IL228031B - Adno vectors for gene therapy in choroideremia - Google Patents
Adno vectors for gene therapy in choroideremiaInfo
- Publication number
- IL228031B IL228031B IL228031A IL22803113A IL228031B IL 228031 B IL228031 B IL 228031B IL 228031 A IL228031 A IL 228031A IL 22803113 A IL22803113 A IL 22803113A IL 228031 B IL228031 B IL 228031B
- Authority
- IL
- Israel
- 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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1103062.4A GB201103062D0 (en) | 2011-02-22 | 2011-02-22 | Method |
| PCT/GB2012/050376 WO2012114090A1 (en) | 2011-02-22 | 2012-02-21 | Aav -vectors for use in gene therapy of choroideremia |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| IL228031A0 IL228031A0 (en) | 2013-09-30 |
| IL228031B true IL228031B (en) | 2018-02-28 |
Family
ID=43881504
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL228031A IL228031B (en) | 2011-02-22 | 2013-08-19 | Adno vectors for gene therapy in choroideremia |
Country Status (18)
| Country | Link |
|---|---|
| US (4) | US9834788B2 (enExample) |
| EP (2) | EP3428283A1 (enExample) |
| JP (4) | JP2014512171A (enExample) |
| KR (3) | KR20210112407A (enExample) |
| CN (2) | CN107881198A (enExample) |
| AU (2) | AU2012220404B2 (enExample) |
| BR (1) | BR112013021318B1 (enExample) |
| CA (1) | CA2827975C (enExample) |
| DK (1) | DK2678435T3 (enExample) |
| ES (1) | ES2676550T5 (enExample) |
| GB (1) | GB201103062D0 (enExample) |
| HK (2) | HK1258917A1 (enExample) |
| IL (1) | IL228031B (enExample) |
| MX (2) | MX356525B (enExample) |
| PL (1) | PL2678435T3 (enExample) |
| PT (1) | PT2678435T (enExample) |
| SG (1) | SG192911A1 (enExample) |
| WO (1) | WO2012114090A1 (enExample) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201103062D0 (en) † | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
| CA2919995A1 (en) | 2013-08-05 | 2015-02-12 | 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 |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| KR20230169197A (ko) | 2014-11-14 | 2023-12-15 | 보이저 테라퓨틱스, 인크. | 근위축성 측삭 경화증(als)을 치료하는 조성물 및 방법 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| US20180043034A1 (en) * | 2015-03-06 | 2018-02-15 | Massachusetts Eye And Ear Infirmary | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
| AU2016370487C1 (en) * | 2015-12-14 | 2022-03-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| US20190017096A1 (en) * | 2016-03-10 | 2019-01-17 | Nightstarx Limited | Prenylation assay |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| CN117904112A (zh) | 2016-05-18 | 2024-04-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| EP3506817A4 (en) | 2016-08-30 | 2020-07-22 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| US10633689B2 (en) | 2016-11-07 | 2020-04-28 | Spark Therapeutics, Inc. | Rab Escort Protein potency assay |
| FI3568483T3 (fi) * | 2017-01-10 | 2025-11-21 | Childrens Medical Res Institute | Polynukleotideja ja vektoreita siirtogeenien ilmentämiseen |
| 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 |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN119491003A (zh) | 2017-05-05 | 2025-02-21 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| 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 |
| MX2020004005A (es) * | 2017-10-03 | 2020-10-05 | Prevail Therapeutics Inc | Terapias genicas para los trastornos lisosomales. |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| AU2018352236B2 (en) | 2017-10-16 | 2025-04-10 | The Curators Of The University Of Missouri | 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 |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| CN112041437A (zh) | 2018-02-19 | 2020-12-04 | 同源药物公司 | 用于恢复f8基因功能的腺相关病毒组合物和其使用方法 |
| SG11202009698PA (en) * | 2018-04-05 | 2020-10-29 | Nightstarx Ltd | Aav compositions, methods of making and methods of use |
| NZ759802A (en) * | 2018-05-07 | 2025-08-29 | Univ North Carolina Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CA3134485A1 (en) * | 2019-04-30 | 2020-11-05 | James M. Wilson | Compositions useful for treatment of pompe disease |
| 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 |
| IL300526A (en) | 2020-08-17 | 2023-04-01 | Massachusetts Inst Technology | Approaches to SHANK3 gene therapy |
| WO2022109247A1 (en) | 2020-11-20 | 2022-05-27 | Biogen Ma Inc. | Viral vector potency assay |
| WO2023164545A1 (en) | 2022-02-23 | 2023-08-31 | Massachusetts Institute Of Technology | Methods for upregulating shank3 expression |
| EP4569124A1 (en) | 2022-08-11 | 2025-06-18 | Institute Of Molecular And Clinical Ophthalmology Basel (Iob) | Promoters for specific expression of genes in cone photoreceptors |
| IL318810A (en) | 2022-08-11 | 2025-04-01 | Inst Of Molecular And Clinical Ophthalmology Basel Iob | Optogenetic structures of human cone photoreceptors |
| 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 |
| US20020045264A1 (en) | 2000-03-14 | 2002-04-18 | During Matthew J. | Production of chimeric capsid vectors |
| EP1381276A4 (en) | 2001-04-13 | 2005-02-02 | Univ Pennsylvania | METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION |
| US20040208847A1 (en) * | 2003-03-28 | 2004-10-21 | Fabienne Rolling | Method and vectors for selectively transducing retinal pigment epithelium cells |
| US8278284B2 (en) * | 2005-02-23 | 2012-10-02 | Masanori Miyazaki | Therapeutic agents for diseases associated with apoptotic degeneration in ocular tissue cells that use SIV-PEDF vectors |
| AU2006242371A1 (en) * | 2005-04-29 | 2006-11-09 | The University Of North Carolina At Chapel Hill | Methods and compositions for regulated expression of nucleic acid at post-transcriptional level |
| US20090022731A1 (en) * | 2006-08-25 | 2009-01-22 | Wyeth | Arthritis-associated B cell gene expression |
| WO2009021293A1 (en) * | 2007-08-16 | 2009-02-19 | St Vincent's Hospital Sydney Limited | Agents and methods for modulating macrophage inhibitory cytokine (mic-1) activity |
| DK2242840T3 (da) * | 2008-01-29 | 2019-10-21 | Applied Genetic Tech Corporation | Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension |
| AU2009212309B2 (en) * | 2008-02-07 | 2014-04-03 | Ceregene, Inc. | Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein |
| PL4342992T3 (pl) * | 2009-05-02 | 2025-09-22 | Genzyme Corporation | Terapia genowa dla zaburzeń neurodegeneracyjnych |
| ES2683695T3 (es) | 2010-01-12 | 2018-09-27 | The University Of North Carolina At Chapel Hill | Repeticiones terminales invertidas restrictivas para vectores virales |
| 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 DK DK12706308.9T patent/DK2678435T3/en active
- 2012-02-21 PT PT127063089T patent/PT2678435T/pt unknown
- 2012-02-21 BR BR112013021318-3A patent/BR112013021318B1/pt not_active IP Right Cessation
- 2012-02-21 SG SG2013063797A patent/SG192911A1/en unknown
- 2012-02-21 EP EP18172129.1A patent/EP3428283A1/en not_active Withdrawn
- 2012-02-21 KR KR1020217028082A patent/KR20210112407A/ko not_active Ceased
- 2012-02-21 KR KR1020207021515A patent/KR102299430B1/ko not_active Expired - Fee Related
- 2012-02-21 AU AU2012220404A patent/AU2012220404B2/en not_active Ceased
- 2012-02-21 CA CA2827975A patent/CA2827975C/en active Active
- 2012-02-21 ES ES12706308T patent/ES2676550T5/es active Active
- 2012-02-21 EP EP12706308.9A patent/EP2678435B2/en active Active
- 2012-02-21 WO PCT/GB2012/050376 patent/WO2012114090A1/en not_active Ceased
- 2012-02-21 MX MX2013009604A patent/MX356525B/es active IP Right Grant
- 2012-02-21 JP JP2013554938A patent/JP2014512171A/ja active Pending
- 2012-02-21 KR KR1020137024782A patent/KR102139203B1/ko not_active Expired - Fee Related
- 2012-02-21 CN CN201710784145.9A patent/CN107881198A/zh active Pending
- 2012-02-21 US US14/000,836 patent/US9834788B2/en active Active
- 2012-02-21 PL PL12706308T patent/PL2678435T3/pl unknown
- 2012-02-21 MX MX2018006733A patent/MX388146B/es unknown
- 2012-02-21 CN CN201280019547.5A patent/CN103562396A/zh active Pending
-
2013
- 2013-08-19 IL IL228031A patent/IL228031B/en active IP Right Grant
-
2014
- 2014-05-23 HK HK19101394.9A patent/HK1258917A1/en unknown
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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
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2018
- 2018-07-13 HK HK18109118.8A patent/HK1249548A1/zh unknown
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2019
- 2019-11-05 JP JP2019201092A patent/JP2020054345A/ja active Pending
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2020
- 2020-01-24 US US16/751,699 patent/US20210032656A1/en not_active Abandoned
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2022
- 2022-10-26 US US17/974,466 patent/US20230332176A1/en active Pending
- 2022-12-02 JP JP2022193831A patent/JP2023036618A/ja active Pending
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FF | Patent granted | ||
| KB | Patent renewed |