ECSP22008949A - Partículas virales para uso en el tratamiento de sinucleinopatías tal como enfermedad de parkinson mediante terapia génica - Google Patents

Partículas virales para uso en el tratamiento de sinucleinopatías tal como enfermedad de parkinson mediante terapia génica

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Publication number
ECSP22008949A
ECSP22008949A ECSENADI20228949A ECDI202208949A ECSP22008949A EC SP22008949 A ECSP22008949 A EC SP22008949A EC SENADI20228949 A ECSENADI20228949 A EC SENADI20228949A EC DI202208949 A ECDI202208949 A EC DI202208949A EC SP22008949 A ECSP22008949 A EC SP22008949A
Authority
EC
Ecuador
Prior art keywords
gene therapy
synucleinopathies
disease
treatment
parkinson
Prior art date
Application number
ECSENADI20228949A
Other languages
English (en)
Inventor
Perez José Luis Lanciego
Aseguinolaza Gloria Gonzalez
Ralph Michael Linden
Original Assignee
UCB Biopharma SRL
Consorcio Centro De Investig Biomedica En Red
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 UCB Biopharma SRL, Consorcio Centro De Investig Biomedica En Red, Fundacion Para La Investig Medica Aplicada filed Critical UCB Biopharma SRL
Publication of ECSP22008949A publication Critical patent/ECSP22008949A/es

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    • 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
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    • C12N15/86—Viral vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01062—Glycosylceramidase (3.2.1.62)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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)
    • C12N9/2405—Glucanases
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • 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
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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

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  • 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)
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  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Psychology (AREA)
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  • Pathology (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Rheumatology (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.
ECSENADI20228949A 2019-08-12 2022-02-04 Partículas virales para uso en el tratamiento de sinucleinopatías tal como enfermedad de parkinson mediante terapia génica ECSP22008949A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP19382706 2019-08-12

Publications (1)

Publication Number Publication Date
ECSP22008949A true ECSP22008949A (es) 2022-03-31

Family

ID=67659024

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI20228949A ECSP22008949A (es) 2019-08-12 2022-02-04 Partículas virales para uso en el tratamiento de sinucleinopatías tal como enfermedad de parkinson mediante terapia génica

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
CN116390773A (zh) * 2020-08-06 2023-07-04 应用医学研究基金会 用于通过基因疗法治疗诸如阿尔茨海默病的Tau蛋白病的病毒颗粒

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
CN111542549A (zh) * 2017-10-03 2020-08-14 普利维尔治疗公司 用于溶酶体障碍的基因疗法
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
US20220298528A1 (en) 2022-09-22
CO2022001192A2 (es) 2022-07-19
IL290357A (en) 2022-04-01
TW202120687A (zh) 2021-06-01
KR20220099944A (ko) 2022-07-14
BR112022002615A2 (pt) 2022-05-03
AR119609A1 (es) 2021-12-29
AU2020328827A1 (en) 2022-03-03
MX2022001676A (es) 2022-05-03
WO2021028299A1 (en) 2021-02-18
CA3149844A1 (en) 2021-02-18
EP4013437A1 (en) 2022-06-22
PE20220601A1 (es) 2022-04-25
CN114786694A (zh) 2022-07-22
CL2022000292A1 (es) 2022-10-21
JP2023500011A (ja) 2023-01-04

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