AR126870A1 - PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GALACTOSIDASA Y SU USO PARA TRATAR LA ENFERMEDAD DE FABRY - Google Patents

PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GALACTOSIDASA Y SU USO PARA TRATAR LA ENFERMEDAD DE FABRY

Info

Publication number
AR126870A1
AR126870A1 ARP220102286A ARP220102286A AR126870A1 AR 126870 A1 AR126870 A1 AR 126870A1 AR P220102286 A ARP220102286 A AR P220102286A AR P220102286 A ARP220102286 A AR P220102286A AR 126870 A1 AR126870 A1 AR 126870A1
Authority
AR
Argentina
Prior art keywords
protein
gal
capsid protein
nucleic acid
acid sequence
Prior art date
Application number
ARP220102286A
Other languages
English (en)
Inventor
Yunxiang Zhu
Peter Pechan
Anannya Banga
Villoslada Susana Gordo
Original Assignee
Canbridge Pharmaceuticals Inc
Logicbio Therapeutics 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 Canbridge Pharmaceuticals Inc, Logicbio Therapeutics Inc filed Critical Canbridge Pharmaceuticals Inc
Publication of AR126870A1 publication Critical patent/AR126870A1/es

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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

Landscapes

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

Abstract

La presente descripción proporciona composiciones que comprenden partículas de virus adenoasociado (AAV) aisladas, por ejemplo, recombinantes, que comprenden una proteína de la cápside hepatotrópica, por ejemplo, una proteína de la cápside sL65, para el suministro de una proteína GAL. La presente descripción también proporciona composiciones que comprenden un primer ácido nucleico que codifica una proteína de la cápside hepatotrópica, por ejemplo, una proteína de la cápside sL65, y un segundo ácido nucleico que comprende un transgén que codifica una proteína GAL. La presente descripción también proporciona métodos para producir partículas de AAV aisladas, por ejemplo, recombinantes, y métodos para suministrar una proteína GAL exógena en un sujeto y/o métodos para tratar a un sujeto que tiene una enfermedad o trastorno asociado con GAL, por ejemplo, un trastorno de almacenamiento lisosomal, por ejemplo, enfermedad de Fabry. Reivindicación 1: Una partícula de virus adenoasociado recombinante (rAAV) aislada caracterizada porque comprende una proteína de la cápside de AAV, en donde la proteína de la cápside comprende una secuencia de aminoácidos de SEQ ID Nº 45, o una secuencia de aminoácidos al menos 85% idéntica a esta, y un ácido nucleico que comprende un transgén que codifica una proteína a-galactosidasa (GAL). Reivindicación 52: Una composición caracterizada porque comprende un primer ácido nucleico que codifica una proteína de la cápside de AAV, en donde la proteína de la cápside comprende una secuencia de aminoácidos de SEQ ID Nº 45, o una secuencia de aminoácidos al menos 85% idéntica a esta, y un segundo ácido nucleico que comprende un transgén que codifica una proteína a-glucosidasa (GAL). Reivindicación 70: Un ácido nucleico aislado caracterizado porque comprende un transgén que codifica una proteína a-glucosidasa (GAL), en donde el transgén que codifica la proteína GAL comprende la secuencia de nucleótidos de una cualquiera de SEQ ID Nº 3 - 5, o una secuencia de nucleótidos al menos 85% idéntica a estas. Reivindicación 87: Una partícula de virus adenoasociado recombinante (rAAV) aislada caracterizada porque comprende un genoma viral de AAV que comprende o consiste en la secuencia de ácidos nucleicos de una cualquiera de SEQ ID Nº 31 - 41 y 51 - 61, y una proteína de la cápside que comprende la secuencia de aminoácidos de SEQ ID Nº 45.
ARP220102286A 2021-08-25 2022-08-25 PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GALACTOSIDASA Y SU USO PARA TRATAR LA ENFERMEDAD DE FABRY AR126870A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163237122P 2021-08-25 2021-08-25

Publications (1)

Publication Number Publication Date
AR126870A1 true AR126870A1 (es) 2023-11-22

Family

ID=85322265

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220102286A AR126870A1 (es) 2021-08-25 2022-08-25 PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GALACTOSIDASA Y SU USO PARA TRATAR LA ENFERMEDAD DE FABRY

Country Status (6)

Country Link
KR (1) KR20240073025A (es)
AR (1) AR126870A1 (es)
AU (1) AU2022332298A1 (es)
CA (1) CA3229998A1 (es)
TW (1) TW202330929A (es)
WO (1) WO2023028566A2 (es)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6083725A (en) * 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
WO2005033321A2 (en) * 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
JP2022516317A (ja) * 2019-01-04 2022-02-25 サンガモ セラピューティクス， インコーポレイテッド ファブリー病の処置のための方法及び組成物

Also Published As

Publication number Publication date
WO2023028566A2 (en) 2023-03-02
AU2022332298A1 (en) 2024-03-14
WO2023028566A3 (en) 2023-04-06
KR20240073025A (ko) 2024-05-24
CA3229998A1 (en) 2023-03-02
TW202330929A (zh) 2023-08-01

Similar Documents

Publication Publication Date Title
JP2022095633A (ja) ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
AU2022235633A1 (en) Modified stem cell memory T cells, methods of making and methods of using same
WO2017205423A1 (en) Pulmonary targeted cas9/crispr for in vivo editing of disease genes
Wang et al. Recoding of the vesicular stomatitis virus L gene by computer-aided design provides a live, attenuated vaccine candidate
JP2012523243A (ja) 改変間葉系幹細胞及びそれを使用した腫瘍の治療方法
CA2418977A1 (en) Gene delivery vectors with cell type specificity for primary human chondrocytes
US20240075109A1 (en) Telomerase reverse transcriptase-based therapies
CA3134523A1 (en) Compositions useful for treatment of pompe disease
KR20210108952A (ko) 미니 뉴클레오솜 코어 단백질 및 핵산 전달에서의 용도
WO2021128692A1 (en) Liver-specific promoter and application thereof
WO2022247873A1 (zh) 工程化的Cas12i核酸酶、效应蛋白及其用途
EP3133923B1 (en) Aav-based gene therapy for multiple sclerosis
AR126870A1 (es) PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GALACTOSIDASA Y SU USO PARA TRATAR LA ENFERMEDAD DE FABRY
AR126877A1 (es) PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GLUCOSIDASA ÁCIDA (GAA) Y SU USO PARA TRATAR LA ENFERMEDAD DE POMPE
CA3206484A1 (en) Engineered t cells
WO2022006306A2 (en) Compositions for genome editing and methods of use thereof
US20230390367A1 (en) Genetic approach to suppress coronaviruses
AU2020411330B2 (en) Liver-specific promoter and application thereof
US20230149563A1 (en) Compositions and methods for expressing factor ix for hemophilia b therapy
CN117003833A (zh) 肝脏特异性的腺相关病毒血清型及应用
CN116497002A (zh) 工程化的CasX核酸酶、效应蛋白及其用途
AR128450A1 (es) Vectores de virus adeno-asociados recombinantes, y métodos de uso de los mismos
WO2023147558A2 (en) Crispr methods for correcting bag3 gene mutations in vivo
WO2024006955A1 (en) Engineered t cells
CN117751185A (zh) 细菌二核苷酸环化酶的表达