JP2018062532A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2018062532A5 JP2018062532A5 JP2018010718A JP2018010718A JP2018062532A5 JP 2018062532 A5 JP2018062532 A5 JP 2018062532A5 JP 2018010718 A JP2018010718 A JP 2018010718A JP 2018010718 A JP2018010718 A JP 2018010718A JP 2018062532 A5 JP2018062532 A5 JP 2018062532A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- administration
- specific promoter
- cell type
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 11
- 210000004027 cells Anatomy 0.000 claims 6
- 230000002207 retinal Effects 0.000 claims 5
- 235000020945 retinal Nutrition 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 4
- 206010064930 Age-related macular degeneration Diseases 0.000 claims 3
- 208000002780 Macular Degeneration Diseases 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- 102000003921 Peroxisome proliferator-activated receptor gamma coactivator 1-alpha Human genes 0.000 claims 2
- 108090000310 Peroxisome proliferator-activated receptor gamma coactivator 1-alpha Proteins 0.000 claims 2
- 210000003583 Retinal Pigment Epithelium Anatomy 0.000 claims 2
- 102100004239 SOD2 Human genes 0.000 claims 2
- 108010045815 superoxide dismutase 2 Proteins 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 229940105657 CATALASE Drugs 0.000 claims 1
- 102000016938 EC 1.11.1.6 Human genes 0.000 claims 1
- 108010053835 EC 1.11.1.6 Proteins 0.000 claims 1
- 102100017234 NFE2L2 Human genes 0.000 claims 1
- 101710031938 NFE2L2 Proteins 0.000 claims 1
- 230000003078 antioxidant Effects 0.000 claims 1
- 239000003963 antioxidant agent Substances 0.000 claims 1
- 235000006708 antioxidants Nutrition 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
Claims (12)
- 被験体における加齢性黄斑変性症（ＡＭＤ）を予防または処置するための医薬組成物であって、Ｎｒｆ２をコードする核酸分子を含む組換えＡＡＶベクターを含む、医薬組成物。
- 前記組換えＡＡＶベクターが、組換えＡＡＶ８ベクターおよび組換えＡＡＶ２／８ベクターからなる群から選択される、請求項１に記載の医薬組成物。
- 前記核酸分子がＣＭＶプロモーターに作動可能に連結している、請求項１または２に記載の医薬組成物。
- 前記核酸分子が網膜細胞型特異的プロモーターに作動可能に連結している、請求項１または２に記載の医薬組成物。
- 前記網膜細胞型特異的プロモーターが、杆体特異的プロモーターである、請求項４に記載の医薬組成物。
- 前記網膜細胞型特異的プロモーターが、錐体特異的プロモーターである、請求項４に記載の医薬組成物。
- 前記網膜細胞型特異的プロモーターが、杆体および錐体特異的プロモーターである、請求項４に記載の医薬組成物。
- 前記網膜細胞型特異的プロモーターが、網膜色素上皮（ＲＰＥ）細胞プロモーターである、請求項４に記載の医薬組成物。
- 前記核酸分子がさらに、カタラーゼ、スーパーオキシドジスムターゼ２（ＳＯＤ２）およびペルオキシソーム増殖因子活性化受容体ガンマコアクチベーター１−アルファ（ＰＧＣ１α）ならびにこれらのいずれかの組合せからなる群より選択される抗酸化防御遺伝子をコードする、請求項１から８のいずれか一項に記載の医薬組成物。
- 前記組成物が、眼内投与に適している、請求項１から９のいずれか一項に記載の医薬組成物。
- 前記眼内投与が、硝子体内投与、網膜下投与、結膜下投与、テノン下投与、眼周囲投与、眼球後投与、脈絡膜上投与および強膜内投与からなる群より選択される、請求項１０に記載の医薬組成物。
- 加齢性黄斑変性症を有する被験体を処置するための医薬の製造における、請求項１から１１のいずれか一項に記載の組成物の使用。
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361896805P | 2013-10-29 | 2013-10-29 | |
| US61/896,805 | 2013-10-29 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016526853A Division JP6317440B2 (ja) | 2013-10-29 | 2014-10-29 | 酸化ストレスを阻害するための方法および組成物 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018217337A Division JP2019048858A (ja) | 2013-10-29 | 2018-11-20 | 酸化ストレスを阻害するための方法および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018062532A JP2018062532A (ja) | 2018-04-19 |
| JP2018062532A5 true JP2018062532A5 (ja) | 2018-06-07 |
| JP6440875B2 JP6440875B2 (ja) | 2018-12-19 |
Family
ID=53005074
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016526853A Active JP6317440B2 (ja) | 2013-10-29 | 2014-10-29 | 酸化ストレスを阻害するための方法および組成物 |
| JP2018010718A Active JP6440875B2 (ja) | 2013-10-29 | 2018-01-25 | 酸化ストレスを阻害するための方法および組成物 |
| JP2018217337A Pending JP2019048858A (ja) | 2013-10-29 | 2018-11-20 | 酸化ストレスを阻害するための方法および組成物 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016526853A Active JP6317440B2 (ja) | 2013-10-29 | 2014-10-29 | 酸化ストレスを阻害するための方法および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018217337A Pending JP2019048858A (ja) | 2013-10-29 | 2018-11-20 | 酸化ストレスを阻害するための方法および組成物 |
Country Status (4)
| Country | Link |
|---|---|
| US (2) | US10980896B2 (ja) |
| EP (3) | EP3777980B1 (ja) |
| JP (3) | JP6317440B2 (ja) |
| WO (1) | WO2015066190A1 (ja) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2651757C2 (ru) * | 2016-01-19 | 2018-04-23 | Селл энд Джин Терапи Лтд | Линейка биологически активных генно-терапевтических субстанций на основе гена sod2 для коррекции патологических состояний клеток органов и тканей и органов и тканей человека, связанных с оксидативным стрессом, способ получения и использования |
| RU2649820C2 (ru) * | 2016-01-20 | 2018-04-04 | Селл энд Джин Терапи Лтд | Средство для коррекции патологических состояний клеток органов и тканей и/или органов и тканей человека на основе гена col1a2, связанных с количественным снижением белка альфа-2 цепи коллагена i типа |
| RU2653491C2 (ru) * | 2016-01-20 | 2018-05-08 | Селл энд Джин Терапи Лтд | Линейка биологически активных генно-терапевтических субстанций на основе гена gpx1 для коррекции патологических состояний клеток органов и тканей и органов и тканей человека, связанных с оксидативным стрессом, способ получения и использования |
| RU2651758C2 (ru) * | 2016-01-20 | 2018-04-23 | Селл энд Джин Терапи Лтд | Средство для коррекции патологических состояний клеток органов и тканей и/или органов и тканей человека, на основе гена gpx3, связанных с оксидативным стрессом, способ получения и использования |
| WO2017136805A1 (en) * | 2016-02-06 | 2017-08-10 | Georgetown University | Compositions and methods for the diagnosis and treatment of age-related macular degeneration |
| RU2651048C1 (ru) * | 2016-11-21 | 2018-04-18 | Общество с ограниченной ответственностью "Аллель Центр Инновационных Биотехнологий" | Средство для лечения состояний человеческого организма, связанных с уменьшением уровня экспрессии гена il-11 и/или уменьшением количества белка интерлейкина-11 на основе генно-терапевтических субстанций с геном il-11, способ получения и использования |
| RU2653487C1 (ru) * | 2016-11-30 | 2018-05-08 | Общество с ограниченной ответственностью "Аллель Центр Инновационных Биотехнологий" | Средство для лечения состояний человеческого организма, связанных с уменьшением уровня экспрессии гена lif и/или уменьшением количества белка фактора, ингибирующего лейкемию, на основе генно-терапевтических субстанций с геном lif, способ получения и использования |
| RU2649814C1 (ru) * | 2017-02-14 | 2018-04-04 | Общество с ограниченной ответственностью "Медсервис" | Средство для лечения состояний человеческого организма, связанных с уменьшением уровня экспрессии гена сат и/или уменьшением активности белка каталазы на основе генно-терапевтических субстанций с геном сат, способ получения и использования |
| JP7105532B2 (ja) * | 2017-05-10 | 2022-07-25 | ロート製薬株式会社 | 後眼部疾患の予防、改善、又は治療用組成物 |
| FI3684423T3 (fi) * | 2017-09-20 | 2023-06-15 | 4D Molecular Therapeutics Inc | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä |
| US20210381002A1 (en) * | 2018-04-03 | 2021-12-09 | Cornell University | Gene therapy for oxidative stress |
| PE20211205A1 (es) | 2018-08-20 | 2021-07-05 | Janssen Pharmaceutica Nv | Inhibidores de la interaccion proteina-proteina keap1-nrf2 |
| CN109055428A (zh) * | 2018-09-19 | 2018-12-21 | 上海市第人民医院 | 一种重组腺相关病毒载体及其制备方法与应用 |
| US20220088222A1 (en) * | 2018-12-20 | 2022-03-24 | President And Fellows Of Harvard College | Compositions and methods for the treatment of degenerative ocular diseases |
| WO2020167770A1 (en) * | 2019-02-11 | 2020-08-20 | President And Fellows Of Harvard College | Txnip and ldhb compositions and methods for the treatment of degenerative ocular diseases |
| BR112022021468A2 (pt) | 2020-04-27 | 2022-12-13 | 4D Molecular Therapeutics Inc | Genes gla otimizados por códon e usos dos mesmos |
| CA3177580A1 (en) * | 2020-05-04 | 2021-11-11 | Sulfagenix, Inc. | Treatment of viral infections |
| US20230250450A1 (en) * | 2020-06-09 | 2023-08-10 | Genethon | NRF2 Activator for Use in Treating Dilated Cardiomyopathies |
| KR102620997B1 (ko) * | 2022-11-10 | 2024-01-04 | 주식회사 아이두젠 | 노각나무 추출물을 유효성분으로 포함하는 시력 보호용 또는 망막 질환의 개선 및 예방용 조성물 |
Family Cites Families (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US4373071A (en) | 1981-04-30 | 1983-02-08 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4401796A (en) | 1981-04-30 | 1983-08-30 | City Of Hope Research Institute | Solid-phase synthesis of polynucleotides |
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| US4598049A (en) | 1983-08-31 | 1986-07-01 | Systec Inc. | General purpose gene synthesizer |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US5116742A (en) | 1986-12-03 | 1992-05-26 | University Patents, Inc. | RNA ribozyme restriction endoribonucleases and methods |
| US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods |
| US5716826A (en) | 1988-03-21 | 1998-02-10 | Chiron Viagene, Inc. | Recombinant retroviruses |
| US5591624A (en) | 1988-03-21 | 1997-01-07 | Chiron Viagene, Inc. | Retroviral packaging cell lines |
| CA1336396C (en) | 1988-07-25 | 1995-07-25 | Kiyoshi Kita | Intraocular anticoagulant including antithrombin iii and method of administration |
| US5223409A (en) | 1988-09-02 | 1993-06-29 | Protein Engineering Corp. | Directed evolution of novel binding proteins |
| US5175384A (en) | 1988-12-05 | 1992-12-29 | Genpharm International | Transgenic mice depleted in mature t-cells and methods for making transgenic mice |
| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| US6075181A (en) | 1990-01-12 | 2000-06-13 | Abgenix, Inc. | Human antibodies derived from immunized xenomice |
| DK0710719T3 (da) | 1990-01-12 | 2007-07-09 | Amgen Fremont Inc | Frembringelse af xenogene antistoffer |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| US5427908A (en) | 1990-05-01 | 1995-06-27 | Affymax Technologies N.V. | Recombinant library screening methods |
| EP0585287B1 (en) | 1990-07-10 | 1999-10-13 | Cambridge Antibody Technology Limited | Methods for producing members of specific binding pairs |
| GB9015198D0 (en) | 1990-07-10 | 1990-08-29 | Brien Caroline J O | Binding substance |
| DK0564531T3 (da) | 1990-12-03 | 1998-09-28 | Genentech Inc | Berigelsesfremgangsmåde for variantproteiner med ændrede bindingsegenskaber |
| CA2105300C (en) | 1991-03-01 | 2008-12-23 | Robert C. Ladner | Process for the development of binding mini-proteins |
| PT100379B (pt) | 1991-04-10 | 1999-01-29 | Scripps Research Inst | Bibliotecas de receptores heterodimericos usando fagomideos |
| DE4122599C2 (de) | 1991-07-08 | 1993-11-11 | Deutsches Krebsforsch | Phagemid zum Screenen von Antikörpern |
| US5756096A (en) | 1991-07-25 | 1998-05-26 | Idec Pharmaceuticals Corporation | Recombinant antibodies for human therapy |
| MX9204374A (es) | 1991-07-25 | 1993-03-01 | Idec Pharma Corp | Anticuerpo recombinante y metodo para su produccion. |
| JPH06509578A (ja) | 1991-07-26 | 1994-10-27 | ユニバーシティ・オブ・ロチェスター | 悪性細胞利用による癌治療法 |
| US5529774A (en) | 1991-08-13 | 1996-06-25 | The Regents Of The University Of California | In vivo transfer of the HSV-TK gene implanted retroviral producer cells |
| JP3571337B2 (ja) | 1992-02-11 | 2004-09-29 | セル ジェネシス，インコーポレーテッド | 遺伝子標的現象による同型遺伝子接合 |
| US5631236A (en) | 1993-08-26 | 1997-05-20 | Baylor College Of Medicine | Gene therapy for solid tumors, using a DNA sequence encoding HSV-Tk or VZV-Tk |
| WO1995006486A1 (en) | 1993-09-03 | 1995-03-09 | Government Of The United States Of America, Secretary Department Of Health And Human Services | Treatment of human tumors by genetic transformation of human tumor cells |
| US5763217A (en) | 1993-11-10 | 1998-06-09 | University Of British Columbia | Method of using, process of preparing and composition comprising recombinant herpesvirus vectors |
| US5714353A (en) | 1994-05-24 | 1998-02-03 | Research Corporation Technologies, Inc. | Safe vectors for gene therapy |
| US5688773A (en) | 1994-08-17 | 1997-11-18 | The General Hospital Corporation | Method of selectively destroying neoplastic cells |
| US5763242A (en) | 1995-02-08 | 1998-06-09 | Thomas Jefferson University | Method for increasing transduction efficiency of recombinant retroviral vectors |
| US5756283A (en) | 1995-06-05 | 1998-05-26 | The Trustees Of The University Of Pennsylvania | Method for improved production of recombinant adeno-associated viruses for gene therapy |
| US5985846A (en) | 1995-06-07 | 1999-11-16 | Baylor College Of Medicine | Gene therapy for muscular dystrophy |
| US5811524A (en) | 1995-06-07 | 1998-09-22 | Idec Pharmaceuticals Corporation | Neutralizing high affinity human monoclonal antibodies specific to RSV F-protein and methods for their manufacture and therapeutic use thereof |
| US5994132A (en) | 1996-10-23 | 1999-11-30 | University Of Michigan | Adenovirus vectors |
| US5981225A (en) | 1998-04-16 | 1999-11-09 | Baylor College Of Medicine | Gene transfer vector, recombinant adenovirus particles containing the same, method for producing the same and method of use of the same |
| WO2000054595A1 (en) | 1999-03-15 | 2000-09-21 | John Guy | Recombinant adeno-associated virus transfer of genes affecting demyelination |
| US20020049176A1 (en) * | 1999-11-10 | 2002-04-25 | Anderson Christen M. | Modulation of mitochondrial mass and function for the treatment of diseases and for target and drug discovery |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| US20070203083A1 (en) | 2003-06-13 | 2007-08-30 | Mootha Vamsi K | Methods Of Regulating Metabolism And Mitochondrial Function |
| US20080274093A1 (en) * | 2004-12-02 | 2008-11-06 | Johnson Jeffrey A | Method of diminishing the symptoms of neurodegenerative disease |
| WO2007005879A2 (en) * | 2005-07-01 | 2007-01-11 | The Johns Hopkins University | Compositions and methods for the treatment or prevention of disorders relating to oxidative stress |
| AU2007324073A1 (en) | 2006-10-23 | 2008-05-29 | John Guy | Suppression of mitochondrial oxidative stress |
| RS63489B1 (sr) | 2007-02-08 | 2022-09-30 | Biogen Ma Inc | Kompozicije i upotrebe za tretiranje multiple skleroze |
| US20120108654A1 (en) * | 2008-06-30 | 2012-05-03 | The Johns Hopkins University | Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa |
| JP6116242B2 (ja) * | 2009-03-16 | 2017-04-19 | クルナ・インコーポレーテッド | 核因子（赤血球由来２）様２（ｎｒｆ２）に対する天然アンチセンス転写物の抑制によるｎｒｆ２関連疾患の治療 |
| WO2010121010A2 (en) | 2009-04-16 | 2010-10-21 | President And Fellows Of Harvard College | Methods for inhibiting starvation of a cell |
| US20130288985A1 (en) * | 2010-07-15 | 2013-10-31 | The Schepens Eye Research Institute Inc. | Compositions and methods of treatment of corneal endothelium disorders |
-
2014
- 2014-10-29 US US15/032,426 patent/US10980896B2/en active Active
- 2014-10-29 EP EP20196298.2A patent/EP3777980B1/en active Active
- 2014-10-29 EP EP14857455.1A patent/EP3062884B1/en active Active
- 2014-10-29 EP EP23213942.8A patent/EP4331590A3/en active Pending
- 2014-10-29 JP JP2016526853A patent/JP6317440B2/ja active Active
- 2014-10-29 WO PCT/US2014/062917 patent/WO2015066190A1/en active Application Filing
-
2018
- 2018-01-25 JP JP2018010718A patent/JP6440875B2/ja active Active
- 2018-11-20 JP JP2018217337A patent/JP2019048858A/ja active Pending
-
2021
- 2021-03-19 US US17/206,212 patent/US20210275688A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2018062532A5 (ja) | ||
| JP2016535034A5 (ja) | ||
| HRP20211024T1 (hr) | Aav vektori za gensku terapiju mrežnice i cns-a | |
| Liu et al. | Gene therapy for ocular diseases | |
| JP2017518271A5 (ja) | ||
| JP2021503914A5 (ja) | ||
| JP2019518427A5 (ja) | ||
| HRP20201842T1 (hr) | Liječenje amd korištenjem aav2 varijante s afliberceptom | |
| RU2018142768A (ru) | Варианты капсидов аденоассоциированного вируса и способы их применения | |
| HRP20201225T1 (hr) | Terapije poboljšavanja gena kod nasljedne degeneracije mrežnice uzrokovanu mutacijama u genu prpf31 | |
| JP2017510296A5 (ja) | ||
| JP2018508519A5 (ja) | ||
| CA2995656A1 (en) | Treatment of retinitis pigmentosa | |
| JP2020528734A5 (ja) | ||
| HRP20201831T1 (hr) | Optimizirani promoter rpe65 i kodirajuće sekvence | |
| RU2013151885A (ru) | Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования | |
| JP2018510620A5 (ja) | ||
| WO2015138628A8 (en) | Use of aav-expressed m013 protein as an anti-inflammatory therapeutic | |
| JP2022529782A (ja) | シュタルガルト病（ａｂｃａ４）の遺伝子治療 | |
| JP2019528793A5 (ja) | ||
| JP7289306B2 (ja) | 網膜障害を治療するための組成物及び方法 | |
| US20150182638A1 (en) | Virus-mediated delivery of bevacizumab for therapeutic applications | |
| JP2021500922A5 (ja) | ||
| Ku et al. | Retinal gene therapy: current progress and future prospects | |
| Liu et al. | Republished review: Gene therapy for ocular diseases |