BR112022002615A2 - Partículas virais e uso das mesmas para fabricar uma composição para tratar sinucleinopatias - Google Patents

Partículas virais e uso das mesmas para fabricar uma composição para tratar sinucleinopatias

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Publication number
BR112022002615A2
BR112022002615A2 BR112022002615A BR112022002615A BR112022002615A2 BR 112022002615 A2 BR112022002615 A2 BR 112022002615A2 BR 112022002615 A BR112022002615 A BR 112022002615A BR 112022002615 A BR112022002615 A BR 112022002615A BR 112022002615 A2 BR112022002615 A2 BR 112022002615A2
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BR
Brazil
Prior art keywords
viral particles
manufacture
composition
synucleinopathies
treat
Prior art date
Application number
BR112022002615A
Other languages
English (en)
Inventor
Gonzalez Aseguinolaza Gloria
Luis Lanciego Perez José
Michael Linden Ralph
Original Assignee
Consorcio Centro De Investig Biomedica En Red
Fundacion Para La Investig Medica Aplicada
UCB Biopharma SRL
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 Consorcio Centro De Investig Biomedica En Red, Fundacion Para La Investig Medica Aplicada, UCB Biopharma SRL filed Critical Consorcio Centro De Investig Biomedica En Red
Publication of BR112022002615A2 publication Critical patent/BR112022002615A2/pt

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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
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    • 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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    • 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
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    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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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)
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  • Molecular Biology (AREA)
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  • Neurosurgery (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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Abstract

partículas virais e uso das mesmas para fabricar uma composição para tratar sinucleinopatias. a presente descrição se refere a partículas virais para uso no tratamento de sinucleinopatias, particularmente doenças de parkinson esporádicas pela terapia de gene. mais especificamente, a presente invenção se refere a uma partícula viral para uso no tratamento de sinucleinopatia pela terapia de gene em um sujeito em necessidade da mesma, a dita partícula viral compreendendo um construto de ácido nucléico incluindo um transgene codificando uma glicocerebrosidase.
BR112022002615A 2019-08-12 2020-08-06 Partículas virais e uso das mesmas para fabricar uma composição para tratar sinucleinopatias BR112022002615A2 (pt)

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
BR112022002615A2 true BR112022002615A2 (pt) 2022-05-03

Family

ID=67659024

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022002615A BR112022002615A2 (pt) 2019-08-12 2020-08-06 Partículas virais e uso das mesmas para fabricar uma composição para tratar sinucleinopatias

Country Status (17)

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

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
AR119609A1 (es) 2021-12-29
AU2020328827A1 (en) 2022-03-03
MX2022001676A (es) 2022-05-03
WO2021028299A1 (en) 2021-02-18
ECSP22008949A (es) 2022-03-31
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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