MX2021008135A - Construcciones de terapia genica para el tratamiento de la enfermedad de wilson. - Google Patents

Construcciones de terapia genica para el tratamiento de la enfermedad de wilson.

Info

Publication number
MX2021008135A
MX2021008135A MX2021008135A MX2021008135A MX2021008135A MX 2021008135 A MX2021008135 A MX 2021008135A MX 2021008135 A MX2021008135 A MX 2021008135A MX 2021008135 A MX2021008135 A MX 2021008135A MX 2021008135 A MX2021008135 A MX 2021008135A
Authority
MX
Mexico
Prior art keywords
gene therapy
wilson disease
atp7b
treating wilson
therapy constructs
Prior art date
Application number
MX2021008135A
Other languages
English (en)
Inventor
Samuel Wadsworth
Christine Livingston
Original Assignee
Ultragenyx Pharmaceutical 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 Ultragenyx Pharmaceutical Inc filed Critical Ultragenyx Pharmaceutical Inc
Publication of MX2021008135A publication Critical patent/MX2021008135A/es

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    • 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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Esta solicitud se refiere generalmente a vectores virales adenoasociados que codifican un ATP7B truncado aunque funcional para usarse en la terapia génica para tratar la enfermedad de Wilson (WD); el ATP7B truncado descrito en este documento tiene varias ventajas sobre el ATP7B de tipo salvaje, tales como una mayor eficacia y un rendimiento de fabricación mejorado.
MX2021008135A 2019-01-04 2020-01-03 Construcciones de terapia genica para el tratamiento de la enfermedad de wilson. MX2021008135A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962788324P 2019-01-04 2019-01-04
US201962834830P 2019-04-16 2019-04-16
PCT/US2020/012131 WO2020142653A1 (en) 2019-01-04 2020-01-03 Gene therapy constructs for treating wilson disease

Publications (1)

Publication Number Publication Date
MX2021008135A true MX2021008135A (es) 2021-08-11

Family

ID=69528954

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021008135A MX2021008135A (es) 2019-01-04 2020-01-03 Construcciones de terapia genica para el tratamiento de la enfermedad de wilson.

Country Status (21)

Country Link
US (1) US20220090131A1 (es)
EP (2) EP3906066B1 (es)
JP (2) JP7531495B2 (es)
KR (1) KR20210112339A (es)
CN (1) CN113518628A (es)
AU (1) AU2020204679A1 (es)
BR (1) BR112021013140A2 (es)
CA (1) CA3124880A1 (es)
CO (1) CO2021010088A2 (es)
DK (1) DK3906066T5 (es)
ES (1) ES2970216T3 (es)
FI (1) FI3906066T3 (es)
HR (1) HRP20231750T1 (es)
HU (1) HUE065782T2 (es)
IL (1) IL284554A (es)
MX (1) MX2021008135A (es)
PL (1) PL3906066T3 (es)
PT (1) PT3906066T (es)
SG (1) SG11202106855YA (es)
TW (1) TWI848038B (es)
WO (1) WO2020142653A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11473106B2 (en) 2016-12-30 2022-10-18 The Trustees Of The University Of Pennsylvania Gene therapy for treating Wilson's disease
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
MX2020010466A (es) 2018-04-03 2021-01-08 Vectores de virus que evitan anticuerpos.
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
MX2021013913A (es) * 2019-05-14 2022-03-17 Univ Duke Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa.
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN113694218A (zh) * 2021-08-30 2021-11-26 昆明理工大学 一种atp7b基因p992l突变的基因修复治疗载体
AU2022407655A1 (en) * 2021-12-10 2024-06-20 Aavantibio, Inc. Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector
AU2022407548A1 (en) * 2021-12-10 2024-07-18 Aavantibio, Inc. Compositions comprising kozak sequences selected for enhanced expression
WO2024193638A1 (en) * 2023-03-22 2024-09-26 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of wilson disease

Family Cites Families (17)

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US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
NZ578982A (en) 2001-11-13 2011-03-31 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
ATE490307T1 (de) 2003-05-21 2010-12-15 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
US8999678B2 (en) 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
CA2713338C (en) 2008-01-29 2021-10-26 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
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AU2011209743B2 (en) 2010-01-28 2016-03-10 The Children's Hospital Of Philadelphia A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
HUE054768T2 (hu) * 2014-05-02 2021-09-28 Genzyme Corp AAV vektorok retina és CNS génterápiára
KR102526711B1 (ko) 2014-09-24 2023-04-27 시티 오브 호프 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법
KR102526616B1 (ko) * 2014-12-17 2023-04-27 푼다시온 파라 라 인베스티가시온 메디카 아플리카다 윌슨병 및 기타 병태의 치료에 사용되기 위한 핵산 구조물 및 유전자 치료용 벡터
US11473106B2 (en) * 2016-12-30 2022-10-18 The Trustees Of The University Of Pennsylvania Gene therapy for treating Wilson's disease
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WO2023230466A1 (en) * 2022-05-23 2023-11-30 Lutsenka Svetlana Compositions and methods for treating wilson's disease

Also Published As

Publication number Publication date
US20220090131A1 (en) 2022-03-24
HRP20231750T1 (hr) 2024-05-24
PT3906066T (pt) 2024-02-06
CA3124880A1 (en) 2020-07-09
EP4269579A3 (en) 2024-01-24
TWI848038B (zh) 2024-07-11
AU2020204679A1 (en) 2021-07-15
DK3906066T3 (da) 2024-02-05
PL3906066T3 (pl) 2024-09-02
EP4269579A2 (en) 2023-11-01
FI3906066T3 (fi) 2024-01-30
CN113518628A (zh) 2021-10-19
WO2020142653A1 (en) 2020-07-09
BR112021013140A2 (pt) 2021-11-30
ES2970216T3 (es) 2024-05-27
DK3906066T5 (da) 2024-08-05
KR20210112339A (ko) 2021-09-14
JP7531495B2 (ja) 2024-08-09
EP3906066A1 (en) 2021-11-10
JP2022516283A (ja) 2022-02-25
SG11202106855YA (en) 2021-07-29
IL284554A (en) 2021-08-31
TW202043479A (zh) 2020-12-01
EP3906066B1 (en) 2023-11-01
HUE065782T2 (hu) 2024-06-28
CO2021010088A2 (es) 2021-09-20
JP2024054426A (ja) 2024-04-16

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