JPWO2020180951A5 - - Google Patents
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- Publication number
- JPWO2020180951A5 JPWO2020180951A5 JP2021551770A JP2021551770A JPWO2020180951A5 JP WO2020180951 A5 JPWO2020180951 A5 JP WO2020180951A5 JP 2021551770 A JP2021551770 A JP 2021551770A JP 2021551770 A JP2021551770 A JP 2021551770A JP WO2020180951 A5 JPWO2020180951 A5 JP WO2020180951A5
- Authority
- JP
- Japan
- Prior art keywords
- unit dose
- pharmaceutical composition
- vector genomes
- seq
- capsid protein
- 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.)
- Granted
Links
- 238000000034 method Methods 0.000 claims description 72
- 239000013598 vector Substances 0.000 claims description 56
- 239000008194 pharmaceutical composition Substances 0.000 claims description 44
- 108090000565 Capsid Proteins Proteins 0.000 claims description 34
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 34
- 239000003795 chemical substances by application Substances 0.000 claims description 22
- 241000702421 Dependoparvovirus Species 0.000 claims description 20
- 208000022873 Ocular disease Diseases 0.000 claims description 18
- 239000002245 particle Substances 0.000 claims description 16
- 238000002347 injection Methods 0.000 claims description 12
- 239000007924 injection Substances 0.000 claims description 12
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 10
- 210000004027 cell Anatomy 0.000 claims description 10
- 108010041308 Endothelial Growth Factors Proteins 0.000 claims description 8
- 102000005789 Vascular Endothelial Growth Factors Human genes 0.000 claims description 8
- 108010019530 Vascular Endothelial Growth Factors Proteins 0.000 claims description 8
- 229960002833 aflibercept Drugs 0.000 claims description 8
- 108010081667 aflibercept Proteins 0.000 claims description 8
- 230000002137 anti-vascular effect Effects 0.000 claims description 8
- 102000039446 nucleic acids Human genes 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 8
- 230000002207 retinal effect Effects 0.000 claims description 8
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 6
- 206010012688 Diabetic retinal oedema Diseases 0.000 claims description 6
- 150000001413 amino acids Chemical class 0.000 claims description 6
- 201000011190 diabetic macular edema Diseases 0.000 claims description 6
- 238000003780 insertion Methods 0.000 claims description 6
- 230000037431 insertion Effects 0.000 claims description 6
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 125000000539 amino acid group Chemical group 0.000 claims description 4
- 210000000234 capsid Anatomy 0.000 claims description 4
- 230000003472 neutralizing effect Effects 0.000 claims description 4
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 4
- 229920001184 polypeptide Polymers 0.000 claims description 4
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 4
- 208000005590 Choroidal Neovascularization Diseases 0.000 claims description 3
- 206010060823 Choroidal neovascularisation Diseases 0.000 claims description 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 2
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 2
- 208000003098 Ganglion Cysts Diseases 0.000 claims description 2
- 208000001344 Macular Edema Diseases 0.000 claims description 2
- 206010025415 Macular oedema Diseases 0.000 claims description 2
- 208000005400 Synovial Cyst Diseases 0.000 claims description 2
- 210000000411 amacrine cell Anatomy 0.000 claims description 2
- 229960000397 bevacizumab Drugs 0.000 claims description 2
- 229950000025 brolucizumab Drugs 0.000 claims description 2
- 210000003986 cell retinal photoreceptor Anatomy 0.000 claims description 2
- 210000002287 horizontal cell Anatomy 0.000 claims description 2
- 201000010230 macular retinal edema Diseases 0.000 claims description 2
- 210000001127 pigmented epithelial cell Anatomy 0.000 claims description 2
- 239000013646 rAAV2 vector Substances 0.000 claims description 2
- 229960003876 ranibizumab Drugs 0.000 claims description 2
- 208000004644 retinal vein occlusion Diseases 0.000 claims description 2
- 210000002966 serum Anatomy 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962813597P | 2019-03-04 | 2019-03-04 | |
| US62/813,597 | 2019-03-04 | ||
| US201962839457P | 2019-04-26 | 2019-04-26 | |
| US62/839,457 | 2019-04-26 | ||
| PCT/US2020/020929 WO2020180951A1 (en) | 2019-03-04 | 2020-03-04 | Sequential intravitreal administration of aav gene therapy to contralateral eyes |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022522776A JP2022522776A (ja) | 2022-04-20 |
| JPWO2020180951A5 true JPWO2020180951A5 (enExample) | 2023-03-13 |
| JP7597380B2 JP7597380B2 (ja) | 2024-12-10 |
Family
ID=70334029
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021551770A Active JP7597380B2 (ja) | 2019-03-04 | 2020-03-04 | 対側眼へのａａｖ遺伝子治療の逐次的硝子体内投与 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20200297869A1 (enExample) |
| EP (1) | EP3934698A1 (enExample) |
| JP (1) | JP7597380B2 (enExample) |
| KR (1) | KR20210135267A (enExample) |
| AU (1) | AU2020231505A1 (enExample) |
| WO (1) | WO2020180951A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN114250227A (zh) * | 2020-09-25 | 2022-03-29 | 北京安龙基因医药技术有限公司 | 用于高水平表达外源基因的表达载体 |
| JP2023550548A (ja) * | 2020-11-10 | 2023-12-01 | アビラマックス・バイオファーマ・インコーポレイテッド | 操作されたウイルスカプシドおよび使用方法 |
| EP4320240A4 (en) * | 2021-04-09 | 2025-07-09 | Avirmax Biopharma Inc | COMPOSITIONS AND METHODS FOR EXPRESSING OCULAR TRANSGENES |
| AU2023220219A1 (en) * | 2022-02-17 | 2024-09-05 | Skyline Therapeutics Limited | Recombinant adeno-associated virus with modified aav capsid polypeptides |
| KR20250022114A (ko) | 2022-06-07 | 2025-02-14 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 시력 회복을 위한 멜라놉신 변이체 |
| WO2024168252A2 (en) * | 2023-02-10 | 2024-08-15 | Yale University | Use of inhibitors of vegf-c signaling for inhibiting immune responses and increasing gene therapy efficacy |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2002248297A1 (en) * | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| DK2699270T3 (en) | 2011-04-22 | 2017-10-02 | Univ California | ADENO-ASSOCIATED VIRUS VIRUSES WITH VARIANT CAPSID AND METHODS FOR USING IT |
| TWI702955B (zh) | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ） |
| PE20170260A1 (es) * | 2014-05-02 | 2017-04-12 | Genzyme Corp | Vectores de aav para la terapia genica de la retina y el snc |
| AU2016226289B2 (en) * | 2015-03-02 | 2021-04-29 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| WO2017190125A1 (en) * | 2016-04-29 | 2017-11-02 | Adverum Biotechnologies, Inc. | Evasion of neutralizing antibodies by a recombinant adeno-associated virus |
| LT3471780T (lt) * | 2016-06-16 | 2021-01-25 | Adverum Biotechnologies, Inc. | Amd gydymas, naudojant aav2 variantą su afliberceptu |
| CA3040179A1 (en) * | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| AU2018227483A1 (en) * | 2017-02-28 | 2019-09-12 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| CA3054942A1 (en) | 2017-03-17 | 2018-09-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression |
-
2020
- 2020-03-04 EP EP20720552.7A patent/EP3934698A1/en not_active Withdrawn
- 2020-03-04 US US16/808,932 patent/US20200297869A1/en not_active Abandoned
- 2020-03-04 KR KR1020217031241A patent/KR20210135267A/ko active Pending
- 2020-03-04 JP JP2021551770A patent/JP7597380B2/ja active Active
- 2020-03-04 AU AU2020231505A patent/AU2020231505A1/en active Pending
- 2020-03-04 WO PCT/US2020/020929 patent/WO2020180951A1/en not_active Ceased
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