PL3436051T3 - W pełni skalowalny sposób kolumnowego wytwarzania raav - Google Patents

W pełni skalowalny sposób kolumnowego wytwarzania raav

Info

Publication number
PL3436051T3
PL3436051T3 PL17776783T PL17776783T PL3436051T3 PL 3436051 T3 PL3436051 T3 PL 3436051T3 PL 17776783 T PL17776783 T PL 17776783T PL 17776783 T PL17776783 T PL 17776783T PL 3436051 T3 PL3436051 T3 PL 3436051T3
Authority
PL
Poland
Prior art keywords
column
manufacturing process
fully scalable
based fully
raav manufacturing
Prior art date
Application number
PL17776783T
Other languages
English (en)
Polish (pl)
Inventor
Guang Qu
Younghoon Oh
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 PL3436051T3 publication Critical patent/PL3436051T3/pl

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • B01D15/34—Size-selective separation, e.g. size-exclusion chromatography; Gel filtration; Permeation
    • 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
    • B01D15/36—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
    • B01D15/361—Ion-exchange
    • B01D15/362—Cation-exchange
    • 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
    • B01D15/36—Selective adsorption, e.g. chromatography characterised by the separation mechanism involving ionic interaction, e.g. ion-exchange, ion-pair, ion-suppression or ion-exclusion
    • B01D15/361—Ion-exchange
    • B01D15/363—Anion-exchange
    • 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 and B01D15/30 - B01D15/36, e.g. affinity, ligand exchange or chiral chromatography
    • B01D15/3804—Affinity chromatography
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D61/00—Processes of separation using semi-permeable membranes, e.g. dialysis, osmosis or ultrafiltration; Apparatus, accessories or auxiliary operations specially adapted therefor
    • B01D61/14—Ultrafiltration; Microfiltration
    • B01D61/145—Ultrafiltration
    • 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
    • C12N1/00—Microorganisms; 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
    • C12N1/06—Lysis of microorganisms
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01D—SEPARATION
    • B01D2315/00—Details relating to the membrane module operation
    • B01D2315/16—Diafiltration
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Water Supply & Treatment (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL17776783T 2016-03-31 2017-03-31 W pełni skalowalny sposób kolumnowego wytwarzania raav PL3436051T3 (pl)

Applications Claiming Priority (3)

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
EP17776783.7A EP3436051B1 (en) 2016-03-31 2017-03-31 Column-based fully scalable raav manufacturing process

Publications (1)

Publication Number Publication Date
PL3436051T3 true PL3436051T3 (pl) 2022-02-07

Family

ID=59966426

Family Applications (1)

Application Number Title Priority Date Filing Date
PL17776783T PL3436051T3 (pl) 2016-03-31 2017-03-31 W pełni skalowalny sposób kolumnowego wytwarzania raav

Country Status (23)

Country Link
US (1) US11702639B2 (enExample)
EP (1) EP3436051B1 (enExample)
JP (2) JP7364306B2 (enExample)
KR (1) KR102405250B1 (enExample)
CN (1) CN109152821A (enExample)
AU (1) AU2017240721B2 (enExample)
CA (1) CA3019133A1 (enExample)
CL (1) CL2018002754A1 (enExample)
CO (1) CO2018011151A2 (enExample)
DK (1) DK3436051T3 (enExample)
ES (1) ES2896080T3 (enExample)
HU (1) HUE056854T2 (enExample)
IL (1) IL261970B2 (enExample)
LT (1) LT3436051T (enExample)
MY (1) MY195391A (enExample)
PE (1) PE20190434A1 (enExample)
PH (1) PH12018502104B1 (enExample)
PL (1) PL3436051T3 (enExample)
PT (1) PT3436051T (enExample)
RU (1) RU2754467C2 (enExample)
SA (1) SA518400146B1 (enExample)
SG (1) SG11201808354PA (enExample)
WO (1) WO2017173283A1 (enExample)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
PH12018502104B1 (en) * 2016-03-31 2024-03-27 Spark Therapeutics Inc Column-based fully scalable raav manufacturing process
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2018044912A1 (en) 2016-08-29 2018-03-08 Wayne State University Identification of mutations in channelopsin variants having improved light sensitivity and methods of use thereof
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3658250A4 (en) * 2017-06-30 2021-10-27 Spark Therapeutics, Inc. AAV VECTOR COLUMN CLEANING PROCEDURE
EP4585229A3 (en) * 2017-11-08 2026-01-14 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
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
JP7478675B2 (ja) 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US20220267796A1 (en) * 2019-06-28 2022-08-25 Takeda Pharmaceutical Company Limited Adeno-associated virus purification methods
WO2021207132A1 (en) * 2020-04-06 2021-10-14 Brown University Formulations and processes for treating water from impacted sources
WO2022045055A1 (ja) * 2020-08-24 2022-03-03 タカラバイオ株式会社 ｐＨの違いによる非エンベロープウイルスベクター粒子の調製方法
WO2023077085A2 (en) * 2021-10-29 2023-05-04 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
AU8200191A (en) 1990-07-09 1992-02-04 United States of America, as represented by the Secretary, U.S. Department of Commerce, The High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
ATE237694T1 (de) 1991-08-20 2003-05-15 Us Gov Health & Human Serv Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
US5981716A (en) * 1995-06-07 1999-11-09 Gruppo Lepettit, S.P.A. Process for the purification of proteins
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
CA2995542A1 (en) * 1997-09-05 1999-03-11 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
AU2001257611A1 (en) 2000-04-28 2001-11-12 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
JP5956331B2 (ja) 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えａａｖベクターの改良された精製方法
EP2325194A1 (en) * 2009-11-24 2011-05-25 Glycotope GmbH Process for the purification of glycoproteins
EP2529020B1 (en) 2010-01-28 2018-04-25 The Children's Hospital of Philadelphia A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
WO2013050523A1 (en) * 2011-10-05 2013-04-11 Molmed Spa Viral vectors purification system
PH12018502104B1 (en) 2016-03-31 2024-03-27 Spark Therapeutics Inc Column-based fully scalable raav manufacturing process

Also Published As

Publication number Publication date
KR102405250B1 (ko) 2022-06-02
JP7364306B2 (ja) 2023-10-18
SA518400146B1 (ar) 2021-10-19
PT3436051T (pt) 2021-11-02
HUE056854T2 (hu) 2022-03-28
CA3019133A1 (en) 2017-10-05
CN109152821A (zh) 2019-01-04
CO2018011151A2 (es) 2019-04-30
EP3436051B1 (en) 2021-07-28
MY195391A (en) 2023-01-18
ES2896080T3 (es) 2022-02-23
IL261970B1 (en) 2025-06-01
US20200299650A1 (en) 2020-09-24
PE20190434A1 (es) 2019-03-21
PH12018502104A1 (en) 2019-07-29
EP3436051A1 (en) 2019-02-06
RU2018138164A3 (enExample) 2020-06-16
IL261970A (en) 2018-10-31
DK3436051T3 (da) 2021-11-01
PH12018502104B1 (en) 2024-03-27
SG11201808354PA (en) 2018-10-30
BR112018070256A2 (pt) 2019-01-29
KR20190006951A (ko) 2019-01-21
US11702639B2 (en) 2023-07-18
LT3436051T (lt) 2021-11-25
RU2754467C2 (ru) 2021-09-02
JP7659523B2 (ja) 2025-04-09
JP2019509745A (ja) 2019-04-11
AU2017240721A1 (en) 2018-10-11
RU2018138164A (ru) 2020-04-30
NZ747126A (en) 2025-05-02
IL261970B2 (en) 2025-10-01
EP3436051A4 (en) 2019-10-16
CL2018002754A1 (es) 2019-02-01
JP2022120011A (ja) 2022-08-17
WO2017173283A1 (en) 2017-10-05
AU2017240721B2 (en) 2023-10-12

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