CL2022000292A1 - Partículas virales para uso en el tratamiento de sinucleinopatías tales como enfermedad de parkinson mediante terapia genética - Google Patents

Partículas virales para uso en el tratamiento de sinucleinopatías tales como enfermedad de parkinson mediante terapia genética

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Publication number
CL2022000292A1
CL2022000292A1 CL2022000292A CL2022000292A CL2022000292A1 CL 2022000292 A1 CL2022000292 A1 CL 2022000292A1 CL 2022000292 A CL2022000292 A CL 2022000292A CL 2022000292 A CL2022000292 A CL 2022000292A CL 2022000292 A1 CL2022000292 A1 CL 2022000292A1
Authority
CL
Chile
Prior art keywords
treatment
gene therapy
synucleinopathies
disease
parkinson
Prior art date
Application number
CL2022000292A
Other languages
English (en)
Inventor
Aseguinolaza Gloria Gonzalez
Perez José Luis Lanciego
Ralph Michael Linden
Original Assignee
UCB Biopharma SRL
Fundacion Para La Investig Medica Aplicada
Consorcio Centro De Investig Biomedica En Red
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, Fundacion Para La Investig Medica Aplicada, Consorcio Centro De Investig Biomedica En Red filed Critical UCB Biopharma SRL
Publication of CL2022000292A1 publication Critical patent/CL2022000292A1/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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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
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    • 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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    • A61K38/46—Hydrolases (3)
    • 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
    • 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)
    • C12N9/2405—Glucanases
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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)
  • 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)
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  • Psychology (AREA)
  • Diabetes (AREA)
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  • Toxicology (AREA)
  • Urology & Nephrology (AREA)
  • Endocrinology (AREA)
  • Mycology (AREA)

Abstract

La presente invención se relaciona con partículas virales para uso en el tratamiento de sinucleinopatías, particularmente enfermedad de Parkinson esporádica mediante terapia genética. Más específicamente, la presente invención se relaciona con una partícula viral para uso en el tratamiento de sinucleinopatía mediante terapia genética en un sujeto que lo necesita, comprendiendo dicha partícula viral un constructo de ácido nucleico que incluye un transgén que codifica una glucocerebrosidasa.
CL2022000292A 2019-08-12 2022-02-04 Partículas virales para uso en el tratamiento de sinucleinopatías tales como enfermedad de parkinson mediante terapia genética CL2022000292A1 (es)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
CL2022000292A1 true CL2022000292A1 (es) 2022-10-21

Family

ID=67659024

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2022000292A CL2022000292A1 (es) 2019-08-12 2022-02-04 Partículas virales para uso en el tratamiento de sinucleinopatías tales como enfermedad de parkinson mediante terapia genética

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
TW202214864A (zh) * 2020-08-06 2022-04-16 應用醫學研究基金會 用於藉基因療法治療諸如阿茲海默症之Ｔａｕ蛋白病變的病毒顆粒

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
JP7254815B2 (ja) * 2017-10-03 2023-04-10 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
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
AR119609A1 (es) 2021-12-29
CO2022001192A2 (es) 2022-07-19
AU2020328827A1 (en) 2022-03-03
TW202120687A (zh) 2021-06-01
KR20220099944A (ko) 2022-07-14
CA3149844A1 (en) 2021-02-18
ECSP22008949A (es) 2022-03-31
PE20220601A1 (es) 2022-04-25
BR112022002615A2 (pt) 2022-05-03
WO2021028299A1 (en) 2021-02-18
MX2022001676A (es) 2022-05-03
EP4013437A1 (en) 2022-06-22
IL290357A (en) 2022-04-01
JP2023500011A (ja) 2023-01-04
CN114786694A (zh) 2022-07-22

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