BR112019002934A2 - métodos para detecção de aav - Google Patents
métodos para detecção de aavInfo
- Publication number
- BR112019002934A2 BR112019002934A2 BR112019002934-6A BR112019002934A BR112019002934A2 BR 112019002934 A2 BR112019002934 A2 BR 112019002934A2 BR 112019002934 A BR112019002934 A BR 112019002934A BR 112019002934 A2 BR112019002934 A2 BR 112019002934A2
- Authority
- BR
- Brazil
- Prior art keywords
- aav
- determining
- detection methods
- particles
- heterogeneity
- Prior art date
Links
- 238000001514 detection method Methods 0.000 title 1
- 239000002245 particle Substances 0.000 abstract 6
- 241000700605 Viruses Species 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 108090000565 Capsid Proteins Proteins 0.000 abstract 1
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 1
- 230000021736 acetylation Effects 0.000 abstract 1
- 238000006640 acetylation reaction Methods 0.000 abstract 1
- 230000009615 deamination Effects 0.000 abstract 1
- 238000006481 deamination reaction Methods 0.000 abstract 1
- 230000026683 transduction Effects 0.000 abstract 1
- 238000010361 transduction Methods 0.000 abstract 1
- 230000003612 virological 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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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N30/00—Investigating or analysing materials by separation into components using adsorption, absorption or similar phenomena or using ion-exchange, e.g. chromatography or field flow fractionation
- G01N30/02—Column chromatography
- G01N30/62—Detectors specially adapted therefor
- G01N30/72—Mass spectrometers
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N30/00—Investigating or analysing materials by separation into components using adsorption, absorption or similar phenomena or using ion-exchange, e.g. chromatography or field flow fractionation
- G01N30/02—Column chromatography
- G01N30/62—Detectors specially adapted therefor
- G01N30/72—Mass spectrometers
- G01N30/7233—Mass spectrometers interfaced to liquid or supercritical fluid chromatograph
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6803—General methods of protein analysis not limited to specific proteins or families of proteins
- G01N33/6848—Methods of protein analysis involving mass spectrometry
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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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- 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
- C12N2750/14123—Virus like particles [VLP]
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N30/00—Investigating or analysing materials by separation into components using adsorption, absorption or similar phenomena or using ion-exchange, e.g. chromatography or field flow fractionation
- G01N30/02—Column chromatography
- G01N2030/022—Column chromatography characterised by the kind of separation mechanism
- G01N2030/027—Liquid chromatography
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/005—Assays involving biological materials from specific organisms or of a specific nature from viruses
- G01N2333/01—DNA viruses
- G01N2333/075—Adenoviridae
Abstract
a presente invenção refere-se a métodos para determinar o sorotipo de uma partícula de vírus e/ou determinar a heterogeneidade de uma partícula de vírus (por exemplo, uma partícula de aav). em outras modalidades, a invenção fornece métodos para determinar a heterogeneidade de partículas de aav. em alguns aspectos, a invenção fornece partículas virais (por exemplo, partículas de raav) com melhor estabilidade e/ou eficiência de transdução melhorada, aumentando a acetilação e/ou desaminação de proteínas do capsídeo.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662375314P | 2016-08-15 | 2016-08-15 | |
US62/375,314 | 2016-08-15 | ||
PCT/US2017/046814 WO2018035059A1 (en) | 2016-08-15 | 2017-08-14 | Methods for detecting aav |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112019002934A2 true BR112019002934A2 (pt) | 2019-05-14 |
Family
ID=59762045
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112019002934-6A BR112019002934A2 (pt) | 2016-08-15 | 2017-08-14 | métodos para detecção de aav |
Country Status (22)
Country | Link |
---|---|
US (2) | US11698377B2 (pt) |
EP (2) | EP3497207B1 (pt) |
JP (3) | JP7021191B2 (pt) |
KR (3) | KR102425289B1 (pt) |
CN (1) | CN110168080A (pt) |
AU (2) | AU2017312951B2 (pt) |
BR (1) | BR112019002934A2 (pt) |
CA (1) | CA3033856A1 (pt) |
CL (2) | CL2019000392A1 (pt) |
CR (1) | CR20190127A (pt) |
CY (1) | CY1124364T1 (pt) |
DK (1) | DK3497207T3 (pt) |
ES (1) | ES2863674T3 (pt) |
HU (1) | HUE053747T2 (pt) |
MA (1) | MA55748A (pt) |
MX (2) | MX2019001938A (pt) |
PH (1) | PH12019500316A1 (pt) |
SG (2) | SG10201913002QA (pt) |
TN (1) | TN2019000047A1 (pt) |
TW (2) | TW202309068A (pt) |
WO (1) | WO2018035059A1 (pt) |
ZA (1) | ZA201900945B (pt) |
Families Citing this family (19)
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GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
RS65241B1 (sr) * | 2017-02-28 | 2024-03-29 | Univ Pennsylvania | Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe |
AU2019228504A1 (en) * | 2018-02-27 | 2020-09-10 | The Trustees Of The University Of Pennsylvania | Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor |
EP3758724A4 (en) * | 2018-02-27 | 2022-07-06 | The Trustees of The University of Pennsylvania | NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSID DEAMIDATION AND USES THEREOF |
EP3762500A1 (en) * | 2018-03-06 | 2021-01-13 | Voyager Therapeutics, Inc. | Insect cell manufactured partial self-complementary aav genomes |
US11391707B2 (en) * | 2018-07-27 | 2022-07-19 | Waters Technologies Corporation | Liquid chromatography/mass spectrometry methods for the analysis of polar molecules |
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 |
CN117969861A (zh) * | 2018-10-25 | 2024-05-03 | 瑞泽恩制药公司 | 用于分析病毒衣壳蛋白组成的方法 |
CA3165911A1 (en) | 2020-01-03 | 2021-07-08 | Sarepta Therapeutics, Inc. | Methods for analyzing aav capsid proteins |
KR20220133941A (ko) | 2020-01-29 | 2022-10-05 | 젠자임 코포레이션 | 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법 |
CN111517359B (zh) * | 2020-04-23 | 2021-08-20 | 江南大学 | 一种手性硫化铜超粒子的合成方法 |
RU2748540C1 (ru) * | 2021-02-08 | 2021-05-26 | Автономная некоммерческая образовательная организация высшего образования "Сколковский институт науки и технологий" | Способ детектирования вируса SARS-CoV-2 методом масс-спектрометрии |
US20220268783A1 (en) * | 2021-02-19 | 2022-08-25 | Waters Technologies Corporation | Methods for peptide mapping of adeno-associated virus (aav) proteins |
JPWO2022224965A1 (pt) * | 2021-04-20 | 2022-10-27 | ||
WO2023287724A1 (en) * | 2021-07-12 | 2023-01-19 | Regeneron Pharmaceuticals, Inc. | Online native mass spectrometry methods for assaying viral particles |
WO2023287725A1 (en) * | 2021-07-12 | 2023-01-19 | Regeneron Pharmaceuticals, Inc. | Liquid chromatography assay for determining aav capsid ratio |
CN113552349B (zh) * | 2021-07-30 | 2022-07-19 | 上海勉亦生物科技有限公司 | Aav蛋白外壳的检测方法 |
WO2023132338A1 (ja) * | 2022-01-06 | 2023-07-13 | 富士フイルム株式会社 | タンパク質における糖が結合したアミノ酸部位を同定する方法、及びキット |
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US6204059B1 (en) * | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
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 |
US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
AU781958C (en) | 1999-08-09 | 2006-03-30 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
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 |
NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
ATE405295T1 (de) | 2002-05-01 | 2008-09-15 | Univ Florida | Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine |
CA2503292A1 (en) * | 2002-11-22 | 2004-06-10 | Caprion Pharmaceuticals, Inc. | Constellation mapping and uses thereof |
WO2004075861A2 (en) | 2003-02-26 | 2004-09-10 | Children's Hospital, Inc. | Recombinant adeno-associated virus production |
PT1625210E (pt) | 2003-05-21 | 2011-03-15 | Genzyme Corp | Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios |
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US9441244B2 (en) * | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
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DK2826860T3 (en) | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
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BR112013020734A2 (pt) | 2011-02-17 | 2017-06-13 | Univ Pennsylvania | composições e métodos para alterar a especificidade do tecido e aprimorar a transferência gênica mediada por aav9 |
EP3795581A3 (en) | 2011-08-24 | 2021-06-09 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
PT2839014T (pt) | 2012-04-18 | 2021-03-19 | Childrens Hospital Philadelphia | ¿composição e métodos para transferência de genes altamente eficiente com a utilização de variantes de capsídeo de aav |
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GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
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2017
- 2017-08-14 TN TNP/2019/000047A patent/TN2019000047A1/en unknown
- 2017-08-14 CA CA3033856A patent/CA3033856A1/en active Pending
- 2017-08-14 KR KR1020197007381A patent/KR102425289B1/ko active IP Right Grant
- 2017-08-14 CR CR20190127A patent/CR20190127A/es unknown
- 2017-08-14 MX MX2019001938A patent/MX2019001938A/es unknown
- 2017-08-14 US US16/325,653 patent/US11698377B2/en active Active
- 2017-08-14 HU HUE17761362A patent/HUE053747T2/hu unknown
- 2017-08-14 JP JP2019508235A patent/JP7021191B2/ja active Active
- 2017-08-14 BR BR112019002934-6A patent/BR112019002934A2/pt unknown
- 2017-08-14 MA MA055748A patent/MA55748A/fr unknown
- 2017-08-14 SG SG10201913002QA patent/SG10201913002QA/en unknown
- 2017-08-14 WO PCT/US2017/046814 patent/WO2018035059A1/en unknown
- 2017-08-14 AU AU2017312951A patent/AU2017312951B2/en active Active
- 2017-08-14 DK DK17761362.7T patent/DK3497207T3/da active
- 2017-08-14 ES ES17761362T patent/ES2863674T3/es active Active
- 2017-08-14 SG SG11201901221YA patent/SG11201901221YA/en unknown
- 2017-08-14 CN CN201780063531.7A patent/CN110168080A/zh active Pending
- 2017-08-14 KR KR1020237017707A patent/KR20230078834A/ko not_active Application Discontinuation
- 2017-08-14 KR KR1020227025409A patent/KR102538037B1/ko active IP Right Grant
- 2017-08-14 EP EP17761362.7A patent/EP3497207B1/en active Active
- 2017-08-14 EP EP20210237.2A patent/EP3851449A1/en active Pending
- 2017-08-15 TW TW111138258A patent/TW202309068A/zh unknown
- 2017-08-15 TW TW106127648A patent/TWI781953B/zh active
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2019
- 2019-02-13 CL CL2019000392A patent/CL2019000392A1/es unknown
- 2019-02-14 ZA ZA2019/00945A patent/ZA201900945B/en unknown
- 2019-02-14 PH PH12019500316A patent/PH12019500316A1/en unknown
- 2019-02-15 MX MX2023009081A patent/MX2023009081A/es unknown
- 2019-10-14 CL CL2019002912A patent/CL2019002912A1/es unknown
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2021
- 2021-03-24 CY CY20211100262T patent/CY1124364T1/el unknown
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2022
- 2022-02-02 JP JP2022014629A patent/JP2022064980A/ja active Pending
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2023
- 2023-05-22 US US18/321,542 patent/US20240044910A1/en active Pending
- 2023-11-30 JP JP2023202414A patent/JP2024023460A/ja active Pending
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2024
- 2024-01-18 AU AU2024200349A patent/AU2024200349A1/en active Pending
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