JPWO2020210724A5 - - Google Patents
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- Publication number
- JPWO2020210724A5 JPWO2020210724A5 JP2021559892A JP2021559892A JPWO2020210724A5 JP WO2020210724 A5 JPWO2020210724 A5 JP WO2020210724A5 JP 2021559892 A JP2021559892 A JP 2021559892A JP 2021559892 A JP2021559892 A JP 2021559892A JP WO2020210724 A5 JPWO2020210724 A5 JP WO2020210724A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- htra1
- protein
- subject
- vector
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 22
- 101001041393 Homo sapiens Serine protease HTRA1 Proteins 0.000 claims 12
- 102100021119 Serine protease HTRA1 Human genes 0.000 claims 12
- 239000003795 chemical substances by application Substances 0.000 claims 7
- 239000013598 vector Substances 0.000 claims 7
- 108700028369 Alleles Proteins 0.000 claims 6
- 206010064930 age-related macular degeneration Diseases 0.000 claims 5
- 108091006106 transcriptional activators Proteins 0.000 claims 5
- 108020005004 Guide RNA Proteins 0.000 claims 4
- 238000002347 injection Methods 0.000 claims 4
- 239000007924 injection Substances 0.000 claims 4
- 208000002780 macular degeneration Diseases 0.000 claims 4
- 238000000034 method Methods 0.000 claims 4
- 108091033319 polynucleotide Proteins 0.000 claims 4
- 239000002157 polynucleotide Substances 0.000 claims 4
- 102000040430 polynucleotide Human genes 0.000 claims 4
- 101150007028 HTRA1 gene Proteins 0.000 claims 3
- 210000000349 chromosome Anatomy 0.000 claims 3
- 108020001507 fusion proteins Proteins 0.000 claims 3
- 102000037865 fusion proteins Human genes 0.000 claims 3
- 108020001580 protein domains Proteins 0.000 claims 3
- 239000013603 viral vector Substances 0.000 claims 3
- 102100022794 Bestrophin-1 Human genes 0.000 claims 2
- 208000005590 Choroidal Neovascularization Diseases 0.000 claims 2
- 206010060823 Choroidal neovascularisation Diseases 0.000 claims 2
- 101000903449 Homo sapiens Bestrophin-1 Proteins 0.000 claims 2
- 101001020544 Homo sapiens LIM/homeobox protein Lhx2 Proteins 0.000 claims 2
- 102100036132 LIM/homeobox protein Lhx2 Human genes 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 102000004389 Ribonucleoproteins Human genes 0.000 claims 2
- 108010081734 Ribonucleoproteins Proteins 0.000 claims 2
- 230000004913 activation Effects 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 238000013518 transcription Methods 0.000 claims 2
- 230000035897 transcription Effects 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 108010040467 CRISPR-Associated Proteins Proteins 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 208000008069 Geographic Atrophy Diseases 0.000 claims 1
- 241000175212 Herpesvirales Species 0.000 claims 1
- 101000729271 Homo sapiens Retinoid isomerohydrolase Proteins 0.000 claims 1
- 101710163270 Nuclease Proteins 0.000 claims 1
- 102100031176 Retinoid isomerohydrolase Human genes 0.000 claims 1
- 208000032023 Signs and Symptoms Diseases 0.000 claims 1
- 230000001419 dependent effect Effects 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims 1
- 230000001177 retroviral effect Effects 0.000 claims 1
- 229920002477 rna polymer Polymers 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- 230000002103 transcriptional effect Effects 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025093119A JP2025175283A (ja) | 2019-04-10 | 2025-06-04 | Ａｍｄの処置のためのｈｔｒａ１調節 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962832182P | 2019-04-10 | 2019-04-10 | |
| US62/832,182 | 2019-04-10 | ||
| PCT/US2020/027802 WO2020210724A1 (en) | 2019-04-10 | 2020-04-10 | Htra1 modulation for treatment of amd |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025093119A Division JP2025175283A (ja) | 2019-04-10 | 2025-06-04 | Ａｍｄの処置のためのｈｔｒａ１調節 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022526429A JP2022526429A (ja) | 2022-05-24 |
| JPWO2020210724A5 true JPWO2020210724A5 (https=) | 2023-04-17 |
| JP2022526429A5 JP2022526429A5 (https=) | 2023-04-17 |
Family
ID=72750902
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021559892A Pending JP2022526429A (ja) | 2019-04-10 | 2020-04-10 | Ａｍｄの処置のためのｈｔｒａ１調節 |
| JP2025093119A Pending JP2025175283A (ja) | 2019-04-10 | 2025-06-04 | Ａｍｄの処置のためのｈｔｒａ１調節 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025093119A Pending JP2025175283A (ja) | 2019-04-10 | 2025-06-04 | Ａｍｄの処置のためのｈｔｒａ１調節 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230165976A1 (https=) |
| EP (1) | EP3953485A4 (https=) |
| JP (2) | JP2022526429A (https=) |
| CN (1) | CN113966401A (https=) |
| CA (1) | CA3136119A1 (https=) |
| WO (1) | WO2020210724A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113512564A (zh) * | 2021-08-05 | 2021-10-19 | 呈诺再生医学科技(珠海横琴新区)有限公司 | 以iPSC来源的细胞为载体、基因治疗湿性年龄相关性黄斑变性 |
| US20240358855A1 (en) * | 2021-08-18 | 2024-10-31 | University Of Utah Research Foundation | Multigene constructs for treatment of age-related macular degeneration and other complement dysregulation-related conditions |
| US20250003989A1 (en) * | 2021-11-15 | 2025-01-02 | The Children`S Medical Center Corporation | Methods for tauopathy diagnosis and treatment |
Family Cites Families (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU8081491A (en) | 1990-06-01 | 1991-12-31 | Cetus Corporation | Compositions and methods for identifying biologically active molecules |
| JP2001524926A (ja) | 1991-09-18 | 2001-12-04 | アフィマックス テクノロジーズ ナームロゼ フェンノートシャップ | オリゴマーの雑多ライブラリーの集合体を合成する方法 |
| US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| ES2204910T3 (es) | 1992-10-01 | 2004-05-01 | The Trustees Of Columbia University In The City Of New York | Bibliotecas quimicas combinatorias complejas codificadas con señales. |
| CN1134156A (zh) | 1993-11-02 | 1996-10-23 | 阿菲马克斯技术公司 | 合成和筛选多种分子 |
| JPH10500112A (ja) | 1994-05-06 | 1998-01-06 | ファーマコピーア，インコーポレイテッド | 組合せ ジヒドロベンゾピラン ライブラリー |
| US5663046A (en) | 1994-06-22 | 1997-09-02 | Pharmacopeia, Inc. | Synthesis of combinatorial libraries |
| DE19710159A1 (de) | 1997-03-12 | 1998-10-01 | Hoechst Ag | In vitro Transkriptionsverfahren zum Screening von Naturstoffen und anderen chemischen Substanzen |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| CA2394850C (en) | 1999-12-06 | 2012-02-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| KR20020086508A (ko) | 2000-02-08 | 2002-11-18 | 상가모 바이오사이언스 인코포레이티드 | 약물 발견용 세포 |
| US20050267061A1 (en) | 2004-04-08 | 2005-12-01 | Sangamo Biosciences, Inc. | Methods and compositions for treating neuropathic and neurodegenerative conditions |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| JP2008506359A (ja) | 2004-04-08 | 2008-03-06 | サンガモ バイオサイエンシズ インコーポレイテッド | ジンクフィンガータンパク質による神経因性疼痛の処置 |
| JP5431151B2 (ja) | 2006-07-13 | 2014-03-05 | ユニバーシティー オブ アイオワ リサーチ ファンデーション | 血管障害および加齢黄斑変性症の治療および診断のための方法および試薬 |
| AU2007277125B2 (en) * | 2006-07-26 | 2013-08-01 | Yale University | Diagnosis and treatment of age related macular degeneration |
| US7972787B2 (en) | 2007-02-16 | 2011-07-05 | Massachusetts Eye And Ear Infirmary | Methods for detecting age-related macular degeneration |
| TW200911290A (en) * | 2007-07-02 | 2009-03-16 | Alcon Res Ltd | RNAI-mediated inhibition of HTRA1 for treatment of macular degeneration |
| EP2108705A1 (en) * | 2008-04-08 | 2009-10-14 | Universität Duisburg-Essen | Method for analysing the epigenetic status of the HtrA 1 gene in a biological sample |
| SG193499A1 (en) * | 2011-03-15 | 2013-10-30 | Univ Utah Res Found | Methods of diagnosing and treating vascular associated maculopathy and symptoms thereof |
| CN103917556B (zh) * | 2011-10-14 | 2018-02-06 | 霍夫曼-拉罗奇有限公司 | 抗HtrA1抗体及使用方法 |
| EP2895624B1 (en) | 2012-09-14 | 2022-06-01 | University Of Utah Research Foundation | Methods of predicting the development of amd based on chromosome 1 and chromosome 10 |
| AU2014346559B2 (en) * | 2013-11-07 | 2020-07-09 | Editas Medicine,Inc. | CRISPR-related methods and compositions with governing gRNAs |
| US9546384B2 (en) * | 2013-12-11 | 2017-01-17 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a mouse genome |
| CA2945335A1 (en) | 2014-04-18 | 2015-10-22 | Editas Medicine, Inc. | Crispr-cas-related methods, compositions and components for cancer immunotherapy |
| AU2017374044B2 (en) | 2016-12-08 | 2023-11-30 | Intellia Therapeutics, Inc. | Modified guide RNAs |
| US11674138B2 (en) * | 2017-03-13 | 2023-06-13 | President And Fellows Of Harvard College | Methods of modulating expression of target nucleic acid sequences in a cell |
| EP3824095A4 (en) | 2018-07-20 | 2022-04-20 | University of Utah Research Foundation | GENE THERAPY FOR MACULATE GENERATION |
| US20200291427A1 (en) * | 2018-11-16 | 2020-09-17 | Gemini Therapeutics Inc. | Crispr-based therapeutics for targeting htra1 and methods of use |
| EP4667578A3 (en) * | 2019-04-03 | 2026-04-01 | REGENXBIO Inc. | Gene therapy for eye pathologies |
-
2020
- 2020-04-10 WO PCT/US2020/027802 patent/WO2020210724A1/en not_active Ceased
- 2020-04-10 EP EP20787458.7A patent/EP3953485A4/en active Pending
- 2020-04-10 CN CN202080042929.4A patent/CN113966401A/zh active Pending
- 2020-04-10 JP JP2021559892A patent/JP2022526429A/ja active Pending
- 2020-04-10 US US17/599,979 patent/US20230165976A1/en not_active Abandoned
- 2020-04-10 CA CA3136119A patent/CA3136119A1/en active Pending
-
2025
- 2025-06-04 JP JP2025093119A patent/JP2025175283A/ja active Pending
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