JP6602788B2 - アデノ随伴ウイルスベクター - Google Patents
アデノ随伴ウイルスベクター Download PDFInfo
- Publication number
- JP6602788B2 JP6602788B2 JP2016569022A JP2016569022A JP6602788B2 JP 6602788 B2 JP6602788 B2 JP 6602788B2 JP 2016569022 A JP2016569022 A JP 2016569022A JP 2016569022 A JP2016569022 A JP 2016569022A JP 6602788 B2 JP6602788 B2 JP 6602788B2
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- variant
- aav
- sequence
- wild 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.)
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Classifications
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- 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
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- 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
-
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
-
- 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
- A61P27/06—Antiglaucoma agents or miotics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C12N15/861—Adenoviral vectors
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Ophthalmology & Optometry (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019186201A JP6942166B2 (ja) | 2014-02-17 | 2019-10-09 | アデノ随伴ウイルスベクター |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461940639P | 2014-02-17 | 2014-02-17 | |
| US61/940,639 | 2014-02-17 | ||
| GB1403684.2 | 2014-03-03 | ||
| GBGB1403684.2A GB201403684D0 (en) | 2014-03-03 | 2014-03-03 | Vector |
| PCT/EP2015/053335 WO2015121501A1 (en) | 2014-02-17 | 2015-02-17 | Adeno-associated virus vector |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019186201A Division JP6942166B2 (ja) | 2014-02-17 | 2019-10-09 | アデノ随伴ウイルスベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017506521A JP2017506521A (ja) | 2017-03-09 |
| JP2017506521A5 JP2017506521A5 (enExample) | 2018-03-29 |
| JP6602788B2 true JP6602788B2 (ja) | 2019-11-06 |
Family
ID=50490674
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016569022A Active JP6602788B2 (ja) | 2014-02-17 | 2015-02-17 | アデノ随伴ウイルスベクター |
| JP2019186201A Active JP6942166B2 (ja) | 2014-02-17 | 2019-10-09 | アデノ随伴ウイルスベクター |
| JP2021086867A Active JP7309780B2 (ja) | 2014-02-17 | 2021-05-24 | アデノ随伴ウイルスベクター |
| JP2023109822A Active JP7612767B2 (ja) | 2014-02-17 | 2023-07-04 | アデノ随伴ウイルスベクター |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019186201A Active JP6942166B2 (ja) | 2014-02-17 | 2019-10-09 | アデノ随伴ウイルスベクター |
| JP2021086867A Active JP7309780B2 (ja) | 2014-02-17 | 2021-05-24 | アデノ随伴ウイルスベクター |
| JP2023109822A Active JP7612767B2 (ja) | 2014-02-17 | 2023-07-04 | アデノ随伴ウイルスベクター |
Country Status (20)
| Country | Link |
|---|---|
| US (4) | US20180135076A1 (enExample) |
| EP (2) | EP3108000B1 (enExample) |
| JP (4) | JP6602788B2 (enExample) |
| KR (1) | KR102082032B1 (enExample) |
| CN (1) | CN106062200B (enExample) |
| AU (3) | AU2015216847B2 (enExample) |
| BR (1) | BR112016018598A2 (enExample) |
| CA (2) | CA3126061C (enExample) |
| DK (1) | DK3108000T3 (enExample) |
| ES (1) | ES2749235T3 (enExample) |
| GB (1) | GB201403684D0 (enExample) |
| HU (1) | HUE045801T2 (enExample) |
| IL (2) | IL247332B (enExample) |
| MX (1) | MX372992B (enExample) |
| PL (1) | PL3108000T3 (enExample) |
| PT (1) | PT3108000T (enExample) |
| RU (1) | RU2743382C2 (enExample) |
| SG (1) | SG11201606365TA (enExample) |
| SI (1) | SI3108000T1 (enExample) |
| WO (1) | WO2015121501A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020022476A (ja) * | 2014-02-17 | 2020-02-13 | キングス カレッジ ロンドンKings College London | アデノ随伴ウイルスベクター |
Families Citing this family (212)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| CA3050894C (en) | 2010-04-23 | 2022-10-18 | University Of Massachusetts | Multicistronic expression constructs |
| DK2986635T3 (en) | 2013-04-18 | 2019-01-28 | Fond Telethon | EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| EP3049527A4 (en) | 2013-09-26 | 2017-08-16 | University of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| JP6756700B2 (ja) | 2014-03-18 | 2020-09-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症を処置するためのrAAVベースの組成物および方法 |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| LT3137497T (lt) * | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| WO2016054557A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Novel high efficiency library-identified aav vectors |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| US20180057839A1 (en) * | 2014-11-26 | 2018-03-01 | The Regents Of The University Of California | Therapeutic compositions comprising transcription factors and methods of making and using the same |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| IL314171A (en) * | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| WO2017070476A2 (en) | 2015-10-22 | 2017-04-27 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav3 capsid library |
| EP3364997B1 (en) | 2015-10-22 | 2024-01-17 | University of Massachusetts | Aspartoacylase gene therapy in the treatment of canavan disease |
| WO2017070516A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| US20180230489A1 (en) | 2015-10-28 | 2018-08-16 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| GB201519086D0 (en) * | 2015-10-28 | 2015-12-09 | Syncona Partners Llp | Gene Therapy |
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| SG10201912763QA (en) | 2015-11-05 | 2020-02-27 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
| AU2016362477A1 (en) | 2015-12-02 | 2018-06-14 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
| AU2016366549B2 (en) * | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| AU2016370630B2 (en) * | 2015-12-14 | 2023-04-13 | The Trustees Of The University Of Pennsylvania | Adeno-associated viral vectors useful in treatment of spinal muscular atropy |
| CA3011939A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| CA3012344A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| US20190071681A1 (en) * | 2016-02-26 | 2019-03-07 | University Of Florida Research Foundation, Incorporated | Aav heparin mutants that display significantly improved eye and brain transduction |
| EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN |
| CN107287238B (zh) * | 2016-04-11 | 2020-10-16 | 厦门继景生物技术有限责任公司 | 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物 |
| IL262207B2 (en) | 2016-04-15 | 2026-01-01 | Univ Pennsylvania | Preparations for the treatment of wet age-related macular degeneration |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US10584337B2 (en) | 2016-05-18 | 2020-03-10 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| MA44546B1 (fr) | 2016-06-15 | 2021-03-31 | Univ California | Virus adéno-associés variants et procédés d'utilisation |
| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| CA2971303C (en) | 2016-06-21 | 2026-03-03 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| US11427838B2 (en) | 2016-06-29 | 2022-08-30 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders |
| CA3029119A1 (en) | 2016-06-29 | 2018-01-04 | Crispr Therapeutics Ag | Materials and methods for treatment of friedreich ataxia and other related disorders |
| WO2018002762A1 (en) | 2016-06-29 | 2018-01-04 | Crispr Therapeutics Ag | Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders |
| EP4650364A3 (en) | 2016-07-06 | 2026-01-21 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of pain related disorders |
| EP3481857B1 (en) | 2016-07-06 | 2026-02-11 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of pain related disorders |
| US11458211B2 (en) | 2016-07-12 | 2022-10-04 | The University Of Manchester | Gene therapy |
| CN109640949B (zh) | 2016-07-29 | 2022-11-25 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体和其使用方法 |
| CA3033125A1 (en) * | 2016-08-16 | 2018-02-22 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
| EP3831281B1 (en) | 2016-08-30 | 2026-02-25 | The Regents of The University of California | Devices and systems for biomedical targeting and delivery |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| US11078238B2 (en) | 2016-09-29 | 2021-08-03 | University Of Florida Research Foundation, Incorporated | AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties |
| KR20190075964A (ko) | 2016-10-13 | 2019-07-01 | 유니버시티 오브 매사추세츠 | Aav 캡시드 설계 |
| AU2017345470B2 (en) * | 2016-10-19 | 2023-08-03 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| CN110300591A (zh) | 2016-11-04 | 2019-10-01 | 百深公司 | 腺相关病毒制剂 |
| JP2020508289A (ja) | 2017-01-31 | 2020-03-19 | レジェンクスバイオ インコーポレーテッド | 完全ヒトグリコシル化ヒトα−L−イズロニダーゼ(IDUA)を用いたムコ多糖症I型の治療 |
| EP3585883A4 (en) * | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| EP3585899A1 (en) | 2017-02-22 | 2020-01-01 | CRISPR Therapeutics AG | Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders |
| WO2018154418A1 (en) | 2017-02-22 | 2018-08-30 | Crispr Therapeutics Ag | Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders |
| WO2018154462A2 (en) | 2017-02-22 | 2018-08-30 | Crispr Therapeutics Ag | Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders |
| WO2018154439A1 (en) | 2017-02-22 | 2018-08-30 | Crispr Therapeutics Ag | Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders |
| AU2018224387B2 (en) | 2017-02-22 | 2024-08-08 | Crispr Therapeutics Ag | Compositions and methods for gene editing |
| KR20240093919A (ko) | 2017-04-14 | 2024-06-24 | 리젠엑스바이오 인크. | 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료 |
| MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| AU2018264996A1 (en) | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
| WO2018209154A1 (en) | 2017-05-10 | 2018-11-15 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modifying assembly-activating protein (app)-dependence of viruses |
| CN110831611A (zh) * | 2017-05-10 | 2020-02-21 | 马萨诸塞眼科耳科诊所 | 用于修饰病毒的组装活化蛋白(aap)依赖性的方法和组合物 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| KR20250023600A (ko) * | 2017-06-30 | 2025-02-18 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법 |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| US11512327B2 (en) | 2017-08-03 | 2022-11-29 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of AAV |
| JP2020533968A (ja) * | 2017-08-25 | 2020-11-26 | オービッド・セラピューティクス・インコーポレイテッドOvid Therapeutics Inc. | 組換えアデノ随伴ベクター |
| MX2020001997A (es) | 2017-08-28 | 2020-07-20 | Univ California | Variantes de capside de virus adenoasociado y metodos de uso de estas. |
| CN118006607A (zh) | 2017-09-22 | 2024-05-10 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| 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 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| KR20200067195A (ko) * | 2017-10-18 | 2020-06-11 | 리젠엑스바이오 인크. | 완전-인간 번역후 변형된 항체 치료제 |
| CA3084633A1 (en) | 2017-12-21 | 2019-06-27 | Crispr Therapeutics Ag | Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp) |
| AU2018393050A1 (en) | 2017-12-21 | 2020-06-18 | Bayer Healthcare Llc | Materials and methods for treatment of Usher Syndrome Type 2A |
| US12558434B2 (en) | 2018-02-20 | 2026-02-24 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| IL277270B2 (en) * | 2018-03-16 | 2025-05-01 | Res Inst Nationwide Childrens Hospital | Increasing tissue-specific gene delivery through capsid modification |
| EP3781187A4 (en) * | 2018-03-29 | 2021-12-22 | Asklepios Biopharmaceutical, Inc. | TROPICAL RECOMBINANT AAV6 LIVER VECTORS TO Bypass NEUTRALIZATION |
| CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| CN108660159B (zh) * | 2018-04-12 | 2020-10-30 | 四川大学 | 重组蝙蝠腺相关病毒载体及其用途 |
| KR102051393B1 (ko) | 2018-05-04 | 2019-12-04 | (주)지뉴인텍 | 아데노부속바이러스 기반 유전자 전달용 재조합 벡터 및 이를 이용한 암질환 예방 또는 치료용 조성물 |
| WO2019221992A1 (en) | 2018-05-15 | 2019-11-21 | President And Fellows Of Harvard College | Viral vectors exhibiting improved gene delivery properties |
| WO2019222329A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| 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 |
| WO2019236995A1 (en) | 2018-06-08 | 2019-12-12 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| MX2020013953A (es) | 2018-07-11 | 2021-03-09 | Takeda Pharmaceuticals Co | Composiciones de virus adenoasociado (aav). |
| BR112021001878A2 (pt) * | 2018-07-31 | 2021-04-27 | Cornell University | métodos de terapia genética para o controle da função do órgão |
| WO2020033842A1 (en) | 2018-08-10 | 2020-02-13 | Regenxbio Inc. | Scalable method for recombinant aav production |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN113166208B (zh) | 2018-10-02 | 2024-11-22 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| US20210371470A1 (en) | 2018-10-12 | 2021-12-02 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| CA3125770A1 (en) | 2019-01-18 | 2020-07-23 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
| GB201900702D0 (en) * | 2019-01-18 | 2019-03-06 | Univ Bristol | Therapy |
| KR20210126096A (ko) * | 2019-02-15 | 2021-10-19 | 상가모 테라퓨틱스, 인코포레이티드 | 재조합 aav를 제조하기 위한 조성물 및 방법 |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| KR20210142725A (ko) * | 2019-03-28 | 2021-11-25 | 오피노 엘엘씨 | 코어 페이징 처리 |
| CN114450411A (zh) | 2019-04-01 | 2022-05-06 | 特纳亚治疗股份有限公司 | 具有工程化衣壳的腺相关病毒 |
| CN114127088B (zh) * | 2019-04-23 | 2024-05-14 | 国家医疗保健研究所 | 变体衣壳蛋白和腺相关病毒aav变体及其药物组合物 |
| CA3132447A1 (en) | 2019-04-24 | 2020-10-29 | Takara Bio Inc. | Aav mutant having brain-targeting property |
| AU2020263392A1 (en) * | 2019-04-26 | 2021-10-28 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding AAV2.5 vector |
| EP3966227A1 (en) | 2019-05-07 | 2022-03-16 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| WO2020261178A1 (en) | 2019-06-27 | 2020-12-30 | Pfizer Inc. | Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy |
| US12173290B2 (en) | 2019-06-28 | 2024-12-24 | Crispr Therapeutics Ag | Materials and methods for controlling gene editing |
| AU2020299026A1 (en) * | 2019-07-04 | 2022-01-20 | Children's Medical Research Institute | Methods and AAV vectors for in vivo transduction |
| JP7371954B2 (ja) * | 2019-07-12 | 2023-10-31 | 株式会社遺伝子治療研究所 | ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| SMT202400486T1 (it) * | 2019-07-15 | 2025-01-14 | Meiragtx Uk Ii Ltd | Proteine di capside avv modificate per il trattamento della malattia artritica |
| EP4010357A2 (en) | 2019-08-09 | 2022-06-15 | University of Florida Research Foundation, Incorporated | Aav capsid variants for targeting human glioblastoma cells |
| JP7785352B2 (ja) * | 2019-08-14 | 2025-12-15 | ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. | 遺伝子治療のためのaavカプシドバリアント |
| AU2020336332A1 (en) * | 2019-08-29 | 2022-03-03 | The Board Of Trustees Of The Leland Stanford Junior University | Adeno-associated viral vectors for crossing the human blood brain barrier |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| IL291944A (en) | 2019-10-10 | 2022-06-01 | Solid Biosciences Inc | Modified aav capsids and uses thereof |
| WO2021073568A1 (en) * | 2019-10-16 | 2021-04-22 | Wuxi Apptec (Shanghai) Co., Ltd. | A novel aav variant |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| MX2022004353A (es) | 2019-10-17 | 2022-07-19 | Casete de transferencia de aav. | |
| EP4055034A4 (en) | 2019-11-08 | 2024-05-29 | President And Fellows Of Harvard College | VIRAL CAPSID POLYPEPTIDES |
| EP4055030A4 (en) | 2019-11-08 | 2024-05-22 | President And Fellows Of Harvard College | VIRAL CAPSID POLYPEPTIDES |
| WO2021108467A1 (en) | 2019-11-25 | 2021-06-03 | University Of Florida Research Foundation, Incorporated | Novel aav3b variants that target human hepatocytes and evade the humoral immune response |
| AU2020392243A1 (en) | 2019-11-28 | 2022-06-16 | Regenxbio Inc. | Microdystrophin gene therapy constructs and uses thereof |
| WO2021155137A1 (en) * | 2020-01-29 | 2021-08-05 | Genzyme Corporation | Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof |
| CA3180222A1 (en) | 2020-04-15 | 2021-10-21 | Voyager Therapeutics, Inc. | Tau binding compounds |
| US12630842B2 (en) | 2020-05-13 | 2026-05-19 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| GB202010009D0 (en) * | 2020-06-30 | 2020-08-12 | Syncona Investment Man Ltd | Vector |
| BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
| US20230285596A1 (en) | 2020-07-27 | 2023-09-14 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| CN111825772B (zh) * | 2020-07-30 | 2023-10-20 | 中国科学院精密测量科学与技术创新研究院 | 具有变异衣壳蛋白的腺相关病毒及其应用 |
| CA3181024A1 (en) | 2020-08-06 | 2022-02-10 | Fundacion Para La Investigacion Medica Aplicada | Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy |
| MX2023001701A (es) | 2020-08-12 | 2023-03-09 | UCB Biopharma SRL | Terapia genica utilizando constructos de acido nucleico que comprenden secuencias promotoras de la proteina de union a metil cpg 2 (mecp2). |
| JP2023542614A (ja) | 2020-08-19 | 2023-10-11 | サレプタ セラピューティクス, インコーポレイテッド | レット症候群の治療のためのアデノ随伴ウイルスベクター |
| IL300738A (en) * | 2020-08-28 | 2023-04-01 | Biocad Joint Stock Co | A vaccine for SARS-COV-2 based on AAV5 |
| CN112194706B (zh) * | 2020-09-30 | 2022-03-08 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| EP4225777A2 (en) * | 2020-10-07 | 2023-08-16 | RegenxBio Inc. | Adeno-associated viruses for ocular delivery of gene therapy |
| JP7415042B2 (ja) | 2020-10-09 | 2024-01-16 | テナヤ セラピューティクス, インコーポレイテッド | プラコフィリン2遺伝子治療の方法および組成物 |
| IL301255A (en) | 2020-10-09 | 2023-05-01 | UCB Biopharma SRL | Nucleic acid structures, viral vectors and viral particles |
| CN116490514A (zh) | 2020-10-28 | 2023-07-25 | 北卡罗来纳-查佩尔山大学 | 用于双重聚糖结合aav2.5载体的方法和组合物 |
| AU2021375404A1 (en) | 2020-11-03 | 2023-06-08 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| MX2023005446A (es) | 2020-11-11 | 2023-07-18 | European Molecular Biology Laboratory | Particulas virales modificadas para terapia genica. |
| WO2022130172A1 (en) | 2020-12-15 | 2022-06-23 | Pfizer Inc. | Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins |
| CA3205588A1 (en) | 2020-12-21 | 2022-06-30 | Pfizer Inc. | Methods and systems for improved cell transfection |
| JP2024503234A (ja) | 2020-12-23 | 2024-01-25 | ファイザー・インク | 親和性クロマトグラフィーによるaavベクターの精製方法 |
| AU2022221284A1 (en) | 2021-02-10 | 2023-08-17 | Regenxbio Inc. | Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) |
| GB202101958D0 (en) | 2021-02-12 | 2021-03-31 | Ucl Business Ltd | Gene therapy for dopamine transporter deficiency syndrome |
| CN112961219B (zh) * | 2021-02-24 | 2022-08-12 | 中国农业科学院生物技术研究所 | 重组腺相关病毒、其突变体及其构建方法和应用 |
| CN115044614B (zh) * | 2021-03-09 | 2023-10-20 | 上海目镜生物医药科技有限公司 | 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用 |
| WO2022208342A1 (en) | 2021-04-01 | 2022-10-06 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| CA3216420A1 (en) | 2021-04-26 | 2022-11-03 | Seungil Han | Adeno-associated viral vector capsids with improved tissue tropism |
| BR112023023249A2 (pt) * | 2021-05-07 | 2024-01-23 | Oyster Point Pharma Inc | Fator neurotrófico de codificação de vírion aav e usos do mesmo |
| CN117377771A (zh) | 2021-05-12 | 2024-01-09 | 特莱索恩基金会 | 载体系统 |
| MX2023015329A (es) | 2021-06-22 | 2024-01-23 | Pfizer | Produccion de vector de virus adeno-asociado en celulas de insecto. |
| WO2023283962A1 (en) | 2021-07-16 | 2023-01-19 | Huigene Therapeutics Co., Ltd. | Modified aav capsid for gene therapy and methods thereof |
| US20230241118A1 (en) | 2021-10-20 | 2023-08-03 | University Of Rochester | Rejuvenation treatment of age-related white matter loss |
| IL312340A (en) | 2021-10-28 | 2024-06-01 | UCB Biopharma SRL | Nucleic acid constructs, viral vectors and viral particles |
| EP4426716A1 (en) | 2021-11-02 | 2024-09-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| CA3236365A1 (en) | 2021-11-02 | 2023-05-11 | University Of Rochester | Tcf7l2 mediated remyelination in the brain |
| CA3238867A1 (en) | 2021-11-29 | 2023-06-01 | Shanghai Regenelead Therapies Co., Ltd. | Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease |
| WO2023114816A1 (en) | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Recombinant optimized galc constructs and methods for treating galc-associated disorders |
| WO2023125481A1 (zh) * | 2021-12-28 | 2023-07-06 | 成都弘基生物科技有限公司 | 经过修饰的aav衣壳蛋白及其用途 |
| GB202201242D0 (en) | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| WO2023155918A1 (zh) | 2022-02-21 | 2023-08-24 | 上海瑞宏迪医药有限公司 | Vegf结合分子及其医药用途 |
| WO2023166425A1 (en) | 2022-03-01 | 2023-09-07 | Crispr Therapeutics Ag | Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions |
| AU2023250649A1 (en) | 2022-04-04 | 2024-11-14 | The Regents Of The University Of California | Genetic complementation compositions and methods |
| GB202206336D0 (en) | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| CA3255627A1 (en) | 2022-05-06 | 2023-11-09 | Novartis Ag | NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV |
| WO2024003687A1 (en) | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| WO2024011112A1 (en) * | 2022-07-06 | 2024-01-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
| US20260041792A1 (en) * | 2022-07-12 | 2026-02-12 | University Of Florida Research Foundation, Incorporated | Novel aav3b capsid variants with enhanced hepatocyte tropism |
| KR20240014846A (ko) * | 2022-07-26 | 2024-02-02 | 숙명여자대학교산학협력단 | Aav2-anks1a 재조합 바이러스를 포함하는 알츠하이머 질병 치료용 조성물 |
| WO2024038365A1 (en) | 2022-08-16 | 2024-02-22 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| CN116063404B (zh) * | 2022-09-27 | 2025-08-29 | 广州译码基因科技有限公司 | 可提高AAV包装能力的衣壳蛋白MutD及其应用 |
| GB202216168D0 (en) | 2022-10-31 | 2022-12-14 | UCB Biopharma SRL | Route of administration |
| CN116041443B (zh) * | 2022-12-30 | 2023-09-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN115925819B (zh) * | 2022-12-30 | 2023-10-13 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| JP2026501732A (ja) * | 2023-01-05 | 2026-01-16 | エミュジェン セラピューティクス エルエルシー | キメラaavおよびその使用 |
| EP4658294A2 (en) | 2023-02-02 | 2025-12-10 | University of Rochester | Competitive replacement of glial cells |
| AU2023427408A1 (en) | 2023-02-02 | 2025-09-04 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency |
| JP2026509301A (ja) | 2023-03-14 | 2026-03-17 | ユーシービー バイオファルマ エスアールエル | 遺伝子治療 |
| GB202304767D0 (en) * | 2023-03-30 | 2023-05-17 | Ucl Business Ltd | Synthetic AAV Capsid |
| CN121311595A (zh) | 2023-04-17 | 2026-01-09 | 阿斯克生物公司 | 转基因在灵长类动物中可调节的受控表达 |
| WO2024238853A1 (en) * | 2023-05-16 | 2024-11-21 | Regenxbio Inc. | Adeno-associated viruses for ocular delivery of gene therapy |
| WO2025022290A1 (en) | 2023-07-21 | 2025-01-30 | Crispr Therapeutics Ag | Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene |
| TW202508610A (zh) | 2023-08-23 | 2025-03-01 | 大陸商上海瑞宏迪醫藥有限公司 | 醫藥組成物及其用途 |
| WO2025049917A1 (en) * | 2023-08-31 | 2025-03-06 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025090427A1 (en) | 2023-10-23 | 2025-05-01 | University Of Rochester | Glial-targeted relief of hyperexcitability in neurodegenerative diseases |
| AU2024386366A1 (en) | 2023-11-23 | 2026-06-04 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Gene therapy compositions and methods for treating glioma |
| WO2025113676A1 (en) | 2023-11-29 | 2025-06-05 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treating stroke in primates |
| CN118440161B (zh) * | 2024-01-16 | 2025-02-25 | 沈阳兴齐眼药股份有限公司 | 提高aav病毒视网膜穿透能力的衣壳蛋白突变体及其应用 |
| WO2025186726A1 (en) | 2024-03-05 | 2025-09-12 | Crispr Therapeutics Ag | Modulating expression of agt (angiotensinogen) gene |
| WO2025228976A1 (en) | 2024-04-29 | 2025-11-06 | UCB Biopharma SRL | Gene therapy |
| GB202407038D0 (en) | 2024-05-17 | 2024-07-03 | Axovia Therapeutics Ltd | Novel Gene Therapy |
| WO2025250454A1 (en) | 2024-05-28 | 2025-12-04 | University Of Rochester | Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo |
| WO2025262203A1 (en) | 2024-06-21 | 2025-12-26 | UCB Biopharma SRL | Nucleic acid sequences |
| WO2026017965A1 (en) | 2024-07-19 | 2026-01-22 | The University Court Of The University Of Edinburgh | Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions |
| WO2026114267A1 (en) | 2024-11-29 | 2026-06-04 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treating alzheimer's disease in primates |
| WO2026150302A1 (en) | 2025-01-09 | 2026-07-16 | Novartis Ag | Novel aav rep/cap encoding constructs |
Family Cites Families (56)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5639872A (en) | 1993-07-27 | 1997-06-17 | Hybridon, Inc. | Human VEGF-specific oligonucleotides |
| WO1997020925A1 (en) | 1995-12-08 | 1997-06-12 | Hybridon, Inc. | Modified vegf antisense oligonucleotides for treatment of skin disorders |
| AU4645697A (en) | 1996-09-11 | 1998-04-02 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| DE69939169D1 (de) | 1998-05-28 | 2008-09-04 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| DE69936104T2 (de) | 1998-11-05 | 2008-01-17 | The Trustees Of The University Of Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
| US20030144221A1 (en) | 2001-07-17 | 2003-07-31 | Isis Pharmaceuticals Inc. | Antisense modulation of BCL2-associated X protein expression |
| EP1572893B1 (en) | 2001-11-09 | 2009-01-07 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES | Production of adeno-associated virus in insect cells |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| EP1463805B1 (en) | 2001-12-17 | 2014-10-22 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| US20050106558A1 (en) | 2001-12-21 | 2005-05-19 | Luca Perabo | Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism |
| SI1496944T1 (sl) | 2002-05-01 | 2009-02-28 | Univ Florida | Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov |
| US7148342B2 (en) | 2002-07-24 | 2006-12-12 | The Trustees Of The University Of Pennyslvania | Compositions and methods for sirna inhibition of angiogenesis |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| PL2657248T3 (pl) | 2003-06-19 | 2017-09-29 | Genzyme Corporation | Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| EP1668143B1 (en) * | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| ES2423060T3 (es) | 2004-03-12 | 2013-09-17 | Alnylam Pharmaceuticals, Inc. | Agentes iRNA que tienen como diana al VEGF |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| DE23193606T1 (de) * | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| US7543116B2 (en) | 2006-01-30 | 2009-06-02 | International Business Machines Corporation | Data processing system, cache system and method for handling a flush operation in a data processing system having multiple coherency domains |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| JP2009535339A (ja) * | 2006-04-28 | 2009-10-01 | 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 | 低下されたキャプシド免疫原性を有する改変aavベクターおよびその使用 |
| CN101528916B (zh) | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
| ES2385679T3 (es) | 2006-08-24 | 2012-07-30 | Virovek, Inc. | Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos |
| US7872118B2 (en) | 2006-09-08 | 2011-01-18 | Opko Ophthalmics, Llc | siRNA and methods of manufacture |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
| EP2012122A1 (en) | 2007-07-06 | 2009-01-07 | Medigene AG | Mutated parvovirus structural proteins as vaccines |
| WO2009108274A2 (en) | 2008-02-26 | 2009-09-03 | The University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| EP2396343B1 (en) * | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| DK2425000T3 (da) | 2009-04-30 | 2019-05-13 | Univ Pennsylvania | Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US9133479B2 (en) | 2009-06-03 | 2015-09-15 | Cedars-Sinai Medical Center | Effective vector platform for gene transfer and gene therapy |
| WO2011011572A1 (en) | 2009-07-22 | 2011-01-27 | Aware, Inc. | Improved packet detector |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| CN102071206A (zh) * | 2010-10-22 | 2011-05-25 | 中山大学 | 一种腺相关病毒衣壳蛋白基因、相应的蛋白及其应用 |
| DK3693025T3 (da) * | 2011-04-22 | 2022-01-10 | Univ California | Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf |
| EP3795581A3 (en) | 2011-08-24 | 2021-06-09 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
| JP6342886B2 (ja) * | 2012-04-18 | 2018-06-13 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | Aavカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法 |
| EP3470523A1 (en) | 2012-05-09 | 2019-04-17 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| TWI698240B (zh) | 2012-05-15 | 2020-07-11 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| WO2014186746A1 (en) | 2013-05-16 | 2014-11-20 | University Of Florida Research Foundation, Inc. | HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| EP3113787B1 (en) | 2014-03-04 | 2019-12-04 | University of Florida Research Foundation, Inc. | Improved raav vectors and methods for transduction of photoreceptors and rpe cells |
| US10648000B2 (en) | 2015-02-16 | 2020-05-12 | University Of Florida Research Foundation, Incorporated | rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes |
| IL314171A (en) | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| WO2017066764A2 (en) | 2015-10-16 | 2017-04-20 | William Marsh Rice University | Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US11458211B2 (en) | 2016-07-12 | 2022-10-04 | The University Of Manchester | Gene therapy |
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Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020022476A (ja) * | 2014-02-17 | 2020-02-13 | キングス カレッジ ロンドンKings College London | アデノ随伴ウイルスベクター |
| US11802293B2 (en) | 2014-02-17 | 2023-10-31 | King's College London | Adeno-associated virus vector |
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