PT2678435T - Vetores aav para utilização na terapia genética de coroideremia - Google Patents
Vetores aav para utilização na terapia genética de coroideremiaInfo
- Publication number
- PT2678435T PT2678435T PT127063089T PT12706308T PT2678435T PT 2678435 T PT2678435 T PT 2678435T PT 127063089 T PT127063089 T PT 127063089T PT 12706308 T PT12706308 T PT 12706308T PT 2678435 T PT2678435 T PT 2678435T
- Authority
- PT
- Portugal
- Prior art keywords
- choroideremia
- aav
- vectors
- gene therapy
- therapy
- Prior art date
Links
- 208000033810 Choroidal dystrophy Diseases 0.000 title 1
- 208000003571 choroideremia Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1103062.4A GB201103062D0 (en) | 2011-02-22 | 2011-02-22 | Method |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT2678435T true PT2678435T (pt) | 2018-07-17 |
Family
ID=43881504
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT127063089T PT2678435T (pt) | 2011-02-22 | 2012-02-21 | Vetores aav para utilização na terapia genética de coroideremia |
Country Status (18)
| Country | Link |
|---|---|
| US (4) | US9834788B2 (https=) |
| EP (2) | EP3428283A1 (https=) |
| JP (4) | JP2014512171A (https=) |
| KR (3) | KR102139203B1 (https=) |
| CN (2) | CN103562396A (https=) |
| AU (2) | AU2012220404B2 (https=) |
| BR (1) | BR112013021318B1 (https=) |
| CA (1) | CA2827975C (https=) |
| DK (1) | DK2678435T3 (https=) |
| ES (1) | ES2676550T5 (https=) |
| GB (1) | GB201103062D0 (https=) |
| HK (2) | HK1258917A1 (https=) |
| IL (1) | IL228031B (https=) |
| MX (2) | MX356525B (https=) |
| PL (1) | PL2678435T3 (https=) |
| PT (1) | PT2678435T (https=) |
| SG (1) | SG192911A1 (https=) |
| WO (1) | WO2012114090A1 (https=) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| AU2014305218B2 (en) | 2013-08-05 | 2020-01-16 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant AAV-Crumbs homologue composition and methods for treating LCA-8 and progressive RP |
| KR102281881B1 (ko) * | 2013-12-06 | 2021-07-27 | 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 (인쎄름) | 대상체의 망막 색소 상피에서 목적 폴리뉴클레오티드를 발현시키기 위한 방법 및 약학적 조성물 |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| EP3748007B1 (en) * | 2015-03-06 | 2024-06-26 | Massachusetts Eye & Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| CA3008264A1 (en) * | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| GB201604146D0 (en) * | 2016-03-10 | 2016-04-27 | Nightstarx Ltd | Prenylation assay |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| MX2019005334A (es) | 2016-11-07 | 2019-10-04 | Spark Therapeutics Inc | Ensayo de potencia de proteina escolta rab. |
| PT3568483T (pt) * | 2017-01-10 | 2025-11-18 | Childrens Medical Res Institute | Polinucleótidos e vetores para a expressão de transgenes |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| JP7766393B2 (ja) | 2017-06-14 | 2025-11-10 | 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 | 眼疾患のための遺伝子療法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| CN112501208A (zh) * | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| EP3697923A1 (en) | 2017-10-17 | 2020-08-26 | Nightstarx Limited | Prenylation assay |
| KR20200119782A (ko) * | 2017-12-06 | 2020-10-20 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 mir101 또는 mir128의 용도 |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| WO2019195729A1 (en) * | 2018-04-05 | 2019-10-10 | Nightstarx Ltd. | Aav compositions, methods of making and methods of use |
| AU2018422759B2 (en) * | 2018-05-07 | 2026-01-22 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| JP7585233B2 (ja) * | 2019-04-30 | 2024-11-18 | 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 | ポンペ病の治療のために有用な組成物 |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| CN116406305A (zh) | 2020-08-17 | 2023-07-07 | 麻省理工学院 | Shank3基因治疗方法 |
| WO2022109247A1 (en) | 2020-11-20 | 2022-05-27 | Biogen Ma Inc. | Viral vector potency assay |
| EP4482970A1 (en) | 2022-02-23 | 2025-01-01 | Massachusetts Institute of Technology | Methods for upregulating shank3 expression |
| CN119998456A (zh) | 2022-08-11 | 2025-05-13 | 巴塞尔分子与临床眼科研究所(Iob) | 视锥光感受器中基因特异性表达的启动子 |
| EP4568710A1 (en) | 2022-08-11 | 2025-06-18 | Institute Of Molecular And Clinical Ophthalmology Basel (Iob) | Human cone photoreceptor optogenetic constructs |
| WO2024233422A1 (en) | 2023-05-05 | 2024-11-14 | Massachusetts Institute Of Technology | Shank3 gene therapy approaches |
| WO2025090942A1 (en) * | 2023-10-26 | 2025-05-01 | University Of Notre Dame Du Lac | Gene therapy for treatment of neurometabolic disease |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0856585A1 (en) | 1997-01-29 | 1998-08-05 | Introgene B.V. | A conditional replication and expression system |
| CA2373110A1 (en) * | 2000-03-14 | 2001-09-20 | Neurologix, Inc. | Production of chimeric capsid vectors |
| WO2002082904A2 (en) | 2001-04-13 | 2002-10-24 | The Trustees Of The University Of Pennsylvania | Method of treating or retarding the development of blindness |
| US20040208847A1 (en) * | 2003-03-28 | 2004-10-21 | Fabienne Rolling | Method and vectors for selectively transducing retinal pigment epithelium cells |
| CN101180082B (zh) * | 2005-02-23 | 2012-02-08 | 生物载体株式会社 | 利用siv-pedf载体治疗伴随眼组织细胞凋亡变性的疾患的药物 |
| JP2008539698A (ja) * | 2005-04-29 | 2008-11-20 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 転写後レベルでの核酸発現調節のための方法および組成物 |
| US20090022731A1 (en) * | 2006-08-25 | 2009-01-22 | Wyeth | Arthritis-associated B cell gene expression |
| US20100278843A1 (en) * | 2007-08-16 | 2010-11-04 | St. Vincent's Hospital Sydney Limited | Agents and methods for modulating macrophage inhibitory cytokine (mic-1) activity |
| ES2751999T3 (es) * | 2008-01-29 | 2020-04-02 | Applied Genetic Tech Corporation | Producción recombinante de virus adeno-asociados usando células BHK en suspensión |
| CN101980668A (zh) * | 2008-02-07 | 2011-02-23 | 赛拉基因公司 | 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器 |
| HRP20250897T1 (hr) * | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
-
2011
- 2011-02-22 GB GBGB1103062.4A patent/GB201103062D0/en not_active Ceased
-
2012
- 2012-02-21 AU AU2012220404A patent/AU2012220404B2/en not_active Ceased
- 2012-02-21 CA CA2827975A patent/CA2827975C/en active Active
- 2012-02-21 EP EP18172129.1A patent/EP3428283A1/en not_active Withdrawn
- 2012-02-21 PL PL12706308T patent/PL2678435T3/pl unknown
- 2012-02-21 ES ES12706308T patent/ES2676550T5/es active Active
- 2012-02-21 CN CN201280019547.5A patent/CN103562396A/zh active Pending
- 2012-02-21 CN CN201710784145.9A patent/CN107881198A/zh active Pending
- 2012-02-21 EP EP12706308.9A patent/EP2678435B2/en active Active
- 2012-02-21 KR KR1020137024782A patent/KR102139203B1/ko not_active Expired - Fee Related
- 2012-02-21 JP JP2013554938A patent/JP2014512171A/ja active Pending
- 2012-02-21 PT PT127063089T patent/PT2678435T/pt unknown
- 2012-02-21 KR KR1020207021515A patent/KR102299430B1/ko not_active Expired - Fee Related
- 2012-02-21 MX MX2013009604A patent/MX356525B/es active IP Right Grant
- 2012-02-21 SG SG2013063797A patent/SG192911A1/en unknown
- 2012-02-21 DK DK12706308.9T patent/DK2678435T3/en active
- 2012-02-21 MX MX2018006733A patent/MX388146B/es unknown
- 2012-02-21 US US14/000,836 patent/US9834788B2/en active Active
- 2012-02-21 KR KR1020217028082A patent/KR20210112407A/ko not_active Ceased
- 2012-02-21 WO PCT/GB2012/050376 patent/WO2012114090A1/en not_active Ceased
- 2012-02-21 BR BR112013021318-3A patent/BR112013021318B1/pt not_active IP Right Cessation
-
2013
- 2013-08-19 IL IL228031A patent/IL228031B/en active IP Right Grant
-
2014
- 2014-05-23 HK HK19101394.9A patent/HK1258917A1/en unknown
-
2017
- 2017-06-14 JP JP2017117286A patent/JP2017221192A/ja active Pending
- 2017-07-20 AU AU2017206254A patent/AU2017206254A1/en not_active Abandoned
- 2017-10-31 US US15/799,852 patent/US20180135078A1/en not_active Abandoned
-
2018
- 2018-07-13 HK HK18109118.8A patent/HK1249548A1/zh unknown
-
2019
- 2019-11-05 JP JP2019201092A patent/JP2020054345A/ja active Pending
-
2020
- 2020-01-24 US US16/751,699 patent/US20210032656A1/en not_active Abandoned
-
2022
- 2022-10-26 US US17/974,466 patent/US20230332176A1/en not_active Abandoned
- 2022-12-02 JP JP2022193831A patent/JP2023036618A/ja active Pending
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| GB201102208D0 (en) | Inventions and therapy | |
| GB201102228D0 (en) | Inventions and therapy | |
| GB201102210D0 (en) | Inventions and therapy | |
| GB201102226D0 (en) | Inventions and therapy | |
| GB201102212D0 (en) | Inventions and therapy |