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WO2025051970A3 - Méthodes de traitement de la cardiomyopathie hypertrophique avec des vecteurs de thérapie génique vaa et des formulations thérapeutiques - Google Patents

Méthodes de traitement de la cardiomyopathie hypertrophique avec des vecteurs de thérapie génique vaa et des formulations thérapeutiques Download PDF

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Publication number
WO2025051970A3
WO2025051970A3 PCT/EP2024/075017 EP2024075017W WO2025051970A3 WO 2025051970 A3 WO2025051970 A3 WO 2025051970A3 EP 2024075017 W EP2024075017 W EP 2024075017W WO 2025051970 A3 WO2025051970 A3 WO 2025051970A3
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WIPO (PCT)
Prior art keywords
methods
treatment
gene therapy
hypertrophic cardiomyopathy
therapy vectors
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/EP2024/075017
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English (en)
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WO2025051970A2 (fr
Inventor
Pooja Agarwal
Peter Cameron Colosi
Justin ISHIDA
Jonathan KALTMAN
Edward KOO
Saeed Moshashaee
Marius P. SUMANDEA
Stephanie KISHBAUGH VARGAS
Catherine VITELLI
Wesley Yonemoto
Éduard AYUSO LÓPEZ
Thomas Voit
Valeria RICOTTI
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.)
Dinaqor AG
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Dinaqor AG
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Publication of WO2025051970A2 publication Critical patent/WO2025051970A2/fr
Publication of WO2025051970A3 publication Critical patent/WO2025051970A3/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1767—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from invertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • 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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Mycology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Inorganic Chemistry (AREA)
  • Manufacturing & Machinery (AREA)
  • Endocrinology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Plant Pathology (AREA)
  • Cardiology (AREA)

Abstract

L'invention concerne des compositions pharmaceutiques et des méthodes de traitement de la cardiomyopathie hypertrophique chez un sujet humain l'utilisant.
PCT/EP2024/075017 2023-09-06 2024-09-06 Méthodes de traitement de la cardiomyopathie hypertrophique avec des vecteurs de thérapie génique vaa et des formulations thérapeutiques Pending WO2025051970A2 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202363580741P 2023-09-06 2023-09-06
US63/580,741 2023-09-06
US202363591172P 2023-10-18 2023-10-18
US63/591,172 2023-10-18

Publications (2)

Publication Number Publication Date
WO2025051970A2 WO2025051970A2 (fr) 2025-03-13
WO2025051970A3 true WO2025051970A3 (fr) 2025-04-17

Family

ID=92710669

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2024/075017 Pending WO2025051970A2 (fr) 2023-09-06 2024-09-06 Méthodes de traitement de la cardiomyopathie hypertrophique avec des vecteurs de thérapie génique vaa et des formulations thérapeutiques

Country Status (2)

Country Link
TW (1) TW202525313A (fr)
WO (1) WO2025051970A2 (fr)

Citations (9)

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Publication number Priority date Publication date Assignee Title
WO2021163357A2 (fr) * 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Vecteurs de thérapie génique de traitement de maladie cardiaque
WO2022031914A2 (fr) * 2020-08-05 2022-02-10 Asklepios Biopharmaceutical, Inc. Méthodes permettant de traiter des troubles cardiaques et une insuffisance cardiaque congestive et d'administrer des vecteurs vaa
WO2022208342A1 (fr) * 2021-04-01 2022-10-06 Pfizer Inc. Compositions pharmaceutiques contenant un vecteur viral adéno-associé
WO2023282796A2 (fr) * 2021-07-08 2023-01-12 Limited Liability Company «ANABION» Composition pharmaceutique de virus non enveloppé
WO2023023590A1 (fr) * 2021-08-18 2023-02-23 Ultragenyx Pharmaceutical Inc. Formulations d'aav recombinants
WO2023028513A1 (fr) * 2021-08-24 2023-03-02 Oxford Biomedica Solutions Llc Formulations de virus adéno-associés
WO2023056436A2 (fr) * 2021-10-01 2023-04-06 Biomarin Pharmaceutical Inc. Traitement de l'oedème de quincke héréditaire avec des vecteurs de thérapie génique aav et des formulations thérapeutiques
WO2023108157A1 (fr) * 2021-12-10 2023-06-15 University Of Florida Research Foundation, Incorporated Méthodes et compositions pour traiter une cardiomyopathie hypertrophique liée à la mybpc3 avec un vecteur viral
WO2023108130A2 (fr) * 2021-12-10 2023-06-15 Aavantibio, Inc. Compositions comprenant des séquences kozak sélectionnées pour produire une expression améliorée

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US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003074714A1 (fr) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Systeme d'expression de baculovirus
IL263801B2 (en) 2016-07-26 2024-01-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
CN114729333A (zh) 2019-09-27 2022-07-08 生物马林药物股份有限公司 使用尺寸排阻色谱与多角度光散射技术表征基因治疗病毒颗粒
MY209860A (en) 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
JP2024534897A (ja) 2021-09-03 2024-09-26 ビオマリン プハルマセウトイカル インコーポレイテッド Ａａｖカプシド組成物及び送達方法
WO2023034980A1 (fr) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Compositions capsidiques de vaa et méthodes d'administration
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WO2021163357A2 (fr) * 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Vecteurs de thérapie génique de traitement de maladie cardiaque
WO2022031914A2 (fr) * 2020-08-05 2022-02-10 Asklepios Biopharmaceutical, Inc. Méthodes permettant de traiter des troubles cardiaques et une insuffisance cardiaque congestive et d'administrer des vecteurs vaa
WO2022208342A1 (fr) * 2021-04-01 2022-10-06 Pfizer Inc. Compositions pharmaceutiques contenant un vecteur viral adéno-associé
WO2023282796A2 (fr) * 2021-07-08 2023-01-12 Limited Liability Company «ANABION» Composition pharmaceutique de virus non enveloppé
WO2023023590A1 (fr) * 2021-08-18 2023-02-23 Ultragenyx Pharmaceutical Inc. Formulations d'aav recombinants
WO2023028513A1 (fr) * 2021-08-24 2023-03-02 Oxford Biomedica Solutions Llc Formulations de virus adéno-associés
WO2023056436A2 (fr) * 2021-10-01 2023-04-06 Biomarin Pharmaceutical Inc. Traitement de l'oedème de quincke héréditaire avec des vecteurs de thérapie génique aav et des formulations thérapeutiques
WO2023108157A1 (fr) * 2021-12-10 2023-06-15 University Of Florida Research Foundation, Incorporated Méthodes et compositions pour traiter une cardiomyopathie hypertrophique liée à la mybpc3 avec un vecteur viral
WO2023108130A2 (fr) * 2021-12-10 2023-06-15 Aavantibio, Inc. Compositions comprenant des séquences kozak sélectionnées pour produire une expression améliorée

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CHU WING SUM ET AL: "Immunomodulation in Administration of rAAV: Preclinical and Clinical Adjuvant Pharmacotherapies", FRONTIERS IN IMMUNOLOGY, vol. 12, 1 April 2021 (2021-04-01), XP093015702, DOI: 10.3389/fimmu.2021.658038 *
ELEZABY ALY ET AL: "Cardiovascular effects of immunosuppression agents", FRONTIERS IN CARDIOVASCULAR MEDICINE, vol. 9, 21 September 2022 (2022-09-21), XP093253697, ISSN: 2297-055X, DOI: 10.3389/fcvm.2022.981838 *
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MEARINI GIULIA ET AL: "Mybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in mice", NATURE COMMUNICATIONS, vol. 5, no. 1, 2 December 2014 (2014-12-02), XP055851801, Retrieved from the Internet <URL:https://www.nature.com/articles/ncomms6515.pdf> DOI: 10.1038/ncomms6515 *

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TW202525313A (zh) 2025-07-01
WO2025051970A2 (fr) 2025-03-13

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