CA3130217A1 - Variant aav capsids for intravitreal delivery - Google Patents

Variant aav capsids for intravitreal delivery Download PDF

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Publication number
CA3130217A1
CA3130217A1 CA3130217A CA3130217A CA3130217A1 CA 3130217 A1 CA3130217 A1 CA 3130217A1 CA 3130217 A CA3130217 A CA 3130217A CA 3130217 A CA3130217 A CA 3130217A CA 3130217 A1 CA3130217 A1 CA 3130217A1
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CA
Canada
Prior art keywords
aav
capsid protein
virion
cell
retinal
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Pending
Application number
CA3130217A
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English (en)
French (fr)
Inventor
Annahita KERAVALA
Diana CEPEDA
Mehdi Gasmi
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Adverum Biotechnologies Inc
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Adverum Biotechnologies Inc
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Publication of CA3130217A1 publication Critical patent/CA3130217A1/en
Pending legal-status Critical Current

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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
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    • 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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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Medicinal Chemistry (AREA)
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  • Molecular Biology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3130217A 2019-04-26 2020-04-24 Variant aav capsids for intravitreal delivery Pending CA3130217A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962839548P 2019-04-26 2019-04-26
US62/839,548 2019-04-26
US201962923924P 2019-10-21 2019-10-21
US62/923,924 2019-10-21
PCT/US2020/029895 WO2020219933A1 (en) 2019-04-26 2020-04-24 Variant aav capsids for intravitreal delivery

Publications (1)

Publication Number Publication Date
CA3130217A1 true CA3130217A1 (en) 2020-10-29

Family

ID=70740759

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3130217A Pending CA3130217A1 (en) 2019-04-26 2020-04-24 Variant aav capsids for intravitreal delivery

Country Status (14)

Country Link
US (1) US11510950B2 (https=)
EP (1) EP3958911A1 (https=)
JP (1) JP7623703B2 (https=)
KR (1) KR20220004114A (https=)
CN (2) CN113766934B (https=)
AU (1) AU2020262948A1 (https=)
BR (1) BR112021020957A2 (https=)
CA (1) CA3130217A1 (https=)
IL (1) IL287438A (https=)
MX (2) MX2021012311A (https=)
SG (1) SG11202108928XA (https=)
TW (2) TW202532428A (https=)
WO (1) WO2020219933A1 (https=)
ZA (1) ZA202105975B (https=)

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TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
CN115044614B (zh) * 2021-03-09 2023-10-20 上海目镜生物医药科技有限公司 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用
US12385064B2 (en) * 2021-03-26 2025-08-12 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
CN113648432B (zh) * 2021-03-31 2024-01-19 中山大学中山眼科中心 rAAV2/Retro作为针对视网膜感光细胞的递送系统及其在制备治疗视网膜疾病药物中的应用
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4688807A1 (en) * 2023-03-31 2026-02-11 Exegenesis Bio Singapore Pte. Ltd. Variant aav9 capsid proteins and uses thereof
EP4689184A1 (en) 2023-04-05 2026-02-11 Adverum Biotechnologies, Inc. Immune landscape signatures for ocular inflammation
KR20260039752A (ko) 2023-07-13 2026-03-20 크리야 테라퓨틱스, 인크. 보체 경로 억제제 융합체 및 그의 사용 방법
CN117247434B (zh) * 2023-11-10 2024-02-02 上海朗昇生物科技有限公司 衣壳修饰型病毒载体及其制备和用途
KR20260014488A (ko) * 2024-07-22 2026-01-30 한국생명공학연구원 변이체 캡시드를 포함하는 아데노-연관 바이러스 및 이의 용도
CN118955654B (zh) * 2024-10-15 2025-05-16 朗信启昇(苏州)生物制药有限公司 具有变异衣壳的腺相关病毒病毒体及其用途
WO2026084101A1 (ko) * 2024-10-18 2026-04-23 주식회사 아바타테라퓨틱스 아데노연관바이러스 변이체

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Also Published As

Publication number Publication date
EP3958911A1 (en) 2022-03-02
TWI862583B (zh) 2024-11-21
TW202532428A (zh) 2025-08-16
MX2021012311A (es) 2021-11-12
CN119708162A (zh) 2025-03-28
KR20220004114A (ko) 2022-01-11
MX2025015023A (es) 2026-02-03
WO2020219933A1 (en) 2020-10-29
IL287438A (en) 2021-12-01
TW202106699A (zh) 2021-02-16
JP2022529775A (ja) 2022-06-24
US20200338146A1 (en) 2020-10-29
CN113766934A (zh) 2021-12-07
ZA202105975B (en) 2024-12-18
SG11202108928XA (en) 2021-09-29
US11510950B2 (en) 2022-11-29
CN113766934B (zh) 2024-11-08
BR112021020957A2 (pt) 2021-12-14
AU2020262948A1 (en) 2021-09-30
JP7623703B2 (ja) 2025-01-29

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