WO2026069251A1 - Anticorps capsidiques de virus adéno-associés et fragments de ceux-ci - Google Patents

Anticorps capsidiques de virus adéno-associés et fragments de ceux-ci

Info

Publication number
WO2026069251A1
WO2026069251A1 PCT/IB2025/059792 IB2025059792W WO2026069251A1 WO 2026069251 A1 WO2026069251 A1 WO 2026069251A1 IB 2025059792 W IB2025059792 W IB 2025059792W WO 2026069251 A1 WO2026069251 A1 WO 2026069251A1
Authority
WO
WIPO (PCT)
Prior art keywords
seq
antibody
acid sequence
amino acid
binding fragment
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
PCT/IB2025/059792
Other languages
English (en)
Inventor
Wenjing XU
Sohrab Khan
Behnam SAYANJALI
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.)
Sarepta Therapeutics Inc
Original Assignee
Sarepta 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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Publication of WO2026069251A1 publication Critical patent/WO2026069251A1/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/081—DNA viruses
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/569—Immunoassay; Biospecific binding assay; Materials therefor for microorganisms, e.g. protozoa, bacteria, viruses
    • G01N33/56983—Viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/33—Crossreactivity, e.g. for species or epitope, or lack of said crossreactivity
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/005—Assays involving biological materials from specific organisms or of a specific nature from viruses
    • G01N2333/01—DNA viruses
    • G01N2333/075—Adenoviridae
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2469/00—Immunoassays for the detection of microorganisms
    • G01N2469/10—Detection of antigens from microorganism in sample from host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Urology & Nephrology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Food Science & Technology (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Biotechnology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La présente divulgation concerne des compositions associées à la liaison de diverses protéines capsidiques de MyoAAV, qui sont pertinentes pour leur rôle dans des thérapies géniques orientées sur les muscles. La divulgation concerne des protéines, des anticorps et/ou des fragments de ceux-ci et des constructions polynucléotidiques associées. La divulgation concerne en outre des procédés de fabrication desdites compositions et leurs utilisations.
PCT/IB2025/059792 2024-09-30 2025-09-29 Anticorps capsidiques de virus adéno-associés et fragments de ceux-ci Pending WO2026069251A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202463701010P 2024-09-30 2024-09-30
US63/701,010 2024-09-30

Publications (1)

Publication Number Publication Date
WO2026069251A1 true WO2026069251A1 (fr) 2026-04-02

Family

ID=97445659

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2025/059792 Pending WO2026069251A1 (fr) 2024-09-30 2025-09-29 Anticorps capsidiques de virus adéno-associés et fragments de ceux-ci

Country Status (1)

Country Link
WO (1) WO2026069251A1 (fr)

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US5225539A (en) 1986-03-27 1993-07-06 Medical Research Council Recombinant altered antibodies and methods of making altered antibodies
US5530101A (en) 1988-12-28 1996-06-25 Protein Design Labs, Inc. Humanized immunoglobulins
US20210388064A1 (en) * 2020-06-15 2021-12-16 Sarepta Therapeutics, Inc. Adeno-associated virus antibodies and fragments thereof
WO2022020616A1 (fr) * 2020-07-22 2022-01-27 The Broad Institute, Inc. Compositions de ciblage musculaire modifiées
WO2022226374A1 (fr) * 2021-04-23 2022-10-27 Locanabio, Inc. Capsides d'aav modifiées ciblant des tissus et leurs méthodes d'utilisation
US20230159949A1 (en) 2020-07-22 2023-05-25 The Broad Institute, Inc. Engineered muscle targeting compositions
WO2024054864A1 (fr) * 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Thérapies cardioprotectrices contre les maladies cardiaques

Patent Citations (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US5225539A (en) 1986-03-27 1993-07-06 Medical Research Council Recombinant altered antibodies and methods of making altered antibodies
US5530101A (en) 1988-12-28 1996-06-25 Protein Design Labs, Inc. Humanized immunoglobulins
US5585089A (en) 1988-12-28 1996-12-17 Protein Design Labs, Inc. Humanized immunoglobulins
US5693762A (en) 1988-12-28 1997-12-02 Protein Design Labs, Inc. Humanized immunoglobulins
US6180370B1 (en) 1988-12-28 2001-01-30 Protein Design Labs, Inc. Humanized immunoglobulins and methods of making the same
US20210388064A1 (en) * 2020-06-15 2021-12-16 Sarepta Therapeutics, Inc. Adeno-associated virus antibodies and fragments thereof
WO2022020616A1 (fr) * 2020-07-22 2022-01-27 The Broad Institute, Inc. Compositions de ciblage musculaire modifiées
US20230159949A1 (en) 2020-07-22 2023-05-25 The Broad Institute, Inc. Engineered muscle targeting compositions
WO2022226374A1 (fr) * 2021-04-23 2022-10-27 Locanabio, Inc. Capsides d'aav modifiées ciblant des tissus et leurs méthodes d'utilisation
WO2024054864A1 (fr) * 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Thérapies cardioprotectrices contre les maladies cardiaques

Non-Patent Citations (28)

* Cited by examiner, † Cited by third party
Title
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"Gene Expression Technology, Methods in Enzymology", vol. 185, 1990, ACADEMIC PRESS
"Handbook of Experimental Immunology", vol. 4, 1986, BLACKWELL SCIENTIFIC PUBLISHERS
APRIL R. GILES ET AL: "Mapping an Adeno-associated Virus 9-Specific Neutralizing Epitope To Develop Next-Generation Gene Delivery Vectors", JOURNAL OF VIROLOGY, vol. 92, no. 20, 8 August 2018 (2018-08-08), US, XP055699487, ISSN: 0022-538X, DOI: 10.1128/JVI.01011-18 *
BIRD RE ET AL., SCIENCE, vol. 242, 1988, pages 423 - 426
BLACKLOW ET AL., J NATL CANCER INST, vol. 40, no. 2, 1968, pages 319
BOUTAGY NABIL E. ET AL: "Noninvasive In Vivo Quantification of Adeno-Associated Virus Serotype 9-Mediated Expression of the Sodium/Iodide Symporter Under Hindlimb Ischemia and Neuraminidase Desialylation in Skeletal Muscle Using Single-Photon Emission Computed Tomography/Computed Tomography", CIRCULATION. CARDIOVASCULAR IMAGING, vol. 12, no. 7, 1 July 2019 (2019-07-01), US, XP093340917, ISSN: 1941-9651, Retrieved from the Internet <URL:https://www.ahajournals.org/doi/pdf/10.1161/CIRCIMAGING.119.009063> DOI: 10.1161/CIRCIMAGING.119.009063 *
BOUTIN ET AL., HUM. GENE THER., vol. 21, 2010, pages 704 - 712
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CARTER P ET AL., BIO/ TECHNOLOGY, vol. 10, 1992, pages 163 - 167
CHAN CHRISTOPHER ET AL: "Rational Design of AAV-rh74, AAV3B, and AAV8 with Limited Liver Targeting", VIRUSES, vol. 15, no. 11, 28 October 2023 (2023-10-28), CH, pages 2168, XP093340914, ISSN: 1999-4915, Retrieved from the Internet <URL:https://pmc.ncbi.nlm.nih.gov/articles/PMC10675213/pdf/viruses-15-02168.pdf> DOI: 10.3390/v15112168 *
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GOROVITS BORIS ET AL: "Evaluation of the Humoral Response to Adeno-Associated Virus-Based Gene Therapy Modalities Using Total Antibody Assays", THE AAPS JOURNAL, SPRINGER INTERNATIONAL PUBLISHING, CHAM, vol. 23, no. 6, 16 September 2021 (2021-09-16), XP037631291, [retrieved on 20210916], DOI: 10.1208/S12248-021-00628-3 *
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MORIMOTO K ET AL., J. BIOCHEM. & BIOPHYSICAL METHODS, vol. 24, 1992, pages 107 - 117
NEIL S GREENSPAN ET AL: "Defining epitopes: It's not as easy as it seems", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 17, no. 10, 1 October 1999 (1999-10-01), pages 936 - 937, XP008118453, ISSN: 1087-0156, DOI: 10.1038/13590 *
PARKS ET AL., INFECT IMMUN, vol. 2, no. 6, 1970, pages 716
SHANAN N. EMMANUEL: "Structurally Mapping Antigenic Epitopes of Adeno-associated Virus 9: Development of Antibody Escape Variants", JOURNAL OF VIROLOGY, vol. 96, no. 3, 9 February 2022 (2022-02-09), US, XP093166994, ISSN: 0022-538X, DOI: 10.1128/JVI.01251-21 *
SIMMONS EMMA ET AL: "A sensitive and drug tolerant assay for detecting anti-AAV9 antibodies using affinity capture elution", JOURNAL OF IMMUNOLOGICAL METHODS, vol. 512, 1 January 2023 (2023-01-01), NL, pages 113397, XP093341004, ISSN: 0022-1759, Retrieved from the Internet <URL:https://pdf.sciencedirectassets.com/271049/1-s2.0-S0022175922X00124/1-s2.0-S0022175922001843/main.pdf?hash=b968cb93fc18d31e4001a284d9acbdb40d8a2f293ce2737b6405712d6b1d8432&host=68042c943591013ac2b2430a89b270f6af2c76d8dfd086a07176afe7c76c2c61&pii=S0022175922001843&tid=spdf-80ad2add-dbaf-44fa-a66c-322> DOI: 10.1016/j.jim.2022.113397 *
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