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

Info

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

Links

Classifications

    • 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
    • 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
    • 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
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • 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
    • 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)
    • C12Y302/01045—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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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

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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Rheumatology (AREA)
  • Endocrinology (AREA)
  • Urology & Nephrology (AREA)
  • Diabetes (AREA)
  • Immunology (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
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
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
CL2022000292A1 (es) 2022-10-21

Similar Documents

Publication Publication Date Title
PH12019502793A1 (en) Aadc polynucleotides for the treatment of parkinson`s disease
WO2017191274A3 (en) Rna encoding a therapeutic protein
PH12017500746A1 (en) Carbidopa and l-dopa prodrugs and their use to treat parkinson's disease
MX2020003965A (es) Terapias genicas para los trastornos lisosomales.
MX2017005834A (es) Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
MX2018012880A (es) Metodos para proporcionar arn de hebra sencilla.
SA516380328B1 (ar) مركبات مستبدلة-8،6-دايوكسابيسيكلو[3-2-1]أوكتان-3،2-دايول asgpr كعوامل استهداف
UY33480A (es) Compuestos terapeuticos, composicion farmaceutica que lo comprende, metodo de tratamiento y su uso para la manufactura de un medicamento.
MX2020004005A (es) Terapias genicas para los trastornos lisosomales.
CL2015001241A1 (es) Uso de interleucina 2 (il-2) para tratar trastornos inflamatorios o relacionados con la autoinmunidad (div. sol. n° 2608-13)
MX355267B (es) Análogos de carba-nucleósido 2´-sustituidos para tratamiento antivírico.
CR20130560A (es) Compuestos de bezotiazol y su uso farmacéutico
UY32629A (es) Compuestos antivirales, composiciones y uso para la manufactura de un medicamento para el tratamiento de trastornos asociados con hepatitis c.
MX2020010694A (es) Particulas de raav que codifican mir-708 y usos del mismo.
AR100560A1 (es) Inhibición o regulación por disminución de la glucógeno sintasa mediante la creación de codones de terminación prematuros utilizando oligonucléotidos antisentido
BR112019001253A2 (pt) composto, composição farmacêutica, e, métodos de tratamento de câncer ou de atrofia muscular e de doença de parkinson.
MX2017017171A (es) Compuestos terapeuticos y composiciones para el tratamiento de trastornos sociales y trastornos de uso de sustancias.
BR112018017077A2 (pt) terapias de combinação para tratamento de atrofia muscular espinhal
ECSP22008949A (es) Partículas virales para uso en el tratamiento de sinucleinopatías tal como enfermedad de parkinson mediante terapia génica
BR112014026817A2 (pt) derivados do pantotenato para o tratamento de transtornos neurológicos
MX2018006636A (es) Fosfatos ciclicos y fosforoamidatos ciclicos para el tratamiento de trastornos neurologicos.
BR112015010196A2 (pt) métodos de tratar doença do fígado
BR112015022308A8 (pt) oligonucleotídeos antissenso para o tratamento de células-tronco cancerosas
MX2021005435A (es) Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2).
MX2021004154A (es) Transgen de codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 3 (pfic3).