EP4185702A4 - GENETICALLY ENGINEERED MUSCLE TARGET COMPOSITIONS - Google Patents

GENETICALLY ENGINEERED MUSCLE TARGET COMPOSITIONS Download PDF

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Publication number
EP4185702A4
EP4185702A4 EP21846426.1A EP21846426A EP4185702A4 EP 4185702 A4 EP4185702 A4 EP 4185702A4 EP 21846426 A EP21846426 A EP 21846426A EP 4185702 A4 EP4185702 A4 EP 4185702A4
Authority
EP
European Patent Office
Prior art keywords
muscle targeting
targeting compositions
modified muscle
modified
compositions
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
EP21846426.1A
Other languages
German (de)
English (en)
French (fr)
Other versions
EP4185702A1 (en
Inventor
Pardis SABETI
Mohammadsharif TABEBORDBAR
Simon YE
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.)
Massachusetts Institute of Technology
Broad Institute Inc
Harvard University
Original Assignee
Massachusetts Institute of Technology
Broad Institute Inc
Harvard University
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 Massachusetts Institute of Technology, Broad Institute Inc, Harvard University filed Critical Massachusetts Institute of Technology
Publication of EP4185702A1 publication Critical patent/EP4185702A1/en
Publication of EP4185702A4 publication Critical patent/EP4185702A4/en
Pending legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • A61K48/0016—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 nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1058—Directional evolution of libraries, e.g. evolution of libraries is achieved by mutagenesis and screening or selection of mixed population of organisms
    • 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
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Ecology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
EP21846426.1A 2020-07-22 2021-07-22 GENETICALLY ENGINEERED MUSCLE TARGET COMPOSITIONS Pending EP4185702A4 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063055265P 2020-07-22 2020-07-22
US202063107394P 2020-10-29 2020-10-29
US202163183038P 2021-05-02 2021-05-02
PCT/US2021/042812 WO2022020616A1 (en) 2020-07-22 2021-07-22 Engineered muscle targeting compositions

Publications (2)

Publication Number Publication Date
EP4185702A1 EP4185702A1 (en) 2023-05-31
EP4185702A4 true EP4185702A4 (en) 2024-08-28

Family

ID=79728337

Family Applications (1)

Application Number Title Priority Date Filing Date
EP21846426.1A Pending EP4185702A4 (en) 2020-07-22 2021-07-22 GENETICALLY ENGINEERED MUSCLE TARGET COMPOSITIONS

Country Status (13)

Country Link
EP (1) EP4185702A4 (https=)
JP (1) JP2023534999A (https=)
KR (1) KR20230053591A (https=)
CN (1) CN116194153A (https=)
AU (1) AU2021314256A1 (https=)
BR (1) BR112023000909A2 (https=)
CA (1) CA3183530A1 (https=)
CL (2) CL2023000204A1 (https=)
CO (1) CO2023000866A2 (https=)
IL (1) IL299431A (https=)
MX (1) MX2023000998A (https=)
TW (1) TW202217003A (https=)
WO (1) WO2022020616A1 (https=)

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IL297391A (en) 2020-04-20 2022-12-01 Tenaya Therapeutics Inc An adeno-associated virus with an engineered capsid
WO2022173847A2 (en) * 2021-02-09 2022-08-18 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
US20250090688A1 (en) * 2021-08-05 2025-03-20 The Broad Institute, Inc. Engineered muscle targeting compositions
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
US20250249052A1 (en) * 2022-04-07 2025-08-07 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2023198828A1 (en) * 2022-04-13 2023-10-19 Universitat Autònoma De Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
EP4707399A2 (en) * 2022-06-10 2026-03-11 Généthon Peptide-modified aav capsid with enhanced muscle transduction efficiency
KR20250135916A (ko) * 2022-12-17 2025-09-15 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물
TW202502803A (zh) 2023-03-10 2025-01-16 美商戴諾治療公司 衣殼多肽及其使用方法
EP4680741A2 (en) 2023-03-15 2026-01-21 RegenxBio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
WO2025090858A1 (en) * 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
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
US20250276095A1 (en) 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
CN118667855A (zh) * 2024-05-31 2024-09-20 扬州大学 一种具有肌肉靶向性促进骨形成的噬菌体的构建方法及其应用
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026041758A1 (en) 2024-08-22 2026-02-26 Genethon Modified aav capsid with enhanced transduction efficiency
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064739A1 (en) * 2024-09-23 2026-03-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2026069251A1 (en) * 2024-09-30 2026-04-02 Sarepta Therapeutics, Inc. Adeno-associated virus capsid antibodies and fragments thereof

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Patent Citations (3)

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WO2020023462A1 (en) * 2018-07-23 2020-01-30 Georgia Tech Research Corporation Synthetic hydrogel carriers for muscle repair
WO2021050974A1 (en) * 2019-09-12 2021-03-18 The Broad Institute, Inc. Engineered adeno-associated virus capsids

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Title
HILDEGARD BÜNING ET AL: "Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors", MOLECULAR THERAPY- METHODS & CLINICAL DEVELOPMENT, vol. 12, 1 March 2019 (2019-03-01), GB, pages 248 - 265, XP055593479, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2019.01.008 *
JORGE BOUCAS ET AL: "Engineering adeno-associated virus serotype 2-based targeting vectors using a new insertion site-position 453-and single point mutations", THE JOURNAL OF GENE MEDICINE, vol. 11, no. 12, 1 December 2009 (2009-12-01), US, pages 1103 - 1113, XP055421746, ISSN: 1099-498X, DOI: 10.1002/jgm.1392 *
See also references of WO2022020616A1 *
TABEBORDBAR MOHAMMADSHARIF ET AL: "Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species", CELL, ELSEVIER, AMSTERDAM NL, vol. 184, no. 19, 9 September 2021 (2021-09-09), pages 4919, XP086784024, ISSN: 0092-8674, [retrieved on 20210909], DOI: 10.1016/J.CELL.2021.08.028 *
VARADI K ET AL: "Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors", GENE THERAPY, NATURE PUBLISHING GROUP, LONDON, GB, vol. 19, no. 8, 29 September 2011 (2011-09-29), pages 800 - 809, XP037770845, ISSN: 0969-7128, [retrieved on 20110929], DOI: 10.1038/GT.2011.143 *

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Publication number Publication date
AU2021314256A1 (en) 2023-02-16
CN116194153A (zh) 2023-05-30
EP4185702A1 (en) 2023-05-31
CO2023000866A2 (es) 2023-02-16
CL2024000275A1 (es) 2024-06-14
JP2023534999A (ja) 2023-08-15
BR112023000909A2 (pt) 2023-03-28
MX2023000998A (es) 2023-03-01
KR20230053591A (ko) 2023-04-21
CA3183530A1 (en) 2022-01-27
WO2022020616A1 (en) 2022-01-27
CL2023000204A1 (es) 2023-08-11
TW202217003A (zh) 2022-05-01
IL299431A (en) 2023-02-01

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