MX2022001676A - Particulas virales para uso en el tratamiento de sinucleinopatias tal como enfermedad de parkinson mediante terapia genica. - Google Patents

Particulas virales para uso en el tratamiento de sinucleinopatias tal como enfermedad de parkinson mediante terapia genica.

Info

Publication number
MX2022001676A
MX2022001676A MX2022001676A MX2022001676A MX2022001676A MX 2022001676 A MX2022001676 A MX 2022001676A MX 2022001676 A MX2022001676 A MX 2022001676A MX 2022001676 A MX2022001676 A MX 2022001676A MX 2022001676 A MX2022001676 A MX 2022001676A
Authority
MX
Mexico
Prior art keywords
gene therapy
diseases
viral particles
parkinson
treating
Prior art date
Application number
MX2022001676A
Other languages
English (en)
Inventor
Ralph Michael Linden
Aseguinolaza Gloria Gonzalez
Perez José Luis Lanciego
Original Assignee
Fundacion Para La Investig Medica Aplicada
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 Fundacion Para La Investig Medica Aplicada filed Critical Fundacion Para La Investig Medica Aplicada
Publication of MX2022001676A publication Critical patent/MX2022001676A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01062Glycosylceramidase (3.2.1.62)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/0004Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405Glucanases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychology (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Rheumatology (AREA)
  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Endocrinology (AREA)
  • Mycology (AREA)

Abstract

La presente descripción se relaciona con partículas virales para uso en el tratamiento de sinucleinopatías, particularmente enfermedad de Parkinson esporádica mediante terapia génica. Más específicamente, la presente invención se relaciona con una partícula viral para uso en el tratamiento de sinucleinopatía mediante terapia génica en un sujeto que lo necesita, comprendiendo dicha partícula viral un constructo de ácido nucleico que incluye un transgén que codifica una glucocerebrosidasa.
MX2022001676A 2019-08-12 2020-08-06 Particulas virales para uso en el tratamiento de sinucleinopatias tal como enfermedad de parkinson mediante terapia genica. MX2022001676A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP19382706 2019-08-12
PCT/EP2020/072087 WO2021028299A1 (en) 2019-08-12 2020-08-06 Viral particles for use in treating synucleinopathies such as parkinson's diseases by gene therapy

Publications (1)

Publication Number Publication Date
MX2022001676A true MX2022001676A (es) 2022-05-03

Family

ID=67659024

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022001676A MX2022001676A (es) 2019-08-12 2020-08-06 Particulas virales para uso en el tratamiento de sinucleinopatias tal como enfermedad de parkinson mediante terapia genica.

Country Status (17)

Country Link
US (1) US20220298528A1 (es)
EP (1) EP4013437A1 (es)
JP (1) JP2023500011A (es)
KR (1) KR20220099944A (es)
CN (1) CN114786694A (es)
AR (1) AR119609A1 (es)
AU (1) AU2020328827A1 (es)
BR (1) BR112022002615A2 (es)
CA (1) CA3149844A1 (es)
CL (1) CL2022000292A1 (es)
CO (1) CO2022001192A2 (es)
EC (1) ECSP22008949A (es)
IL (1) IL290357A (es)
MX (1) MX2022001676A (es)
PE (1) PE20220601A1 (es)
TW (1) TW202120687A (es)
WO (1) WO2021028299A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4192961A2 (en) * 2020-08-06 2023-06-14 Fundacion para la Investigacion Medica Aplicada Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2985235A1 (en) * 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. Glucocerebrosidase gene therapy for parkinson's disease
WO2019070893A1 (en) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISORDERS
WO2019068854A1 (en) * 2017-10-06 2019-04-11 Ospedale San Raffaele S.R.L. GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS

Also Published As

Publication number Publication date
EP4013437A1 (en) 2022-06-22
TW202120687A (zh) 2021-06-01
CL2022000292A1 (es) 2022-10-21
PE20220601A1 (es) 2022-04-25
ECSP22008949A (es) 2022-03-31
JP2023500011A (ja) 2023-01-04
IL290357A (en) 2022-04-01
BR112022002615A2 (pt) 2022-05-03
AR119609A1 (es) 2021-12-29
KR20220099944A (ko) 2022-07-14
WO2021028299A1 (en) 2021-02-18
CA3149844A1 (en) 2021-02-18
CO2022001192A2 (es) 2022-07-19
US20220298528A1 (en) 2022-09-22
CN114786694A (zh) 2022-07-22
AU2020328827A1 (en) 2022-03-03

Similar Documents

Publication Publication Date Title
WO2017191274A3 (en) Rna encoding a therapeutic protein
PH12019502793A1 (en) Aadc polynucleotides for the treatment of parkinson`s disease
MX2022012490A (es) Vectores de aav para la terapia genica de la retina y el snc.
PH12017501436A1 (en) Enhanced delivery of viral particles to the striatum and cortex
MY161415A (en) A homeopathic formulation
MX2020010694A (es) Particulas de raav que codifican mir-708 y usos del mismo.
WO2019070891A8 (en) Gene therapies for lysosomal disorders
EP4021559C0 (en) DELIVERY OF TUMOR TREATMENT FIELDS (TTFIELDS) TO THE NECK
MX2016001204A (es) Vectores virales adeno-asociados (aav) utiles para transducir tejido adiposo.
MX356865B (es) Nuevas soluciones terapéuticas para tratar la enfermedad de parkinson.
MX2021006253A (es) Terapias génicas para enfermedad neurodegenerativa.
MX2020013865A (es) Tratamiento genico combinatorio.
MX2021011958A (es) Terapias genicas para trastornos lisosomales.
MX2010008942A (es) Paramyxovirus eficaz como antitumoral.
MX2022001676A (es) Particulas virales para uso en el tratamiento de sinucleinopatias tal como enfermedad de parkinson mediante terapia genica.
MX2019000982A (es) Compuestos y composiciones y usos de los mismos.
MX2021005435A (es) Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2).
MX2023000968A (es) Particulas virales para usarse en el tratamiento contra tauopatias tales como enfermedad de alzheimer mediante terapia genica.
MX2021004154A (es) Transgen de codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 3 (pfic3).
PT3145553T (pt) Arn de interferência pequeno (arnsi) para a terapêutica da osteopetrose autossómica dominante de tipo 2 (ado2) causada por mutação génica de clcn7 (ado2 dependente de clcn7)
MX2023003994A (es) Construcciones de acido nucleico, vectores virales y particulas virales.
TN2016000460A1 (en) Aav vectors for retinal and cns gene therapy.