proteins 0.000 claims description 5
- 208000024827 Alzheimer disease Diseases 0.000 claims description 4
- 208000002339 Frontotemporal Lobar Degeneration Diseases 0.000 claims description 4
- 210000000234 capsid Anatomy 0.000 claims description 4
- 210000004962 mammalian cell Anatomy 0.000 claims description 4
- 108090000623 proteins and genes Proteins 0.000 claims description 4
- 101150044789 Cap gene Proteins 0.000 claims description 3
- 239000013607 AAV vector Substances 0.000 claims description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 238000009825 accumulation Methods 0.000 claims description 2
- 230000003542 behavioural effect Effects 0.000 claims description 2
- 210000003169 central nervous system Anatomy 0.000 claims description 2
- 208000010877 cognitive disease Diseases 0.000 claims description 2
- 239000002299 complementary DNA Substances 0.000 claims description 2
- 230000002950 deficient Effects 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 2
- 210000005260 human cell Anatomy 0.000 claims description 2
- 230000002132 lysosomal effect Effects 0.000 claims description 2
- 201000007605 neuronal ceroid lipofuscinosis 11 Diseases 0.000 claims description 2
- 102000004169 proteins and genes Human genes 0.000 claims description 2
- 239000000725 suspension Substances 0.000 claims description 2
- 210000002845 virion Anatomy 0.000 claims description 2
- 108020004705 Codon Proteins 0.000 claims 1
- 241000701022 Cytomegalovirus Species 0.000 description 2
- 241000701085 Human alphaherpesvirus 3 Species 0.000 description 2
- 241000701044 Human gammaherpesvirus 4 Species 0.000 description 2
- 208000003322 Coinfection Diseases 0.000 description 1
- 101001027324 Homo sapiens Progranulin Proteins 0.000 description 1
- 102000054121 human GRN Human genes 0.000 description 1
- 238000007913 intrathecal administration Methods 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 238000007914 intraventricular administration Methods 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 229920001184 polypeptide Polymers 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 108090000765 processed proteins & peptides Proteins 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962924340P | 2019-10-22 | 2019-10-22 | |
| US62/924,340 | 2019-10-22 | ||
| PCT/US2020/056860 WO2021081201A1 (en) | 2019-10-22 | 2020-10-22 | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022553307A JP2022553307A (ja) | 2022-12-22 |
| JPWO2021081201A5 true JPWO2021081201A5 (https=) | 2023-10-30 |
| JP2022553307A5 JP2022553307A5 (https=) | 2023-10-30 |
Family
ID=75620215
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022523408A Pending JP2022553307A (ja) | 2019-10-22 | 2020-10-22 | プログラニュリン関連神経変性疾患または障害の治療のためのアデノ随伴ウイルス（ａａｖ）システム |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20210147872A1 (https=) |
| EP (1) | EP4048799A4 (https=) |
| JP (1) | JP2022553307A (https=) |
| KR (1) | KR20230019402A (https=) |
| CN (1) | CN115715327A (https=) |
| AU (1) | AU2020371662A1 (https=) |
| CA (1) | CA3158516A1 (https=) |
| IL (1) | IL292382A (https=) |
| MX (1) | MX2022004812A (https=) |
| WO (1) | WO2021081201A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2021533126A (ja) * | 2018-07-31 | 2021-12-02 | コーネル・ユニバーシティーCornell University | 臓器機能を制御するための遺伝子治療方法 |
| KR20230128001A (ko) * | 2020-12-01 | 2023-09-01 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도 |
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| WO2024035649A1 (en) * | 2022-08-08 | 2024-02-15 | Shape Therapeutics Inc. | Compositions for progranulin expression and methods of use thereof |
| KR20250131772A (ko) * | 2022-11-13 | 2025-09-03 | 알렉시온 파마 인터내셔널 오퍼레이션즈 리미티드 | 전두측두엽 치매에 대한 유전자 요법 |
| WO2025251041A1 (en) * | 2024-05-31 | 2025-12-04 | Bluerock Therapeutics Lp | METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009089635A1 (en) * | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| JP5879256B2 (ja) * | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| WO2016125330A1 (ja) * | 2015-02-02 | 2016-08-11 | 原 英彰 | 網膜再生促進薬 |
| WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| WO2018136680A1 (en) * | 2017-01-19 | 2018-07-26 | New York University | Progranulin and progranulin derivatives in treating impaired fracture healing |
| EP3692075A4 (en) * | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| KR102709597B1 (ko) * | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| US20220111005A1 (en) * | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| BR112021015817A2 (pt) * | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
-
2020
- 2020-10-22 IL IL292382A patent/IL292382A/en unknown
- 2020-10-22 EP EP20880235.5A patent/EP4048799A4/en active Pending
- 2020-10-22 MX MX2022004812A patent/MX2022004812A/es unknown
- 2020-10-22 CA CA3158516A patent/CA3158516A1/en active Pending
- 2020-10-22 WO PCT/US2020/056860 patent/WO2021081201A1/en not_active Ceased
- 2020-10-22 US US17/077,680 patent/US20210147872A1/en not_active Abandoned
- 2020-10-22 CN CN202080089386.1A patent/CN115715327A/zh active Pending
- 2020-10-22 AU AU2020371662A patent/AU2020371662A1/en not_active Abandoned
- 2020-10-22 KR KR1020227017064A patent/KR20230019402A/ko not_active Ceased
- 2020-10-22 JP JP2022523408A patent/JP2022553307A/ja active Pending
-
2023
- 2023-09-26 US US18/372,868 patent/US20240318199A1/en active Pending
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