CL2023002764A1 - Novedosas proteínas de la cápside del virus adenoasociado - Google Patents
Novedosas proteínas de la cápside del virus adenoasociadoInfo
- Publication number
- CL2023002764A1 CL2023002764A1 CL2023002764A CL2023002764A CL2023002764A1 CL 2023002764 A1 CL2023002764 A1 CL 2023002764A1 CL 2023002764 A CL2023002764 A CL 2023002764A CL 2023002764 A CL2023002764 A CL 2023002764A CL 2023002764 A1 CL2023002764 A1 CL 2023002764A1
- Authority
- CL
- Chile
- Prior art keywords
- capsid proteins
- associated virus
- novel
- virus capsid
- novel adeno
- Prior art date
Links
- 108090000565 Capsid Proteins Proteins 0.000 title abstract 6
- 102100023321 Ceruloplasmin Human genes 0.000 title abstract 6
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 239000013607 AAV vector Substances 0.000 abstract 2
- 239000002245 particle Substances 0.000 abstract 1
- 102000040430 polynucleotide Human genes 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 239000002157 polynucleotide Substances 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Toxicology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Abstract
La invención se refiere a novedosas proteínas de la cápside del virus adenoasociado (AAV), partículas de AAV que comprenden una novedosa proteína de la cápside, polinucleótidos que codifican estas proteínas de la cápside y vectores de AAV que expresan estas proteínas de la cápside. La invención también se refiere a métodos para elaborar los vectores de AAV descritos en la presente que expresan las novedosas proteínas de la cápside de la invención y sus usos terapéuticos asociados.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662366838P | 2016-07-26 | 2016-07-26 |
Publications (1)
Publication Number | Publication Date |
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CL2023002764A1 true CL2023002764A1 (es) | 2024-04-05 |
Family
ID=59558468
Family Applications (6)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2019000167A CL2019000167A1 (es) | 2016-07-26 | 2019-01-22 | Novedosas proteínas de la cápside del virus adenoasociado. |
CL2020003176A CL2020003176A1 (es) | 2016-07-26 | 2020-12-04 | Novedosas proteínas de la cápside del virus adenoasociado (divisional sol. 201900167). |
CL2023000165A CL2023000165A1 (es) | 2016-07-26 | 2023-01-18 | Novedosas proteínas de la cápside del virus adenoasociado |
CL2023002764A CL2023002764A1 (es) | 2016-07-26 | 2023-09-15 | Novedosas proteínas de la cápside del virus adenoasociado |
CL2023003001A CL2023003001A1 (es) | 2016-07-26 | 2023-10-06 | Novedosas proteínas de la cápside del virus adenoasociado |
CL2023003010A CL2023003010A1 (es) | 2016-07-26 | 2023-10-06 | Novedosas proteínas de la cápside del virus adenoasociado |
Family Applications Before (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2019000167A CL2019000167A1 (es) | 2016-07-26 | 2019-01-22 | Novedosas proteínas de la cápside del virus adenoasociado. |
CL2020003176A CL2020003176A1 (es) | 2016-07-26 | 2020-12-04 | Novedosas proteínas de la cápside del virus adenoasociado (divisional sol. 201900167). |
CL2023000165A CL2023000165A1 (es) | 2016-07-26 | 2023-01-18 | Novedosas proteínas de la cápside del virus adenoasociado |
Family Applications After (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2023003001A CL2023003001A1 (es) | 2016-07-26 | 2023-10-06 | Novedosas proteínas de la cápside del virus adenoasociado |
CL2023003010A CL2023003010A1 (es) | 2016-07-26 | 2023-10-06 | Novedosas proteínas de la cápside del virus adenoasociado |
Country Status (15)
Country | Link |
---|---|
US (2) | US11584780B2 (es) |
EP (1) | EP3491008A2 (es) |
JP (4) | JP6994018B2 (es) |
KR (2) | KR20230161535A (es) |
CN (1) | CN109715650B (es) |
AU (2) | AU2017301600B2 (es) |
BR (1) | BR112019001532A2 (es) |
CA (1) | CA3031026A1 (es) |
CL (6) | CL2019000167A1 (es) |
IL (2) | IL263801B2 (es) |
MX (1) | MX2019000962A (es) |
PE (1) | PE20190401A1 (es) |
PH (1) | PH12019500068A1 (es) |
SG (1) | SG11201811603WA (es) |
WO (1) | WO2018022608A2 (es) |
Families Citing this family (41)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
SG10202107733QA (en) | 2015-09-28 | 2021-09-29 | Univ North Carolina Chapel Hill | Methods and compositions for antibody-evading virus vectors |
JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
BR112020017278A2 (pt) * | 2018-02-27 | 2020-12-22 | The Trustees Of The University Of Pennsylvania | Vetores de vírus adenoassociado (aav), vetores de aav tendo desamidação de capsídeos reduzida e usos para os mesmos |
WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
TN2020000187A1 (en) * | 2018-04-05 | 2022-04-04 | Univ Devry Val Dessonne | Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism |
AU2019265560A1 (en) | 2018-05-09 | 2020-11-26 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
KR20210010491A (ko) * | 2018-05-11 | 2021-01-27 | 매사추세츠 아이 앤드 이어 인퍼머리 | 아데노-연관 바이러스의 간-특이적 향성 |
TW202005978A (zh) * | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
JP7451497B2 (ja) * | 2018-08-21 | 2024-03-18 | マサチューセッツ アイ アンド イヤー インファーマリー | アデノ随伴ウイルスの形質導入効率を調節するための組成物および方法 |
EP3890786A4 (en) * | 2018-12-05 | 2022-08-31 | Abeona Therapeutics Inc. | RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR FOR GENE RELEASE |
EP3914229A4 (en) * | 2019-01-23 | 2022-11-02 | University of Florida Research Foundation, Incorporated | HIGHLY EFFICIENT TRANSDUCTION AND LATERAL SPREADING IN THE RETINA BY A NOVEL ENHANCED AAV VIRUS WITH RATIONAL DESIGN |
MX2021011468A (es) | 2019-03-21 | 2021-12-15 | Vectores de virus adenoasociados recombinantes. | |
JP2022527917A (ja) * | 2019-03-28 | 2022-06-07 | ザ ジェネラル ホスピタル コーポレイション | 導入遺伝子発現のための操作されたアデノ随伴(aav)ベクター |
TW202104592A (zh) | 2019-05-14 | 2021-02-01 | 美商拜奧馬林製藥公司 | 重複投予基因療法載體之方法 |
WO2021073568A1 (en) * | 2019-10-16 | 2021-04-22 | Wuxi Apptec (Shanghai) Co., Ltd. | A novel aav variant |
WO2021076925A1 (en) | 2019-10-17 | 2021-04-22 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
EP4058475A1 (en) | 2019-11-14 | 2022-09-21 | BioMarin Pharmaceutical Inc. | Treatment of hereditary angioedema with liver-specific gene therapy vectors |
BR112022016596A2 (pt) | 2020-02-21 | 2022-11-16 | Akouos Inc | Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano |
TW202142554A (zh) * | 2020-02-25 | 2021-11-16 | 澳洲兒童醫學研究所 | 腺相關病毒蛋白殼多肽及載體 |
WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
TW202144575A (zh) | 2020-04-03 | 2021-12-01 | 美商拜奧馬林製藥公司 | 使用aav及治療調配物之苯酮尿症治療 |
AR121954A1 (es) | 2020-04-29 | 2022-07-27 | Bristol Myers Squibb Co | Distrofinas miniaturizadas con dominios de fusión de espectrina y usos de las mismas |
WO2021236908A2 (en) | 2020-05-20 | 2021-11-25 | Biomarin Pharmaceutical Inc. | Use of regulatory proteins for the production of adeno-associated virus |
CN116209768A (zh) * | 2020-07-03 | 2023-06-02 | 吉尼松公司 | 用于通过高变区交换工程化新杂合aav衣壳的方法 |
JP2023544683A (ja) * | 2020-09-10 | 2023-10-25 | ジェネトン | ペプチド改変されたaavカプシド |
CA3195553A1 (en) * | 2020-10-18 | 2022-04-21 | Qiang Wang | Improved adeno-associated virus (aav) vector and uses therefor |
EP4237545A1 (en) | 2020-11-02 | 2023-09-06 | BioMarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
EP4396199A1 (en) | 2021-09-03 | 2024-07-10 | BioMarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
EP4396202A1 (en) | 2021-09-03 | 2024-07-10 | BioMarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034994A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034989A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
TW202332472A (zh) | 2021-10-01 | 2023-08-16 | 美商拜奧馬林製藥公司 | 利用aav基因療法載體進行之遺傳性血管水腫治療及治療性調配物 |
CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
WO2023183623A1 (en) * | 2022-03-25 | 2023-09-28 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
WO2024064863A2 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
WO2024064856A1 (en) | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of cardiomyopathy with aav gene therapy vectors |
CN115925819B (zh) * | 2022-12-30 | 2023-10-13 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
Family Cites Families (71)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DE3485810T2 (de) | 1983-05-27 | 1992-12-10 | Texas A & M University Syst | Verfahren zur herstellung eines rekombinanten baculovirus-expressionsvektors. |
US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
DK518384A (da) | 1984-01-31 | 1985-07-01 | Idaho Res Found | Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse |
US4994371A (en) | 1987-08-28 | 1991-02-19 | Davie Earl W | DNA preparation of Christmas factor and use of DNA sequences |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US6309853B1 (en) | 1994-08-17 | 2001-10-30 | The Rockfeller University | Modulators of body weight, corresponding nucleic acids and proteins, and diagnostic and therapeutic uses thereof |
RU2273645C9 (ru) | 1994-08-17 | 2006-11-27 | Дзе Рокефеллер Юниверсити | Полипептид ожирения (ов)(варианты), его аналог (варианты) и слитый белок (варианты), изолированная молекула нуклеиновой кислоты, молекула днк, рекомбинантный вектор клонирования, рекомбинантный вектор экспрессии, фармацевтическая композиция, моноклональное и поликлональное антитело |
EP0933997A4 (en) | 1996-09-06 | 2000-08-16 | Chiron Corp | METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS |
WO1998010088A1 (en) | 1996-09-06 | 1998-03-12 | Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
WO1999003496A1 (en) | 1997-07-21 | 1999-01-28 | The University Of North Carolina At Chapel Hill | Factor ix antihemophilic factor with increased clotting activity |
IL138857A0 (en) | 1998-04-21 | 2001-10-31 | Micromet Ges For Biomedizinisc | Cd19xcd3 specific polypeptides and uses thereof |
AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
EP1447448B1 (en) | 2001-10-16 | 2011-02-02 | National Institute of Advanced Industrial Science and Technology | Novel n-acetylglucosamine transferase, nucleic acid encoding the same, antibody against the same and use thereof for diagnosing cancer or tumor |
US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
SG157224A1 (en) | 2001-11-13 | 2009-12-29 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
ES2664505T3 (es) | 2001-12-17 | 2018-04-19 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas |
WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
US20030198620A1 (en) | 2002-04-16 | 2003-10-23 | Keiya Ozawa | Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions |
WO2003093436A2 (en) | 2002-04-30 | 2003-11-13 | University Of Florida | Treatment for phenylketonuria |
DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
EP1776364A2 (en) | 2003-11-17 | 2007-04-25 | BioMarin Pharmaceutical Inc. | Processes for preparing tetrahydrobiopterin, and analogs of tetrahydrobiopterin |
WO2006073496A2 (en) | 2004-07-30 | 2006-07-13 | Targeted Genetics Corporation | Recombinant aav based vaccine methods |
WO2006055511A2 (en) | 2004-11-17 | 2006-05-26 | Biomarin Pharmaceutical Inc. | Stable tablet formulation of tetrahydrobiopterin |
DK2359867T3 (en) | 2005-04-07 | 2015-01-05 | Univ Pennsylvania | A method for increasing an AAV vector function |
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 |
US7534595B2 (en) | 2006-06-12 | 2009-05-19 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof |
US7531341B1 (en) | 2006-06-12 | 2009-05-12 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof |
US7892824B2 (en) | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
AR066354A1 (es) | 2007-05-01 | 2009-08-12 | Genzyme Corp | La terapia sistematica con factor-1 de crecimiento de tipo insulina reduce la neuropatia periferica diabetica y mejora la funcion renal en la nefropatia diabetica |
EP3093345B8 (en) * | 2007-07-26 | 2019-07-24 | UniQure IP B.V. | Baculoviral vectors comprising repeated coding sequences with differential codon biases |
US7537923B2 (en) | 2007-08-17 | 2009-05-26 | Biomarin Pharmaceutical Inc. | Compositions of prokaryotic phenylalanine ammonia-lyase and methods of treating cancer using compositions thereof |
CN101959891A (zh) | 2008-01-07 | 2011-01-26 | 生物马林药物股份有限公司 | 合成四氢生物蝶呤的方法 |
KR20100113112A (ko) | 2008-01-15 | 2010-10-20 | 아보트 러보러터리즈 | 개선된 포유동물 발현 벡터 및 이의 용도 |
US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
US20110201022A1 (en) | 2008-07-30 | 2011-08-18 | Biomarin Pharmaceutical Inc. | Assays for detection of phenylalanine ammonia-lyase and antibodies to phenylalanine ammonia-lyase |
TR201813067T4 (tr) | 2008-09-15 | 2018-09-21 | Uniqure Biopharma B V | Faktör IX polipeptit mutantı, bunun kullanımları ve bunu üretmeye yönelik bir yöntem. |
US10920244B2 (en) * | 2009-04-30 | 2021-02-16 | The Trustees Of The University Of Pennsylvania | Compositions for targeting conducting airway cells comprising adeno-associated virus constructs |
GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
MX362513B (es) | 2010-03-23 | 2019-01-22 | Intrexon Corp | Vectores que expresan de manera condicional proteinas terapeuticas, celulas hospedadoras que comprenden vectores, y usos de los mismos. |
CA2793633A1 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
AU2013221212B2 (en) | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
EP3747998A1 (en) | 2013-03-15 | 2020-12-09 | The Trustees of the University of Pennsylvania | Compositions for treating mpsi |
US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
CA3209883A1 (en) | 2013-07-22 | 2015-01-29 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
EP3770169A1 (en) | 2013-09-12 | 2021-01-27 | BioMarin Pharmaceutical Inc. | Adeno-associated virus factor viii vectors |
CN115141260A (zh) * | 2013-10-11 | 2022-10-04 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
CA2928186A1 (en) | 2013-10-22 | 2015-04-30 | Shire Human Genetic Therapies, Inc. | Mrna therapy for phenylketonuria |
HUE051311T2 (hu) | 2014-03-09 | 2021-03-01 | Univ Pennsylvania | Ornitin transzkarbamiláz (TC) deficiencia kezelésében alkalmas készítmények |
EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
CN114146063A (zh) | 2014-07-02 | 2022-03-08 | 川斯勒佰尔公司 | 信使rna的包封 |
US10301367B2 (en) | 2014-07-26 | 2019-05-28 | Consiglio Nazionale Delle Ricerche | Compositions and methods for treatment of muscular dystrophy |
WO2016049230A1 (en) | 2014-09-24 | 2016-03-31 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
GB201507842D0 (en) | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
CN108603235A (zh) | 2015-07-30 | 2018-09-28 | 马萨诸塞眼科耳科诊所 | 祖先病毒序列及其用途 |
MA42934A (fr) | 2015-09-24 | 2018-08-01 | Biomarin Pharm Inc | Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci |
WO2017066764A2 (en) | 2015-10-16 | 2017-04-20 | William Marsh Rice University | Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses |
AU2016343887B2 (en) | 2015-10-28 | 2023-04-06 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
PE20231949A1 (es) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
CA3042689A1 (en) | 2016-11-04 | 2018-07-12 | Baxalta Incorporated | Adeno-associated virus formulations |
KR20190100318A (ko) | 2016-12-30 | 2019-08-28 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
KR20230065367A (ko) | 2018-02-01 | 2023-05-11 | 호몰로지 메디슨, 인크. | Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
AU2019265560A1 (en) | 2018-05-09 | 2020-11-26 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
US20200069819A1 (en) | 2018-05-14 | 2020-03-05 | Biomarin Pharmaceutical Inc. | Stable expression of aav vectors in juvenile subjects |
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 |
MX2021012564A (es) | 2019-04-19 | 2022-01-18 | Regenxbio Inc | Formulaciones y métodos de vectores de virus adenoasociados. |
TW202104592A (zh) | 2019-05-14 | 2021-02-01 | 美商拜奧馬林製藥公司 | 重複投予基因療法載體之方法 |
TW202144575A (zh) | 2020-04-03 | 2021-12-01 | 美商拜奧馬林製藥公司 | 使用aav及治療調配物之苯酮尿症治療 |
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