EP4713345A2 - Rekombinante aavs mit verbessertem tropismus und verbesserter spezifität - Google Patents

Rekombinante aavs mit verbessertem tropismus und verbesserter spezifität

Info

Publication number
EP4713345A2
EP4713345A2 EP24734324.7A EP24734324A EP4713345A2 EP 4713345 A2 EP4713345 A2 EP 4713345A2 EP 24734324 A EP24734324 A EP 24734324A EP 4713345 A2 EP4713345 A2 EP 4713345A2
Authority
EP
European Patent Office
Prior art keywords
capsid protein
seq
aav capsid
independently selected
amino acid
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
EP24734324.7A
Other languages
English (en)
French (fr)
Inventor
Charles Francis ALBRIGHT
Kevin C. OLIVIERI
Xiaohong CAO
Jie Tan
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.)
Affinia Therapeutics Inc
Original Assignee
Affinia 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
Priority claimed from PCT/US2023/072424 external-priority patent/WO2024040193A2/en
Application filed by Affinia Therapeutics Inc filed Critical Affinia Therapeutics Inc
Publication of EP4713345A2 publication Critical patent/EP4713345A2/de
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
    • 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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP24734324.7A 2023-05-16 2024-05-16 Rekombinante aavs mit verbessertem tropismus und verbesserter spezifität Pending EP4713345A2 (de)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202363502595P 2023-05-16 2023-05-16
PCT/US2023/072424 WO2024040193A2 (en) 2022-08-17 2023-08-17 Recombinant aavs with improved tropism and specificity
US202463625790P 2024-01-26 2024-01-26
US202463643890P 2024-05-07 2024-05-07
PCT/US2024/029714 WO2024238807A2 (en) 2023-05-16 2024-05-16 Recombinant aavs with improved tropism and specificity

Publications (1)

Publication Number Publication Date
EP4713345A2 true EP4713345A2 (de) 2026-03-25

Family

ID=91585889

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24734324.7A Pending EP4713345A2 (de) 2023-05-16 2024-05-16 Rekombinante aavs mit verbessertem tropismus und verbesserter spezifität

Country Status (4)

Country Link
EP (1) EP4713345A2 (de)
AU (1) AU2024271851A1 (de)
IL (1) IL324687A (de)
WO (1) WO2024238807A2 (de)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121090489B (zh) * 2025-09-08 2026-03-03 河北科技师范学院 一种水污染溯源与应急决策联动方法及系统

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
ATE272123T1 (de) 1993-11-09 2004-08-15 Ohio Med College Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
US5656785A (en) 1995-08-07 1997-08-12 The Charles Stark Draper Laboratory, Inc. Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor
JP4397972B2 (ja) 1996-11-19 2010-01-13 サージックス コーポレイション 過渡電圧保護素子およびその製造方法
US6541258B2 (en) 1996-12-18 2003-04-01 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
BRPI1011236A2 (pt) 2009-06-15 2019-07-09 4S3 Bioscience Inc métodos e composições para tratamento de miopatia miotubular usando polipeptídeos quiméricos compreendendo polipeptídeos miotubularesih 1 (mtm1)
NZ718926A (en) 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
EP3875593A1 (de) 2015-12-11 2021-09-08 California Institute of Technology Zielpeptide zum leiten von adeno-assoziierten viren (aavs)
JP7122091B2 (ja) 2017-07-24 2022-08-19 株式会社Fuji ストッカ、ストッカ管理装置およびストッカ管理方法
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
CA3097375A1 (en) 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
JP7438981B2 (ja) 2018-05-11 2024-02-27 マサチューセッツ アイ アンド イヤー インファーマリー アデノ随伴ウイルスの肝臓特異的向性
EP3861010A1 (de) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Umlenkung des tropismus von aav-kapsiden
TW202102526A (zh) * 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
CN110675371A (zh) 2019-09-05 2020-01-10 北京达佳互联信息技术有限公司 一种场景切换的检测方法、装置、电子设备及存储介质
US12570998B2 (en) 2019-09-09 2026-03-10 Massachusetts Eye & Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (AAV) and the AAV receptor (AAVR) for altered bio-distribution of AAV
JP2022547570A (ja) 2019-09-12 2022-11-14 ザ・ブロード・インスティテュート・インコーポレイテッド 操作済みのアデノ随伴ウイルスキャプシド
CA3153902A1 (en) 2019-10-16 2021-04-22 Pardis SABETI Engineered muscle targeting compositions
JP2023524061A (ja) 2020-05-01 2023-06-08 ザ・ブロード・インスティテュート・インコーポレイテッド 操作された中枢神経系用組成物
US20240197918A1 (en) * 2021-04-23 2024-06-20 Locanabio, Inc. Tissue-targeted modified aav capsids and methods of use thereof
WO2023015297A1 (en) * 2021-08-05 2023-02-09 The Broad Institute, Inc. Engineered muscle targeting compositions
WO2024040193A2 (en) * 2022-08-17 2024-02-22 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity

Also Published As

Publication number Publication date
AU2024271851A1 (en) 2025-12-18
WO2024238807A3 (en) 2024-12-26
WO2024238807A2 (en) 2024-11-21
IL324687A (en) 2026-01-01

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