CA3027737A1 - Treatment of eye disorders using aav2 variant with aflibercept - Google Patents

Treatment of eye disorders using aav2 variant with aflibercept Download PDF

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Publication number
CA3027737A1
CA3027737A1 CA3027737A CA3027737A CA3027737A1 CA 3027737 A1 CA3027737 A1 CA 3027737A1 CA 3027737 A CA3027737 A CA 3027737A CA 3027737 A CA3027737 A CA 3027737A CA 3027737 A1 CA3027737 A1 CA 3027737A1
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CA
Canada
Prior art keywords
aflibercept
fold
pharmaceutical composition
acid sequence
suspension
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
CA3027737A
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English (en)
French (fr)
Inventor
Mark Blumenkranz
Mehdi Gasmi
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.)
Adverum Biotechnologies Inc
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Adverum Biotechnologies Inc
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Filing date
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Application filed by Adverum Biotechnologies Inc filed Critical Adverum Biotechnologies Inc
Publication of CA3027737A1 publication Critical patent/CA3027737A1/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
    • 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
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/178—Syringes
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from 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
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Dispersion Chemistry (AREA)
  • Hematology (AREA)
CA3027737A 2016-06-16 2017-06-16 Treatment of eye disorders using aav2 variant with aflibercept Pending CA3027737A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662351234P 2016-06-16 2016-06-16
US62/351,234 2016-06-16
PCT/US2017/038003 WO2017218974A2 (en) 2016-06-16 2017-06-16 Treatment of amd using aav2 variant with aflibercept

Publications (1)

Publication Number Publication Date
CA3027737A1 true CA3027737A1 (en) 2017-12-21

Family

ID=60663653

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3027737A Pending CA3027737A1 (en) 2016-06-16 2017-06-16 Treatment of eye disorders using aav2 variant with aflibercept

Country Status (24)

Country Link
US (2) US20190151409A1 (enExample)
EP (2) EP3795181B1 (enExample)
JP (4) JP6814822B2 (enExample)
KR (2) KR20210021113A (enExample)
CN (2) CN109641065A (enExample)
AU (3) AU2017286673B2 (enExample)
BR (1) BR112018076124A2 (enExample)
CA (1) CA3027737A1 (enExample)
CY (1) CY1123697T1 (enExample)
DK (2) DK3795181T3 (enExample)
ES (1) ES2840059T3 (enExample)
FI (1) FI3795181T3 (enExample)
HR (1) HRP20201842T1 (enExample)
HU (1) HUE051953T2 (enExample)
IL (3) IL279919B2 (enExample)
LT (1) LT3471780T (enExample)
MX (2) MX391779B (enExample)
PT (2) PT3795181T (enExample)
RS (2) RS67328B1 (enExample)
SG (1) SG11201811232XA (enExample)
SI (2) SI3795181T1 (enExample)
SM (2) SMT202100010T1 (enExample)
WO (1) WO2017218974A2 (enExample)
ZA (1) ZA201808538B (enExample)

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KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
HUE051953T2 (hu) * 2016-06-16 2021-04-28 Adverum Biotechnologies Inc Idõskori makula degeneráció kezelése AAV2 variáns és aflibercept alkalmazásával
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
EP4653536A2 (en) 2017-03-17 2025-11-26 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
CN107661492A (zh) * 2017-08-31 2018-02-06 中山大学中山眼科中心 Vegf‑b的新用途
CN107537026A (zh) * 2017-08-31 2018-01-05 中山大学中山眼科中心 Vegf‑b的应用
AU2018350990A1 (en) * 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
AU2018372235B9 (en) * 2017-11-27 2022-03-10 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
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
WO2019198084A1 (en) * 2018-04-12 2019-10-17 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Anti-aging compositions and methods of use
NZ770284A (en) 2018-05-10 2025-11-28 Regeneron Pharma High concentration vegf receptor fusion protein containing formulations
US20200297869A1 (en) * 2019-03-04 2020-09-24 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
BR112021021156A2 (pt) * 2019-04-24 2022-02-08 Regenxbio Inc Terapêuticos anticorpos totalmente humanos pós-tradução modificados
WO2021050649A1 (en) * 2019-09-11 2021-03-18 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
MX2022002960A (es) * 2019-09-11 2022-04-06 Adverum Biotechnologies Inc Metodos para tratar enfermedades neovasculares oculares usando variantes de aav2 que codifican para aflibercept.
TWI819268B (zh) * 2020-01-16 2023-10-21 詹姆斯 Ｗ 希爾 藉由基因轉導改變眼睛顏色
FR3111913A1 (fr) * 2020-06-30 2021-12-31 Eyevensys Construction d’adn pour le traitement de pathologies oculaires
CN113952473A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
CN113952474A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
AU2021313839A1 (en) * 2020-07-21 2023-03-23 FTGEN Corp Composition and method for treating eye diseases
CN113817775B (zh) * 2020-08-25 2023-02-17 南京吉迈生物技术有限公司 修饰的阿柏西普、组合物、方法及其在基因治疗中的应用
MX2023002695A (es) * 2020-09-03 2023-05-19 Univ Massachusetts Virus adeno-asociado para la administración de kh902 (conbercept) y usos del mismo.
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
JP2021049374A (ja) * 2020-12-09 2021-04-01 株式会社三洋物産 遊技機
CA3215855A1 (en) * 2021-04-27 2022-11-03 Julie Clark Methods of treating ocular diseases using aav2 variants encoding aflibercept
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
WO2025072688A2 (en) * 2023-09-27 2025-04-03 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal cells, for nucleic acid delivery across retinal regions, or for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells
WO2025073297A1 (zh) * 2023-10-06 2025-04-10 甘李药业股份有限公司 表达抗vegf融合蛋白的aav病毒载体及其用途

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RS66184B1 (sr) * 2011-04-22 2024-12-31 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
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JP6607850B2 (ja) 2013-10-18 2019-11-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｖｅｇｆアンタゴニスト及び抗ｃｔｌａ−４抗体の組み合わせを含む方法及び組成物
KR20170137730A (ko) * 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
HUE051953T2 (hu) * 2016-06-16 2021-04-28 Adverum Biotechnologies Inc Idõskori makula degeneráció kezelése AAV2 variáns és aflibercept alkalmazásával

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IL263686B (en) 2021-02-28
JP2020203941A (ja) 2020-12-24
BR112018076124A2 (pt) 2019-03-26
FI3795181T3 (fi) 2025-10-14
PT3471780T (pt) 2020-12-17
MX2018015512A (es) 2019-04-24
WO2017218974A2 (en) 2017-12-21
JP2024113193A (ja) 2024-08-21
JP2022137244A (ja) 2022-09-21
US20190151409A1 (en) 2019-05-23
IL279919A (en) 2021-03-01
IL279919B1 (en) 2024-09-01
KR20210021113A (ko) 2021-02-24
KR102218265B1 (ko) 2021-02-25
LT3471780T (lt) 2021-01-25
SG11201811232XA (en) 2019-01-30
HRP20201842T1 (hr) 2021-04-16
EP3471780A2 (en) 2019-04-24
EP3471780B1 (en) 2020-10-28
AU2017286673A1 (en) 2019-01-31
PT3795181T (pt) 2025-11-05
CN117531025A (zh) 2024-02-09
RS61311B1 (sr) 2021-02-26
JP2019521990A (ja) 2019-08-08
JP6814822B2 (ja) 2021-01-20
EP3795181B1 (en) 2025-08-06
MX2022004786A (es) 2022-05-16
EP3471780A4 (en) 2019-04-24
EP3795181A1 (en) 2021-03-24
KR20190037228A (ko) 2019-04-05
AU2025202930A1 (en) 2025-05-15
IL314568B1 (en) 2025-05-01
SMT202500400T1 (it) 2025-11-10
WO2017218974A3 (en) 2018-01-25
AU2021225178B2 (en) 2025-01-30
HUE051953T2 (hu) 2021-04-28
SI3471780T1 (sl) 2021-03-31
US20220265740A1 (en) 2022-08-25
ES2840059T3 (es) 2021-07-06
RS67328B1 (sr) 2025-11-28
AU2021225178A1 (en) 2021-09-30
IL314568A (en) 2024-09-01
MX391779B (es) 2025-03-21
SMT202100010T1 (it) 2021-03-15
IL279919B2 (en) 2025-01-01
IL314568B2 (en) 2025-09-01
CY1123697T1 (el) 2022-03-24
IL263686A (en) 2019-01-31
DK3795181T3 (da) 2025-10-13
CN109641065A (zh) 2019-04-16
ZA201808538B (en) 2021-04-28
AU2017286673B2 (en) 2021-06-24
DK3471780T3 (da) 2020-11-23
SI3795181T1 (sl) 2025-11-28

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