JPWO2019237067A5 - - Google Patents
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- Publication number
- JPWO2019237067A5 JPWO2019237067A5 JP2020568431A JP2020568431A JPWO2019237067A5 JP WO2019237067 A5 JPWO2019237067 A5 JP WO2019237067A5 JP 2020568431 A JP2020568431 A JP 2020568431A JP 2020568431 A JP2020568431 A JP 2020568431A JP WO2019237067 A5 JPWO2019237067 A5 JP WO2019237067A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- gene
- transgene
- heart
- optionally
- 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
- 108700019146 Transgenes Proteins 0.000 claims description 26
- 108090000623 proteins and genes Proteins 0.000 claims description 18
- 239000013608 rAAV vector Substances 0.000 claims description 15
- 230000000747 cardiac effect Effects 0.000 claims description 9
- 102000004169 proteins and genes Human genes 0.000 claims description 9
- 229940088872 Apoptosis inhibitor Drugs 0.000 claims description 8
- 239000000158 apoptosis inhibitor Substances 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 4
- 102000039446 nucleic acids Human genes 0.000 claims description 4
- 230000008488 polyadenylation Effects 0.000 claims description 3
- 241000894007 species Species 0.000 claims description 3
- 102100023097 Protein S100-A1 Human genes 0.000 claims description 2
- 101710156967 Protein S100-A1 Proteins 0.000 claims description 2
- 102000004987 Troponin T Human genes 0.000 claims description 2
- 108090001108 Troponin T Proteins 0.000 claims description 2
- 230000006907 apoptotic process Effects 0.000 claims description 2
- 239000002245 particle Substances 0.000 claims 11
- 239000000203 mixture Substances 0.000 claims 9
- 208000019622 heart disease Diseases 0.000 claims 5
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 4
- 239000013598 vector Substances 0.000 claims 4
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 241000124008 Mammalia Species 0.000 claims 3
- 241001465754 Metazoa Species 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 108010085238 Actins Proteins 0.000 claims 2
- 108090000565 Capsid Proteins Proteins 0.000 claims 2
- 102100023321 Ceruloplasmin Human genes 0.000 claims 2
- 102000007469 Actins Human genes 0.000 claims 1
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 101710086403 Carbonic anhydrase-related protein Proteins 0.000 claims 1
- 208000031229 Cardiomyopathies Diseases 0.000 claims 1
- 102000011727 Caspases Human genes 0.000 claims 1
- 108010076667 Caspases Proteins 0.000 claims 1
- 206010056370 Congestive cardiomyopathy Diseases 0.000 claims 1
- 201000010046 Dilated cardiomyopathy Diseases 0.000 claims 1
- 241000282326 Felis catus Species 0.000 claims 1
- 206010019280 Heart failures Diseases 0.000 claims 1
- 108090000362 Lymphotoxin-beta Proteins 0.000 claims 1
- 108010067385 Myosin Light Chains Proteins 0.000 claims 1
- 102000016349 Myosin Light Chains Human genes 0.000 claims 1
- 101710098224 Myosin regulatory light chain 2, atrial isoform Proteins 0.000 claims 1
- 101710105127 Myosin regulatory light chain 2, ventricular/cardiac muscle isoform Proteins 0.000 claims 1
- 108700005078 Synthetic Genes Proteins 0.000 claims 1
- 102000013534 Troponin C Human genes 0.000 claims 1
- 102100036859 Troponin I, cardiac muscle Human genes 0.000 claims 1
- 101710128251 Troponin I, cardiac muscle Proteins 0.000 claims 1
- 108010051583 Ventricular Myosins Proteins 0.000 claims 1
- OIPILFWXSMYKGL-UHFFFAOYSA-N acetylcholine Chemical compound CC(=O)OCC[N+](C)(C)C OIPILFWXSMYKGL-UHFFFAOYSA-N 0.000 claims 1
- 229960004373 acetylcholine Drugs 0.000 claims 1
- 230000001154 acute effect Effects 0.000 claims 1
- 206010020871 hypertrophic cardiomyopathy Diseases 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 208000028867 ischemia Diseases 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 230000003551 muscarinic effect Effects 0.000 claims 1
- 230000002107 myocardial effect Effects 0.000 claims 1
- 210000002027 skeletal muscle Anatomy 0.000 claims 1
- 108700026220 vif Genes Proteins 0.000 claims 1
- 108020004684 Internal Ribosome Entry Sites Proteins 0.000 description 10
- 102000021350 Caspase recruitment domains Human genes 0.000 description 3
- 108091011189 Caspase recruitment domains Proteins 0.000 description 3
- 108020004999 messenger RNA Proteins 0.000 description 3
- 108091034057 RNA (poly(A)) Proteins 0.000 description 2
- 230000003205 diastolic effect Effects 0.000 description 2
- 230000000977 initiatory effect Effects 0.000 description 2
- 230000014616 translation Effects 0.000 description 2
- 241000282472 Canis lupus familiaris Species 0.000 description 1
- 108091026890 Coding region Proteins 0.000 description 1
- 241000206602 Eukaryota Species 0.000 description 1
- 108091092195 Intron Proteins 0.000 description 1
- 108091027981 Response element Proteins 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 239000003623 enhancer Substances 0.000 description 1
- 108020001507 fusion proteins Proteins 0.000 description 1
- 102000037865 fusion proteins Human genes 0.000 description 1
- 238000001476 gene delivery Methods 0.000 description 1
- -1 initiation sites Proteins 0.000 description 1
- 239000012212 insulator Substances 0.000 description 1
- 238000000034 method Methods 0.000 description 1
- 230000003387 muscular Effects 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 238000001243 protein synthesis Methods 0.000 description 1
- 238000007634 remodeling Methods 0.000 description 1
- 230000014621 translational initiation Effects 0.000 description 1
- 230000002861 ventricular Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024062211A JP2024097779A (ja) | 2018-06-08 | 2024-04-08 | 心筋症のためのａａｖ心臓遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862682772P | 2018-06-08 | 2018-06-08 | |
| US62/682,772 | 2018-06-08 | ||
| US201962822015P | 2019-03-21 | 2019-03-21 | |
| US62/822,015 | 2019-03-21 | ||
| PCT/US2019/036157 WO2019237067A1 (en) | 2018-06-08 | 2019-06-07 | Aav cardiac gene therapy for cardiomyopathy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024062211A Division JP2024097779A (ja) | 2018-06-08 | 2024-04-08 | 心筋症のためのａａｖ心臓遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021526818A JP2021526818A (ja) | 2021-10-11 |
| JP2021526818A5 JP2021526818A5 (https=) | 2024-05-15 |
| JPWO2019237067A5 true JPWO2019237067A5 (https=) | 2024-05-15 |
Family
ID=68770676
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020568431A Pending JP2021526818A (ja) | 2018-06-08 | 2019-06-07 | 心筋症のためのａａｖ心臓遺伝子治療 |
| JP2024062211A Pending JP2024097779A (ja) | 2018-06-08 | 2024-04-08 | 心筋症のためのａａｖ心臓遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024062211A Pending JP2024097779A (ja) | 2018-06-08 | 2024-04-08 | 心筋症のためのａａｖ心臓遺伝子治療 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20210260215A1 (https=) |
| EP (1) | EP3814512A4 (https=) |
| JP (2) | JP2021526818A (https=) |
| KR (1) | KR20210018902A (https=) |
| CN (1) | CN112272705A (https=) |
| AU (1) | AU2019282822B2 (https=) |
| BR (1) | BR112020024935A2 (https=) |
| CA (1) | CA3100280A1 (https=) |
| CL (1) | CL2020003190A1 (https=) |
| CO (1) | CO2020016718A2 (https=) |
| IL (1) | IL279225B2 (https=) |
| MX (1) | MX421375B (https=) |
| SG (1) | SG11202011061SA (https=) |
| WO (1) | WO2019237067A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112022000898A2 (pt) * | 2019-07-19 | 2022-06-07 | Univ Florida | Terapia de gene cardíaco aav para cardiomiopatia em seres humanos |
| BR112023001336A2 (pt) * | 2020-08-05 | 2023-02-14 | Spacecraft Seven Llc | Terapia gênica de csrp3 (proteína 3 rica em cisteína e glicina) |
| MX2023007602A (es) * | 2020-12-23 | 2023-09-21 | Univ Florida | Mayor eficiencia del empaquetamiento de vectores para terapia genica cardiaca. |
| CA3252359A1 (en) * | 2022-03-02 | 2023-09-07 | Cevec Pharmaceuticals Gmbh | IMPROVED CELL LINES AND PROCESSES FOR THE PRODUCTION OF ADENO-ASSOCIATED VECTORS |
| EP4239063A1 (en) * | 2022-03-02 | 2023-09-06 | CEVEC Pharmaceuticals GmbH | Improved cell lines and methods for the production of adeno-associated vectors |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5962313A (en) | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| CN105452458A (zh) * | 2013-06-07 | 2016-03-30 | 加利福尼亚大学董事会 | 用于治疗心力衰竭和增加心脏功能的不能产生环腺苷酸的腺苷酸环化酶和组合物和方法 |
| DK3405215T3 (da) * | 2016-01-19 | 2022-09-19 | Univ California | Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser |
| US20170348387A1 (en) * | 2016-02-29 | 2017-12-07 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for nphp5 lca-ciliopathy |
-
2019
- 2019-06-07 US US16/972,740 patent/US20210260215A1/en active Pending
- 2019-06-07 WO PCT/US2019/036157 patent/WO2019237067A1/en not_active Ceased
- 2019-06-07 CN CN201980038176.7A patent/CN112272705A/zh active Pending
- 2019-06-07 MX MX2020013313A patent/MX421375B/es unknown
- 2019-06-07 BR BR112020024935-1A patent/BR112020024935A2/pt unknown
- 2019-06-07 CA CA3100280A patent/CA3100280A1/en active Pending
- 2019-06-07 IL IL279225A patent/IL279225B2/en unknown
- 2019-06-07 SG SG11202011061SA patent/SG11202011061SA/en unknown
- 2019-06-07 AU AU2019282822A patent/AU2019282822B2/en active Active
- 2019-06-07 JP JP2020568431A patent/JP2021526818A/ja active Pending
- 2019-06-07 EP EP19815084.9A patent/EP3814512A4/en active Pending
- 2019-06-07 KR KR1020217000163A patent/KR20210018902A/ko not_active Ceased
-
2020
- 2020-12-07 CL CL2020003190A patent/CL2020003190A1/es unknown
- 2020-12-31 CO CONC2020/0016718A patent/CO2020016718A2/es unknown
-
2024
- 2024-04-08 JP JP2024062211A patent/JP2024097779A/ja active Pending
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