DK2675902T3 - Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel - Google Patents
Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel Download PDFInfo
- Publication number
- DK2675902T3 DK2675902T3 DK12705773.5T DK12705773T DK2675902T3 DK 2675902 T3 DK2675902 T3 DK 2675902T3 DK 12705773 T DK12705773 T DK 12705773T DK 2675902 T3 DK2675902 T3 DK 2675902T3
- Authority
- DK
- Denmark
- Prior art keywords
- aav9
- compositions
- improving
- methods
- tissue specificity
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
Classifications
-
- 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)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161443879P | 2011-02-17 | 2011-02-17 | |
| PCT/US2012/025550 WO2012112832A1 (en) | 2011-02-17 | 2012-02-17 | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2675902T3 true DK2675902T3 (da) | 2019-06-03 |
Family
ID=45757234
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12705773.5T DK2675902T3 (da) | 2011-02-17 | 2012-02-17 | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
Country Status (16)
| Country | Link |
|---|---|
| US (5) | US9884071B2 (enExample) |
| EP (1) | EP2675902B1 (enExample) |
| JP (1) | JP6224459B2 (enExample) |
| KR (1) | KR101956885B1 (enExample) |
| CN (1) | CN103502458B (enExample) |
| AU (1) | AU2012219380B2 (enExample) |
| BR (1) | BR112013020734A2 (enExample) |
| CA (1) | CA2826316C (enExample) |
| DK (1) | DK2675902T3 (enExample) |
| ES (1) | ES2724800T3 (enExample) |
| HU (1) | HUE043921T2 (enExample) |
| MX (1) | MX353930B (enExample) |
| PL (1) | PL2675902T3 (enExample) |
| SG (3) | SG192268A1 (enExample) |
| TR (1) | TR201908020T4 (enExample) |
| WO (1) | WO2012112832A1 (enExample) |
Families Citing this family (123)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3211085B1 (en) * | 2003-09-30 | 2021-03-31 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| 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 |
| CA2833912C (en) | 2010-04-23 | 2021-09-21 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| ES2605305T3 (es) | 2010-04-23 | 2017-03-13 | University Of Massachusetts | Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos |
| CA2826273C (en) | 2011-02-10 | 2021-11-02 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| JP6224459B2 (ja) | 2011-02-17 | 2017-11-01 | 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 | 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法 |
| 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 |
| EP2847337A4 (en) * | 2012-05-09 | 2016-04-27 | Univ Oregon Health & Science | ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS |
| 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 |
| CA2905952A1 (en) * | 2013-03-13 | 2014-10-02 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
| CN110885854A (zh) * | 2013-03-15 | 2020-03-17 | 北卡罗来纳-查佩尔山大学 | 双重聚糖结合aav载体的方法和组合物 |
| PL2984166T3 (pl) | 2013-03-15 | 2021-01-11 | The Trustees Of The University Of Pennsylvania | Kompozycje do leczenia mpsi |
| CN115252823A (zh) | 2014-03-21 | 2022-11-01 | 建新公司 | 用于色素性视网膜炎的基因疗法 |
| 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 |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| PL4012035T3 (pl) | 2014-09-16 | 2025-03-24 | Genzyme Corporation | Wektory wirusowe związane z adenowirusami do leczenia jaskry miocylinowej (myoc) |
| EP4012035B1 (en) | 2014-09-16 | 2024-11-06 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | 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 |
| CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| IL284949B2 (en) | 2014-11-14 | 2024-06-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| IL296391B2 (en) | 2015-01-20 | 2024-06-01 | Genzyme Corp | Analytical ultracentrifugation for characterization of recombinant viral particles |
| WO2016130591A2 (en) | 2015-02-10 | 2016-08-18 | Genzyme Corporation | Enhanced delivery of viral particles to the striatum and cortex |
| JP2018506304A (ja) | 2015-02-10 | 2018-03-08 | ジェンザイム・コーポレーション | バリアントＲＮＡｉ |
| US10584321B2 (en) | 2015-02-13 | 2020-03-10 | 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 |
| US11046955B2 (en) | 2015-04-24 | 2021-06-29 | University Of Massachusetts | Modified AAV constructs and uses thereof |
| CA2986021A1 (en) | 2015-05-16 | 2016-11-24 | Genzyme Corporation | Gene editing of deep intronic mutations |
| 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 |
| WO2017070516A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| EP3364997B1 (en) | 2015-10-22 | 2024-01-17 | University of Massachusetts | Aspartoacylase gene therapy in the treatment of canavan disease |
| 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 |
| WO2017100704A1 (en) | 2015-12-11 | 2017-06-15 | 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 |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| AU2016372035B2 (en) | 2015-12-15 | 2023-03-30 | Genzyme Corporation | Adeno-associated viral vectors for treating mucolipidosis type ii |
| WO2017136536A1 (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 |
| SG11201806270XA (en) | 2016-02-03 | 2018-08-30 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis 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 |
| KR20190008237A (ko) | 2016-04-15 | 2019-01-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| RU2725286C2 (ru) | 2016-05-13 | 2020-06-30 | 4Д Молекьюлар Терапьютикс Инк. | Варианты капсидов аденоассоциированного вируса и способы их применения |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| EP3458588B1 (en) | 2016-05-18 | 2025-10-29 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | 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 |
| US11698377B2 (en) | 2016-08-15 | 2023-07-11 | Genzyme Corporation | Methods for detecting AAV |
| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| KR20190075964A (ko) | 2016-10-13 | 2019-07-01 | 유니버시티 오브 매사추세츠 | Aav 캡시드 설계 |
| 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) |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| KR20200053468A (ko) | 2017-07-06 | 2020-05-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | I형 점액다당류증을 치료하기 위한 유전자 요법 |
| AU2018302016A1 (en) | 2017-07-17 | 2020-02-06 | The Regents Of The University Of California | Trajectory array guide system |
| MX2020003176A (es) | 2017-09-20 | 2020-08-20 | 4D Molecular Therapeutics Inc | Variantes de capsides de virus adenoasociados y metodos de uso de estas. |
| AR113134A1 (es) | 2017-09-22 | 2020-01-29 | Genzyme Corp | Arni variante |
| WO2019060662A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF 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 |
| CA3083472A1 (en) | 2017-11-27 | 2019-05-31 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and use for inhibiting angiogenesis |
| PE20210341A1 (es) * | 2018-02-01 | 2021-02-23 | Homology Medicines Inc | Composiciones de virus adeno-asociadas para la transferencia de genes pah y metodos de uso de los mismos |
| PE20212076A1 (es) | 2018-02-01 | 2021-10-26 | Homology Medicines Inc | Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| 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 |
| EP3775173A1 (en) | 2018-04-05 | 2021-02-17 | Genethon | Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism |
| WO2019204303A2 (en) | 2018-04-16 | 2019-10-24 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating duchenne muscular dystrophy |
| WO2019213668A1 (en) * | 2018-05-04 | 2019-11-07 | Oregon Health & Science University | Human anti-aav2 capsid polyclonal antibody epitopes |
| MA52631A (fr) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
| US12163129B2 (en) | 2018-06-08 | 2024-12-10 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| JP2021530548A (ja) | 2018-07-24 | 2021-11-11 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. | 遺伝子治療製剤を生産するための系および方法 |
| PH12021550254A1 (en) | 2018-08-03 | 2023-02-06 | Genzyme Corp | Variant rnai against alpha-synuclein |
| JP7528066B2 (ja) | 2018-09-28 | 2024-08-05 | ボイジャー セラピューティクス インコーポレイテッド | 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 |
| EP3861113A1 (en) | 2018-10-04 | 2021-08-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| CA3115248A1 (en) | 2018-10-05 | 2020-04-09 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| EP3863662A1 (en) | 2018-10-12 | 2021-08-18 | Genzyme Corporation | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
| UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
| TW202028468A (zh) | 2018-10-15 | 2020-08-01 | 美商航海家醫療公司 | 用於桿狀病毒/Sf9系統中rAAV之大規模生產的表現載體 |
| EP3917566A4 (en) | 2019-01-31 | 2022-10-26 | Oregon Health & Science University | METHODS OF USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS |
| WO2020174369A2 (en) | 2019-02-25 | 2020-09-03 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
| JP2022521025A (ja) | 2019-02-25 | 2022-04-04 | 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 |
| CN113557010A (zh) | 2019-02-28 | 2021-10-26 | 瑞泽恩制药公司 | 用于递送治疗剂的腺相关病毒载体 |
| US12398378B2 (en) | 2019-04-12 | 2025-08-26 | University Of Massachusetts | AAV-mediated gene therapy for maple syrup urine disease (MSUD) |
| AU2020278960A1 (en) * | 2019-05-17 | 2021-12-23 | Research Institute At Nationwide Children's Hospital | Optimized gene therapy targeting retinal cells |
| 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 |
| 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 |
| AU2020367532A1 (en) * | 2019-10-17 | 2022-05-12 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick disease type C |
| CA3159620A1 (en) * | 2019-11-01 | 2021-05-06 | 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 |
| MX2023003805A (es) | 2020-10-01 | 2023-04-12 | Genzyme Corp | Casete de expresion de pah humana para el tratamiento de la pku mediante terapia de reemplazo genico dirigida al higado. |
| US20240091380A1 (en) | 2021-02-01 | 2024-03-21 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2023010135A1 (en) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| EP4377459A2 (en) | 2021-07-30 | 2024-06-05 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin (fxn) |
| US20250144243A1 (en) * | 2022-01-25 | 2025-05-08 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
| KR20250007619A (ko) | 2022-05-06 | 2025-01-14 | 노파르티스 아게 | 신규 재조합 aav vp2 융합 폴리펩티드 |
| 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 |
| TW202509211A (zh) | 2023-04-26 | 2025-03-01 | 法商賽諾菲公司 | 宿主細胞系以及鑑定和使用該等宿主細胞系之方法 |
| US20250197862A1 (en) | 2023-12-15 | 2025-06-19 | Genzyme Corporation | ARTIFICIAL microRNAs TARGETING TAU |
| WO2025160434A1 (en) | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting huntington's disease |
| WO2025160429A1 (en) | 2024-01-26 | 2025-07-31 | Genzyme Corporation | Artificial micrornas targeting snca |
| 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 |
| WO2026011008A1 (en) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Expression control by skeletal muscle-expressed mirnas |
| WO2026011009A1 (en) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
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 |
| JPH07106156B2 (ja) | 1983-10-28 | 1995-11-15 | ジェネティックス、インスティチュ−ト | ファクタ−▲ｖｉｉｉ▼および関連生産物の製造 |
| US4757006A (en) | 1983-10-28 | 1988-07-12 | Genetics Institute, Inc. | Human factor VIII:C gene and recombinant methods for production |
| US5045455A (en) | 1984-01-12 | 1991-09-03 | Chiron Corporation | Factor VIII:C cDNA cloning and expression |
| US4965199A (en) | 1984-04-20 | 1990-10-23 | Genentech, Inc. | Preparation of functional human factor VIII in mammalian cells using methotrexate based selection |
| FI86885C (fi) | 1984-04-20 | 1992-10-26 | Genentech Inc | Foerfarande foer framstaellning av human rekombinantfaktor viii och nukleinsyrasekvenser och vektorer anvaend daertill |
| EP0182448A3 (en) | 1984-08-24 | 1987-10-28 | Genetics Institute, Inc. | Production of factor viii and related products |
| FI98829C (fi) | 1986-01-27 | 1997-08-25 | Chiron Corp | Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta |
| US5595886A (en) | 1986-01-27 | 1997-01-21 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
| US5422260A (en) | 1986-05-29 | 1995-06-06 | Genetics Institute, Inc. -Legal Affairs | Human factor VIII:c muteins |
| US5451521A (en) | 1986-05-29 | 1995-09-19 | Genetics Institute, Inc. | Procoagulant proteins |
| 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 |
| EP0670332A3 (en) | 1989-11-17 | 1995-10-25 | Novo Nordisk As | Protein complexes with factor VIII: C activity and their production. |
| SE465222C5 (sv) | 1989-12-15 | 1998-02-10 | Pharmacia & Upjohn Ab | Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning |
| 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 |
| 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 |
| 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 |
| AU5602394A (en) | 1992-11-13 | 1994-06-08 | Duke University | Chimeric blood coagulation proteins |
| US5563045A (en) | 1992-11-13 | 1996-10-08 | Genetics Institute, Inc. | Chimeric procoagulant 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 |
| WO1997003195A1 (en) | 1995-07-11 | 1997-01-30 | Chiron Corporation | Novel factor viii:c polypeptide analogs with altered protease sites |
| AU722624B2 (en) | 1996-09-06 | 2000-08-10 | Trustees Of The University Of Pennsylvania, The | An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase |
| US6096273A (en) | 1996-11-05 | 2000-08-01 | Clinical Micro Sensors | Electrodes linked via conductive oligomers to nucleic acids |
| US6074884A (en) | 1997-10-09 | 2000-06-13 | Coulter International Corp. | Stable protein-nickel particles and methods of production and use thereof |
| US5945293A (en) | 1997-10-09 | 1999-08-31 | Coulter International Corp. | Protein-colloidal metal-aminodextran coated particle and methods of preparation and use |
| 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 |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| EP3211085B1 (en) | 2003-09-30 | 2021-03-31 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| 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 |
| HUE044522T2 (hu) | 2009-04-30 | 2019-10-28 | Univ Pennsylvania | Adeno-asszociált vírus konstrukciókat tartalmazó légúti vezetõ sejtek célzására szolgáló kompozíciók |
| 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 |
| CA2826273C (en) | 2011-02-10 | 2021-11-02 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| JP6224459B2 (ja) | 2011-02-17 | 2017-11-01 | 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 | 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法 |
-
2012
- 2012-02-17 JP JP2013554622A patent/JP6224459B2/ja not_active Expired - Fee Related
- 2012-02-17 HU HUE12705773A patent/HUE043921T2/hu unknown
- 2012-02-17 PL PL12705773T patent/PL2675902T3/pl unknown
- 2012-02-17 SG SG2013058920A patent/SG192268A1/en unknown
- 2012-02-17 MX MX2013009538A patent/MX353930B/es active IP Right Grant
- 2012-02-17 SG SG10202007803QA patent/SG10202007803QA/en unknown
- 2012-02-17 TR TR2019/08020T patent/TR201908020T4/tr unknown
- 2012-02-17 KR KR1020137023589A patent/KR101956885B1/ko not_active Expired - Fee Related
- 2012-02-17 ES ES12705773T patent/ES2724800T3/es active Active
- 2012-02-17 BR BR112013020734A patent/BR112013020734A2/pt not_active IP Right Cessation
- 2012-02-17 WO PCT/US2012/025550 patent/WO2012112832A1/en not_active Ceased
- 2012-02-17 SG SG10201601110VA patent/SG10201601110VA/en unknown
- 2012-02-17 DK DK12705773.5T patent/DK2675902T3/da active
- 2012-02-17 US US13/985,630 patent/US9884071B2/en active Active
- 2012-02-17 EP EP12705773.5A patent/EP2675902B1/en active Active
- 2012-02-17 AU AU2012219380A patent/AU2012219380B2/en not_active Ceased
- 2012-02-17 CN CN201280008862.8A patent/CN103502458B/zh not_active Expired - Fee Related
- 2012-02-17 CA CA2826316A patent/CA2826316C/en active Active
-
2018
- 2018-01-05 US US15/862,999 patent/US10406173B2/en active Active
-
2019
- 2019-09-09 US US16/564,537 patent/US10918658B2/en not_active Expired - Fee Related
-
2021
- 2021-01-27 US US17/159,735 patent/US11766448B2/en active Active
-
2023
- 2023-08-15 US US18/450,130 patent/US20240082288A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| JP2014507154A (ja) | 2014-03-27 |
| US20240082288A1 (en) | 2024-03-14 |
| EP2675902B1 (en) | 2019-03-27 |
| EP2675902A1 (en) | 2013-12-25 |
| CA2826316C (en) | 2021-04-27 |
| KR101956885B1 (ko) | 2019-03-12 |
| CA2826316A1 (en) | 2012-08-23 |
| HUE043921T2 (hu) | 2019-09-30 |
| AU2012219380B2 (en) | 2017-04-13 |
| SG10201601110VA (en) | 2016-03-30 |
| US11766448B2 (en) | 2023-09-26 |
| US10406173B2 (en) | 2019-09-10 |
| JP6224459B2 (ja) | 2017-11-01 |
| MX2013009538A (es) | 2013-09-26 |
| US20180185405A1 (en) | 2018-07-05 |
| US20130323226A1 (en) | 2013-12-05 |
| US20200101099A1 (en) | 2020-04-02 |
| ES2724800T3 (es) | 2019-09-16 |
| PL2675902T3 (pl) | 2019-08-30 |
| CN103502458B (zh) | 2016-11-16 |
| SG10202007803QA (en) | 2020-09-29 |
| WO2012112832A1 (en) | 2012-08-23 |
| TR201908020T4 (tr) | 2019-06-21 |
| BR112013020734A2 (pt) | 2017-06-13 |
| MX353930B (es) | 2018-02-02 |
| US20210145857A1 (en) | 2021-05-20 |
| AU2012219380A1 (en) | 2013-08-29 |
| SG192268A1 (en) | 2013-08-30 |
| US10918658B2 (en) | 2021-02-16 |
| KR20140046407A (ko) | 2014-04-18 |
| US9884071B2 (en) | 2018-02-06 |
| CN103502458A (zh) | 2014-01-08 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| DK2675902T3 (da) | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel | |
| IL280819A (en) | Adeno-associated virus virions with variant capsid and methods of use thereof | |
| DK2914741T3 (da) | Hidtil ukendte sammensætninger og fremgangsmåder til forbedring af PCR-specificitet | |
| ME03766B (me) | Određeni amino-pirimidini, njihove kompozicije, i metode njihove upotrebe | |
| DK3366143T3 (da) | Probiotiske sammensætninger og fremgangsmåder | |
| DK3213738T3 (da) | Sammensætninger og fremgangsmåder til målrettet termomodulering | |
| PL2640375T3 (pl) | Sposób leczenia nefropatii pokontrastowej | |
| DK2688557T3 (da) | Fremgangsmåder og sammensætninger til behandling af forstyrelse af opmærksomhed | |
| EP2714970A4 (en) | Enzyme QUANTIFICATION | |
| DK3333188T3 (da) | Anti-NGF-antistoffer og deres anvendelse | |
| DK3495392T3 (da) | Anti-cgrp-sammensætninger og anvendelse deraf | |
| DK2898061T3 (da) | Probiotiske sammensætninger til behandlingen af fedme og fedme-relaterede tilstande | |
| DK3795573T3 (da) | Bestemte triazolopyraziner, sammensætninger deraf og fremgangsmåder til anvendelse deraf | |
| DK2929031T5 (da) | Pcsk9-irna-sammensætninger og fremgangsmåder til anvendelse deraf | |
| ME03475B (me) | Antiparazitske kompozicije dihidroazola | |
| BR112012016793A2 (pt) | compostos tipo fumagilol e métodos de fazer e usar os mesmos | |
| HUE038759T2 (hu) | Terápiás nukleázkészítmények és eljárások | |
| EP2641343A4 (en) | CONFIGURATION OF A BUILDING | |
| BR112014007029A2 (pt) | métodos aperfeiçoados de terapia gênica | |
| DK2663864T3 (da) | Fremgangsmåde til at vurdere immundiversitet og anvendelse af denne | |
| DK2661274T3 (da) | Sammensætninger og fremgangsmåder med aloepolysaccharider | |
| BR112014006291A2 (pt) | composição e utilização da composição | |
| DK2793895T3 (da) | Fremgangsmåder og sammensætninger til reducering af kropsfedt og adipocytter | |
| DK2408794T3 (da) | Isolering og oprensning af vallebestanddele | |
| DK2600715T3 (da) | Flydende ST-246-formuleringer og fremgangsmåder |