SG11201808354PA - Column-based fully scalable raav manufacturing process - Google Patents

Column-based fully scalable raav manufacturing process

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Publication number
SG11201808354PA
SG11201808354PA SG11201808354PA SG11201808354PA SG11201808354PA SG 11201808354P A SG11201808354P A SG 11201808354PA SG 11201808354P A SG11201808354P A SG 11201808354PA SG 11201808354P A SG11201808354P A SG 11201808354PA SG 11201808354P A SG11201808354P A SG 11201808354PA
Authority
SG
Singapore
Prior art keywords
international
rule
aiex
avb
pennsylvania
Prior art date
Application number
SG11201808354PA
Inventor
Guang Qu
Younghoon Oh
Lin Lu
John Fraser Wright
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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.)
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Publication date
Application filed by Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of SG11201808354PA publication Critical patent/SG11201808354PA/en

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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D15/00Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08Selective adsorption, e.g. chromatography
    • B01D15/26Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/34Size selective separation, e.g. size exclusion chromatography, gel filtration, permeation
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D15/00Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08Selective adsorption, e.g. chromatography
    • B01D15/26Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/36Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction
    • B01D15/361Ion-exchange
    • B01D15/362Cation-exchange
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D15/00Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08Selective adsorption, e.g. chromatography
    • B01D15/26Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/36Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction
    • B01D15/361Ion-exchange
    • B01D15/363Anion-exchange
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D15/00Separating processes involving the treatment of liquids with solid sorbents; Apparatus therefor
    • B01D15/08Selective adsorption, e.g. chromatography
    • B01D15/26Selective adsorption, e.g. chromatography characterised by the separation mechanism
    • B01D15/38Selective adsorption, e.g. chromatography characterised by the separation mechanism involving specific interaction not covered by one or more of groups B01D15/265 - B01D15/36
    • B01D15/3804Affinity chromatography
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D61/00Processes of separation using semi-permeable membranes, e.g. dialysis, osmosis or ultrafiltration; Apparatus, accessories or auxiliary operations specially adapted therefor
    • B01D61/14Ultrafiltration; Microfiltration
    • B01D61/145Ultrafiltration
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N1/00Microorganisms, e.g. protozoa; Compositions thereof; Processes of propagating, maintaining or preserving microorganisms or compositions thereof; Processes of preparing or isolating a composition containing a microorganism; Culture media therefor
    • C12N1/06Lysis of microorganisms
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • BPERFORMING OPERATIONS; TRANSPORTING
    • B01PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01DSEPARATION
    • B01D2315/00Details relating to the membrane module operation
    • B01D2315/16Diafiltration
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Water Supply & Treatment (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • Public Health (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 11111111011110111010101111101011111011101011111001011111111001111101111011111 International Bureau ... ..... ..)d (10) International Publication Number (43) International Publication Date ..... .....::„I WO 2017/173283 Al 5 October 2017 (05.10.2017) IN I PO I P CT (51) International Patent Classification: (US). WRIGHT, John Fraser; 68 River Birch Circle, A61K 38/46 (2006.01) Cl 2N 15/86 (2006.01) Princeton, New Jersey 08540 (US). (21) International Application Number: (74) Agent: BEDGOOD, Robert M.; Pillsbury Winthrop Shaw PCT/US2017/025396 Pittman LLP, P.O. Box 10500, McLean, Virginia 22102 (US). (22) International Filing Date: 31 March 2017 (31.03.2017) (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (25) Filing Language: English AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (26) Publication Language: English BZ, DO, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (30) Priority Data: HN, 62/316,252 31 March 2016 (31.03.2016) US KP, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, (71) Applicant: SPARK THERAPEUTICS, INC. [US/US]; MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, 3737 Market Street, Suite 1300, Philadelphia, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, Pennsylvania 19104 (US). RU, TH, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (72) Inventors: QU, Guang; 22 Beaver Dam Drive, Sick- ZA, ZM, ZW. lerville, New Jersey 08081 (US). OH, Younghoon; 107 Lowrys Lane, Bryn Mawr, Pennsylvania 19010 (US). LU, (84) Designated States (unless otherwise indicated, for every Lin; 439 Robin Hill Road, Wayne, Pennsylvania 19087 kind GM, of regional protection available): ARIPO (BW, GH, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, [Continued on next page] (54) Title: COLUMN-BASED FULLY SCALABLE RAAV MANUFACTURING PROCESS (57) : In accordance with the invention, provided FIG. 3 herein are methods for purifying recombinant adeno-associ- ated (rAAV) vector particles. All column purification process 1 2 3 4 98 62 vpl vp2 vp3 49 1. AVB 2. AVB > CIEX > AIEX Il 3. AVB AIEX > 4. CIEX > UF/DF > SEC > en ot AIEX (50-60 M ei 1 7 50-60% vector recovery IN 1-1 IN 1-1 0 ei 0 WO 2017/173283 Al MIDEDIMOMOIDEIRMEMOMMEINIMMOOMEHOIS TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, — as to the applicant's entitlement to claim the priority of 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, — the earlier application (Rule 4.17(iii)) of inventorship (Rule 4.17(iv)) SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, Published: GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). — with international search report Declarations under Rule 4.17: (Art. 21(3)) — as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii))
SG11201808354PA 2016-03-31 2017-03-31 Column-based fully scalable raav manufacturing process SG11201808354PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662316252P 2016-03-31 2016-03-31
PCT/US2017/025396 WO2017173283A1 (en) 2016-03-31 2017-03-31 Column-based fully scalable raav manufacturing process

Publications (1)

Publication Number Publication Date
SG11201808354PA true SG11201808354PA (en) 2018-10-30

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ID=59966426

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SG11201808354PA SG11201808354PA (en) 2016-03-31 2017-03-31 Column-based fully scalable raav manufacturing process

Country Status (24)

Country Link
US (1) US11702639B2 (en)
EP (1) EP3436051B1 (en)
JP (2) JP7364306B2 (en)
KR (1) KR102405250B1 (en)
CN (1) CN109152821A (en)
AU (1) AU2017240721B2 (en)
BR (1) BR112018070256A2 (en)
CA (1) CA3019133A1 (en)
CL (1) CL2018002754A1 (en)
CO (1) CO2018011151A2 (en)
DK (1) DK3436051T3 (en)
ES (1) ES2896080T3 (en)
HU (1) HUE056854T2 (en)
IL (1) IL261970A (en)
LT (1) LT3436051T (en)
MY (1) MY195391A (en)
PE (1) PE20190434A1 (en)
PH (1) PH12018502104A1 (en)
PL (1) PL3436051T3 (en)
PT (1) PT3436051T (en)
RU (1) RU2754467C2 (en)
SA (1) SA518400146B1 (en)
SG (1) SG11201808354PA (en)
WO (1) WO2017173283A1 (en)

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MX2017005834A (en) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease.
RU2749882C2 (en) 2014-11-14 2021-06-18 Вояджер Терапьютикс, Инк. Modulating polynucleotides
EP3218484A4 (en) 2014-11-14 2018-05-30 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
BR112018070256A2 (en) * 2016-03-31 2019-01-29 Spark Therapeutics Inc fully scalable column-based raav manufacturing process
RU2758488C2 (en) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Modulating polynucleotides
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
KR102669561B1 (en) * 2017-06-30 2024-05-24 스파크 테라퓨틱스, 인코포레이티드 AAV vector column purification method
IL314692A (en) * 2017-11-08 2024-10-01 Novartis Ag Means and method for preparing viral vectors and uses of same
WO2019209632A1 (en) 2018-04-24 2019-10-31 Merck Sharp & Dohme Corp. Scalable chromatography process for purification of human cytomegalovirus
JPWO2022045055A1 (en) * 2020-08-24 2022-03-03
WO2023077085A2 (en) * 2021-10-29 2023-05-04 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus

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Publication number Publication date
CL2018002754A1 (en) 2019-02-01
PL3436051T3 (en) 2022-02-07
EP3436051B1 (en) 2021-07-28
SA518400146B1 (en) 2021-10-19
IL261970A (en) 2018-10-31
RU2018138164A3 (en) 2020-06-16
US11702639B2 (en) 2023-07-18
JP2022120011A (en) 2022-08-17
BR112018070256A2 (en) 2019-01-29
JP2019509745A (en) 2019-04-11
RU2018138164A (en) 2020-04-30
JP7364306B2 (en) 2023-10-18
KR20190006951A (en) 2019-01-21
DK3436051T3 (en) 2021-11-01
HUE056854T2 (en) 2022-03-28
CA3019133A1 (en) 2017-10-05
AU2017240721A1 (en) 2018-10-11
CN109152821A (en) 2019-01-04
AU2017240721B2 (en) 2023-10-12
PE20190434A1 (en) 2019-03-21
WO2017173283A1 (en) 2017-10-05
KR102405250B1 (en) 2022-06-02
US20200299650A1 (en) 2020-09-24
PT3436051T (en) 2021-11-02
LT3436051T (en) 2021-11-25
ES2896080T3 (en) 2022-02-23
CO2018011151A2 (en) 2019-04-30
EP3436051A1 (en) 2019-02-06
RU2754467C2 (en) 2021-09-02
MY195391A (en) 2023-01-18
EP3436051A4 (en) 2019-10-16
PH12018502104A1 (en) 2019-07-29

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