SG10202007803QA - Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer - Google Patents
Compositions and methods for altering tissue specificity and improving aav9-mediated gene transferInfo
- Publication number
- SG10202007803QA SG10202007803QA SG10202007803QA SG10202007803QA SG10202007803QA SG 10202007803Q A SG10202007803Q A SG 10202007803QA SG 10202007803Q A SG10202007803Q A SG 10202007803QA SG 10202007803Q A SG10202007803Q A SG 10202007803QA SG 10202007803Q A SG10202007803Q A SG 10202007803QA
- Authority
- SG
- Singapore
- Prior art keywords
- aav9
- compositions
- improving
- methods
- gene transfer
- Prior art date
Links
- 230000001404 mediated effect Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
Classifications
-
- 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
- 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
- A61K31/66—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)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (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 |
|---|---|
| SG10202007803QA true SG10202007803QA (en) | 2020-09-29 |
Family
ID=45757234
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG10202007803QA SG10202007803QA (en) | 2011-02-17 | 2012-02-17 | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| SG2013058920A SG192268A1 (en) | 2011-02-17 | 2012-02-17 | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| SG10201601110VA SG10201601110VA (en) | 2011-02-17 | 2012-02-17 | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG2013058920A SG192268A1 (en) | 2011-02-17 | 2012-02-17 | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| SG10201601110VA SG10201601110VA (en) | 2011-02-17 | 2012-02-17 | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
Country Status (16)
| Country | Link |
|---|---|
| US (5) | US9884071B2 (https=) |
| EP (1) | EP2675902B1 (https=) |
| JP (1) | JP6224459B2 (https=) |
| KR (1) | KR101956885B1 (https=) |
| CN (1) | CN103502458B (https=) |
| AU (1) | AU2012219380B2 (https=) |
| BR (1) | BR112013020734A2 (https=) |
| CA (1) | CA2826316C (https=) |
| DK (1) | DK2675902T3 (https=) |
| ES (1) | ES2724800T3 (https=) |
| HU (1) | HUE043921T2 (https=) |
| MX (1) | MX353930B (https=) |
| PL (1) | PL2675902T3 (https=) |
| SG (3) | SG10202007803QA (https=) |
| TR (1) | TR201908020T4 (https=) |
| WO (1) | WO2012112832A1 (https=) |
Families Citing this family (126)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2345731B1 (en) * | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| 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 |
| EP3444346B1 (en) | 2010-04-23 | 2022-07-27 | University of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | 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 |
| TR201908020T4 (tr) | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| EP3318635A1 (en) | 2011-04-21 | 2018-05-09 | University of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | 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 |
| AU2014235096B2 (en) | 2013-03-15 | 2020-03-19 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| ES2926774T3 (es) * | 2013-03-15 | 2022-10-28 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores AAV de doble unión de glicano |
| PL3628334T3 (pl) | 2014-03-21 | 2023-12-18 | Genzyme Corporation | Terapia genowa w retinopatii barwnikowej |
| WO2015164757A1 (en) * | 2014-04-25 | 2015-10-29 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | 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 |
| EP3200830B1 (en) | 2014-10-03 | 2020-09-09 | 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 |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| EA201791630A1 (ru) | 2015-01-20 | 2018-04-30 | Джензим Корпорейшн | Аналитическое ультрацентрифугирование для определения характеристик рекомбинантных вирусных частиц |
| CR20170406A (es) | 2015-02-10 | 2017-11-14 | Genzyme Corp | Arni variante |
| CN107530447A (zh) | 2015-02-10 | 2018-01-02 | 建新公司 | 病毒颗粒至纹状体和皮质的增强递送 |
| EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | 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 |
| BR112017024514A2 (pt) | 2015-05-16 | 2018-07-24 | Genzyme Corporation | edição genética de mutações intrônicas profundas |
| 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 |
| EP3384035A4 (en) | 2015-12-02 | 2019-08-07 | Voyager Therapeutics, Inc. | ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| MX2018007330A (es) | 2015-12-15 | 2019-03-14 | Genzyme Corp | Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. |
| 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 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| CA3012344A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| US11207426B2 (en) | 2016-04-05 | 2021-12-28 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A 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 |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| 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 |
| CA3033856A1 (en) | 2016-08-15 | 2018-02-22 | Genzyme Corporation | Methods for detecting aav |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| WO2018119330A2 (en) * | 2016-12-22 | 2018-06-28 | Oregon Health & Science University | Adeno associated viral vectors |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CA3068328A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| CA3070087A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| RU2770922C2 (ru) | 2017-09-20 | 2022-04-25 | 4Д Молекьюлар Терапьютикс Инк. | Капсиды вариантов аденоассоциированных вирусов и методы их применения |
| CN111356763B (zh) | 2017-09-22 | 2024-03-12 | 建新公司 | 变体RNAi |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| 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 |
| CN111770999A (zh) | 2017-11-27 | 2020-10-13 | 4D分子治疗有限公司 | 腺相关病毒变体衣壳和用于抑制血管生成的应用 |
| KR20230065367A (ko) * | 2018-02-01 | 2023-05-11 | 호몰로지 메디슨, 인크. | Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| MA51353B1 (fr) | 2018-04-05 | 2022-09-30 | Univ Sorbonne | Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit |
| US12460226B2 (en) | 2018-04-16 | 2025-11-04 | 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 |
| KR20210019996A (ko) | 2018-05-15 | 2021-02-23 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 조성물 및 방법 |
| WO2019236995A1 (en) | 2018-06-08 | 2019-12-12 | 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 |
| 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 |
| KR102931619B1 (ko) | 2018-08-03 | 2026-02-26 | 젠자임 코포레이션 | 알파-시뉴클레인에 대한 변이형 RNAi |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020072849A1 (en) | 2018-10-04 | 2020-04-09 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| WO2020072844A1 (en) | 2018-10-05 | 2020-04-09 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| AU2019357039B2 (en) | 2018-10-12 | 2025-10-09 | 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 |
| CN113166781A (zh) | 2018-10-15 | 2021-07-23 | 沃雅戈治疗公司 | 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体 |
| WO2020160508A1 (en) * | 2019-01-31 | 2020-08-06 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
| US20220154211A1 (en) | 2019-02-25 | 2022-05-19 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
| SG11202108044YA (en) | 2019-02-25 | 2021-09-29 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
| US20220047618A1 (en) | 2019-02-28 | 2022-02-17 | Regeneron Pharmaceuticals, Inc. | Adeno-associated virus vectors for the delivery of therapeutics |
| 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 |
| US20220233655A1 (en) * | 2019-05-17 | 2022-07-28 | Research Institute At Nationwide Children's Hospital | Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme |
| 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 |
| JP2022551739A (ja) * | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| WO2021087361A1 (en) * | 2019-11-01 | 2021-05-06 | Sangamo Therapeutics, Inc. | Zinc finger nuclease variants for treating or preventing lysosomal storage diseases |
| 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 |
| US12064894B2 (en) | 2020-09-04 | 2024-08-20 | 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. |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| US20240252684A1 (en) | 2021-07-30 | 2024-08-01 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| CA3227103A1 (en) | 2021-07-30 | 2023-02-02 | Matthew P. GEMBERLING | Compositions and methods for modulating expression of frataxin (fxn) |
| CN119317718A (zh) * | 2022-01-25 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于改善心脏转导和肝脏去靶向的aav衣壳 |
| US20250353883A1 (en) | 2022-05-06 | 2025-11-20 | Novartis Ag | Novel recombinant aav vp2 fusion polypeptides |
| CA3261865A1 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS |
| WO2024163678A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| 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) |
| CN121586778A (zh) | 2023-04-26 | 2026-02-27 | 赛诺菲 | 宿主细胞系以及用于鉴定和使用此类宿主细胞系的方法 |
| 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 |
| US20250276095A1 (en) | 2024-03-04 | 2025-09-04 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy |
| WO2025226842A1 (en) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Expression control by drg-expressed mirnas |
| WO2025226841A1 (en) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Gene therapy approach for treating disorders associated with tnnt2 |
| 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 |
| WO2026030242A1 (en) | 2024-07-29 | 2026-02-05 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy |
| WO2026030244A1 (en) | 2024-07-30 | 2026-02-05 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a |
| WO2026050402A1 (en) | 2024-08-29 | 2026-03-05 | Kate Therapeutics, Inc. | Liver de-targeted muscle tropic capsids |
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 |
| 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 |
| KR960701899A (ko) | 1989-11-17 | 1996-03-28 | 안네 제케르 | 인자 viii:c활성을 갖는 단백질 복합체 및 그의 제조 |
| 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 |
| SE468050C (sv) | 1991-03-15 | 1998-04-27 | Pharmacia & Upjohn Ab | Rekombinant derivat av human faktor VIII |
| US5661008A (en) | 1991-03-15 | 1997-08-26 | Kabi Pharmacia Ab | Recombinant human factor VIII derivatives |
| 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 |
| WO1994011013A1 (en) | 1992-11-13 | 1994-05-26 | Duke University | Chimeric blood coagulation proteins |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| ES2226029T3 (es) | 1993-06-10 | 2005-03-16 | Bayer Corporation | Vector y linea celular de mamifero con productividad mejorada. |
| JPH10507997A (ja) | 1993-06-11 | 1998-08-04 | クールター コーポレイション | Ｔ細胞の活性化および増殖を誘導するための抗ｃｄ３抗体−アミノデキストラン結合体 |
| 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 |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | 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 |
| EP1048723B1 (en) | 1998-09-09 | 2004-04-14 | 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 |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| 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 |
| EP2425000B1 (en) * | 2009-04-30 | 2019-02-20 | 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 |
| TR201908020T4 (tr) | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
-
2012
- 2012-02-17 TR TR2019/08020T patent/TR201908020T4/tr unknown
- 2012-02-17 WO PCT/US2012/025550 patent/WO2012112832A1/en not_active Ceased
- 2012-02-17 PL PL12705773T patent/PL2675902T3/pl unknown
- 2012-02-17 JP JP2013554622A patent/JP6224459B2/ja not_active Expired - Fee Related
- 2012-02-17 KR KR1020137023589A patent/KR101956885B1/ko not_active Expired - Fee Related
- 2012-02-17 AU AU2012219380A patent/AU2012219380B2/en not_active Ceased
- 2012-02-17 BR BR112013020734A patent/BR112013020734A2/pt not_active IP Right Cessation
- 2012-02-17 HU HUE12705773A patent/HUE043921T2/hu unknown
- 2012-02-17 CN CN201280008862.8A patent/CN103502458B/zh not_active Expired - Fee Related
- 2012-02-17 SG SG10202007803QA patent/SG10202007803QA/en unknown
- 2012-02-17 SG SG2013058920A patent/SG192268A1/en unknown
- 2012-02-17 ES ES12705773T patent/ES2724800T3/es active Active
- 2012-02-17 CA CA2826316A patent/CA2826316C/en active Active
- 2012-02-17 SG SG10201601110VA patent/SG10201601110VA/en unknown
- 2012-02-17 DK DK12705773.5T patent/DK2675902T3/da active
- 2012-02-17 EP EP12705773.5A patent/EP2675902B1/en active Active
- 2012-02-17 US US13/985,630 patent/US9884071B2/en not_active Expired - Fee Related
- 2012-02-17 MX MX2013009538A patent/MX353930B/es active IP Right Grant
-
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 |
|---|---|
| US20210145857A1 (en) | 2021-05-20 |
| PL2675902T3 (pl) | 2019-08-30 |
| JP6224459B2 (ja) | 2017-11-01 |
| HUE043921T2 (hu) | 2019-09-30 |
| CA2826316C (en) | 2021-04-27 |
| KR20140046407A (ko) | 2014-04-18 |
| US10918658B2 (en) | 2021-02-16 |
| US11766448B2 (en) | 2023-09-26 |
| EP2675902B1 (en) | 2019-03-27 |
| CA2826316A1 (en) | 2012-08-23 |
| US10406173B2 (en) | 2019-09-10 |
| KR101956885B1 (ko) | 2019-03-12 |
| ES2724800T3 (es) | 2019-09-16 |
| WO2012112832A1 (en) | 2012-08-23 |
| US20240082288A1 (en) | 2024-03-14 |
| EP2675902A1 (en) | 2013-12-25 |
| AU2012219380B2 (en) | 2017-04-13 |
| CN103502458B (zh) | 2016-11-16 |
| SG10201601110VA (en) | 2016-03-30 |
| JP2014507154A (ja) | 2014-03-27 |
| US20130323226A1 (en) | 2013-12-05 |
| BR112013020734A2 (pt) | 2017-06-13 |
| DK2675902T3 (da) | 2019-06-03 |
| SG192268A1 (en) | 2013-08-30 |
| AU2012219380A1 (en) | 2013-08-29 |
| TR201908020T4 (tr) | 2019-06-21 |
| MX2013009538A (es) | 2013-09-26 |
| US20200101099A1 (en) | 2020-04-02 |
| US9884071B2 (en) | 2018-02-06 |
| CN103502458A (zh) | 2014-01-08 |
| US20180185405A1 (en) | 2018-07-05 |
| MX353930B (es) | 2018-02-02 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| SG10202007803QA (en) | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer | |
| AP3907A (en) | 3-Pyrimidin-4-yl-oxazolidin-2-ones as inhibitors of mutant IDH | |
| PH12013501969A1 (en) | Compositions and methods for inhibiting expression of tmprss6 gene | |
| IL235102A0 (en) | Compositions and methods for efficient gene transfer using aav caspid variants | |
| IL224390B (en) | Compositions and methods for targeted thermal modulation | |
| EP2785837A4 (en) | TARGETED ENZYMATIC COMPOUNDS AND ITS USES | |
| EP2673017A4 (en) | DEVICES AND METHOD FOR TISSUE TRANSMISSION | |
| EP2462230A4 (en) | METHOD AND COMPOSITIONS FOR TARGETED GENE MODIFICATION | |
| PT3366143T (pt) | Composições e métodos probióticos | |
| EP2705107A4 (en) | METHODS AND COMPOSITIONS FOR HEAT TRANSFER | |
| SG10201601023UA (en) | Inhibitors of nedd8-activating enzyme | |
| GB201120550D0 (en) | Photobioreactor | |
| ZA201207226B (en) | Compositions and methods for treating viral diseases | |
| HUE050699T2 (hu) | Készítmények és eljárások Spirocheták tenyésztésére | |
| EP2768941A4 (en) | photobioreactor | |
| GB201114017D0 (en) | Inhibitors against endosomal/ysosomal enzymes | |
| IL231490A0 (en) | Preparations and methods for the production and use of human cholinesterases | |
| SG11201402899TA (en) | Methods and compositions for treating viral diseases | |
| EP2747765A4 (en) | COMPOSITIONS AND METHODS FOR THE TREATMENT OF VIRAL DISEASES | |
| GB201110658D0 (en) | Improved process and compositions | |
| GB201114751D0 (en) | Enzyme inhibition | |
| GB201114748D0 (en) | Enzyme inhibition |