CA3148376A1 - Methods of treating retinal neovascular diseases using aav2 variants encoding aflibercept________________________________ - Google Patents

Methods of treating retinal neovascular diseases using aav2 variants encoding aflibercept________________________________ Download PDF

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Publication number
CA3148376A1
CA3148376A1 CA3148376A CA3148376A CA3148376A1 CA 3148376 A1 CA3148376 A1 CA 3148376A1 CA 3148376 A CA3148376 A CA 3148376A CA 3148376 A CA3148376 A CA 3148376A CA 3148376 A1 CA3148376 A1 CA 3148376A1
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Prior art keywords
eye
unit dose
raav particles
weeks
administration
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Pending
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CA3148376A
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English (en)
French (fr)
Inventor
Mehdi Gasmi
Szilard Kiss
Aaron Osborne
Adam TURPCU
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Adverum Biotechnologies Inc
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Adverum Biotechnologies Inc
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Priority claimed from PCT/US2019/062066 external-priority patent/WO2021050094A1/en
Application filed by Adverum Biotechnologies Inc filed Critical Adverum Biotechnologies Inc
Publication of CA3148376A1 publication Critical patent/CA3148376A1/en
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/179Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/56Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/56Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
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    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
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    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
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    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
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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
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    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14111Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141Use of virus, viral particle or viral elements as a vector
    • C12N2710/14144Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Cell Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
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  • Mycology (AREA)
  • Inorganic Chemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3148376A 2019-09-11 2020-09-10 Methods of treating retinal neovascular diseases using aav2 variants encoding aflibercept________________________________ Pending CA3148376A1 (en)

Applications Claiming Priority (17)

Application Number Priority Date Filing Date Title
US201962899070P 2019-09-11 2019-09-11
US62/899,070 2019-09-11
US201962913648P 2019-10-10 2019-10-10
US62/913,648 2019-10-10
PCT/US2019/062066 WO2021050094A1 (en) 2019-09-11 2019-11-18 Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
USPCT/US2019/062066 2019-11-18
US202062959784P 2020-01-10 2020-01-10
US62/959,784 2020-01-10
US202062971835P 2020-02-07 2020-02-07
US62/971,835 2020-02-07
US202063019190P 2020-05-01 2020-05-01
US63/019,190 2020-05-01
US202063030819P 2020-05-27 2020-05-27
US63/030,819 2020-05-27
US202063063203P 2020-08-07 2020-08-07
US63/063,203 2020-08-07
PCT/US2020/050079 WO2021050649A1 (en) 2019-09-11 2020-09-10 Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept

Publications (1)

Publication Number Publication Date
CA3148376A1 true CA3148376A1 (en) 2021-03-18

Family

ID=72644941

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3148376A Pending CA3148376A1 (en) 2019-09-11 2020-09-10 Methods of treating retinal neovascular diseases using aav2 variants encoding aflibercept________________________________

Country Status (10)

Country Link
EP (1) EP4027983A1 (es)
JP (1) JP2022547541A (es)
KR (1) KR20220062353A (es)
CN (1) CN114375195A (es)
AU (1) AU2020345915A1 (es)
BR (1) BR112022004027A2 (es)
CA (1) CA3148376A1 (es)
IL (1) IL291182A (es)
MX (1) MX2022002961A (es)
WO (1) WO2021050649A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023540464A (ja) * 2020-07-21 2023-09-25 インスピラール リミテッド 眼疾患の処置のための組成物および方法
US20240197915A1 (en) 2021-04-27 2024-06-20 Adverum Biotechnologies, Inc. Methods of treating ocular diseases using aav2 variants encoding aflibercept
EP4349852A1 (en) * 2021-05-28 2024-04-10 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
WO2023028457A1 (en) * 2021-08-23 2023-03-02 Duke University Compositions for and methods of long-term integration of circuits using connexins
AU2023221321A1 (en) * 2022-02-16 2024-09-26 Adverum Biotechnologies, Inc. Method of reducing cst fluctuation in neovascular amd by a recombinant adeno-associated virus

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG194583A1 (en) 2011-04-22 2013-12-30 Univ California Adeno-associated virus virions with variant capsid and methods of use thereof
DK3471780T3 (da) * 2016-06-16 2020-11-23 Adverum Biotechnologies Inc Behandling af amd med aav2-variant med aflibercept
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법

Also Published As

Publication number Publication date
JP2022547541A (ja) 2022-11-14
BR112022004027A2 (pt) 2022-05-31
CN114375195A (zh) 2022-04-19
IL291182A (en) 2022-05-01
MX2022002961A (es) 2022-04-06
KR20220062353A (ko) 2022-05-16
WO2021050649A1 (en) 2021-03-18
EP4027983A1 (en) 2022-07-20
AU2020345915A1 (en) 2022-03-24

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