AR120171A1 - Composición farmacéutica de vector de virus adenoasociado y métodos - Google Patents

Composición farmacéutica de vector de virus adenoasociado y métodos

Info

Publication number
AR120171A1
AR120171A1 ARP200102778A ARP200102778A AR120171A1 AR 120171 A1 AR120171 A1 AR 120171A1 AR P200102778 A ARP200102778 A AR P200102778A AR P200102778 A ARP200102778 A AR P200102778A AR 120171 A1 AR120171 A1 AR 120171A1
Authority
AR
Argentina
Prior art keywords
pharmaceutical composition
associated virus
adeno
methods
virus vector
Prior art date
Application number
ARP200102778A
Other languages
English (en)
Inventor
Tristan Marshall
Jared Bee
Kristin Oberry
Yu Zhang
Roberto Depaz
Original Assignee
Regenxbio 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 Regenxbio Inc filed Critical Regenxbio Inc
Publication of AR120171A1 publication Critical patent/AR120171A1/es

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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • 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
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • 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
    • A61K47/26—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
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5256—Virus expressing foreign proteins
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Inorganic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

Reivindicación 1: Una composición farmacéutica caracterizada porque comprende: (a) un virus adenoasociado recombinante (AAV), (b) cloruro de potasio, (c) fosfato de potasio monobásico, (d) cloruro de sodio, (e) fosfato de sodio dibásico anhidro, (f) sacarosa, y (e) poloxámero 188, polisorbato 20, o polisorbato 80. Reivindicación 3: La composición farmacéutica de acuerdo con cualquiera de las reivindicaciones 1 - 2, caracterizada porque el AAV recombinante es AAV8.
ARP200102778A 2019-10-07 2020-10-07 Composición farmacéutica de vector de virus adenoasociado y métodos AR120171A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962911968P 2019-10-07 2019-10-07

Publications (1)

Publication Number Publication Date
AR120171A1 true AR120171A1 (es) 2022-02-02

Family

ID=73131798

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200102778A AR120171A1 (es) 2019-10-07 2020-10-07 Composición farmacéutica de vector de virus adenoasociado y métodos

Country Status (13)

Country Link
US (1) US20240108669A1 (es)
EP (1) EP4041292A1 (es)
JP (1) JP2022552262A (es)
KR (1) KR20220081365A (es)
CN (1) CN114728049A (es)
AR (1) AR120171A1 (es)
AU (1) AU2020362119A1 (es)
BR (1) BR112022006718A2 (es)
CA (1) CA3156984A1 (es)
IL (1) IL291930A (es)
MX (1) MX2022004146A (es)
TW (1) TW202126319A (es)
WO (1) WO2021071835A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021506861A (ja) 2017-12-19 2021-02-22 アコーオス インコーポレイテッド 内耳への治療用抗体のａａｖ媒介送達
CN116710149A (zh) * 2020-12-18 2023-09-05 桑格摩生物治疗股份有限公司 含有腺相关病毒载体的改善的药物组合物
US20230293724A1 (en) * 2022-03-14 2023-09-21 Regeneron Pharmaceuticals, Inc. Lyophilized Formulations of AAV Drug Products
TW202404651A (zh) * 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
WO2024073669A1 (en) 2022-09-30 2024-04-04 Regenxbio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
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
CN115869425B (zh) * 2022-12-12 2024-08-20 北京生物制品研究所有限责任公司 一种aav眼用注射液及其制备方法和应用
WO2024138129A2 (en) * 2022-12-23 2024-06-27 Spark Therapeutics, Inc. Adeno-associated virus formulations

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994018317A1 (en) 1993-02-12 1994-08-18 The Board Of Trustees Of The Leland Stanford Junior University Regulated transcription of targeted genes and other biological events
ATE544776T1 (de) 1994-12-29 2012-02-15 Massachusetts Inst Technology Chimäre dna-bindeproteine
WO1996041865A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamcycin-based regulation of biological events
JP2003524368A (ja) 1997-08-26 2003-08-19 アリアド ジーン セラピューティクス インコーポレイテッド ニ量化ドメイン、三量化ドメインまたは四量化ドメインおよび補足的非相同転写活性化ドメイン、転写抑制ドメイン、ｄｎａ結合ドメインまたはリガンド結合ドメインを含む融合蛋白
WO1999010508A1 (en) 1997-08-27 1999-03-04 Ariad Gene Therapeutics, Inc. Chimeric transcriptional activators and compositions and uses related thereto
WO1999036553A2 (en) 1998-01-15 1999-07-22 Ariad Gene Therapeutics, Inc. Regulation of biological events using multimeric chimeric proteins
AU766513B2 (en) 1998-02-13 2003-10-16 Board Of Trustees Of The Leland Stanford Junior University Novel dimerizing agents, their production and use
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
NZ578982A (en) 2001-11-13 2011-03-31 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
CA2569244C (en) 2004-06-01 2017-02-14 Avigen, Inc. Compositions and methods to prevent aav vector aggregation
US8999678B2 (en) 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
JP5704361B2 (ja) 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
EP4234571A3 (en) 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
RS62795B1 (sr) 2011-04-22 2022-02-28 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
WO2013029030A1 (en) 2011-08-24 2013-02-28 The Board Of Trustees Of The Leland Stanford Junior University New aav capsid proteins for nucleic acid transfer
US9677088B2 (en) 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
JP2016514152A (ja) 2013-03-13 2016-05-19 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア アデノ随伴ウイルスベクターおよびその使用の方法
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
CA3182790A1 (en) 2013-10-11 2015-04-16 Massachusetts Eye & Ear Infirmary Ancestral adeno-associated virus sequences and uses thereof
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
US10064752B2 (en) 2014-09-11 2018-09-04 Orbit Biomedical Limited Motorized suprachoroidal injection of therapeutic agent
KR102574810B1 (ko) 2016-04-15 2023-09-08 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 습성 연령 관련 황반 변성의 치료를 위한 조성물
US20190127455A1 (en) 2016-04-15 2019-05-02 Regenxbio Inc. Treatment of Ocular Diseases with Fully- Human Post-Translationally Modified Anti-VEGF Fab
EP4083203B1 (en) * 2016-06-16 2024-08-07 Adverum Biotechnologies, Inc. Compositions and methods for reducing ocular neovascularization
AU2018342094A1 (en) 2017-09-27 2020-04-02 Regenxbio Inc. Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF Fab
BR112021009370A2 (pt) * 2018-11-14 2021-08-17 Regenxbio Inc. método de tratamento da doença de batten cln2, composição farmacêutica e kit

Also Published As

Publication number Publication date
KR20220081365A (ko) 2022-06-15
JP2022552262A (ja) 2022-12-15
TW202126319A (zh) 2021-07-16
US20240108669A1 (en) 2024-04-04
WO2021071835A1 (en) 2021-04-15
CA3156984A1 (en) 2021-04-15
CN114728049A (zh) 2022-07-08
BR112022006718A2 (pt) 2022-07-12
MX2022004146A (es) 2022-09-19
IL291930A (en) 2022-06-01
AU2020362119A1 (en) 2022-05-26
EP4041292A1 (en) 2022-08-17

Similar Documents

Publication Publication Date Title
AR120171A1 (es) Composición farmacéutica de vector de virus adenoasociado y métodos
PE20241346A1 (es) Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones que los comprenden
MX2019005088A (es) Formulaciones de virus adeno-asociados.
CO2017013231A2 (es) Composiciones que comprenden una cepa bacteriana del género parabacteroides
AR127361A2 (es) Composición farmacéutica de partículas de lipoplejos y arn, composición acuosa y método de preparación
EA201890355A1 (ru) Новые способы индукции иммунного ответа
EA201791936A8 (ru) Рекомбинантные конструкции glut1 аденоассоциированного вирусного вектора и способы восстановления экспрессии glut1 на их основе
AR074958A2 (es) Composicion farmaceutica liofilizada de anticuerpos monoclonales o policlonales y procedimiento para su produccion
MX2018013215A (es) Composicion farmaceutica.
BR112018075785A2 (pt) formulação de vacina para o hiv
CU20170176A7 (es) Composición farmacéutica que comprende péptido tipo apl
HRP20220011T1 (hr) Nova stabilna formulacija za fxia protutijela
AR108631A1 (es) Formulación de neurotoxinas
CO2020016147A2 (es) Composiciones de virus adenoasociado (aav)
CO2020013552A2 (es) Formulación de anticuerpos
BR112022006926A2 (pt) Vetor de vírus oncolítico que codifica o polipeptídeo variante de interleucina 2 (vil-2)
PE20220570A1 (es) Anticuerpo anti-epha4 humano
MX2023006471A (es) Composiciones farmaceuticas mejoradas que contienen vector viral adenoasociado.
AR086672A1 (es) Formulaciones de furina recombinante
AR126417A1 (es) Composición farmacéutica de virus no encapsulado
BR112022005615A2 (pt) Peptídeos de staphylococcus e métodos de uso
MX2020001851A (es) Composiciones farmaceuticas y regimenes de dosificacion que contienen anticuerpos anti-alfa(v)beta(6).
AR107430A1 (es) Mutantes de proteína f de rsv (virus sincicial respiratorio)
MX2022006085A (es) Formulaciones parenterales estables de duloxetina.
AR106131A1 (es) Vectores del factor viii del virus adenoasociado, partículas víricas asociadas y formulaciones terapéuticas que las comprenden, método