AU2020231505A1 - Sequential intravitreal administration of AAV gene therapy to contralateral eyes - Google Patents

Sequential intravitreal administration of AAV gene therapy to contralateral eyes Download PDF

Info

Publication number
AU2020231505A1
AU2020231505A1 AU2020231505A AU2020231505A AU2020231505A1 AU 2020231505 A1 AU2020231505 A1 AU 2020231505A1 AU 2020231505 A AU2020231505 A AU 2020231505A AU 2020231505 A AU2020231505 A AU 2020231505A AU 2020231505 A1 AU2020231505 A1 AU 2020231505A1
Authority
AU
Australia
Prior art keywords
unit dose
seq
fold
capsid protein
vector genomes
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
AU2020231505A
Other languages
English (en)
Other versions
AU2020231505A8 (en
Inventor
Diana CEPEDA
Mehdi Gasmi
Annahita KERAVALA
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
Original Assignee
Adverum Biotechnologies 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 Adverum Biotechnologies Inc filed Critical Adverum Biotechnologies Inc
Publication of AU2020231505A1 publication Critical patent/AU2020231505A1/en
Publication of AU2020231505A8 publication Critical patent/AU2020231505A8/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
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • 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 [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IG], 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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
AU2020231505A 2019-03-04 2020-03-04 Sequential intravitreal administration of AAV gene therapy to contralateral eyes Pending AU2020231505A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962813597P 2019-03-04 2019-03-04
US62/813,597 2019-03-04
US201962839457P 2019-04-26 2019-04-26
US62/839,457 2019-04-26
PCT/US2020/020929 WO2020180951A1 (en) 2019-03-04 2020-03-04 Sequential intravitreal administration of aav gene therapy to contralateral eyes

Publications (2)

Publication Number Publication Date
AU2020231505A1 true AU2020231505A1 (en) 2021-08-19
AU2020231505A8 AU2020231505A8 (en) 2021-09-02

Family

ID=70334029

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020231505A Pending AU2020231505A1 (en) 2019-03-04 2020-03-04 Sequential intravitreal administration of AAV gene therapy to contralateral eyes

Country Status (6)

Country Link
US (1) US20200297869A1 (https=)
EP (1) EP3934698A1 (https=)
JP (1) JP7597380B2 (https=)
KR (1) KR20210135267A (https=)
AU (1) AU2020231505A1 (https=)
WO (1) WO2020180951A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3886946A1 (en) 2019-06-05 2021-10-06 Regeneron Pharmaceuticals, Inc. Devices and methods for precision dose delivery
CN114250227B (zh) * 2020-09-25 2026-04-03 北京安龙生物医药有限公司 用于高水平表达外源基因的表达载体
JP2023550548A (ja) * 2020-11-10 2023-12-01 アビラマックス・バイオファーマ・インコーポレイテッド 操作されたウイルスカプシドおよび使用方法
BR112023020490A2 (pt) * 2021-04-09 2023-11-21 Avirmax Biopharma Inc Composições e métodos para expressão de transgenes oculares
AU2023220219A1 (en) 2022-02-17 2024-09-05 Skyline Therapeutics Limited Recombinant adeno-associated virus with modified aav capsid polypeptides
JP2025519378A (ja) 2022-06-07 2025-06-26 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 視覚回復のためのメラノプシンバリアント
USD1120314S1 (en) 2022-11-30 2026-03-24 Regeneron Pharmaceuticals, Inc. Dose delivery device
WO2024168252A2 (en) * 2023-02-10 2024-08-15 Yale University Use of inhibitors of vegf-c signaling for inhibiting immune responses and increasing gene therapy efficacy

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
CN105755044A (zh) 2011-04-22 2016-07-13 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
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US11021519B2 (en) * 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
AU2017257169B2 (en) * 2016-04-29 2021-07-08 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
ES2840059T3 (es) * 2016-06-16 2021-07-06 Adverum Biotechnologies Inc Tratamiento de la DMRE mediante el uso de la variante AAV2 con aflibercept
CA3040179A1 (en) * 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
JP2020510424A (ja) * 2017-02-28 2020-04-09 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 改変ａａｖカプシドおよびその使用
EP4653536A3 (en) 2017-03-17 2026-02-25 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression

Also Published As

Publication number Publication date
AU2020231505A8 (en) 2021-09-02
US20200297869A1 (en) 2020-09-24
JP2022522776A (ja) 2022-04-20
WO2020180951A1 (en) 2020-09-10
EP3934698A1 (en) 2022-01-12
JP7597380B2 (ja) 2024-12-10
KR20210135267A (ko) 2021-11-12

Similar Documents

Publication Publication Date Title
AU2021225178B2 (en) Treatment of AMD using AAV2 variant with aflibercept
JP7606245B2 (ja) 眼性新血管形成を低減するための組成物および方法
US20200297869A1 (en) Sequential intravitreal administration of aav gene therapy to contralateral eyes
AU2023202277B2 (en) Compositions and methods for reducing ocular neovascularization
HK40082757B (en) Compositions and methods for reducing ocular neovascularization
HK40082757A (en) Compositions and methods for reducing ocular neovascularization
HK40115902A (en) Compositions and methods for reducing ocular neovascularization
HK40049262A (en) Treatment of amd using aav2 variant with aflibercept
HK40049262B (en) Treatment of amd using aav2 variant with aflibercept
HK40008120A (en) Compositions and methods for reducing ocular neovascularization
HK40008120B (en) Compositions and methods for reducing ocular neovascularization
HK40007499A (en) Treatment of amd using aav2 variant with aflibercept

Legal Events

Date Code Title Description
TH Corrigenda

Free format text: IN VOL 35 , NO 33 , PAGE(S) 6739 UNDER THE HEADING PCT APPLICATIONS THAT HAVE ENTERED THE NATIONAL PHASE - NAME INDEX UNDER THE NAME ADVERUM BIOTECHNOLOGIES, INC., APPLICATION NO. 2020231505, UNDER INID (72) ADD CO-INVENTOR KERAVALA, ANNAHITA