JPWO2021016126A5 - - Google Patents
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- Publication number
- JPWO2021016126A5 JPWO2021016126A5 JP2022503802A JP2022503802A JPWO2021016126A5 JP WO2021016126 A5 JPWO2021016126 A5 JP WO2021016126A5 JP 2022503802 A JP2022503802 A JP 2022503802A JP 2022503802 A JP2022503802 A JP 2022503802A JP WO2021016126 A5 JPWO2021016126 A5 JP WO2021016126A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- vector
- raav vector
- seq
- composition
- 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
- 239000013608 rAAV vector Substances 0.000 claims 25
- 108700019146 Transgenes Proteins 0.000 claims 16
- 108090000623 proteins and genes Proteins 0.000 claims 13
- 239000000203 mixture Substances 0.000 claims 11
- 239000002773 nucleotide Substances 0.000 claims 8
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 230000000747 cardiac effect Effects 0.000 claims 7
- 239000013598 vector Substances 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 6
- 208000019622 heart disease Diseases 0.000 claims 5
- 239000002245 particle Substances 0.000 claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 4
- 210000000234 capsid Anatomy 0.000 claims 3
- 108091033319 polynucleotide Proteins 0.000 claims 3
- 239000002157 polynucleotide Substances 0.000 claims 3
- 102000040430 polynucleotide Human genes 0.000 claims 3
- 108010085238 Actins Proteins 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 108090000565 Capsid Proteins Proteins 0.000 claims 2
- 102100023321 Ceruloplasmin Human genes 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 230000004217 heart function Effects 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 102000007469 Actins Human genes 0.000 claims 1
- 241001655883 Adeno-associated virus - 1 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
- 206010056370 Congestive cardiomyopathy Diseases 0.000 claims 1
- 201000010046 Dilated cardiomyopathy Diseases 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
- 102100023097 Protein S100-A1 Human genes 0.000 claims 1
- 101710156967 Protein S100-A1 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
- 102000004987 Troponin T Human genes 0.000 claims 1
- 108090001108 Troponin T 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
- 230000006907 apoptotic process Effects 0.000 claims 1
- 210000004351 coronary vessel Anatomy 0.000 claims 1
- 206010020871 hypertrophic cardiomyopathy Diseases 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
- 210000001908 sarcoplasmic reticulum Anatomy 0.000 claims 1
- 210000002027 skeletal muscle Anatomy 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962876540P | 2019-07-19 | 2019-07-19 | |
| US62/876,540 | 2019-07-19 | ||
| PCT/US2020/042663 WO2021016126A1 (en) | 2019-07-19 | 2020-07-17 | Aav cardiac gene therapy for cardiomyopathy in humans |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022541793A JP2022541793A (ja) | 2022-09-27 |
| JPWO2021016126A5 true JPWO2021016126A5 (https=) | 2023-07-26 |
| JP2022541793A5 JP2022541793A5 (https=) | 2023-07-26 |
Family
ID=74193792
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022503802A Pending JP2022541793A (ja) | 2019-07-19 | 2020-07-17 | ヒトにおける心筋症のためのａａｖ心臓遺伝子治療 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20220265858A1 (https=) |
| EP (1) | EP3999074A4 (https=) |
| JP (1) | JP2022541793A (https=) |
| KR (1) | KR20220034801A (https=) |
| CN (1) | CN114206351A (https=) |
| AR (1) | AR122289A1 (https=) |
| AU (1) | AU2020316339A1 (https=) |
| BR (1) | BR112022000898A2 (https=) |
| CA (1) | CA3142534A1 (https=) |
| CL (1) | CL2022000115A1 (https=) |
| CO (1) | CO2022001686A2 (https=) |
| IL (1) | IL289667A (https=) |
| MX (1) | MX2022000767A (https=) |
| SG (1) | SG11202112140SA (https=) |
| TW (1) | TW202117017A (https=) |
| WO (1) | WO2021016126A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2023007602A (es) * | 2020-12-23 | 2023-09-21 | Univ Florida | Mayor eficiencia del empaquetamiento de vectores para terapia genica cardiaca. |
| US20250144243A1 (en) | 2022-01-25 | 2025-05-08 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| KR20250135916A (ko) | 2022-12-17 | 2025-09-15 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물 |
| AR133098A1 (es) | 2023-06-29 | 2025-08-27 | Univ Pennsylvania | Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE19915485A1 (de) * | 1999-04-07 | 2000-10-19 | Hugo A Katus | Therapie der Herzinsuffizienz |
| CA2412847A1 (en) * | 2000-06-30 | 2002-01-10 | Collateral Therapeutics, Inc. | Dual recombinant gene therapy compositions and methods of use |
| WO2008054713A2 (en) * | 2006-10-30 | 2008-05-08 | Thomas Jefferson University | Tissue specific gene therapy treatment |
| EP2295072A1 (en) * | 2009-09-15 | 2011-03-16 | Max-Delbrück-Centrum für Molekulare Medizin (MDC) | Use of ARC for inhibiting cell death during liver failure |
| CA2925691A1 (en) * | 2013-10-01 | 2015-04-09 | Ruprecht-Karls-Univeristat Heidelberg | S100 based treatment of cardiac power failure |
| EP3452101A2 (en) * | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
| EP3694996A1 (en) * | 2017-10-12 | 2020-08-19 | Universität Heidelberg | Kcnk3-based gene therapy of cardiac arrhythmia |
| US20210260215A1 (en) * | 2018-06-08 | 2021-08-26 | University Of Florida Research Foundation, Incorporated | Aav cardiac gene therapy for cardiomyopathy |
-
2020
- 2020-07-17 BR BR112022000898A patent/BR112022000898A2/pt unknown
- 2020-07-17 US US17/628,139 patent/US20220265858A1/en active Pending
- 2020-07-17 EP EP20844818.3A patent/EP3999074A4/en active Pending
- 2020-07-17 CA CA3142534A patent/CA3142534A1/en active Pending
- 2020-07-17 TW TW109124287A patent/TW202117017A/zh unknown
- 2020-07-17 AU AU2020316339A patent/AU2020316339A1/en not_active Abandoned
- 2020-07-17 JP JP2022503802A patent/JP2022541793A/ja active Pending
- 2020-07-17 CN CN202080052456.6A patent/CN114206351A/zh active Pending
- 2020-07-17 SG SG11202112140SA patent/SG11202112140SA/en unknown
- 2020-07-17 WO PCT/US2020/042663 patent/WO2021016126A1/en not_active Ceased
- 2020-07-17 MX MX2022000767A patent/MX2022000767A/es unknown
- 2020-07-17 AR ARP200102021A patent/AR122289A1/es not_active Application Discontinuation
- 2020-07-17 KR KR1020227002673A patent/KR20220034801A/ko not_active Ceased
-
2022
- 2022-01-06 IL IL289667A patent/IL289667A/en unknown
- 2022-01-17 CL CL2022000115A patent/CL2022000115A1/es unknown
- 2022-02-17 CO CONC2022/0001686A patent/CO2022001686A2/es unknown
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