SG11201804400SA - Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use - Google Patents

Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use

Info

Publication number
SG11201804400SA
SG11201804400SA SG11201804400SA SG11201804400SA SG11201804400SA SG 11201804400S A SG11201804400S A SG 11201804400SA SG 11201804400S A SG11201804400S A SG 11201804400SA SG 11201804400S A SG11201804400S A SG 11201804400SA SG 11201804400S A SG11201804400S A SG 11201804400SA
Authority
SG
Singapore
Prior art keywords
international
rule
free
methods
disclosed
Prior art date
Application number
SG11201804400SA
Inventor
Guang Qu
Lin Lu
John Fraser Wright
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 SG11201804400SA publication Critical patent/SG11201804400SA/en

Links

Classifications

    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/0018—Culture media for cell or tissue culture
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0603—Embryonic cells ; Embryoid bodies
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0684—Cells of the urinary tract or kidneys
    • C12N5/0686—Kidney cells
    • 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
    • C12Q1/70—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322—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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Reproductive Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -' Organization International Bureau rist0) (43) International Publication Date .... .....r ....) 8 June 2017 (08.06.2017) W I P0 1 PCT ID Hit (10) WO 11111111111111111111111111 International II 2017/096039 III 1111111111 Publication Ell III Number Al IIIIIIIIIItIIIIIIIIIIIIIII (51) International Patent Classification: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, C12N 5/10 (2006.01) A61K 48/00 (2006.01) HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, C12N 7/00 (2006.01) A61K 31/7088 (2006.01) KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, C12N 7/02 (2006.01) C12Q 1/70 (2006.01) MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, C12N 7/01 (2006.01) 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, (21) International Application Number: TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, PCT/US2016/064414 ZW. (22) International Filing Date: (84) Designated States (unless otherwise indicated, for every 1 December 2016 (01.12.2016) kind of regional protection available): ARIPO (BW, GH, (25) Filing Language: English GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, (26) Publication Language: English TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, (30) Priority Data: DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, 62/261,815 1 December 2015 (01.12.2015) US 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, (71) Applicant: SPARK THERAPEUTICS, INC. [US/US]; GW, KM, ML, MR, NE, SN, TD, TG). 3737 Market Street, Suite 1300, Philadelphia, Pennsylvania 19104 (US). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a (72) Inventors: QU, Guang; 22 Beaver Dam Drive, Sick- lerville, New Jersey 08081 (US). LU, Lin; 439 Robin Hill patent (Rule 4.170) Road, Wayne, Pennsylvania 19087 WRIGHT, John — as to the applicant's entitlement to claim the of the (US). priority Fraser; 68 River Birch Circle, Princeton, New Jersey earlier application (Rule 4.17(iii)) 08540 (US). — of inventorship (Rule 4.17(iv)) (74) Agent: BEDGOOD, Robert; Pillsbury Winthrop Shaw Published: Pittman LLP, P.O. Box 10500, McLean, Virginia 22102 (US). — with international search report (Art 21(3)) before the expiration of the time limit for amending the (81) Designated States (unless otherwise indicated, for every claims and to be republished in the event of receipt of kind of national available): AE, AG, AL, AM, protection 48.2(h)) AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, amendments (Rule BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, (54) Title: SCALABLE METHODS FOR PRODUCING RECOMBINANT ADENO-ASSOCIATED VIRAL (AAV) VECTOR IN SERUMSUSPENSION CELL CULTURE SYSTEM SUITABLE FOR CLINICAL USE = -FREE No free PEI Add 1/2 free PEI Add 1/3 free PEI , :,,,,* ';' ••;\ .4.!A. , 0 N..;,44.• k:4 1 / 4 c) re:::\t, , ,,` .x., „ ,• ..,:„---, ..t., ‘, ., .\\''''• , th: cr, o it-- 1-1 N (57) : Methods and compositions for transfecting compositions are disclosed in which transfection efficiency significantly increased by contacting the cells being transduced with Q polyethyleneimine (PEI) that is free of nucleic acid during vectors generated according to the disclosed methods and compositions are also disclosed. . is .:.\ FIG. cells the \ A' ‘ • I,VN \ , ' 4* N - \ \ \ - • \•: '' ' ., ':s •c•`,,,, \ s4 ,, 4, = ' , '. \ '''‘.; \ I :'+. ., %. • s , .. \ • ..•, \ . „.„\:.. .- - .:\ --,, , ,„:„ 3A with plasmids are disclosed. In certain embodiments, methods and transfection process. Therapeutically useful adeno-associated viral
SG11201804400SA 2015-12-01 2016-12-01 Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use SG11201804400SA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562261815P 2015-12-01 2015-12-01
PCT/US2016/064414 WO2017096039A1 (en) 2015-12-01 2016-12-01 Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use

Publications (1)

Publication Number Publication Date
SG11201804400SA true SG11201804400SA (en) 2018-06-28

Family

ID=58797705

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201804400SA SG11201804400SA (en) 2015-12-01 2016-12-01 Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
SG10202106287YA SG10202106287YA (en) 2015-12-01 2016-12-01 Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG10202106287YA SG10202106287YA (en) 2015-12-01 2016-12-01 Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use

Country Status (16)

Country Link
US (1) US20190292561A1 (en)
EP (1) EP3384015A4 (en)
JP (2) JP7444521B2 (en)
KR (1) KR20180091863A (en)
CN (1) CN108603174A (en)
AU (2) AU2016362317B2 (en)
BR (1) BR112018011193A2 (en)
CA (1) CA3006309A1 (en)
CO (1) CO2018006699A2 (en)
IL (1) IL259595B2 (en)
MX (2) MX2018006682A (en)
PE (2) PE20240371A1 (en)
PH (1) PH12018501168A1 (en)
RU (1) RU2766583C2 (en)
SG (2) SG11201804400SA (en)
WO (1) WO2017096039A1 (en)

Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
RU2020109343A (en) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. AADC POLINUCLEOTIDES FOR TREATMENT OF PARKINSON'S DISEASE
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
DK3218386T3 (en) 2014-11-14 2021-06-07 Voyager Therapeutics Inc MODULATORY POLYNUCLEOTID
MX2017006216A (en) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als).
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
BR112018011193A2 (en) * 2015-12-01 2018-11-21 Spark Therapeutics Inc scalable methods for producing recombinant adeno-associated viral vector (aav) in a serum-free suspension cell culture system suitable for clinical use
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN109831916B (en) 2016-05-18 2023-07-21 沃雅戈治疗公司 Compositions and methods for treating huntington's disease
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc Modulatory polynucleotides
CN110225975A (en) 2016-08-23 2019-09-10 阿库斯股份有限公司 For treating the composition and method of non-age-dependent hearing impairment in people experimenter
EP3831281A1 (en) 2016-08-30 2021-06-09 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
CN110913866A (en) 2017-05-05 2020-03-24 沃雅戈治疗公司 Compositions and methods for treating Amyotrophic Lateral Sclerosis (ALS)
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3068622A1 (en) * 2017-06-30 2019-01-03 Spark Therapeutics, Inc. Aav vector column purification methods
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7502991B2 (en) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド Treatment of amyotrophic lateral sclerosis (ALS)
CA3079568A1 (en) * 2017-10-18 2019-04-25 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
EP3720500A4 (en) * 2017-12-05 2021-08-25 Applied Genetic Technologies Corporation Formulation optimization for viral particles
JP7406677B2 (en) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド Viral vectors that evade antibodies
CN108866011A (en) * 2018-07-31 2018-11-23 深圳生生凡非基因技术有限公司 A kind of method of synchronous packaging rAAV
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CN110437317B (en) * 2019-01-30 2023-05-02 上海科技大学 Adeno-associated virus with variant capsid proteins and uses thereof
JP7305115B2 (en) * 2019-02-11 2023-07-10 エクセルジェネ エス．ア． A novel eukaryotic cell transformation system and related methods
TW202102525A (en) 2019-03-21 2021-01-16 美商史崔德生物公司 Recombinant adeno-associated virus vectors
WO2021028837A1 (en) 2019-08-13 2021-02-18 Waters Technologies Corporation Affinity resins and sample preparation devices based on cartilaginous fish ignar derived binding domains
TW202128736A (en) 2019-10-17 2021-08-01 美商史崔德生物公司 Adeno-associated viral vectors for treatment of niemann-pick disease type c
CN110894494B (en) * 2019-11-22 2022-09-27 广西梧州制药（集团）股份有限公司 Method for large-scale high-density suspension culture of 293 cell high-yield adenovirus
CN110938654A (en) * 2019-12-11 2020-03-31 和元生物技术（上海）股份有限公司 Cell transfection reagent and application thereof
CN115315518A (en) * 2020-01-17 2022-11-08 阿斯克肋匹奥生物制药公司 Production of recombinant AAV
WO2021168362A1 (en) 2020-02-21 2021-08-26 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2022043926A1 (en) * 2020-08-31 2022-03-03 Intas Pharmaceuticals Ltd. Process for preparation of recombinant adeno-associated virus particle
CN116406425A (en) 2020-10-15 2023-07-07 豪夫迈·罗氏有限公司 Nucleic acid constructs for VA RNA transcription
WO2022079082A1 (en) 2020-10-15 2022-04-21 F. Hoffmann-La Roche Ag Nucleic acid constructs for simultaneous gene activation
TW202309294A (en) * 2021-04-27 2023-03-01 瑞士商諾華公司 Viral vector production system
WO2023198685A1 (en) 2022-04-13 2023-10-19 F. Hoffmann-La Roche Ag Method for determining aav genomes
EP4279599A1 (en) * 2022-05-18 2023-11-22 Branca Bunus Limited Ordered formulation method to construct polyplex with core-aggregate structure
WO2023227438A1 (en) 2022-05-23 2023-11-30 F. Hoffmann-La Roche Ag Raman-based method for the differentiation of aav particle serotype and aav particle loading status
WO2023232922A1 (en) 2022-06-03 2023-12-07 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
WO2024013239A1 (en) 2022-07-14 2024-01-18 F. Hoffmann-La Roche Ag Method for producing recombinant aav particles
WO2024056561A1 (en) 2022-09-12 2024-03-21 F. Hoffmann-La Roche Ag Method for separating full and empty aav particles

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2995542A1 (en) * 1997-09-05 1999-03-11 Genzyme Corporation Methods for generating high titer helper-free preparations of 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
US6593123B1 (en) * 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US7829694B2 (en) * 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
RU2303064C2 (en) * 2005-04-06 2007-07-20 ГУ НИИ вирусных препаратов им. О.Г. Анджапаридзе РАМН Molecular complex for transfection of mammalian cells, containing plasmide dna, modified polyethyleneimine, and ligand molecules
US20150111955A1 (en) * 2012-02-17 2015-04-23 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
ES2681434T3 (en) * 2013-03-15 2018-09-13 The Children's Hospital Of Philadelphia Scalable manufacturing process to produce recombinant lentiviral vectors in a serum-free suspension cell culture system
WO2014201252A2 (en) * 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
CN103329852B (en) * 2013-06-19 2015-10-07 中国医学科学院病原生物学研究所 A kind of HBV persistent infection and fibrosis Establishment of mouse model
DK3013964T3 (en) * 2013-06-28 2020-06-15 Ethris Gmbh COMPOSITIONS FOR INTRODUCING RNA IN CELLS
SG10201809075XA (en) * 2013-07-22 2018-11-29 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
US10246560B2 (en) * 2013-08-13 2019-04-02 Baylor College Of Medicine PLGA-modified polyethylenimine self-assembly nanotechnology for nucleic acid and drug delivery
BR112018011193A2 (en) * 2015-12-01 2018-11-21 Spark Therapeutics Inc scalable methods for producing recombinant adeno-associated viral vector (aav) in a serum-free suspension cell culture system suitable for clinical use
WO2018226887A1 (en) * 2017-06-07 2018-12-13 Spark Therapeutics, Inc. ENHANCING AGENTS FOR IMPROVED CELL TRANSFECTION AND/OR rAAV VECTOR PRODUCTION

Also Published As

Publication number Publication date
IL259595B1 (en) 2023-09-01
PH12018501168A1 (en) 2019-01-21
KR20180091863A (en) 2018-08-16
IL259595A (en) 2018-07-31
IL259595B2 (en) 2024-01-01
JP2022071049A (en) 2022-05-13
RU2018123502A3 (en) 2020-04-20
AU2016362317B2 (en) 2023-03-16
CA3006309A1 (en) 2017-06-08
RU2766583C2 (en) 2022-03-15
PE20240371A1 (en) 2024-03-05
AU2016362317A1 (en) 2018-06-14
PE20181534A1 (en) 2018-09-26
MX2023012498A (en) 2023-11-03
JP2018535682A (en) 2018-12-06
RU2018123502A (en) 2020-01-14
EP3384015A1 (en) 2018-10-10
EP3384015A4 (en) 2019-05-29
BR112018011193A2 (en) 2018-11-21
JP7444521B2 (en) 2024-03-06
CO2018006699A2 (en) 2018-09-20
MX2018006682A (en) 2018-09-26
US20190292561A1 (en) 2019-09-26
WO2017096039A1 (en) 2017-06-08
SG10202106287YA (en) 2021-07-29
AU2023200992A1 (en) 2023-05-18
CN108603174A (en) 2018-09-28

Similar Documents

Publication Publication Date Title
SG11201804400SA (en) Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
SG11201909432SA (en) Compounds for increasing genome editing efficiency
SG11201808398PA (en) Cell line for recombinant protein and/or viral vector production
SG11201807489PA (en) Cells expressing multiple chimeric antigen receptor (car) molecules and uses therefore
SG11201906297QA (en) Nucleic acids encoding crispr-associated proteins and uses thereof
SG11201909868YA (en) Compositions and methods of treating huntington's disease
SG11201909955XA (en) Formulations of anti-lag3 antibodies and co-formulations of anti-lag3 antibodies and anti-pd-1 antibodies
SG11201907714UA (en) Adeno-associated virus (aav) clade f vector and uses therefor
SG11201804070XA (en) Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
SG11201901531TA (en) Regulation of gene expression using engineered nucleases
SG11201803593QA (en) Engineered nucleic-acid targeting nucleic acids
SG11201805320XA (en) Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
SG11201909777YA (en) Modulatory polynucleotides
SG11201809912UA (en) Hybrid carriers for nucleic acid cargo
SG11201809643UA (en) Compositions and methods of treating huntington's disease
SG11201803701YA (en) Methods and compositions for gene editing in hematopoietic stem cells
SG11201809700YA (en) Gdf15 fusion proteins and uses thereof
SG11201810327XA (en) Single domain serum albumin binding protein
SG11201808622SA (en) Chimeric receptors to flt3 and methods of use thereof
SG11201804729RA (en) Methods and compositions for treating a serpinc1-associated disorder
SG11201900447SA (en) Methods and compositions for modifying genomic dna
SG11201906147VA (en) Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences
SG11201407866XA (en) Method for expression of heterologous proteins using a recombinant negative-strand rna virus vector
SG11201408095XA (en) Fibroblast growth factor 21 proteins
SG11201811232XA (en) Treatment of amd using aav2 variant with aflibercept