JPWO2022079453A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2022079453A5 JPWO2022079453A5 JP2022573176A JP2022573176A JPWO2022079453A5 JP WO2022079453 A5 JPWO2022079453 A5 JP WO2022079453A5 JP 2022573176 A JP2022573176 A JP 2022573176A JP 2022573176 A JP2022573176 A JP 2022573176A JP WO2022079453 A5 JPWO2022079453 A5 JP WO2022079453A5
- Authority
- JP
- Japan
- Prior art keywords
- isolated polynucleotide
- amd
- vector
- nucleotide sequence
- subject
- 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
- 108091033319 polynucleotide Proteins 0.000 claims description 59
- 239000002157 polynucleotide Substances 0.000 claims description 59
- 102000040430 polynucleotide Human genes 0.000 claims description 59
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 36
- 239000013598 vector Substances 0.000 claims description 30
- 239000002773 nucleotide Substances 0.000 claims description 28
- 125000003729 nucleotide group Chemical group 0.000 claims description 28
- 239000008194 pharmaceutical composition Substances 0.000 claims description 22
- 208000002780 macular degeneration Diseases 0.000 claims description 20
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims description 18
- 208000011325 dry age related macular degeneration Diseases 0.000 claims description 18
- 230000000295 complement effect Effects 0.000 claims description 17
- 208000008069 Geographic Atrophy Diseases 0.000 claims description 16
- 108090000044 Complement Factor I Proteins 0.000 claims description 14
- 102000016550 Complement Factor H Human genes 0.000 claims description 12
- 108010053085 Complement Factor H Proteins 0.000 claims description 12
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims description 12
- 229960002833 aflibercept Drugs 0.000 claims description 10
- 108010081667 aflibercept Proteins 0.000 claims description 10
- 108010009575 CD55 Antigens Proteins 0.000 claims description 8
- 102000006912 Complement C4b-Binding Protein Human genes 0.000 claims description 8
- 108010047548 Complement C4b-Binding Protein Proteins 0.000 claims description 8
- 102000050019 Membrane Cofactor Human genes 0.000 claims description 8
- 101710146216 Membrane cofactor protein Proteins 0.000 claims description 8
- 102000006834 complement receptors Human genes 0.000 claims description 8
- 108010047295 complement receptors Proteins 0.000 claims description 8
- 239000012634 fragment Substances 0.000 claims description 8
- 238000002347 injection Methods 0.000 claims description 8
- 239000007924 injection Substances 0.000 claims description 8
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 8
- 108090000623 proteins and genes Proteins 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 8
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 6
- WLCZTRVUXYALDD-IBGZPJMESA-N 7-[[(2s)-2,6-bis(2-methoxyethoxycarbonylamino)hexanoyl]amino]heptoxy-methylphosphinic acid Chemical compound COCCOC(=O)NCCCC[C@H](NC(=O)OCCOC)C(=O)NCCCCCCCOP(C)(O)=O WLCZTRVUXYALDD-IBGZPJMESA-N 0.000 claims description 4
- 108010089414 Anaphylatoxins Proteins 0.000 claims description 4
- 108091023037 Aptamer Proteins 0.000 claims description 4
- 108091033409 CRISPR Proteins 0.000 claims description 4
- 238000010354 CRISPR gene editing Methods 0.000 claims description 4
- 108090000565 Capsid Proteins Proteins 0.000 claims description 4
- 208000003569 Central serous chorioretinopathy Diseases 0.000 claims description 4
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 4
- 102000003780 Clusterin Human genes 0.000 claims description 4
- 108090000197 Clusterin Proteins 0.000 claims description 4
- 108700040183 Complement C1 Inhibitor Proteins 0.000 claims description 4
- 102000055157 Complement C1 Inhibitor Human genes 0.000 claims description 4
- 108010078015 Complement C3b Proteins 0.000 claims description 4
- 108010034753 Complement Membrane Attack Complex Proteins 0.000 claims description 4
- 102100025680 Complement decay-accelerating factor Human genes 0.000 claims description 4
- 206010012688 Diabetic retinal oedema Diseases 0.000 claims description 4
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 4
- 208000010412 Glaucoma Diseases 0.000 claims description 4
- 206010029113 Neovascularisation Diseases 0.000 claims description 4
- 108091034117 Oligonucleotide Proteins 0.000 claims description 4
- 201000010183 Papilledema Diseases 0.000 claims description 4
- 206010063381 Polypoidal choroidal vasculopathy Diseases 0.000 claims description 4
- 206010038886 Retinal oedema Diseases 0.000 claims description 4
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 4
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 4
- 108020004459 Small interfering RNA Proteins 0.000 claims description 4
- 208000027073 Stargardt disease Diseases 0.000 claims description 4
- 206010046851 Uveitis Diseases 0.000 claims description 4
- 102100035140 Vitronectin Human genes 0.000 claims description 4
- 108010031318 Vitronectin Proteins 0.000 claims description 4
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 4
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 4
- 229960000397 bevacizumab Drugs 0.000 claims description 4
- 230000015572 biosynthetic process Effects 0.000 claims description 4
- 229950000025 brolucizumab Drugs 0.000 claims description 4
- 230000000593 degrading effect Effects 0.000 claims description 4
- 230000000694 effects Effects 0.000 claims description 4
- 108020001507 fusion proteins Proteins 0.000 claims description 4
- 102000037865 fusion proteins Human genes 0.000 claims description 4
- 230000002779 inactivation Effects 0.000 claims description 4
- 239000003112 inhibitor Substances 0.000 claims description 4
- 230000002401 inhibitory effect Effects 0.000 claims description 4
- 230000001404 mediated effect Effects 0.000 claims description 4
- 239000002245 particle Substances 0.000 claims description 4
- 229960003407 pegaptanib Drugs 0.000 claims description 4
- 210000002467 pigment epithelium of eye Anatomy 0.000 claims description 4
- 229920001184 polypeptide Polymers 0.000 claims description 4
- 230000002265 prevention Effects 0.000 claims description 4
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 4
- 229960003876 ranibizumab Drugs 0.000 claims description 4
- 201000011195 retinal edema Diseases 0.000 claims description 4
- 208000004644 retinal vein occlusion Diseases 0.000 claims description 4
- 239000004055 small Interfering RNA Substances 0.000 claims description 4
- 230000002792 vascular Effects 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 3
- 201000011190 diabetic macular edema Diseases 0.000 claims description 3
- 239000013607 AAV vector Substances 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 102100038651 Four and a half LIM domains protein 1 Human genes 0.000 claims description 2
- 101710127220 Four and a half LIM domains protein 1 Proteins 0.000 claims description 2
- 239000003085 diluting agent Substances 0.000 claims description 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 2
- 208000035475 disorder Diseases 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 208000030533 eye disease Diseases 0.000 claims description 2
- BTCSSZJGUNDROE-UHFFFAOYSA-N gamma-aminobutyric acid Chemical group NCCCC(O)=O BTCSSZJGUNDROE-UHFFFAOYSA-N 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 238000002560 therapeutic procedure Methods 0.000 claims description 2
- 239000013603 viral vector Substances 0.000 claims description 2
- 102100035431 Complement factor I Human genes 0.000 claims 7
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 5
- 108010006025 bovine growth hormone Proteins 0.000 claims 2
- 238000011144 upstream manufacturing Methods 0.000 claims 2
- 208000015181 infectious disease Diseases 0.000 claims 1
- 230000010415 tropism Effects 0.000 claims 1
- 102000003689 Complement Factor I Human genes 0.000 description 7
- 238000012986 modification Methods 0.000 description 2
- 230000004048 modification Effects 0.000 description 2
- 239000003795 chemical substances by application Substances 0.000 description 1
- 238000000034 method Methods 0.000 description 1
- 239000000203 mixture Substances 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB2016463.8A GB202016463D0 (en) | 2020-10-16 | 2020-10-16 | Product |
| GB2016463.8 | 2020-10-16 | ||
| GBGB2104148.8A GB202104148D0 (en) | 2021-03-24 | 2021-03-24 | Product |
| GB2104148.8 | 2021-03-24 | ||
| PCT/GB2021/052689 WO2022079453A1 (en) | 2020-10-16 | 2021-10-18 | Nucleic acid encoding an anti-vegf entity and a negative complement regulator and uses thereof for the treatment of age-related macular degeneration |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023545229A JP2023545229A (ja) | 2023-10-27 |
| JP2023545229A5 JP2023545229A5 (https=) | 2024-10-28 |
| JPWO2022079453A5 true JPWO2022079453A5 (https=) | 2024-10-28 |
Family
ID=78463555
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022573176A Pending JP2023545229A (ja) | 2020-10-16 | 2021-10-18 | 抗ｖｅｇｆ実体および負の補体調節因子をコードする核酸、ならびに加齢黄斑変性の処置のためのその使用 |
Country Status (18)
| Country | Link |
|---|---|
| US (1) | US20230212275A1 (https=) |
| EP (1) | EP4229076A1 (https=) |
| JP (1) | JP2023545229A (https=) |
| KR (1) | KR20230088630A (https=) |
| CN (1) | CN119585299A (https=) |
| AU (1) | AU2021362770A1 (https=) |
| CA (1) | CA3182337A1 (https=) |
| CL (1) | CL2023001072A1 (https=) |
| CO (1) | CO2023005981A2 (https=) |
| CR (1) | CR20230202A (https=) |
| DO (1) | DOP2023000076A (https=) |
| EC (1) | ECSP23034907A (https=) |
| IL (1) | IL298527A (https=) |
| JO (1) | JOP20230076A1 (https=) |
| MX (1) | MX2023004377A (https=) |
| PE (1) | PE20231747A1 (https=) |
| PH (1) | PH12023551015A1 (https=) |
| WO (1) | WO2022079453A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201800620D0 (en) | 2018-01-15 | 2018-02-28 | Univ Manchester | C3b Binding Polypeptide |
| GB201821089D0 (en) | 2018-12-21 | 2019-02-06 | Gyroscope Therapeutics Ltd | Codon-optimised complement factor I |
| JP7842082B2 (ja) * | 2020-07-21 | 2026-04-07 | エフティージェン コーポレーション | 眼疾患を処置するための組成物および方法 |
| WO2025083169A1 (en) * | 2023-10-17 | 2025-04-24 | Tenpoint Therapeutics Limited | Combination of a vegf inhibitor and a complement pathway inhibitor for treating ocular disorders |
| KR20260004635A (ko) * | 2024-07-01 | 2026-01-09 | 삼성바이오에피스 주식회사 | 애플리버셉트를 암호화하는 코돈-최적화된 폴리뉴클레오티드를 포함하는 발현 카세트 및 이를 포함하는 rAAV 생산용 플라스미드 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2191001T3 (en) | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| BR112014013205A2 (pt) * | 2011-12-01 | 2020-10-27 | Protevobio, Inc. | proteína de fusão, seu uso e seu método de produção, composição farmacêutica, ácido nucleico, e kit |
| JP7046828B2 (ja) * | 2016-04-15 | 2022-04-04 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 滲出型加齢性黄斑変性の治療のための組成物 |
| AU2018350990A1 (en) * | 2017-10-18 | 2020-05-21 | Regenxbio Inc. | Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap |
| CN111788311A (zh) * | 2017-10-20 | 2020-10-16 | 双子治疗公司 | 用于治疗年龄相关性黄斑变性的组合物和方法 |
| US12558434B2 (en) | 2018-02-20 | 2026-02-24 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| GB201821089D0 (en) * | 2018-12-21 | 2019-02-06 | Gyroscope Therapeutics Ltd | Codon-optimised complement factor I |
| GB201821082D0 (en) * | 2018-12-21 | 2019-02-06 | Gyroscope Therapeutics Ltd | Combination of complement factors i and h, and vector encoding thereof |
| EP4048318A4 (en) * | 2019-10-23 | 2023-11-22 | Gemini Therapeutics Sub, Inc. | METHOD FOR TREATING PATIENTS WITH CFH MUTATIONS USING RECOMBINANT CFH PROTEINS |
-
2021
- 2021-10-18 MX MX2023004377A patent/MX2023004377A/es unknown
- 2021-10-18 CR CR20230202A patent/CR20230202A/es unknown
- 2021-10-18 CA CA3182337A patent/CA3182337A1/en active Pending
- 2021-10-18 KR KR1020227041935A patent/KR20230088630A/ko active Pending
- 2021-10-18 CN CN202180039494.2A patent/CN119585299A/zh active Pending
- 2021-10-18 AU AU2021362770A patent/AU2021362770A1/en active Pending
- 2021-10-18 US US17/928,803 patent/US20230212275A1/en active Pending
- 2021-10-18 WO PCT/GB2021/052689 patent/WO2022079453A1/en not_active Ceased
- 2021-10-18 EP EP21801205.2A patent/EP4229076A1/en active Pending
- 2021-10-18 JP JP2022573176A patent/JP2023545229A/ja active Pending
- 2021-10-18 IL IL298527A patent/IL298527A/en unknown
- 2021-10-18 PE PE2023001418A patent/PE20231747A1/es unknown
- 2021-10-18 PH PH1/2023/551015A patent/PH12023551015A1/en unknown
-
2023
- 2023-04-14 DO DO2023000076A patent/DOP2023000076A/es unknown
- 2023-04-14 CL CL2023001072A patent/CL2023001072A1/es unknown
- 2023-04-16 JO JOJO/P/2023/0076A patent/JOP20230076A1/ar unknown
- 2023-05-10 CO CONC2023/0005981A patent/CO2023005981A2/es unknown
- 2023-05-11 EC ECSENADI202334907A patent/ECSP23034907A/es unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| de Guimaraes et al. | Gene therapy for neovascular age-related macular degeneration: rationale, clinical trials and future directions | |
| JP2021503914A5 (https=) | ||
| JP2022141670A5 (https=) | ||
| JP6387350B2 (ja) | 希突起神経膠細胞を標的とするアデノ随伴ウイルスベクター | |
| JP2021522842A5 (https=) | ||
| RU2018142768A (ru) | Варианты капсидов аденоассоциированного вируса и способы их применения | |
| US20150182638A1 (en) | Virus-mediated delivery of bevacizumab for therapeutic applications | |
| CN117467025B (zh) | 一种抗vegf和补体双功能融合蛋白及其应用 | |
| JP2021500922A5 (https=) | ||
| CN113286878A (zh) | 补体因子i和补体因子i辅因子、编码它们的载体和治疗用途 | |
| JP2021511030A5 (https=) | ||
| CN118108862B (zh) | 抗血管新生的融合蛋白及其应用 | |
| US20230212275A1 (en) | Nucleic acid encoding an anti-vegf entity and a negative complement regulator and uses thereof for the treatment of age-related macular degeneration | |
| JP2026041876A (ja) | コドン最適化補体因子ｉ | |
| KR20260005328A (ko) | 항vegf 단백질을 코딩하는 핵산, 폴리뉴클레오타이드 발현 카세트 및 재조합 아데노 관련 바이러스 | |
| JPWO2022247917A5 (https=) | ||
| JPWO2022079453A5 (https=) | ||
| US20240358855A1 (en) | Multigene constructs for treatment of age-related macular degeneration and other complement dysregulation-related conditions | |
| EP4640699A2 (en) | Retinal disorders | |
| JPWO2020010042A5 (https=) | ||
| CN117587027A (zh) | 编码抗vegf的抗体或抗原结合片段的多核苷酸表达盒及质粒 | |
| WO2022256820A1 (en) | Multispecific antagonists | |
| RU2021119359A (ru) | Фактор комплемента i и кофактор фактора комплемента i, кодирующие их векторы и применение в терапевтических целях | |
| CN118085112B (zh) | 抗多个vegf家族蛋白的融合蛋白及其应用 | |
| CN115997012B (zh) | Rpe65相关眼睛疾病和障碍的治疗 |