BR112022009279A2 - Tratamento de angioedema hereditário com vetores de terapia gênica específicos do fígado - Google Patents

Tratamento de angioedema hereditário com vetores de terapia gênica específicos do fígado

Info

Publication number
BR112022009279A2
BR112022009279A2 BR112022009279A BR112022009279A BR112022009279A2 BR 112022009279 A2 BR112022009279 A2 BR 112022009279A2 BR 112022009279 A BR112022009279 A BR 112022009279A BR 112022009279 A BR112022009279 A BR 112022009279A BR 112022009279 A2 BR112022009279 A2 BR 112022009279A2
Authority
BR
Brazil
Prior art keywords
liver
treatment
gene therapy
specific gene
therapy vectors
Prior art date
Application number
BR112022009279A
Other languages
English (en)
Inventor
Colosi Peter
Original Assignee
Biomarin Pharm 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 Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of BR112022009279A2 publication Critical patent/BR112022009279A2/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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/005—Enzyme inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/005—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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/10—Antioedematous agents; Diuretics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • C12N2750/14122—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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

TRATAMENTO DE ANGIOEDEMA HEREDITÁRIO COM VETORES DE TERAPIA GÊNICA ESPECÍFICOS DO FÍGADO. A presente invenção refere-se a composições e métodos para tratar uma deficiência de inibidor de C1 esterase normalizando os níveis da proteína inibidora de C1 esterase em um indivíduo com HAE.
BR112022009279A 2019-11-14 2020-11-13 Tratamento de angioedema hereditário com vetores de terapia gênica específicos do fígado BR112022009279A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962935359P 2019-11-14 2019-11-14
US202063016365P 2020-04-28 2020-04-28
PCT/US2020/060337 WO2021097157A1 (en) 2019-11-14 2020-11-13 Treatment of hereditary angioedema with liver-specific gene therapy vectors

Publications (1)

Publication Number Publication Date
BR112022009279A2 true BR112022009279A2 (pt) 2022-09-06

Family

ID=73834586

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022009279A BR112022009279A2 (pt) 2019-11-14 2020-11-13 Tratamento de angioedema hereditário com vetores de terapia gênica específicos do fígado

Country Status (14)

Country Link
US (1) US20230340078A1 (pt)
EP (1) EP4058475A1 (pt)
JP (1) JP2023503850A (pt)
KR (1) KR20220098210A (pt)
CN (1) CN114829391A (pt)
AU (1) AU2020384294A1 (pt)
BR (1) BR112022009279A2 (pt)
CA (1) CA3161154A1 (pt)
CL (1) CL2022001260A1 (pt)
CO (1) CO2022005641A2 (pt)
IL (1) IL292717A (pt)
MX (1) MX2022005869A (pt)
TW (1) TW202128733A (pt)
WO (1) WO2021097157A1 (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3196778A1 (en) 2020-11-02 2022-05-05 Biomarin Pharmaceutical, Inc. Process for enriching adeno-associated virus
EP4355887A2 (en) * 2021-06-17 2024-04-24 Meiragtx UK II Limited Aav manufacturing methods
TW202332472A (zh) 2021-10-01 2023-08-16 美商拜奧馬林製藥公司 利用ａａｖ基因療法載體進行之遺傳性血管水腫治療及治療性調配物

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
ATE78293T1 (de) 1983-05-27 1992-08-15 Texas A & M Univ Sys Verfahren zur herstellung eines rekombinanten baculovirus-expressionsvektors.
DK518384A (da) 1984-01-31 1985-07-01 Idaho Res Found Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
WO2003033710A1 (fr) 2001-10-16 2003-04-24 National Institute Of Advanced Industrial Science And Technology Nouvelle n-acetylglucosamine transferase, acide nucleique codant cette enzyme, anticorps dirige contre cette enzyme et utilisation de l'enzyme pour diagnostiquer un cancer ou une tumeur
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
CA2406745C (en) 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
AU2003212708A1 (en) 2002-03-05 2003-09-16 Stichting Voor De Technische Wetenschappen Baculovirus expression system
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
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
DK3024498T3 (da) * 2013-07-22 2020-03-02 Childrens Hospital Philadelphia Aav-variant og sammensætninger, fremgangsmåder og anvendelser til genoverførsel til celler, organer og væv
CA2987103A1 (en) * 2015-05-28 2016-12-01 Cornell University Adeno-associated virus mediated delivery of c1ei as a therapy for angioedema
US11584780B2 (en) 2016-07-26 2023-02-21 Biomarin Pharmaceutical Inc. Adeno-associated virus capsid proteins
AU2019209770A1 (en) * 2018-01-16 2020-09-03 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity

Also Published As

Publication number Publication date
CL2022001260A1 (es) 2023-03-10
JP2023503850A (ja) 2023-02-01
KR20220098210A (ko) 2022-07-11
WO2021097157A1 (en) 2021-05-20
CA3161154A1 (en) 2021-05-20
CO2022005641A2 (es) 2022-05-20
EP4058475A1 (en) 2022-09-21
CN114829391A (zh) 2022-07-29
IL292717A (en) 2022-07-01
US20230340078A1 (en) 2023-10-26
MX2022005869A (es) 2022-06-14
TW202128733A (zh) 2021-08-01
AU2020384294A1 (en) 2022-06-02

Similar Documents

Publication Publication Date Title
BR112022009279A2 (pt) Tratamento de angioedema hereditário com vetores de terapia gênica específicos do fígado
MX2018014256A (es) Metodos de terapia genica para enfermedades y condiciones relacionadas con la edad.
EP4085919A3 (en) Compositions and methods to treat cancer
BR112017016068A2 (pt) composições para modulação de expressão de c9orf72
BR112018007453A2 (pt) genes modificados de ataxia de friedreich e vetores para a terapia gênica
BR112018007447A2 (pt) terapia de combinação para tratamento de malignidades
BR112021017430A2 (pt) Inibidores da proteína tirosina fosfatase e métodos de uso dos mesmos
BR112018007304A2 (pt) terapia de combinação para tratamento de malignidades
BR112018007656A2 (pt) terapia de combinação para tratamento de doenças malignas
DOP2007000015A (es) Usos terapéuticos de inhibidores de rtp801
MX2022002198A (es) Composiciones y métodos para modular el empalme y la expresión de proteínas.
BR112018007671A2 (pt) terapia de combinação para tratamento de malignidades
BR112022007010A2 (pt) Terapia gênica para doença de alzheimer
BR112018008344A2 (pt) molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição
BR112021021047A2 (pt) Métodos e composições para modulação de emenda e tradução
MX2022001004A (es) Inhibidores de enzimas.
MX2020013313A (es) Terapia genica cardiaca con aav para cardiomiopatia.
BR112022005757A2 (pt) Uricase aperfeiçoada e método de tratamento de hiperuricemia usando a mesma
CL2021001488A1 (es) Transposasa de piggybac mutada
BR112021024080A2 (pt) Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos
BR112023018944A2 (pt) Composição de terapia gênica e tratamento de cardiomiopatia arritmogênica ventricular direita
UY32896A (es) Composiciones de enzimas pancreáticas y métodos para tratar pancreatitis e insuficiencia pancreática
BR112022009710A2 (pt) Terapia de combinação compreendendo um inibidor de alk2 e um inibidor de jak2
BR112019011365A2 (pt) métodos para tratar câncer
BR112023000428A2 (pt) Métodos e composições para tratar epilepsia