CO2022004652A2 - Casete de transferencia de aav - Google Patents

Casete de transferencia de aav

Info

Publication number
CO2022004652A2
CO2022004652A2 CONC2022/0004652A CO2022004652A CO2022004652A2 CO 2022004652 A2 CO2022004652 A2 CO 2022004652A2 CO 2022004652 A CO2022004652 A CO 2022004652A CO 2022004652 A2 CO2022004652 A2 CO 2022004652A2
Authority
CO
Colombia
Prior art keywords
aav transfer
transfer cassette
plasmids
cassettes
npc1
Prior art date
Application number
CONC2022/0004652A
Other languages
English (en)
Inventor
David Dismuke
Original Assignee
Stridebio 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 Stridebio Inc filed Critical Stridebio Inc
Publication of CO2022004652A2 publication Critical patent/CO2022004652A2/es

Links

Classifications

    • 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
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14044—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
    • C12N2750/14141—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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invención describe casetes de transferencia de AAV y plásmidos que se utilizan en la producción de vectores virales adenoasociados recombinantes (rAAV). Los casetes y plásmidos divulgados comprenden uno o más transgenes que tienen eficacia terapéutica en la mejora, tratamiento y/o prevención de una o más enfermedades o trastornos, como la enfermedad de Niemann-Pick, Tipo C1 (NPC1).
CONC2022/0004652A 2019-10-17 2022-04-12 Casete de transferencia de aav CO2022004652A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962916749P 2019-10-17 2019-10-17
US201962923253P 2019-10-18 2019-10-18
PCT/US2020/056015 WO2021076911A1 (en) 2019-10-17 2020-10-16 Aav transfer cassette

Publications (1)

Publication Number Publication Date
CO2022004652A2 true CO2022004652A2 (es) 2022-04-29

Family

ID=73344131

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2022/0004652A CO2022004652A2 (es) 2019-10-17 2022-04-12 Casete de transferencia de aav

Country Status (14)

Country Link
US (2) US20240131185A1 (es)
EP (1) EP4045524A1 (es)
JP (1) JP2022551744A (es)
KR (1) KR20220083708A (es)
CN (1) CN114585743A (es)
AU (1) AU2020367825A1 (es)
BR (1) BR112022006946A2 (es)
CA (1) CA3157702A1 (es)
CL (1) CL2022000938A1 (es)
CO (1) CO2022004652A2 (es)
IL (1) IL292264A (es)
MX (1) MX2022004353A (es)
TW (1) TW202128737A (es)
WO (1) WO2021076911A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease
WO2023221942A1 (en) * 2022-05-16 2023-11-23 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating glutaric aciduria type i

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2982129A1 (en) * 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
EP3285788A4 (en) * 2015-04-23 2018-12-05 University of Massachusetts Modulation of aav vector transgene expression
ES2869284T3 (es) * 2015-12-11 2021-10-25 California Inst Of Techn Péptidos de diana para dirigir virus adenoasociados (AAV)
AU2018311504A1 (en) * 2017-07-31 2020-03-12 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases

Also Published As

Publication number Publication date
IL292264A (en) 2022-06-01
MX2022004353A (es) 2022-07-19
EP4045524A1 (en) 2022-08-24
US20210128652A1 (en) 2021-05-06
TW202128737A (zh) 2021-08-01
US20240131185A1 (en) 2024-04-25
CL2022000938A1 (es) 2023-01-20
KR20220083708A (ko) 2022-06-20
CN114585743A (zh) 2022-06-03
BR112022006946A2 (pt) 2022-08-23
JP2022551744A (ja) 2022-12-13
WO2021076911A1 (en) 2021-04-22
AU2020367825A1 (en) 2022-05-12
CA3157702A1 (en) 2021-04-22

Similar Documents

Publication Publication Date Title
CO2022004652A2 (es) Casete de transferencia de aav
CO2021008120A2 (es) Vectores virales recombinantes y ácidos nucleicos para producirlos
CL2020001360A1 (es) Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesis.
CO2020004738A2 (es) Variantes de cápsides de virus adenoasociados y métodos de uso de estas
PE20190129A1 (es) Capsides variantes de virus adenoasociados y metodos de uso de estas
CL2018000170A1 (es) Vectores de aav para la terapia génica de la retina y el snc (divisional de solicitud 201602713)
CO2018000134A2 (es) Factor ix modificado, y composiciones para la transferencia de genes a células, órganos y tejidos
WO2015158749A3 (de) Viraler vektor für den zielgerichteten gentransfer in gehirn und rückenmark
CL2018000740A1 (es) Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden
DOP2016000237A (es) Terapia genica para la retinitis pigmentaria
MX2018007234A (es) Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.
NZ726316A (en) Adenoassociated virus vectors for the treatment of lysosomal storage disorders
BR112022006530A2 (pt) Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c
CL2023000629A1 (es) Partículas virales modificadas y usos de estas
BR112018075692A2 (pt) genes cln1 otimizados e cassetes de expressão e seu uso
EP4055174A4 (en) TRANSGENIC CASSETTES, AAV VECTORS AND AAV VIRAL VECTORS FOR EXPRESSION OF THE SLC6A1 GENE WITH OPTIMIZED HUMAN CODONS
CO2022001833A2 (es) Proteína vp1 modificada aislada de la cápside del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en la misma.
CL2021002236A1 (es) Inserción de vectores de virus adeno-asociados de b-sarcoglicano y el tratamiento de distrofia muscular
CO2021009575A2 (es) Composiciones y métodos para la administración de polipéptidos cftr
AR120252A1 (es) Casete de transferencia de aav
CO2017013361A2 (es) Angiogénesis usando células madre placentarias estimuladas
WO2019222314A8 (en) Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a
BR112017022621A2 (pt) liberação do gene de smad7 como uma substância terapêutica
PH12020551319A1 (en) Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
UY38238A (es) Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida