BR112018072268A2 - ensaio de potência relativa para vetor viral que codifica isomerohidrolases - Google Patents

ensaio de potência relativa para vetor viral que codifica isomerohidrolases

Info

Publication number
BR112018072268A2
BR112018072268A2 BR112018072268-5A BR112018072268A BR112018072268A2 BR 112018072268 A2 BR112018072268 A2 BR 112018072268A2 BR 112018072268 A BR112018072268 A BR 112018072268A BR 112018072268 A2 BR112018072268 A2 BR 112018072268A2
Authority
BR
Brazil
Prior art keywords
isomerohydrolases
viral vector
vector encoding
relative potency
potency assay
Prior art date
Application number
BR112018072268-5A
Other languages
English (en)
Inventor
Couto Linda
Original Assignee
Spark Therapeutics, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Spark Therapeutics, Inc. filed Critical Spark Therapeutics, Inc.
Publication of BR112018072268A2 publication Critical patent/BR112018072268A2/pt

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • C12Q1/44—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase involving esterase
    • 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
    • A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
    • A61K31/07—Retinol compounds, e.g. vitamin A
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/32—Bonded phase chromatography
    • B01D15/325—Reversed phase
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D15/00—Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08—Selective adsorption, e.g. chromatography
    • B01D15/26—Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/38—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 - B01D15/36
    • B01D15/3804—Affinity chromatography
    • B01D15/3819—Affinity chromatography of the nucleic acid-nucleic acid binding protein type
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18—Carboxylic ester hydrolases (3.1.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/533—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving isomerase
    • 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
    • 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
    • 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
    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01135—Phosphatidylcholine--retinol O-acyltransferase (2.3.1.135)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01064—Retinoid isomerohydrolase (3.1.1.64)
    • 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
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/916—Hydrolases (3) acting on ester bonds (3.1), e.g. phosphatases (3.1.3), phospholipases C or phospholipases D (3.1.4)
    • G01N2333/918—Carboxylic ester hydrolases (3.1.1)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/16—Ophthalmology
    • G01N2800/164—Retinal disorders, e.g. retinopathy

Abstract

são fornecidos métodos para ensaio da função e/ou da atividade e/ou da potência de proteínas isomerohidrolase.
BR112018072268-5A 2016-04-28 2017-04-28 ensaio de potência relativa para vetor viral que codifica isomerohidrolases BR112018072268A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662328916P 2016-04-28 2016-04-28
US62/328.916 2016-04-28
PCT/US2017/030254 WO2017190081A1 (en) 2016-04-28 2017-04-28 Relative potency assay for viral vector encoding isomerhydrolases

Publications (1)

Publication Number Publication Date
BR112018072268A2 true BR112018072268A2 (pt) 2019-02-12

Family

ID=60161213

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018072268-5A BR112018072268A2 (pt) 2016-04-28 2017-04-28 ensaio de potência relativa para vetor viral que codifica isomerohidrolases

Country Status (18)

Country Link
US (1) US20210348210A1 (pt)
EP (1) EP3448371A4 (pt)
JP (1) JP7067843B2 (pt)
KR (1) KR20190020661A (pt)
CN (1) CN109689033A (pt)
AU (2) AU2017258776B2 (pt)
BR (1) BR112018072268A2 (pt)
CA (1) CA3022386A1 (pt)
CL (1) CL2018003079A1 (pt)
CO (1) CO2018012509A2 (pt)
IL (1) IL262583B2 (pt)
MX (1) MX2018013205A (pt)
MY (1) MY194096A (pt)
PE (1) PE20190393A1 (pt)
PH (1) PH12018502291A1 (pt)
RU (1) RU2756403C2 (pt)
SG (1) SG11201809388SA (pt)
WO (1) WO2017190081A1 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021064695A1 (en) 2019-10-04 2021-04-08 Novartis Ag Methods for measuring cralbp activity
WO2023099793A1 (en) * 2021-12-03 2023-06-08 Dsm Ip Assets B.V. Novel compositions comprising retinoids
WO2023099791A1 (en) * 2021-12-03 2023-06-08 Dsm Ip Assets B.V. Novel compositions comprising trans retinol

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SU873121A1 (ru) * 1979-08-20 1981-10-15 Институт биохимии АН ЛитССР Способ определени активности гидролаз
WO1998013020A1 (en) * 1996-09-27 1998-04-02 Unilever Plc Skin care compositions containing combinations of compounds for mimicking the effect on skin of retinoic acid
US6071410A (en) * 1998-11-16 2000-06-06 Varian, Inc. Recovery of organic solutes from aqueous solutions
CN1997359A (zh) * 2004-02-17 2007-07-11 哈佛大学校长及研究员协会 包括黄斑变性在内的眼科疾病的处理
HUE036596T2 (hu) * 2004-06-18 2018-07-30 Univ Washington Retinaszármazékok és módszerek ezek felhasználására látászavarok kezeléséhez
US20080249042A1 (en) * 2004-09-09 2008-10-09 University Of Washington All-Trans-Retinol: All-Trans-13,14-Dihydroretinol Saturase and Methods of Its Use
JP5663025B2 (ja) * 2009-09-15 2015-02-04 キューエルティー インコーポレイテッド ９−ｃｉｓ−レチニルエステルを脂質ビヒクル中に含む医薬製剤
US20140357611A1 (en) * 2009-12-08 2014-12-04 Case Western Reserve University Compounds and methods of treating ocular disorders
CA2907799A1 (en) * 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2015184453A1 (en) 2014-05-30 2015-12-03 Case Western Reserve University Retinylamine derivitives for treatment of ocular disorders
CN106794262A (zh) * 2014-07-31 2017-05-31 俄克拉何马大学董事会 哺乳动物rpe65的高异构水解酶活性突变体
GB201502137D0 (en) * 2015-02-09 2015-03-25 Ucl Business Plc Treatment

Also Published As

Publication number Publication date
EP3448371A1 (en) 2019-03-06
SG11201809388SA (en) 2018-11-29
PE20190393A1 (es) 2019-03-13
AU2017258776A1 (en) 2018-11-29
AU2023203939A1 (en) 2023-09-14
IL262583B1 (en) 2023-06-01
US20210348210A1 (en) 2021-11-11
JP2019514375A (ja) 2019-06-06
IL262583B2 (en) 2023-10-01
JP7067843B2 (ja) 2022-05-16
CN109689033A (zh) 2019-04-26
RU2756403C2 (ru) 2021-09-30
CA3022386A1 (en) 2017-11-02
MX2018013205A (es) 2019-06-24
WO2017190081A1 (en) 2017-11-02
MY194096A (en) 2022-11-11
EP3448371A4 (en) 2019-09-11
CO2018012509A2 (es) 2019-04-30
RU2018141675A3 (pt) 2020-09-14
AU2017258776B2 (en) 2023-07-13
IL262583A (en) 2018-12-31
PH12018502291A1 (en) 2019-07-08
RU2018141675A (ru) 2020-05-29
KR20190020661A (ko) 2019-03-04
CL2018003079A1 (es) 2019-03-29

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Legal Events

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B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI N? 10196/2001, QUE MODIFICOU A LEI N? 9279/96, A CONCESS?O DA PATENTE EST? CONDICIONADA ? ANU?NCIA PR?VIA DA ANVISA. CONSIDERANDO A APROVA??O DOS TERMOS DO PARECER N? 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL N? 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVID?NCIAS CAB?VEIS.

B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B06I Publication of requirement cancelled [chapter 6.9 patent gazette]

Free format text: ANULADA A PUBLICACAO CODIGO 6.6.1 NA RPI NO 2631 DE 08/06/2021 POR TER SIDO INDEVIDA.

B07G Grant request does not fulfill article 229-c lpi (prior consent of anvisa) [chapter 7.7 patent gazette]
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]