BR112021015751A2 - Vetores de terapia genética para tratamento da doença de danon - Google Patents

Vetores de terapia genética para tratamento da doença de danon

Info

Publication number
BR112021015751A2
BR112021015751A2 BR112021015751A BR112021015751A BR112021015751A2 BR 112021015751 A2 BR112021015751 A2 BR 112021015751A2 BR 112021015751 A BR112021015751 A BR 112021015751A BR 112021015751 A BR112021015751 A BR 112021015751A BR 112021015751 A2 BR112021015751 A2 BR 112021015751A2
Authority
BR
Brazil
Prior art keywords
danon
disease
gene therapy
therapy vectors
treatment
Prior art date
Application number
BR112021015751A
Other languages
English (en)
Portuguese (pt)
Inventor
Annahita Keravala
Gaurav Shah
Naveen Yalamanchi
Piratip Pratumsuwan
Raj Prabhakar
Roderick Wong
Original Assignee
Spacecraft Seven Llc
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 Spacecraft Seven Llc filed Critical Spacecraft Seven Llc
Publication of BR112021015751A2 publication Critical patent/BR112021015751A2/pt

Links

Classifications

    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • A61P9/06—Antiarrhythmics
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112021015751A 2019-02-12 2020-02-12 Vetores de terapia genética para tratamento da doença de danon BR112021015751A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962804521P 2019-02-12 2019-02-12
US201962934928P 2019-11-13 2019-11-13
PCT/US2020/017987 WO2020167996A1 (en) 2019-02-12 2020-02-12 Gene therapy vectors for treatment of danon disease

Publications (1)

Publication Number Publication Date
BR112021015751A2 true BR112021015751A2 (pt) 2022-02-08

Family

ID=72043957

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021015751A BR112021015751A2 (pt) 2019-02-12 2020-02-12 Vetores de terapia genética para tratamento da doença de danon

Country Status (12)

Country Link
US (1) US20220143215A1 (https=)
EP (1) EP3924371A4 (https=)
JP (2) JP2022520232A (https=)
KR (1) KR20210125999A (https=)
CN (2) CN113508130A (https=)
AU (3) AU2020221842A1 (https=)
BR (1) BR112021015751A2 (https=)
CA (1) CA3128514A1 (https=)
IL (1) IL285238A (https=)
MX (1) MX2021009696A (https=)
SG (1) SG11202107744SA (https=)
WO (1) WO2020167996A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3021572A1 (en) 2016-04-20 2017-10-26 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas Compositions and methods for enhanced gene expression of pklr
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR ADMINISTRATION OF PKLR TO TREAT PYRUVATE KINASE DEFECTS
BR112020020841A2 (pt) 2018-04-11 2021-01-19 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus Composições e métodos para transplante de célulastronco
CA3096293A1 (en) 2018-04-27 2019-10-31 Rocket Pharmaceuticals, Ltd. Gene therapy for cns degeneration
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
CA3201247A1 (en) * 2020-12-07 2022-06-16 Gaurav Shah Treatment of danon disease
CN114874990A (zh) * 2021-02-05 2022-08-09 中国科学院苏州纳米技术与纳米仿生研究所 一种功能化外泌体及其制备方法和应用
JP7564368B2 (ja) 2021-09-23 2024-10-08 エルジー エナジー ソリューション リミテッド 高耐熱性コネクター及びこれを含むバッテリーモジュール、バッテリーパック、自動車
CN114457112A (zh) * 2022-02-07 2022-05-10 苏州市立医院 一种特异性神经靶向mr分子探针及其制备方法和应用
CN115975051B (zh) * 2022-11-17 2025-09-12 东南大学 靶向外泌体及制备方法、应用、药物和药物递送系统

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7985553B2 (en) * 2001-10-29 2011-07-26 Nathaniel Heintz Method for isolating cell type-specific mRNAs
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US9434928B2 (en) * 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
AU2013221212B2 (en) * 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
DK3405215T3 (da) * 2016-01-19 2022-09-19 Univ California Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser
WO2017136536A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
US11713468B2 (en) * 2017-08-02 2023-08-01 Dna Twopointo Inc. DNA vectors and elements for sustained gene expression in eukaryotic cells
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
EP3833745A1 (en) * 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production

Also Published As

Publication number Publication date
WO2020167996A1 (en) 2020-08-20
JP2022520232A (ja) 2022-03-29
EP3924371A4 (en) 2023-01-11
EP3924371A1 (en) 2021-12-22
CN120324641A (zh) 2025-07-18
JP2025063228A (ja) 2025-04-15
AU2025205318A1 (en) 2025-07-31
CA3128514A1 (en) 2020-08-20
US20220143215A1 (en) 2022-05-12
KR20210125999A (ko) 2021-10-19
AU2020221842A1 (en) 2021-08-12
MX2021009696A (es) 2021-09-23
IL285238A (en) 2021-09-30
AU2023201237B2 (en) 2025-04-10
AU2023201237A1 (en) 2023-04-13
CN113508130A (zh) 2021-10-15
SG11202107744SA (en) 2021-08-30

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