JPWO2022053630A5 - - Google Patents
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- Publication number
- JPWO2022053630A5 JPWO2022053630A5 JP2023515813A JP2023515813A JPWO2022053630A5 JP WO2022053630 A5 JPWO2022053630 A5 JP WO2022053630A5 JP 2023515813 A JP2023515813 A JP 2023515813A JP 2023515813 A JP2023515813 A JP 2023515813A JP WO2022053630 A5 JPWO2022053630 A5 JP WO2022053630A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- sequence
- gene
- aav capsid
- seq
- 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
- 108090000565 Capsid Proteins Proteins 0.000 claims 15
- 102100023321 Ceruloplasmin Human genes 0.000 claims 15
- 108090000623 proteins and genes Proteins 0.000 claims 8
- 108090000765 processed proteins & peptides Proteins 0.000 claims 7
- 150000001413 amino acids Chemical class 0.000 claims 6
- 230000037431 insertion Effects 0.000 claims 6
- 238000003780 insertion Methods 0.000 claims 6
- 230000008685 targeting Effects 0.000 claims 5
- 239000013607 AAV vector Substances 0.000 claims 4
- 210000003205 muscle Anatomy 0.000 claims 4
- 239000002245 particle Substances 0.000 claims 4
- 230000001225 therapeutic effect Effects 0.000 claims 4
- 125000003275 alpha amino acid group Chemical group 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- 208000029578 Muscle disease Diseases 0.000 claims 2
- 238000001415 gene therapy Methods 0.000 claims 2
- 238000010362 genome editing Methods 0.000 claims 2
- 108091033319 polynucleotide Proteins 0.000 claims 2
- 102000040430 polynucleotide Human genes 0.000 claims 2
- 239000002157 polynucleotide Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 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
- 102100030685 Alpha-sarcoglycan Human genes 0.000 claims 1
- 102100036524 Anoctamin-5 Human genes 0.000 claims 1
- 108020005544 Antisense RNA Proteins 0.000 claims 1
- 102100030686 Beta-sarcoglycan Human genes 0.000 claims 1
- 102100032539 Calpain-3 Human genes 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 102100035425 DnaJ homolog subfamily B member 6 Human genes 0.000 claims 1
- 102100032248 Dysferlin Human genes 0.000 claims 1
- 102100024108 Dystrophin Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 102100021792 Gamma-sarcoglycan Human genes 0.000 claims 1
- 108020005004 Guide RNA Proteins 0.000 claims 1
- 101000703500 Homo sapiens Alpha-sarcoglycan Proteins 0.000 claims 1
- 101000928364 Homo sapiens Anoctamin-5 Proteins 0.000 claims 1
- 101000703495 Homo sapiens Beta-sarcoglycan Proteins 0.000 claims 1
- 101000867715 Homo sapiens Calpain-3 Proteins 0.000 claims 1
- 101000804112 Homo sapiens DnaJ homolog subfamily B member 6 Proteins 0.000 claims 1
- 101001016184 Homo sapiens Dysferlin Proteins 0.000 claims 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 1
- 101000616435 Homo sapiens Gamma-sarcoglycan Proteins 0.000 claims 1
- 101000846198 Homo sapiens Ribitol 5-phosphate transferase FKRP Proteins 0.000 claims 1
- 108010021625 Immunoglobulin Fragments Proteins 0.000 claims 1
- 102000008394 Immunoglobulin Fragments Human genes 0.000 claims 1
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims 1
- 102100031774 Ribitol 5-phosphate transferase FKRP Human genes 0.000 claims 1
- 101001053942 Saccharolobus solfataricus (strain ATCC 35092 / DSM 1617 / JCM 11322 / P2) Diphosphomevalonate decarboxylase Proteins 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 239000003184 complementary RNA Substances 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 230000002452 interceptive effect Effects 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 210000004165 myocardium Anatomy 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 208000022587 qualitative or quantitative defects of dystrophin Diseases 0.000 claims 1
- 210000002027 skeletal muscle Anatomy 0.000 claims 1
- 238000011144 upstream manufacturing Methods 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP20306005.8 | 2020-09-10 | ||
| EP20306005 | 2020-09-10 | ||
| PCT/EP2021/074964 WO2022053630A1 (fr) | 2020-09-10 | 2021-09-10 | Capside de aav modifiée par peptide |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023544683A JP2023544683A (ja) | 2023-10-25 |
| JPWO2022053630A5 true JPWO2022053630A5 (fr) | 2024-09-19 |
Family
ID=72717827
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023515813A Pending JP2023544683A (ja) | 2020-09-10 | 2021-09-10 | ペプチド改変されたａａｖカプシド |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240209030A1 (fr) |
| EP (2) | EP4211231B1 (fr) |
| JP (1) | JP2023544683A (fr) |
| CN (1) | CN116096431A (fr) |
| BR (1) | BR112023004176A2 (fr) |
| CA (1) | CA3193128A1 (fr) |
| DK (1) | DK4211231T3 (fr) |
| FI (1) | FI4211231T3 (fr) |
| PT (1) | PT4211231T (fr) |
| WO (1) | WO2022053630A1 (fr) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022204476A1 (fr) | 2021-03-26 | 2022-09-29 | The Board Of Regents Of The University Of Texas System | Édition de nucléotides pour remettre en phase des transcrits de la dmd par édition de base et édition génomique prémium (« prime editing ») |
| JP2024529540A (ja) | 2021-08-04 | 2024-08-06 | ジェネトン | 筋肉及びｃｎｓにおける遺伝子発現のためのハイブリッドプロモーター |
| WO2023237731A1 (fr) | 2022-06-09 | 2023-12-14 | Genethon | Gde tronquée au niveau du terminal n pour le traitement de la maladie de stockage du glycogène de type iii |
| WO2023237748A1 (fr) | 2022-06-10 | 2023-12-14 | Genethon | Capside d'aav modifiée par un peptide présentant une efficacité de transduction musculaire améliorée |
| WO2024074727A1 (fr) | 2022-10-07 | 2024-04-11 | Genethon | Immunothérapie cellulaire par car-t anti-fap pour traiter des myopathies squelettiques |
| WO2024080311A1 (fr) * | 2022-10-12 | 2024-04-18 | 昌和 栗田 | Procédé d'introduction de gène, procédé de thérapie génique et procédé de régénération de tissus |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN116004721A (zh) * | 2013-07-12 | 2023-04-25 | 费城儿童医院 | Aav载体和用于抗aav（腺相关病毒）中和抗体的检测 |
| SG11201811603WA (en) * | 2016-07-26 | 2019-02-27 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| MA51353B1 (fr) | 2018-04-05 | 2022-09-30 | Univ Sorbonne | Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit |
| CN112040988A (zh) | 2018-04-27 | 2020-12-04 | 海德堡大学 | 修饰的aav衣壳多肽用于治疗肌肉疾病 |
-
2021
- 2021-09-10 CN CN202180055064.XA patent/CN116096431A/zh active Pending
- 2021-09-10 JP JP2023515813A patent/JP2023544683A/ja active Pending
- 2021-09-10 DK DK21777271.4T patent/DK4211231T3/da active
- 2021-09-10 WO PCT/EP2021/074964 patent/WO2022053630A1/fr active Search and Examination
- 2021-09-10 EP EP21777271.4A patent/EP4211231B1/fr active Active
- 2021-09-10 US US18/024,103 patent/US20240209030A1/en active Pending
- 2021-09-10 BR BR112023004176A patent/BR112023004176A2/pt unknown
- 2021-09-10 EP EP24183857.2A patent/EP4421179A2/fr active Pending
- 2021-09-10 CA CA3193128A patent/CA3193128A1/fr active Pending
- 2021-09-10 FI FIEP21777271.4T patent/FI4211231T3/fi active
- 2021-09-10 PT PT217772714T patent/PT4211231T/pt unknown
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