SG11201811603WA - Novel adeno-associated virus capsid proteins - Google Patents
Novel adeno-associated virus capsid proteinsInfo
- Publication number
- SG11201811603WA SG11201811603WA SG11201811603WA SG11201811603WA SG11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA SG 11201811603W A SG11201811603W A SG 11201811603WA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- capsid proteins
- novato
- novel
- digital drive
- Prior art date
Links
- 108090000565 Capsid Proteins Proteins 0.000 title abstract 6
- 102100023321 Ceruloplasmin Human genes 0.000 title abstract 5
- 241000702421 Dependoparvovirus Species 0.000 title abstract 3
- 239000013607 AAV vector Substances 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 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
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
Classifications
-
- 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
-
- 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
-
- 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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—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
-
- 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
-
- 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
-
- 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
Landscapes
- 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)
- Biophysics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662366838P | 2016-07-26 | 2016-07-26 | |
| PCT/US2017/043703 WO2018022608A2 (en) | 2016-07-26 | 2017-07-25 | Novel adeno-associated virus capsid proteins |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201811603WA true SG11201811603WA (en) | 2019-02-27 |
Family
ID=59558468
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201811603WA SG11201811603WA (en) | 2016-07-26 | 2017-07-25 | Novel adeno-associated virus capsid proteins |
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) | IL305149A (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 |
| PT3356390T (pt) | 2015-09-28 | 2021-04-21 | Univ Florida | Métodos e composições para vetores virais que se evadem a anticorpos |
| CA3034527A1 (en) | 2016-08-23 | 2018-03-01 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
| KR20210006327A (ko) * | 2018-02-27 | 2021-01-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 캡시드 탈아미드화가 감소된 신규 아데노-연관 바이러스(aav) 벡터 및 이의 용도 |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| BR112020020223A2 (pt) * | 2018-04-05 | 2021-01-19 | Genethon | Sorotipo de vírus adeno-associado híbrido recombinante entre aav9 e aavrh74 com tropismo pelo fígado reduzido |
| CN113164762A (zh) | 2018-05-09 | 2021-07-23 | 生物马林药物股份有限公司 | 苯丙酮尿症的治疗方法 |
| US11987801B2 (en) | 2018-05-11 | 2024-05-21 | Massachusetts Eye And Ear Infirmary | Altering tissue tropism of adeno-associated viruses |
| 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 |
| US20210317478A1 (en) * | 2018-08-21 | 2021-10-14 | Massachusetts Eye And Ear Infirmary | Compositions and methods for modulating transduction efficiency of adeno-associated viruses |
| JP2022515338A (ja) * | 2018-12-05 | 2022-02-18 | アビオナ・セラピュ―ティクス・インコーポレイテッド | 遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
| JP2022523050A (ja) * | 2019-01-23 | 2022-04-21 | ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． | 合理的設計により増強された新規なａａｖウイルスによる網膜における高効率の形質導入および側方への広がり |
| CA3133453A1 (en) | 2019-03-21 | 2020-09-24 | Daniel Mccoy | Recombinant adeno-associated virus vectors |
| KR20210143869A (ko) * | 2019-03-28 | 2021-11-29 | 더 제너럴 하스피탈 코포레이션 | 트랜스진 발현을 위한 조작된 아데노-연관 (aav) 벡터 |
| AR118928A1 (es) | 2019-05-14 | 2021-11-10 | Biomarin Pharm Inc | Métodos de redosificación de vectores de terapia génica |
| EP4045664A1 (en) * | 2019-10-16 | 2022-08-24 | Wuxi Apptec (Shanghai) Co., Ltd. | A novel aav variant |
| BR112022006530A2 (pt) | 2019-10-17 | 2022-07-05 | Stridebio Inc | Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c |
| CN114829391A (zh) | 2019-11-14 | 2022-07-29 | 生物马林药物股份有限公司 | 用肝特异性基因疗法载体治疗遗传性血管性水肿 |
| IL295747A (en) | 2020-02-21 | 2022-10-01 | Akouos Inc | Preparations and methods for the treatment of hearing impairment that is not related to age in humans |
| EP4110792A1 (en) * | 2020-02-25 | 2023-01-04 | Children's Medical Research Institute | Adeno-associated virus capsid polypeptides and vectors |
| 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 |
| WO2021222332A1 (en) | 2020-04-29 | 2021-11-04 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
| WO2021236908A2 (en) | 2020-05-20 | 2021-11-25 | Biomarin Pharmaceutical Inc. | Use of regulatory proteins for the production of adeno-associated virus |
| CA3187635A1 (en) * | 2020-07-03 | 2022-01-06 | Genethon | Method for engineering novel hybrid aav capsids through hypervariable regions swapping |
| 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 |
| IL302127A (en) * | 2020-10-18 | 2023-06-01 | Univ Pennsylvania | An improved adeno-associated virus (AAV) vector and its uses |
| WO2022094461A1 (en) | 2020-11-02 | 2022-05-05 | 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 |
| WO2023034997A1 (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 |
| WO2023034996A1 (en) | 2021-09-03 | 2023-03-09 | 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 |
| AR127217A1 (es) | 2021-10-01 | 2023-12-27 | Biomarin Pharm Inc | Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas |
| 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 |
|---|---|---|---|---|
| ATE78293T1 (de) | 1983-05-27 | 1992-08-15 | Texas A & M Univ Sys | 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 |
| JP2001500376A (ja) | 1996-09-06 | 2001-01-16 | カイロン コーポレイション | 組換えａａｖベクターを使用する治療分子の肝臓特異的送達のための方法および組成物 |
| 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 |
| US6531298B2 (en) | 1997-07-21 | 2003-03-11 | The University Of North Carolina At Chapel Hill | Factor IX antihemophilic factor with increased clotting activity |
| BRPI9909860B8 (pt) | 1998-04-21 | 2021-05-25 | Amgen Res Munich Gmbh | polipeptídeo multifuncional de cadeia simples, polinucleotídeo, vetor, célula procariótica, de levedura ou unicelular, composição, usos de polipeptídeo e polinucleotídeo e métodos para preparo do referido polipeptídeo e para identificação de ativadores ou inibidores de ativação ou estimulação das células t |
| WO2001083692A2 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| ATE497536T1 (de) | 2001-10-16 | 2011-02-15 | Nat Inst Of Advanced Ind Scien | Neue n-acetylglucosamin transferase, codierende nukleinsäuren, spezifische antikörper, sowie verwendung zur krebsdiagnose |
| 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 |
| NZ600121A (en) * | 2001-11-13 | 2013-12-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| AU2002359284A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
| 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 |
| JP5054975B2 (ja) | 2003-09-30 | 2012-10-24 | 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 | アデノ随伴ウイルス（ａａｖ）の同源系統群、配列、それらを含有するベクターおよびそれらの用途 |
| EP1776364A2 (en) | 2003-11-17 | 2007-04-25 | BioMarin Pharmaceutical Inc. | Processes for preparing tetrahydrobiopterin, and analogs of tetrahydrobiopterin |
| EP1771571A2 (en) | 2004-07-30 | 2007-04-11 | Targeted Genetics Corporation | Recombinant aav based vaccine methods |
| WO2006055511A2 (en) | 2004-11-17 | 2006-05-26 | Biomarin Pharmaceutical Inc. | Stable tablet formulation of tetrahydrobiopterin |
| JP5702519B2 (ja) | 2005-04-07 | 2015-04-15 | 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 | Ａａｖベクターの機能を上げる方法 |
| 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 |
| EP2125006B1 (en) | 2007-01-18 | 2013-10-16 | 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 |
| WO2009014445A2 (en) * | 2007-07-26 | 2009-01-29 | Amsterdam Molecular Therapeutics 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 |
| CL2009000007A1 (es) | 2008-01-07 | 2010-02-19 | Biomarin Pharm Inc | Metodo para preparar biopterina por hidrolisis de diacetilbiopterina en presencia de una base en una mezcla bifasica de agua y un solvente organico inmiscible en agua a un ph de 10. |
| 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 |
| DK2337849T3 (en) | 2008-09-15 | 2018-10-01 | Uniqure Biopharma B V | FACTOR IX POLYPEPTIME MUTANT, APPLICATIONS THEREOF AND METHOD OF PRODUCING THEREOF |
| CN102439157B (zh) * | 2009-04-30 | 2015-09-16 | 宾夕法尼亚大学托管会 | 包含腺伴随病毒构建体的靶向传导气道细胞组合物 |
| GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
| CA2794196A1 (en) | 2010-03-23 | 2011-09-29 | Intrexon Corporation | Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | 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 |
| ES2752191T3 (es) | 2012-02-17 | 2020-04-03 | Childrens Hospital Philadelphia | Composiciones y métodos con vectores de AAV para la transferencia de genes a células, órganos y tejidos |
| GB201210357D0 (en) | 2012-06-12 | 2012-07-25 | Ucl Business Plc | Factor VIII sequences |
| KR102346455B1 (ko) | 2013-03-15 | 2022-01-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ｍｐｓｉ 치료를 위한 조성물 및 방법 |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| KR102380265B1 (ko) * | 2013-07-22 | 2022-03-29 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| HUE050484T2 (hu) | 2013-09-12 | 2020-12-28 | Biomarin Pharm Inc | VIII. faktort kódoló gént tartalmazó AAV vektorok |
| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 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 |
| EA034103B1 (ru) | 2013-10-22 | 2019-12-27 | Транслейт Био, Инк. | СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК |
| PT3116900T (pt) | 2014-03-09 | 2020-10-08 | Univ Pennsylvania | Composições úteis no tratamento da deficiência de ornitina transcarbamilase (otc) |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| CN114344275A (zh) | 2014-07-02 | 2022-04-15 | 川斯勒佰尔公司 | 信使rna的包封 |
| WO2016016119A1 (en) | 2014-07-26 | 2016-02-04 | Consiglio Nazionale Delle Ricerche | Compositions and methods for treatment of muscular dystrophy |
| PT3198018T (pt) * | 2014-09-24 | 2021-02-24 | Hope City | Variantes dos vetores de vírus adenoassociados para edição do genoma de alta eficácia e métodos da mesma |
| GB201507842D0 (en) | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| US10738087B2 (en) * | 2015-07-30 | 2020-08-11 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
| TWI756185B (zh) | 2015-09-24 | 2022-03-01 | 美商拜奧馬林製藥公司 | 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 |
| 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 |
| BR112019013576A2 (pt) | 2016-12-30 | 2020-02-04 | Univ Pennsylvania | terapia genica para o tratamento da fenilcetonuria |
| 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 |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| CN113164762A (zh) | 2018-05-09 | 2021-07-23 | 生物马林药物股份有限公司 | 苯丙酮尿症的治疗方法 |
| 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 |
| KR20210008491A (ko) | 2018-05-14 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 청소년 대상체에서 aav 벡터의 안정한 발현 |
| 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 |
| CA3136939A1 (en) | 2019-04-19 | 2020-10-22 | Regenxbio Inc. | Adeno-associated virus vector formulations and methods |
| AR118928A1 (es) | 2019-05-14 | 2021-11-10 | Biomarin Pharm Inc | Métodos de redosificación de vectores de terapia génica |
| 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 |
-
2017
- 2017-07-25 IL IL305149A patent/IL305149A/en unknown
- 2017-07-25 IL IL263801A patent/IL263801B2/en unknown
- 2017-07-25 SG SG11201811603WA patent/SG11201811603WA/en unknown
- 2017-07-25 KR KR1020237039466A patent/KR20230161535A/ko active Search and Examination
- 2017-07-25 KR KR1020197001930A patent/KR102604510B1/ko active IP Right Grant
- 2017-07-25 BR BR112019001532A patent/BR112019001532A2/pt unknown
- 2017-07-25 US US16/319,877 patent/US11584780B2/en active Active
- 2017-07-25 PE PE2019000266A patent/PE20190401A1/es unknown
- 2017-07-25 AU AU2017301600A patent/AU2017301600B2/en active Active
- 2017-07-25 WO PCT/US2017/043703 patent/WO2018022608A2/en active Application Filing
- 2017-07-25 MX MX2019000962A patent/MX2019000962A/es unknown
- 2017-07-25 CN CN201780045741.3A patent/CN109715650B/zh active Active
- 2017-07-25 JP JP2019503445A patent/JP6994018B2/ja active Active
- 2017-07-25 CA CA3031026A patent/CA3031026A1/en active Pending
- 2017-07-25 EP EP17749265.9A patent/EP3491008A2/en active Pending
-
2019
- 2019-01-10 PH PH12019500068A patent/PH12019500068A1/en unknown
- 2019-01-22 CL CL2019000167A patent/CL2019000167A1/es unknown
-
2020
- 2020-12-04 CL CL2020003176A patent/CL2020003176A1/es unknown
-
2021
- 2021-10-07 JP JP2021165270A patent/JP7395550B2/ja active Active
- 2021-12-29 JP JP2021215368A patent/JP7303287B2/ja active Active
-
2022
- 2022-10-27 AU AU2022259813A patent/AU2022259813A1/en active Pending
- 2022-12-08 US US18/063,589 patent/US20230322864A1/en active Pending
-
2023
- 2023-01-18 CL CL2023000165A patent/CL2023000165A1/es unknown
- 2023-04-04 JP JP2023060936A patent/JP2023076606A/ja active Pending
- 2023-09-15 CL CL2023002764A patent/CL2023002764A1/es unknown
- 2023-10-06 CL CL2023003010A patent/CL2023003010A1/es unknown
- 2023-10-06 CL CL2023003001A patent/CL2023003001A1/es unknown
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| SG11201811603WA (en) | Novel adeno-associated virus capsid proteins | |
| SG11201906297QA (en) | Nucleic acids encoding crispr-associated proteins and uses thereof | |
| SG11201901206SA (en) | Trimer stabilizing hiv envelope protein mutations | |
| SG11201811432WA (en) | Rna for cancer therapy | |
| SG11201811431VA (en) | Multispecific antibodies against cd40 and cd137 | |
| SG11201807573VA (en) | Methods for providing single-stranded rna | |
| SG11201808750PA (en) | T cell receptors | |
| SG11201907298WA (en) | Methods and compositions for gene transfer across the vasculature | |
| SG11201811338VA (en) | A novel botulinum neurotoxin and its derivatives | |
| SG11201809029RA (en) | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy | |
| SG11201809344QA (en) | Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde | |
| SG11201809912UA (en) | Hybrid carriers for nucleic acid cargo | |
| SG11201901077RA (en) | Anti-tigit antibodies, anti-pvrig antibodies and combinations thereof | |
| SG11201804814YA (en) | Materials and methods for delivering nucleic acids to cochlear and vestibular cells | |
| SG11201804070XA (en) | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a | |
| SG11201804411TA (en) | Human immunodeficiency virus antigens, vectors, compositions, and methods of use thereof | |
| SG11201803958WA (en) | Nucleic acids encoding repetitive amino acid sequences rich in proline and alanine residues that have low repetitive nucleotide sequences | |
| SG11201804443UA (en) | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome | |
| SG11201900400QA (en) | Bispecific antibody-like binding proteins specifically binding to cd3 and cd123 | |
| SG11201906853QA (en) | Anti-rsv monoclonal antibody formulation | |
| SG11201809908TA (en) | Stabilized glycopeptide antibiotic formulations | |
| SG11201909520VA (en) | Rna for treatment of autoimmune diseases | |
| SG11201811230RA (en) | Compositions and methods for reducing ocular neovascularization | |
| SG11201808592PA (en) | Method for selection of high m6p recombinant proteins | |
| SG11201811554SA (en) | Antisense oligonucleotides for modulating htra1 expression |