CA3173450A1 - High throughput engineering of functional aav capsids - Google Patents

High throughput engineering of functional aav capsids Download PDF

Info

Publication number
CA3173450A1
CA3173450A1 CA3173450A CA3173450A CA3173450A1 CA 3173450 A1 CA3173450 A1 CA 3173450A1 CA 3173450 A CA3173450 A CA 3173450A CA 3173450 A CA3173450 A CA 3173450A CA 3173450 A1 CA3173450 A1 CA 3173450A1
Authority
CA
Canada
Prior art keywords
seq
amino acid
capsid polypeptide
aav5
aav
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
CA3173450A
Other languages
English (en)
French (fr)
Inventor
Francois Vigneault
Thomas Packard
David Jeffrey HUSS
Kevin Christopher STEIN
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.)
Shape Therapeutics Inc
Original Assignee
Shape Therapeutics 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 Shape Therapeutics Inc filed Critical Shape Therapeutics Inc
Publication of CA3173450A1 publication Critical patent/CA3173450A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/02—Libraries contained in or displayed by microorganisms, e.g. bacteria or animal cells; Libraries contained in or displayed by vectors, e.g. plasmids; Libraries containing only microorganisms or vectors
    • 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
    • A61K48/0008—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 '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
    • 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
    • A61K48/0008—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 '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
    • A61K48/0025—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 '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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 '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 wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3173450A 2020-05-26 2021-05-26 High throughput engineering of functional aav capsids Pending CA3173450A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063030038P 2020-05-26 2020-05-26
US63/030,038 2020-05-26
US202063119554P 2020-11-30 2020-11-30
US63/119,554 2020-11-30
US202163134885P 2021-01-07 2021-01-07
US63/134,885 2021-01-07
US202163181037P 2021-04-28 2021-04-28
US63/181,037 2021-04-28
PCT/US2021/034329 WO2021242909A1 (en) 2020-05-26 2021-05-26 High throughput engineering of functional aav capsids

Publications (1)

Publication Number Publication Date
CA3173450A1 true CA3173450A1 (en) 2021-12-02

Family

ID=78722738

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3173450A Pending CA3173450A1 (en) 2020-05-26 2021-05-26 High throughput engineering of functional aav capsids

Country Status (7)

Country Link
US (2) US20230048732A1 (https=)
EP (1) EP4158037A1 (https=)
JP (1) JP2023529315A (https=)
CN (1) CN116390934A (https=)
AU (1) AU2021282257A1 (https=)
CA (1) CA3173450A1 (https=)
WO (1) WO2021242909A1 (https=)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
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
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CA3249551A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
WO2023152229A1 (en) 2022-02-09 2023-08-17 Teitur Trophics Aps Novel peptides
CA3259902A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
US20250388626A1 (en) 2022-07-06 2025-12-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4626906A1 (en) * 2022-11-29 2025-10-08 Vacino Biotech Co., Ltd. Transporter peptides and application thereof
CN118307648A (zh) * 2023-01-09 2024-07-09 广东克冠达医药科技有限公司 腺相关病毒衣壳蛋白衍生多肽及其用途
EP4410988A1 (en) * 2023-01-31 2024-08-07 Universitätsklinikum Hamburg-Eppendorf An aav2-vector variant for targeted transfer of genes
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
TW202523846A (zh) 2023-08-16 2025-06-16 美商航海家醫療公司 用於治療與共濟蛋白(frataxin)不足有關之疾病之組合物及方法
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025038795A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
EP3003391B1 (en) * 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2015048534A1 (en) * 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3200830B1 (en) * 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
SG11201808812RA (en) * 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
PT3589730T (pt) 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
JP2020534788A (ja) * 2017-08-28 2020-12-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルスカプシド変異体及びその使用方法
EP3833745A1 (en) 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production

Also Published As

Publication number Publication date
US20260002177A1 (en) 2026-01-01
EP4158037A1 (en) 2023-04-05
WO2021242909A1 (en) 2021-12-02
US20230048732A1 (en) 2023-02-16
JP2023529315A (ja) 2023-07-10
CN116390934A (zh) 2023-07-04
AU2021282257A1 (en) 2022-12-01

Similar Documents

Publication Publication Date Title
CA3173450A1 (en) High throughput engineering of functional aav capsids
US12565685B2 (en) Engineering AAV
US20220396813A1 (en) Recombinase compositions and methods of use
AU2014293253B2 (en) Variant AAV and compositions, methods and uses for gene transfer to cells, organs and tissues
EP2970943B1 (en) Vectors comprising stuffer/filler polynucleotide sequences and methods of use
AU2022200502A1 (en) Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US20210363192A1 (en) Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
WO2013173512A2 (en) High-transduction-efficiency raav vectors, compositions, and methods of use
KR20230117157A (ko) 조직 특이적 표적화 모티프를 갖는 신규 조성물 및 이를 포함하는 조성물
US20230045478A1 (en) Liver-specific promoter and application thereof
CN119403823A (zh) 具有增强的肌肉转导效率的肽修饰的aav衣壳
CN121127599A (zh) 具有心肌和骨骼肌特异性靶向基序的重组aav突变载体及含有其的组合物
JP7244547B2 (ja) Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
WO2023147558A2 (en) Crispr methods for correcting bag3 gene mutations in vivo
US12569573B2 (en) Enhanced transduction of AAV vectors encoding micrornas
US20260041794A1 (en) Expression cassette for target gene and use thereof
EP4684808A1 (en) Plasmids, transgenes, vectors and medical uses comprising adamts13
RU2820088C1 (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
US20250135032A1 (en) Crispr methods for correcting bag3 gene mutations in vivo
WO2025193808A1 (en) Cns-specific gene replacement therapy for genetic forms of als/ftd
WO2025235666A1 (en) Peptides targeting ldlr for blood-brain barrier crossing
WO2025217117A1 (en) Compositions with cardiac and skeletal muscle-specific targeting motifs and uses thereof
WO2025023319A1 (ja) 脳指向性ａａｖ変異体
CN121752725A (zh) 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220926

B12 Application deemed to be withdrawn, abandoned or lapsed

Free format text: ST27 STATUS EVENT CODE: N-6-6-B10-B12-B303 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO AN EXAMINER'S REQUISITION

Effective date: 20240806

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251113

U13 Renewal or maintenance fee not paid

Free format text: ST27 STATUS EVENT CODE: N-2-6-U10-U13-U300 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO MAINTENANCE FEE NOTICE

Effective date: 20260113