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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Canada
Prior art keywords
eye
unit dose
raav particles
weeks
administration
Prior art date
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Pending
Application number
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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Filing date
Publication date
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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    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/179—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds 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
    • A61K31/573—Compounds 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/575—Compounds 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • 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
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    • 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
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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
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    • 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
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Immunology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Plant Pathology (AREA)
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  • 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
USPCT/US2019/062066 2019-11-18
PCT/US2019/062066 WO2021050094A1 (en) 2019-09-11 2019-11-18 Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
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 (19)

Country Link
EP (2) EP4027983B1 (OSRAM)
JP (2) JP2022547541A (OSRAM)
KR (1) KR20220062353A (OSRAM)
CN (1) CN114375195A (OSRAM)
AU (1) AU2020345915A1 (OSRAM)
BR (1) BR112022004027A2 (OSRAM)
CA (1) CA3148376A1 (OSRAM)
DK (1) DK4027983T3 (OSRAM)
ES (1) ES3063491T3 (OSRAM)
FI (1) FI4027983T3 (OSRAM)
HR (1) HRP20260137T1 (OSRAM)
IL (1) IL291182A (OSRAM)
LT (1) LT4027983T (OSRAM)
MX (1) MX2022002961A (OSRAM)
PL (1) PL4027983T3 (OSRAM)
PT (1) PT4027983T (OSRAM)
RS (1) RS67774B1 (OSRAM)
SM (1) SMT202600070T1 (OSRAM)
WO (1) WO2021050649A1 (OSRAM)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA54325A (fr) 2019-06-05 2022-05-11 Regeneron Pharma Dispositifs et procédés pour l'administration précise de doses
AU2021313839A1 (en) * 2020-07-21 2023-03-23 FTGEN Corp Composition and method for treating eye diseases
BR112023019056A2 (pt) 2021-04-27 2024-01-16 Adverum Biotechnologies Inc Métodos para tratar doenças oculares usando variantes de aav2 que codificam aflibercepte
WO2022247917A1 (zh) * 2021-05-28 2022-12-01 上海瑞宏迪医药有限公司 衣壳变异的重组腺相关病毒及其应用
US20250186620A1 (en) * 2021-08-23 2025-06-12 Duke University Compositions for and methods of long-term integration of circuits using connexins
WO2023158990A1 (en) * 2022-02-16 2023-08-24 Adverum Biotechnologies, Inc. Method of reducing cst fluctuation in neovascular amd by a recombinant adeno-associated virus
USD1120314S1 (en) 2022-11-30 2026-03-24 Regeneron Pharmaceuticals, Inc. Dose delivery device
WO2025026182A1 (zh) 2023-08-01 2025-02-06 北京因诺惟康医药科技有限公司 融合多肽、包含其编码基因的表达盒、基因递送载体、药物组合物及用途
KR20260004635A (ko) * 2024-07-01 2026-01-09 삼성바이오에피스 주식회사 애플리버셉트를 암호화하는 코돈-최적화된 폴리뉴클레오티드를 포함하는 발현 카세트 및 이를 포함하는 rAAV 생산용 플라스미드

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2544136B2 (ja) * 1986-05-23 1996-10-16 第一製薬株式会社 硫酸化多糖体ｄｓ４１５２を含有する血管新生抑制剤及び抗腫瘍剤
TW201105363A (en) * 2009-07-14 2011-02-16 Univ Yamagata Eye drop for macular edema treatment
CN107012171B (zh) 2011-04-22 2021-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
AU2014252807A1 (en) * 2013-04-09 2015-11-12 Cresset Biomolecular Discovery Ltd The local treatment of inflammatory ophthalmic diseases
SI3471780T1 (sl) * 2016-06-16 2021-03-31 Adverum Biotechnologies, Inc. Zdravljenje SDM-ja z uporabo različice AAV2 z afliberceptom
EP4653536A3 (en) 2017-03-17 2026-02-25 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression

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Publication number Publication date
JP2022547541A (ja) 2022-11-14
KR20220062353A (ko) 2022-05-16
DK4027983T3 (da) 2026-02-09
ES3063491T3 (en) 2026-04-16
RS67774B1 (sr) 2026-03-31
CN114375195A (zh) 2022-04-19
EP4696318A1 (en) 2026-02-18
PT4027983T (pt) 2026-02-25
MX2022002961A (es) 2022-04-06
EP4027983A1 (en) 2022-07-20
IL291182A (en) 2022-05-01
FI4027983T3 (fi) 2026-02-03
EP4027983B1 (en) 2025-11-19
HRP20260137T1 (hr) 2026-03-13
PL4027983T3 (pl) 2026-05-11
SMT202600070T1 (it) 2026-03-09
AU2020345915A1 (en) 2022-03-24
BR112022004027A2 (pt) 2022-05-31
WO2021050649A1 (en) 2021-03-18
LT4027983T (lt) 2026-02-25
JP2025118798A (ja) 2025-08-13

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