JPWO2022099007A5 - - Google Patents
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- Publication number
- JPWO2022099007A5 JPWO2022099007A5 JP2023527351A JP2023527351A JPWO2022099007A5 JP WO2022099007 A5 JPWO2022099007 A5 JP WO2022099007A5 JP 2023527351 A JP2023527351 A JP 2023527351A JP 2023527351 A JP2023527351 A JP 2023527351A JP WO2022099007 A5 JPWO2022099007 A5 JP WO2022099007A5
- Authority
- JP
- Japan
- Prior art keywords
- acid sequence
- composition
- capsid polypeptide
- variant
- gjb2
- 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
- 210000000234 capsid Anatomy 0.000 claims description 62
- 229920001184 polypeptide Polymers 0.000 claims description 60
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 60
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 60
- 101000954092 Homo sapiens Gap junction beta-2 protein Proteins 0.000 claims description 50
- 102100037156 Gap junction beta-2 protein Human genes 0.000 claims description 49
- 241000702421 Dependoparvovirus Species 0.000 claims description 38
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 32
- 150000007523 nucleic acids Chemical group 0.000 claims description 28
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 27
- 230000008488 polyadenylation Effects 0.000 claims description 11
- 230000001105 regulatory effect Effects 0.000 claims description 10
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 9
- 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
- 210000004027 cell Anatomy 0.000 claims description 7
- 108091033319 polynucleotide Proteins 0.000 claims description 7
- 102000040430 polynucleotide Human genes 0.000 claims description 7
- 239000002157 polynucleotide Substances 0.000 claims description 7
- 238000006467 substitution reaction Methods 0.000 claims description 6
- 108020005345 3' Untranslated Regions Proteins 0.000 claims description 5
- 241001492404 Woodchuck hepatitis virus Species 0.000 claims description 5
- 230000001124 posttranscriptional effect Effects 0.000 claims description 5
- 108020004705 Codon Proteins 0.000 claims description 4
- 206010011878 Deafness Diseases 0.000 claims description 4
- 101100118093 Drosophila melanogaster eEF1alpha2 gene Proteins 0.000 claims description 4
- 102100039289 Glial fibrillary acidic protein Human genes 0.000 claims description 4
- 101710193519 Glial fibrillary acidic protein Proteins 0.000 claims description 4
- 238000012217 deletion Methods 0.000 claims description 4
- 230000037430 deletion Effects 0.000 claims description 4
- 210000005046 glial fibrillary acidic protein Anatomy 0.000 claims description 4
- 230000010370 hearing loss Effects 0.000 claims description 4
- 231100000888 hearing loss Toxicity 0.000 claims description 4
- 208000016354 hearing loss disease Diseases 0.000 claims description 4
- 238000003780 insertion Methods 0.000 claims description 4
- 230000037431 insertion Effects 0.000 claims description 4
- 108090000623 proteins and genes Proteins 0.000 claims description 4
- 239000002299 complementary DNA Substances 0.000 claims description 3
- 244000309715 mini pig Species 0.000 claims description 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
- 241000580270 Adeno-associated virus - 4 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
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 2
- 241000649047 Adeno-associated virus 12 Species 0.000 claims description 2
- 101100061838 Rattus norvegicus Gjb2 gene Proteins 0.000 claims description 2
- 239000004393 Stigmasterol-rich plant sterol Substances 0.000 claims description 2
- 125000000539 amino acid group Chemical group 0.000 claims description 2
- 210000003027 ear inner Anatomy 0.000 claims description 2
- 238000012224 gene deletion Methods 0.000 claims description 2
- 239000000395 magnesium oxide Substances 0.000 claims description 2
- 239000000441 potassium aluminium silicate Substances 0.000 claims description 2
- 210000001519 tissue Anatomy 0.000 claims description 2
- 230000010415 tropism Effects 0.000 claims description 2
- 210000004899 c-terminal region Anatomy 0.000 description 3
- 239000002773 nucleotide Substances 0.000 description 3
- 125000003729 nucleotide group Chemical group 0.000 description 3
- 101710154606 Hemagglutinin Proteins 0.000 description 2
- 241000699666 Mus <mouse, genus> Species 0.000 description 2
- 101710093908 Outer capsid protein VP4 Proteins 0.000 description 2
- 101710135467 Outer capsid protein sigma-1 Proteins 0.000 description 2
- 101710176177 Protein A56 Proteins 0.000 description 2
- 239000000185 hemagglutinin Substances 0.000 description 2
- 241000700199 Cavia porcellus Species 0.000 description 1
- 241000282326 Felis catus Species 0.000 description 1
- HVLSXIKZNLPZJJ-TXZCQADKSA-N HA peptide Chemical compound C([C@@H](C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](C(C)C)C(=O)N1[C@@H](CCC1)C(=O)N[C@@H](CC(O)=O)C(=O)N[C@@H](CC=1C=CC(O)=CC=1)C(=O)N[C@@H](C)C(O)=O)NC(=O)[C@H]1N(CCC1)C(=O)[C@@H](N)CC=1C=CC(O)=CC=1)C1=CC=C(O)C=C1 HVLSXIKZNLPZJJ-TXZCQADKSA-N 0.000 description 1
- 241001494479 Pecora Species 0.000 description 1
- 241000700159 Rattus Species 0.000 description 1
- 241000282898 Sus scrofa Species 0.000 description 1
- 102000048085 human GJB2 Human genes 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063110697P | 2020-11-06 | 2020-11-06 | |
| US63/110,697 | 2020-11-06 | ||
| US202163146269P | 2021-02-05 | 2021-02-05 | |
| US63/146,269 | 2021-02-05 | ||
| PCT/US2021/058255 WO2022099007A1 (en) | 2020-11-06 | 2021-11-05 | Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023549124A JP2023549124A (ja) | 2023-11-22 |
| JPWO2022099007A5 true JPWO2022099007A5 (https=) | 2024-10-31 |
| JP2023549124A5 JP2023549124A5 (https=) | 2024-10-31 |
Family
ID=81457402
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023527351A Pending JP2023549124A (ja) | 2020-11-06 | 2021-11-05 | 難聴の治療のためのバリアントアデノ随伴ウイルス（ａａｖ）カプシドポリペプチド、及びその遺伝子治療薬 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20240226330A9 (https=) |
| JP (1) | JP2023549124A (https=) |
| KR (1) | KR20230117731A (https=) |
| AU (1) | AU2021376225A1 (https=) |
| CA (1) | CA3197592A1 (https=) |
| IL (1) | IL302653A (https=) |
| MX (1) | MX2023005344A (https=) |
| WO (1) | WO2022099007A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7807380B2 (ja) | 2020-02-21 | 2026-01-27 | アコーオス インコーポレイテッド | ヒト対象において非加齢関連聴力障害を処置するための組成物および方法 |
| WO2024164318A1 (zh) * | 2023-02-10 | 2024-08-15 | 苏州星奥拓维生物技术有限公司 | Gch1抑制剂在制备用于预防和治疗听力损伤的药物中的用途 |
| CN116925239B (zh) * | 2023-07-17 | 2024-10-18 | 苏州星奥拓维生物技术有限公司 | 双载体系统表达Otof基因的组合物和方法 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10294281B2 (en) * | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| US12054724B2 (en) * | 2018-04-10 | 2024-08-06 | President And Fellows Of Harvard College | AAV vectors encoding clarin-1 or GJB2 and uses thereof |
| CN118497276A (zh) * | 2018-11-07 | 2024-08-16 | 阿库斯股份有限公司 | 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途 |
| WO2020148458A1 (en) * | 2019-01-18 | 2020-07-23 | Institut Pasteur | Aav-mediated gene therapy restoring the otoferlin gene |
| CA3153133A1 (en) * | 2019-09-30 | 2021-04-08 | Chris Bartolome | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
-
2021
- 2021-11-05 AU AU2021376225A patent/AU2021376225A1/en active Pending
- 2021-11-05 MX MX2023005344A patent/MX2023005344A/es unknown
- 2021-11-05 IL IL302653A patent/IL302653A/en unknown
- 2021-11-05 CA CA3197592A patent/CA3197592A1/en active Pending
- 2021-11-05 JP JP2023527351A patent/JP2023549124A/ja active Pending
- 2021-11-05 KR KR1020237018930A patent/KR20230117731A/ko active Pending
- 2021-11-05 WO PCT/US2021/058255 patent/WO2022099007A1/en not_active Ceased
-
2023
- 2023-05-05 US US18/143,902 patent/US20240226330A9/en active Pending