JPWO2022031756A5 - - Google Patents
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- Publication number
- JPWO2022031756A5 JPWO2022031756A5 JP2023507388A JP2023507388A JPWO2022031756A5 JP WO2022031756 A5 JPWO2022031756 A5 JP WO2022031756A5 JP 2023507388 A JP2023507388 A JP 2023507388A JP 2023507388 A JP2023507388 A JP 2023507388A JP WO2022031756 A5 JPWO2022031756 A5 JP WO2022031756A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- mlp
- polynucleotide
- seq
- vector
- 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
- 108010023942 cysteine and glycine-rich protein 3 Proteins 0.000 claims description 63
- 102000040430 polynucleotide Human genes 0.000 claims description 63
- 108091033319 polynucleotide Proteins 0.000 claims description 63
- 239000002157 polynucleotide Substances 0.000 claims description 63
- 102100031620 Cysteine and glycine-rich protein 3 Human genes 0.000 claims description 59
- 239000013598 vector Substances 0.000 claims description 32
- 238000000034 method Methods 0.000 claims description 27
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 20
- 239000013608 rAAV vector Substances 0.000 claims description 12
- 239000008194 pharmaceutical composition Substances 0.000 claims description 11
- 201000010099 disease Diseases 0.000 claims description 10
- 208000035475 disorder Diseases 0.000 claims description 10
- 230000000747 cardiac effect Effects 0.000 claims description 8
- 210000004027 cell Anatomy 0.000 claims description 8
- 210000004413 cardiac myocyte Anatomy 0.000 claims description 7
- 108090000565 Capsid Proteins Proteins 0.000 claims description 5
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 5
- 230000000295 complement effect Effects 0.000 claims description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- 101100402552 Homo sapiens MARCKSL1 gene Proteins 0.000 claims description 4
- 102000002265 Human Growth Hormone Human genes 0.000 claims description 4
- 108010000521 Human Growth Hormone Proteins 0.000 claims description 4
- 239000000854 Human Growth Hormone Substances 0.000 claims description 4
- 102000004987 Troponin T Human genes 0.000 claims description 4
- 108090001108 Troponin T Proteins 0.000 claims description 4
- 238000001415 gene therapy Methods 0.000 claims description 4
- 102000001708 Protein Isoforms Human genes 0.000 claims description 3
- 108010029485 Protein Isoforms Proteins 0.000 claims description 3
- VYXXMAGSIYIYGD-NWAYQTQBSA-N propan-2-yl 2-[[[(2R)-1-(6-aminopurin-9-yl)propan-2-yl]oxymethyl-(pyrimidine-4-carbonylamino)phosphoryl]amino]-2-methylpropanoate Chemical compound CC(C)OC(=O)C(C)(C)NP(=O)(CO[C@H](C)Cn1cnc2c(N)ncnc12)NC(=O)c1ccncn1 VYXXMAGSIYIYGD-NWAYQTQBSA-N 0.000 claims description 3
- 239000013607 AAV vector Substances 0.000 claims description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 101150062275 CSRP3 gene Proteins 0.000 claims description 2
- 208000020446 Cardiac disease Diseases 0.000 claims description 2
- 206010056370 Congestive cardiomyopathy Diseases 0.000 claims description 2
- 201000010046 Dilated cardiomyopathy Diseases 0.000 claims description 2
- 206010019280 Heart failures Diseases 0.000 claims description 2
- 241000124008 Mammalia Species 0.000 claims description 2
- 241000288906 Primates Species 0.000 claims description 2
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims description 2
- 150000001413 amino acids Chemical group 0.000 claims description 2
- 102220149772 c.164A>G Human genes 0.000 claims description 2
- 208000019622 heart disease Diseases 0.000 claims description 2
- 206010020871 hypertrophic cardiomyopathy Diseases 0.000 claims description 2
- 238000001802 infusion Methods 0.000 claims description 2
- 239000007925 intracardiac injection Substances 0.000 claims description 2
- 238000010253 intravenous injection Methods 0.000 claims description 2
- 210000003205 muscle Anatomy 0.000 claims description 2
- 230000035772 mutation Effects 0.000 claims description 2
- 230000001124 posttranscriptional effect Effects 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 230000001105 regulatory effect Effects 0.000 claims description 2
- 102200026988 rs104894204 Human genes 0.000 claims description 2
- 102200026998 rs104894205 Human genes 0.000 claims description 2
- 102200026990 rs137852764 Human genes 0.000 claims description 2
- 102200079915 rs193302898 Human genes 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 230000001969 hypertrophic effect Effects 0.000 claims 1
- 239000000203 mixture Substances 0.000 description 2
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063061727P | 2020-08-05 | 2020-08-05 | |
| US63/061,727 | 2020-08-05 | ||
| PCT/US2021/044412 WO2022031756A1 (en) | 2020-08-05 | 2021-08-03 | Csrp3 (cysteine and glycine rich protein 3) gene therapy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023536618A JP2023536618A (ja) | 2023-08-28 |
| JP2023536618A5 JP2023536618A5 (https=) | 2024-08-08 |
| JPWO2022031756A5 true JPWO2022031756A5 (https=) | 2024-08-08 |
Family
ID=80118758
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023507388A Pending JP2023536618A (ja) | 2020-08-05 | 2021-08-03 | Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230257431A1 (https=) |
| EP (1) | EP4192962A4 (https=) |
| JP (1) | JP2023536618A (https=) |
| KR (1) | KR20230042468A (https=) |
| CN (1) | CN116234916A (https=) |
| AU (1) | AU2021321410A1 (https=) |
| BR (1) | BR112023001336A2 (https=) |
| CA (1) | CA3184983A1 (https=) |
| IL (1) | IL300187A (https=) |
| MX (1) | MX2023000994A (https=) |
| WO (1) | WO2022031756A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3021572A1 (en) | 2016-04-20 | 2017-10-26 | Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas | Compositions and methods for enhanced gene expression of pklr |
| EP3697452A4 (en) | 2017-10-16 | 2021-11-24 | Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. | LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS |
| BR112020020841A2 (pt) | 2018-04-11 | 2021-01-19 | Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus | Composições e métodos para transplante de célulastronco |
| CA3096293A1 (en) | 2018-04-27 | 2019-10-31 | Rocket Pharmaceuticals, Ltd. | Gene therapy for cns degeneration |
| MY209860A (en) | 2020-02-13 | 2025-08-07 | Tenaya Therapeutics Inc | Gene therapy vectors for treating heart disease |
| JP2023538519A (ja) | 2020-08-07 | 2023-09-08 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療 |
| EP4479543A1 (en) * | 2022-02-18 | 2024-12-25 | Ginkgo Bioworks, Inc. | Gene therapy for arrhythmogenic cardiomyopathy |
| CA3255650A1 (en) * | 2022-03-18 | 2023-09-21 | Aavantibio, Inc. | METHODS AND COMPOSITIONS FOR TREATING RBM20-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE10004857A1 (de) * | 2000-02-03 | 2001-08-16 | Schering Ag | MLP-Gen, Nukleinsäuren, Polypeptide und deren Verwendung |
| WO2009012176A2 (en) | 2007-07-14 | 2009-01-22 | The University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP2792742A1 (en) * | 2013-04-17 | 2014-10-22 | Universitätsklinikum Hamburg-Eppendorf (UKE) | Gene-therapy vectors for treating cardiomyopathy |
| US9585971B2 (en) | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| MX2019011046A (es) * | 2017-03-17 | 2019-10-17 | Res Inst Nationwide Childrens Hospital | Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular. |
| SG11202002276VA (en) * | 2017-09-20 | 2020-04-29 | 4D Molecular Therapeutics Inc | Adeno-associated virus variant capsids and methods of use thereof |
| AU2019258830B2 (en) * | 2018-04-27 | 2025-12-18 | Universität Heidelberg | Modified AAV capsid polypeptides for treatment of muscular diseases |
| US20210260215A1 (en) * | 2018-06-08 | 2021-08-26 | University Of Florida Research Foundation, Incorporated | Aav cardiac gene therapy for cardiomyopathy |
| CN114450411A (zh) * | 2019-04-01 | 2022-05-06 | 特纳亚治疗股份有限公司 | 具有工程化衣壳的腺相关病毒 |
-
2021
- 2021-08-03 BR BR112023001336A patent/BR112023001336A2/pt unknown
- 2021-08-03 IL IL300187A patent/IL300187A/en unknown
- 2021-08-03 JP JP2023507388A patent/JP2023536618A/ja active Pending
- 2021-08-03 CA CA3184983A patent/CA3184983A1/en active Pending
- 2021-08-03 KR KR1020237003436A patent/KR20230042468A/ko active Pending
- 2021-08-03 AU AU2021321410A patent/AU2021321410A1/en active Pending
- 2021-08-03 EP EP21852527.7A patent/EP4192962A4/en active Pending
- 2021-08-03 US US18/019,396 patent/US20230257431A1/en active Pending
- 2021-08-03 WO PCT/US2021/044412 patent/WO2022031756A1/en not_active Ceased
- 2021-08-03 MX MX2023000994A patent/MX2023000994A/es unknown
- 2021-08-03 CN CN202180057650.8A patent/CN116234916A/zh active Pending
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