HUE043921T2 - Készítmények és módszerek szövetspecifitás módosítására és AAV9-mediált géntranszfer javítására - Google Patents
Készítmények és módszerek szövetspecifitás módosítására és AAV9-mediált géntranszfer javításáraInfo
- Publication number
- HUE043921T2 HUE043921T2 HUE12705773A HUE12705773A HUE043921T2 HU E043921 T2 HUE043921 T2 HU E043921T2 HU E12705773 A HUE12705773 A HU E12705773A HU E12705773 A HUE12705773 A HU E12705773A HU E043921 T2 HUE043921 T2 HU E043921T2
- Authority
- HU
- Hungary
- Prior art keywords
- aav9
- compositions
- improving
- methods
- gene transfer
- Prior art date
Links
Classifications
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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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/66—Phosphorus compounds
- A61K31/683—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols
- A61K31/685—Diesters of a phosphorus acid with two hydroxy compounds, e.g. phosphatidylinositols one of the hydroxy compounds having nitrogen atoms, e.g. phosphatidylserine, lecithin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14045—Special targeting system for viral 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161443879P | 2011-02-17 | 2011-02-17 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE043921T2 true HUE043921T2 (hu) | 2019-09-30 |
Family
ID=45757234
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE12705773A HUE043921T2 (hu) | 2011-02-17 | 2012-02-17 | Készítmények és módszerek szövetspecifitás módosítására és AAV9-mediált géntranszfer javítására |
Country Status (16)
| Country | Link |
|---|---|
| US (5) | US9884071B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP2675902B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP6224459B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR101956885B1 (cg-RX-API-DMAC7.html) |
| CN (1) | CN103502458B (cg-RX-API-DMAC7.html) |
| AU (1) | AU2012219380B2 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112013020734A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2826316C (cg-RX-API-DMAC7.html) |
| DK (1) | DK2675902T3 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2724800T3 (cg-RX-API-DMAC7.html) |
| HU (1) | HUE043921T2 (cg-RX-API-DMAC7.html) |
| MX (1) | MX353930B (cg-RX-API-DMAC7.html) |
| PL (1) | PL2675902T3 (cg-RX-API-DMAC7.html) |
| SG (3) | SG192268A1 (cg-RX-API-DMAC7.html) |
| TR (1) | TR201908020T4 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2012112832A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (121)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| 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 |
| JP2013533847A (ja) | 2010-04-23 | 2013-08-29 | ユニバーシティ オブ マサチューセッツ | コレステロール関連障害のａａｖベースの治療 |
| CA2833908C (en) | 2010-04-23 | 2021-02-09 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| WO2012109570A1 (en) * | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| EP3318634A1 (en) | 2011-04-21 | 2018-05-09 | University of Massachusetts | Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations |
| JP6385920B2 (ja) * | 2012-05-09 | 2018-09-05 | オレゴン ヘルス アンド サイエンス ユニバーシティー | アデノ随伴ウイルスプラスミド及びベクター |
| EP2749569A1 (en) | 2012-12-28 | 2014-07-02 | Annibale Alessandro Puca | Variant of BPIFB4 protein |
| US20140219964A1 (en) * | 2013-02-07 | 2014-08-07 | Children's Medical Center Corporation | Methods for inducing cardiomyocyte proliferation |
| JP2016514152A (ja) * | 2013-03-13 | 2016-05-19 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | アデノ随伴ウイルスベクターおよびその使用の方法 |
| PT2968605T (pt) * | 2013-03-15 | 2022-09-22 | Univ North Carolina Chapel Hill | Métodos e composições para vetores aav de ligação dupla de glicano |
| JP6591956B2 (ja) | 2013-03-15 | 2019-10-16 | 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 | Ｍｐｓ１を治療するための組成物および方法 |
| PT3119437T (pt) | 2014-03-21 | 2019-12-12 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
| US10746742B2 (en) | 2014-04-25 | 2020-08-18 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| 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 |
| PT4012035T (pt) | 2014-09-16 | 2025-02-11 | Genzyme Corp | Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc) |
| EP4012035B1 (en) | 2014-09-16 | 2024-11-06 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
| EP3209311B1 (en) | 2014-10-21 | 2024-03-06 | University of Massachusetts | Recombinant aav variants and uses thereof |
| RU2020109343A (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) |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| EP3247793A1 (en) | 2015-01-20 | 2017-11-29 | Genzyme Corporation | Analytical ultracentrifugation for characterization of recombinant viral particles |
| KR102863734B1 (ko) | 2015-02-10 | 2025-09-25 | 젠자임 코포레이션 | 선조체 및 피질로의 바이러스 입자의 향상된 전달 |
| EA201791805A1 (ru) | 2015-02-10 | 2018-05-31 | Джензим Корпорейшн | ВАРИАНТ СРЕДСТВА ДЛЯ RNAi |
| WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| EP3285780A4 (en) | 2015-04-24 | 2018-12-19 | University of Massachusetts | Modified aav constructions and uses thereof |
| KR102584873B1 (ko) | 2015-05-16 | 2023-10-10 | 젠자임 코포레이션 | 심부 인트론 돌연변이의 유전자 편집 |
| WO2017019876A1 (en) * | 2015-07-28 | 2017-02-02 | University Of Massachusetts | Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector |
| DK3364997T5 (da) | 2015-10-22 | 2024-09-30 | Univ Massachusetts | Aspartoacylase genterapi til behandling af canavans sygdom |
| US11426469B2 (en) | 2015-10-22 | 2022-08-30 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| WO2017096162A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| EP3387118B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| SG10201912935WA (en) | 2015-12-15 | 2020-02-27 | Genzyme Corp | Adeno-associated viral vectors for treating mucolipidosis type ii |
| US11826433B2 (en) | 2016-02-02 | 2023-11-28 | University Of Massachusetts | Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| WO2017139643A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| MA44874A (fr) | 2016-04-15 | 2019-03-13 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type ii |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11364308B2 (en) | 2016-05-13 | 2022-06-21 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| IL302748B1 (en) | 2016-05-18 | 2025-09-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| WO2017218852A1 (en) | 2016-06-15 | 2017-12-21 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| CN106093369B (zh) * | 2016-06-29 | 2017-10-20 | 福建农林大学 | Bt水稻对非靶标害虫褐飞虱的安全性评价方法 |
| EP3497207B1 (en) | 2016-08-15 | 2021-01-06 | Genzyme Corporation | Methods for detecting aav |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| EP3526333A4 (en) | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
| WO2018119330A2 (en) * | 2016-12-22 | 2018-06-28 | Oregon Health & Science University | Adeno associated viral vectors |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| WO2019010335A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I |
| EP3654860A1 (en) | 2017-07-17 | 2020-05-27 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| DK3684423T5 (da) | 2017-09-20 | 2024-08-26 | 4D Molecular Therapeutics Inc | Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf |
| AU2018335410B2 (en) | 2017-09-22 | 2025-04-03 | Genzyme Corporation | Variant RNAi |
| IL273427B2 (en) | 2017-09-22 | 2025-04-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous polycythemia vera type II |
| WO2019067840A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| EP3717636B1 (en) | 2017-11-27 | 2023-03-08 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| WO2019152843A1 (en) | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| CN111886021A (zh) * | 2018-02-01 | 2020-11-03 | 同源药物公司 | 用于pah基因转移的腺伴随病毒组合物及其使用方法 |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| BR112020020223A2 (pt) | 2018-04-05 | 2021-01-19 | Genethon | Sorotipo de vírus adeno-associado híbrido recombinante entre aav9 e aavrh74 com tropismo pelo fígado reduzido |
| JP7575273B2 (ja) | 2018-04-16 | 2024-10-29 | 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 | デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法 |
| WO2019213668A1 (en) * | 2018-05-04 | 2019-11-07 | Oregon Health & Science University | Human anti-aav2 capsid polyclonal antibody epitopes |
| CA3099306A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| US12163129B2 (en) | 2018-06-08 | 2024-12-10 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| US12060567B2 (en) | 2018-06-13 | 2024-08-13 | Voyager Therapeutics, Inc. | Engineered untranslated regions (UTR) for AAV production |
| CN112770812A (zh) | 2018-07-24 | 2021-05-07 | 沃雅戈治疗公司 | 产生基因治疗制剂的系统和方法 |
| PH12021550254A1 (en) | 2018-08-03 | 2023-02-06 | Genzyme Corp | Variant rnai against alpha-synuclein |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| TW202035689A (zh) | 2018-10-04 | 2020-10-01 | 美商航海家醫療公司 | 測量病毒載體粒子的效價及強度之方法 |
| AU2019354793A1 (en) | 2018-10-05 | 2021-05-13 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding AAV production proteins |
| JP2022504740A (ja) | 2018-10-12 | 2022-01-13 | ジェンザイム・コーポレーション | 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成 |
| JP2022505106A (ja) | 2018-10-15 | 2022-01-14 | ボイジャー セラピューティクス インコーポレイテッド | バキュロウイルス／Ｓｆ９システムにおけるｒＡＡＶの大規模産生のための発現ベクター |
| UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
| AU2020216480A1 (en) | 2019-01-31 | 2021-09-09 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of AAV capsids |
| JP2022520875A (ja) | 2019-02-25 | 2022-04-01 | ノバルティス アーゲー | Ｂｉｅｔｔｉクリスタリン網膜症を治療するための組成物及び方法 |
| PH12021552038A1 (en) | 2019-02-25 | 2023-01-04 | Friedrich Miescher Institute For Biomedical Res | Compositions and methods to treat bietti crystalline dystrophy |
| JP2022522756A (ja) | 2019-02-28 | 2022-04-20 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | 治療薬の送達のためのアデノ随伴ウイルスベクター |
| JP2022528010A (ja) | 2019-04-12 | 2022-06-07 | ユニバーシティ オブ マサチューセッツ | メープルシロップ尿症（ｍｓｕｄ）のａａｖ媒介遺伝子治療 |
| KR20220009427A (ko) * | 2019-05-17 | 2022-01-24 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 글리코시드 가수분해효소를 사용하는 망막 세포에 대한 유전자 치료 벡터의 개선된 전달 |
| US10801042B1 (en) | 2019-07-15 | 2020-10-13 | Vigene Biosciences, Inc. | Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus |
| US10653731B1 (en) | 2019-07-15 | 2020-05-19 | Vigene Biosciences Inc. | Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency |
| US10557149B1 (en) | 2019-07-15 | 2020-02-11 | Vigene Biosciences, Inc. | Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus |
| 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 |
| AU2020376048A1 (en) * | 2019-11-01 | 2022-06-02 | Sangamo Therapeutics, Inc. | Compositions and methods for genome engineering |
| 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 |
| WO2022051468A1 (en) | 2020-09-04 | 2022-03-10 | Milwaukee Electric Tool Corporation | Chainsaw |
| BR112023003929A2 (pt) | 2020-10-01 | 2023-04-11 | Genzyme Corp | Cassete de expressão de pah humano para tratamento de pku por terapia de reposição gênica direcionada ao fígado |
| JP2024505257A (ja) | 2021-02-01 | 2024-02-05 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| WO2023010133A2 (en) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin (fxn) |
| WO2023010135A1 (en) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| AU2023211652A1 (en) * | 2022-01-25 | 2024-08-08 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
| IL316327A (en) | 2022-05-06 | 2024-12-01 | Novartis Ag | AAV VP2 fusion polypeptide novel recombinant |
| WO2024015881A2 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | Compositions, systems, and methods for targeted transcriptional activation |
| WO2024163683A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist) |
| WO2024163678A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| WO2024224361A1 (en) | 2023-04-26 | 2024-10-31 | Sanofi | Host cell lines and methods for identifying and using such host cell lines |
| US20250197862A1 (en) | 2023-12-15 | 2025-06-19 | Genzyme Corporation | ARTIFICIAL microRNAs TARGETING TAU |
| WO2025160429A1 (en) | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
| WO2025160434A1 (en) | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting huntington's disease |
| WO2025188755A1 (en) | 2024-03-04 | 2025-09-12 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy |
| WO2025226841A1 (en) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Gene therapy approach for treating disorders associated with tnnt2 |
| WO2025226842A1 (en) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Expression control by drg-expressed mirnas |
Family Cites Families (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS5774090A (en) | 1980-10-27 | 1982-05-10 | Shionogi & Co Ltd | Novel beta-galactosidase inhibitor, gt-2558, its derivative, and production |
| US4886876A (en) | 1983-03-31 | 1989-12-12 | Scripps Clinic And Research Foundation | Factor VIII coagulant polypeptides |
| US4542104A (en) | 1983-04-06 | 1985-09-17 | The Board Of Trustees Of The Leland Stanford Jr. Univ. | Phycobiliprotein fluorescent conjugates |
| US4757006A (en) | 1983-10-28 | 1988-07-12 | Genetics Institute, Inc. | Human factor VIII:C gene and recombinant methods for production |
| WO1985001961A1 (en) | 1983-10-28 | 1985-05-09 | Genetics Institute | Production of factor viii and related products |
| US5045455A (en) | 1984-01-12 | 1991-09-03 | Chiron Corporation | Factor VIII:C cDNA cloning and expression |
| FI86885C (fi) | 1984-04-20 | 1992-10-26 | Genentech Inc | Foerfarande foer framstaellning av human rekombinantfaktor viii och nukleinsyrasekvenser och vektorer anvaend daertill |
| US4965199A (en) | 1984-04-20 | 1990-10-23 | Genentech, Inc. | Preparation of functional human factor VIII in mammalian cells using methotrexate based selection |
| EP0182448A3 (en) | 1984-08-24 | 1987-10-28 | Genetics Institute, Inc. | Production of factor viii and related products |
| US5595886A (en) | 1986-01-27 | 1997-01-21 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
| FI98829C (fi) | 1986-01-27 | 1997-08-25 | Chiron Corp | Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta |
| US5451521A (en) | 1986-05-29 | 1995-09-19 | Genetics Institute, Inc. | Procoagulant proteins |
| US5422260A (en) | 1986-05-29 | 1995-06-06 | Genetics Institute, Inc. -Legal Affairs | Human factor VIII:c muteins |
| US5149637A (en) | 1987-04-06 | 1992-09-22 | Scripps Clinic & Research Foundation | Recombinant Factor VIIIC fragments |
| IL86693A (en) | 1987-06-12 | 1994-06-24 | Stichting Centraal Lab | Proteins with factor VIII activity, process for their preparation using genetically engineered cells and pharmaceutical compositions containing them |
| FR2619314B1 (fr) | 1987-08-11 | 1990-06-15 | Transgene Sa | Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant |
| US5004803A (en) | 1988-11-14 | 1991-04-02 | Genetics Institute, Inc. | Production of procoagulant proteins |
| CA2068728A1 (en) | 1989-11-17 | 1991-05-18 | Barbara Chapman | Protein complexes having factor viii:c activity and production thereof |
| SE465222C5 (sv) | 1989-12-15 | 1998-02-10 | Pharmacia & Upjohn Ab | Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning |
| US5272257A (en) | 1990-05-21 | 1993-12-21 | Coulter Corporation | Method of preferential labelling of a phycobiliprotein with an amine-reactive dye for use in a multiple color assay and product for such use |
| US5171846A (en) | 1990-05-21 | 1992-12-15 | Coulter Corporation | Method of preferential labelling of a phycobiliprotein with a second dye for use in a multiple color assay and product for such use |
| US5240640A (en) | 1990-06-04 | 1993-08-31 | Coulter Corporation | In situ use of gelatin or an aminodextran in the preparation of uniform ferrite particles |
| US5639620A (en) | 1990-10-31 | 1997-06-17 | Coulter Corporation | Polymeric particles having a biodegradable gelatin or aminodextran coating and process for making same |
| US5466609A (en) | 1990-10-31 | 1995-11-14 | Coulter Corporation | Biodegradable gelatin-aminodextran particle coatings of and processes for making same |
| US5661008A (en) | 1991-03-15 | 1997-08-26 | Kabi Pharmacia Ab | Recombinant human factor VIII derivatives |
| SE468050C (sv) | 1991-03-15 | 1998-04-27 | Pharmacia & Upjohn Ab | Rekombinant derivat av human faktor VIII |
| US5248771A (en) | 1991-06-26 | 1993-09-28 | Triumf | Process for preparing no-carrier-added radiohalogenated vinyl nucleosides |
| CA2078721A1 (en) | 1991-09-24 | 1993-03-25 | Hiroshi Yonemura | Process for preparing human coagulation factor viii protein complex |
| US5248772A (en) | 1992-01-29 | 1993-09-28 | Coulter Corporation | Formation of colloidal metal dispersions using aminodextrans as reductants and protective agents |
| US5563045A (en) | 1992-11-13 | 1996-10-08 | Genetics Institute, Inc. | Chimeric procoagulant proteins |
| AU5602394A (en) | 1992-11-13 | 1994-06-08 | Duke University | Chimeric blood coagulation proteins |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| EP0629700A3 (en) | 1993-06-10 | 1995-02-15 | Miles Inc | Mammalian vector and cell lines that have increased productivity. |
| CA2164646A1 (en) | 1993-06-11 | 1994-12-22 | Wade E. Bolton | Anti-cd3 antibody-aminodextran conjugates for induction of t-cell activation and proliferation |
| US5681746A (en) | 1994-12-30 | 1997-10-28 | Chiron Viagene, Inc. | Retroviral delivery of full length factor VIII |
| AU6486196A (en) | 1995-07-11 | 1997-02-10 | Chiron Corporation | Novel factor viii:c polypeptide analogs with altered protease sites |
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | 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 | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| US6096273A (en) | 1996-11-05 | 2000-08-01 | Clinical Micro Sensors | Electrodes linked via conductive oligomers to nucleic acids |
| US5945293A (en) | 1997-10-09 | 1999-08-31 | Coulter International Corp. | Protein-colloidal metal-aminodextran coated particle and methods of preparation and use |
| US6074884A (en) | 1997-10-09 | 2000-06-13 | Coulter International Corp. | Stable protein-nickel particles and methods of production and use thereof |
| US6117643A (en) | 1997-11-25 | 2000-09-12 | Ut Battelle, Llc | Bioluminescent bioreporter integrated circuit |
| US6197503B1 (en) | 1997-11-26 | 2001-03-06 | Ut-Battelle, Llc | Integrated circuit biochip microsystem containing lens |
| US6287857B1 (en) * | 1998-02-09 | 2001-09-11 | Genzyme Corporation | Nucleic acid delivery vehicles |
| WO2000014197A1 (en) | 1998-09-09 | 2000-03-16 | Hitachi Software Engineering Co., Ltd. | Biochip and method of using biochip |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| CN102199626B (zh) * | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006119432A2 (en) * | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| WO2010127097A1 (en) | 2009-04-30 | 2010-11-04 | The Trustees Of The University Of Pennsylvania | Compositions for targeting conducting airway cells comprising adeno-associated virus constructs |
| JP5458245B2 (ja) | 2010-06-25 | 2014-04-02 | 国立大学法人九州工業大学 | 動作制御装置、その方法、及びプログラム |
| WO2012109214A1 (en) * | 2011-02-07 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Targeted transduction of aav vectors |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
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| EP2675902A1 (en) | 2013-12-25 |
| BR112013020734A2 (pt) | 2017-06-13 |
| US20210145857A1 (en) | 2021-05-20 |
| SG10201601110VA (en) | 2016-03-30 |
| KR20140046407A (ko) | 2014-04-18 |
| US20200101099A1 (en) | 2020-04-02 |
| AU2012219380B2 (en) | 2017-04-13 |
| US10406173B2 (en) | 2019-09-10 |
| MX353930B (es) | 2018-02-02 |
| US11766448B2 (en) | 2023-09-26 |
| JP6224459B2 (ja) | 2017-11-01 |
| US9884071B2 (en) | 2018-02-06 |
| US20240082288A1 (en) | 2024-03-14 |
| SG192268A1 (en) | 2013-08-30 |
| US20180185405A1 (en) | 2018-07-05 |
| SG10202007803QA (en) | 2020-09-29 |
| CA2826316C (en) | 2021-04-27 |
| EP2675902B1 (en) | 2019-03-27 |
| JP2014507154A (ja) | 2014-03-27 |
| ES2724800T3 (es) | 2019-09-16 |
| CN103502458A (zh) | 2014-01-08 |
| AU2012219380A1 (en) | 2013-08-29 |
| CA2826316A1 (en) | 2012-08-23 |
| TR201908020T4 (tr) | 2019-06-21 |
| DK2675902T3 (da) | 2019-06-03 |
| US10918658B2 (en) | 2021-02-16 |
| PL2675902T3 (pl) | 2019-08-30 |
| US20130323226A1 (en) | 2013-12-05 |
| WO2012112832A1 (en) | 2012-08-23 |
| CN103502458B (zh) | 2016-11-16 |
| KR101956885B1 (ko) | 2019-03-12 |
| MX2013009538A (es) | 2013-09-26 |
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