SG11201908055WA - Methods for determining potency of adeno-associated virus preparations - Google Patents

Methods for determining potency of adeno-associated virus preparations

Info

Publication number
SG11201908055WA
SG11201908055WA SG11201908055WA SG11201908055WA SG11201908055WA SG 11201908055W A SG11201908055W A SG 11201908055WA SG 11201908055W A SG11201908055W A SG 11201908055WA SG 11201908055W A SG11201908055W A SG 11201908055WA SG 11201908055W A SG11201908055W A SG 11201908055WA
Authority
SG
Singapore
Prior art keywords
international
vienna
methods
preparations
adeno
Prior art date
Application number
SG11201908055WA
Inventor
Marian Bendik
Roman Necina
Ernst Boehm
Michael Graninger
Christian Fiedler
Original Assignee
Baxalta Inc
Baxalta GmbH
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 Baxalta Inc, Baxalta GmbH filed Critical Baxalta Inc
Publication of SG11201908055WA publication Critical patent/SG11201908055WA/en

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Classifications

    • 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
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/569—Immunoassay; Biospecific binding assay; Materials therefor for microorganisms, e.g. protozoa, bacteria, viruses
    • G01N33/56983—Viruses
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N1/00—Sampling; Preparing specimens for investigation
    • G01N1/28—Preparing specimens for investigation including physical details of (bio-)chemical methods covered elsewhere, e.g. G01N33/50, C12Q
    • G01N1/36—Embedding or analogous mounting of samples
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N1/00—Sampling; Preparing specimens for investigation
    • G01N1/28—Preparing specimens for investigation including physical details of (bio-)chemical methods covered elsewhere, e.g. G01N33/50, C12Q
    • G01N1/42—Low-temperature sample treatment, e.g. cryofixation
    • 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
    • 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
    • G01N33/483—Physical analysis of biological material
    • G01N33/4833—Physical analysis of biological material of solid biological material, e.g. tissue samples, cell cultures
    • 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
    • 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
    • G01N2333/015—Parvoviridae, e.g. feline panleukopenia virus, human Parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Urology & Nephrology (AREA)
  • Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Cell Biology (AREA)
  • Optics & Photonics (AREA)
  • Epidemiology (AREA)
  • Manufacturing & Machinery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 07 September 2018 (07.09.2018) WIP0 I PCT o °nolo momioolioimo in oimIE (10) International Publication Number WO 2018/160975 Al (51) International Patent Classification: GO1N 33/569 (2006.01) GO1N 33/48 (2006.01) C12N 15/864 (2006.01) (21) International Application Number: PCT/US2018/020676 (22) International Filing Date: 02 March 2018 (02.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/467,045 03 March 2017 (03.03.2017) US (71) Applicants: BAXALTA INCORPORATED [US/US]; 1200 Lakeside Drive, Bannockburn, IL 60015 (US). BAX- ALTA GMBH [CH/CH]; Zaehlerweg 4, 6300 Zug (CH). (72) Inventors: BENDIK, Marian; Gabcikova 4, 84105 Bratislava (SK). NECINA, Roman; Mengergasse 44/2, 1210 Vienna (AT). BOEHM, Ernst; Muehlweg 94, 1210 Vienna (AT). GRANINGER, Michael; Gernotgasse 3/11, 1150 Vienna (AT). FIEDLER, Christian; Raaberbahn- gasse 18/36, 1100 Vienna (AT). (74) Agent: SULLIVAN, Nicole R.; Troutman Sanders LLP, 875 Third Avenue, New York, NY 10022 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. = (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: with international search report (Art. 21(3)) O 1-1 (54) Title: METHODS FOR DETERMINING POTENCY OF ADENO-ASSOCIATED VIRUS PREPARATIONS (57) : Provided herein are methods of measuring the qualitative and/ or quantity attributes of gene therapy vector preparations. O In certain embodiments, the gene therapy vector preparations are Adeno-associated virus (AAV) preparations (e.g., AAV8). In certain \" embodiments the methods comprise determining the potency or dose of the AAV preparation using ELISA, or ELISA in combination 0 with cryogenic transmission electron microscopy (CryoTEM).
SG11201908055WA 2017-03-03 2018-03-02 Methods for determining potency of adeno-associated virus preparations SG11201908055WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762467045P 2017-03-03 2017-03-03
PCT/US2018/020676 WO2018160975A1 (en) 2017-03-03 2018-03-02 Methods for determining potency of adeno-associated virus preparations

Publications (1)

Publication Number Publication Date
SG11201908055WA true SG11201908055WA (en) 2019-09-27

Family

ID=61692094

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201908055WA SG11201908055WA (en) 2017-03-03 2018-03-02 Methods for determining potency of adeno-associated virus preparations

Country Status (12)

Country Link
US (1) US20200018752A1 (en)
EP (1) EP3589956A1 (en)
JP (1) JP7232766B2 (en)
KR (1) KR102590519B1 (en)
CN (1) CN110352353B (en)
AU (1) AU2018227883B2 (en)
BR (1) BR112019018216A2 (en)
CA (1) CA3053176A1 (en)
IL (1) IL268600A (en)
MX (1) MX2019010045A (en)
SG (1) SG11201908055WA (en)
WO (1) WO2018160975A1 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021153659A1 (en) * 2020-01-31 2021-08-05 Ａｇｃ株式会社 Method for quantifying adeno-associated virus (aav)
CN113552349B (en) * 2021-07-30 2022-07-19 上海勉亦生物科技有限公司 Detection method of AAV protein coat

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030149235A1 (en) * 2000-11-17 2003-08-07 Baker Andrew Howard Targeting peptides
AU2008248904B2 (en) 2007-05-04 2014-10-09 Takeda Pharmaceutical Company Limited Formulation of sugar solutions for continuous ultracentrifugation for virus purification
US8906863B2 (en) * 2009-02-27 2014-12-09 The Regents Of The University Of California Proteolysis-resistant capsid of chimeric hepatitis E virus as an oral delivery vector
RU2561035C2 (en) * 2009-07-01 2015-08-20 Конинклейке Филипс Электроникс Н.В. Carriers for mpv-regulated delivery of medication, sensitive to stimulating factor
GB0913433D0 (en) * 2009-07-31 2009-09-16 Isis Innovation Method
AU2013202339A1 (en) * 2010-03-19 2013-05-02 Massachusetts Institute Of Technology Lipid vesicle compositions and methods of use
US20130191037A1 (en) * 2011-07-26 2013-07-25 Nanoimaging Services Characterization of particulates using electron microscopy and image processing methods
EP2771455B1 (en) * 2011-10-28 2016-10-05 The University of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
EP2963646A1 (en) * 2014-07-01 2016-01-06 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Decoder and method for decoding an audio signal, encoder and method for encoding an audio signal
JP6608969B2 (en) * 2015-06-23 2019-11-20 ドイッチェス・クレープスフォルシュングスツェントルム Methods for large-scale production and purification of parvovirus

Also Published As

Publication number Publication date
JP2020511630A (en) 2020-04-16
CA3053176A1 (en) 2018-09-07
AU2018227883A1 (en) 2019-08-29
EP3589956A1 (en) 2020-01-08
MX2019010045A (en) 2019-10-02
BR112019018216A2 (en) 2020-06-23
JP7232766B2 (en) 2023-03-03
AU2018227883B2 (en) 2022-08-04
IL268600A (en) 2019-09-26
KR20190122245A (en) 2019-10-29
RU2019127780A (en) 2021-04-05
CN110352353B (en) 2023-12-15
WO2018160975A1 (en) 2018-09-07
KR102590519B1 (en) 2023-10-17
US20200018752A1 (en) 2020-01-16
CN110352353A (en) 2019-10-18
RU2019127780A3 (en) 2021-12-22

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