CA3104196A1 - Aav compositions - Google Patents

Aav compositions Download PDF

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Publication number
CA3104196A1
CA3104196A1 CA3104196A CA3104196A CA3104196A1 CA 3104196 A1 CA3104196 A1 CA 3104196A1 CA 3104196 A CA3104196 A CA 3104196A CA 3104196 A CA3104196 A CA 3104196A CA 3104196 A1 CA3104196 A1 CA 3104196A1
Authority
CA
Canada
Prior art keywords
composition
certain embodiments
aav
formulation
months
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3104196A
Other languages
English (en)
French (fr)
Inventor
Eva FRITSCHER
Christian Fiedler
Tanvir TABISH
Dominik MITTERGRADNEGGER
Stefan REUBERGER
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Takeda Pharmaceutical Co Ltd
Original Assignee
Baxalta GmbH
Baxalta 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 Baxalta GmbH, Baxalta Inc filed Critical Baxalta GmbH
Publication of CA3104196A1 publication Critical patent/CA3104196A1/en
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Dermatology (AREA)
  • Psychiatry (AREA)
  • Hematology (AREA)
  • Inorganic Chemistry (AREA)
  • Diabetes (AREA)
  • Hospice & Palliative Care (AREA)
CA3104196A 2018-07-11 2019-07-11 Aav compositions Pending CA3104196A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862696792P 2018-07-11 2018-07-11
US62/696,792 2018-07-11
PCT/US2019/041398 WO2020014479A1 (en) 2018-07-11 2019-07-11 Aav compositions

Publications (1)

Publication Number Publication Date
CA3104196A1 true CA3104196A1 (en) 2020-01-16

Family

ID=69142590

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3104196A Pending CA3104196A1 (en) 2018-07-11 2019-07-11 Aav compositions

Country Status (13)

Country Link
US (1) US12553062B2 (https=)
EP (1) EP3820490A4 (https=)
JP (1) JP2021532071A (https=)
KR (1) KR20210032984A (https=)
CN (1) CN112469427A (https=)
AU (1) AU2019299964A1 (https=)
BR (1) BR112021000327A2 (https=)
CA (1) CA3104196A1 (https=)
CO (1) CO2020016147A2 (https=)
IL (1) IL279537A (https=)
MX (1) MX2020013953A (https=)
SG (1) SG11202012946RA (https=)
WO (1) WO2020014479A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012018628A1 (en) 2010-07-26 2012-02-09 Board Of Regents, The University Of Texas System Methods for inducing an immune response via buccal and/or sublingual administration of a vaccine
US9974850B2 (en) 2013-01-25 2018-05-22 Board Of Regents, The University Of Texas System Immunogenic compositions and uses thereof
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
AU2021265768B2 (en) * 2020-04-27 2023-03-30 4D Molecular Therapeutics Inc. Adeno-associated variants, formulations and methods for pulmonary delivery
WO2022236008A1 (en) * 2021-05-07 2022-11-10 Board Of Regents, The University Of Texas System Methods and compositions for transport, storage, and delivery of adeno-associated viral vector and other molecules
CN117980471A (zh) 2021-08-04 2024-05-03 武田药品工业株式会社 阳离子交换剂上的腺相关病毒分离
US20230090654A1 (en) * 2021-08-24 2023-03-23 Homology Medicines, Inc. Adeno-associated virus formulations
CN116350801A (zh) * 2022-11-22 2023-06-30 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
WO2025076729A1 (zh) * 2023-10-11 2025-04-17 朗信启昇(苏州)生物制药有限公司 包含aav的药物组合物及其制备方法

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8801338D0 (en) 1988-01-21 1988-02-17 Quadrant Bioresources Ltd Preservation of viruses
EP0728195A1 (en) 1993-10-12 1996-08-28 Chiron Corporation Methods for preserving recombinant viruses
PT1061955E (pt) 1998-03-13 2005-08-31 Wyeth Corp Composicao de polinucleotido, metodo de preparacao e sua utilizacao
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
JP5118798B2 (ja) 2000-03-07 2013-01-16 メルク・シャープ・エンド・ドーム・コーポレイション アデノウイルス製剤
US20030153065A1 (en) 2002-01-14 2003-08-14 Genvec, Inc. Composition and method for maintaining non-enveloped viral vectors
BRPI0511764B8 (pt) 2004-06-01 2021-05-25 Avigen Inc método de prevenção de agregação de vírions de vírus adeno-associado recombinante (raav) em uma preparação purificada de virions raav
CA2722238C (en) 2008-04-22 2017-11-28 Life Sciences Research Partners Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
PL4218797T3 (pl) * 2009-09-21 2025-02-10 Takeda Pharmaceutical Company Limited Stabilizowane płynne i liofilizowane preparaty adamts13
IT1405793B1 (it) 2010-10-15 2014-01-24 Univ Ferrara Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica
EP2646051A4 (en) * 2010-12-02 2014-05-28 Oncolytics Biotech Inc FORMULATIONS OF LIQUID VIRUSES
MX349294B (es) 2010-12-02 2017-07-21 Oncolytics Biotech Inc Formulaciones virales liofilizadas.
WO2013036961A1 (en) 2011-09-09 2013-03-14 Api Genesis Llc A pain relief composition, comprising a trpv1 selective agonist, and manufacture and uses thereof
US10093947B2 (en) 2012-02-28 2018-10-09 Cornell University AAV-directed persistent expression of an anti-nicotine antibody gene for smoking cessation
US9254332B2 (en) 2013-03-15 2016-02-09 Arecor Limited Stable aqueous formulations of adenovirus vectors
WO2015040234A1 (en) * 2013-09-23 2015-03-26 Crucell Holland B.V. Adenovirus formulations
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2016004319A1 (en) 2014-07-02 2016-01-07 University Of Florida Research Foundation, Inc. Compositions and methods for purifying recombinant adeno-associated virus
WO2016154055A1 (en) 2015-03-20 2016-09-29 Bluebird Bio, Inc. Vector formulations
EP3384015A4 (en) * 2015-12-01 2019-05-29 Spark Therapeutics, Inc. SCALABLE PROCESS FOR THE PREPARATION OF A RECOMBINANT ADENO ASSOCIATED VIRAL VECTOR IN A SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR THE CLINICAL APPLICATION
BR112019009113A2 (pt) * 2016-11-04 2019-07-16 Baxalta Incorporated formulações de vírus adenoassociado
CA3040288A1 (en) * 2016-11-04 2018-07-12 Baxalta Incorporated Adeno-associated virus purification methods
JP2021505610A (ja) * 2017-12-05 2021-02-18 アプライド ジェネティック テクノロジーズ コーポレイション ウイルス粒子のための製剤最適化
EP3731863A4 (en) * 2017-12-29 2021-10-27 Baxalta Incorporated ADENO-ASSOCIATED VIRUS CLEANSING PROCEDURES

Also Published As

Publication number Publication date
WO2020014479A1 (en) 2020-01-16
US20210163990A1 (en) 2021-06-03
CO2020016147A2 (es) 2021-01-29
EP3820490A4 (en) 2022-06-08
US12553062B2 (en) 2026-02-17
MX2020013953A (es) 2021-03-09
BR112021000327A2 (pt) 2021-04-06
SG11202012946RA (en) 2021-02-25
EP3820490A1 (en) 2021-05-19
JP2021532071A (ja) 2021-11-25
AU2019299964A1 (en) 2021-01-21
IL279537A (en) 2021-01-31
KR20210032984A (ko) 2021-03-25
CN112469427A (zh) 2021-03-09

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