EA202192543A1 - Вектор и способ для лечения синдрома ангельмана - Google Patents

Вектор и способ для лечения синдрома ангельмана

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Publication number
EA202192543A1
EA202192543A1 EA202192543A EA202192543A EA202192543A1 EA 202192543 A1 EA202192543 A1 EA 202192543A1 EA 202192543 A EA202192543 A EA 202192543A EA 202192543 A EA202192543 A EA 202192543A EA 202192543 A1 EA202192543 A1 EA 202192543A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
vector
angelman
syndrome
raav
Prior art date
Application number
EA202192543A
Other languages
English (en)
Inventor
Антонио Аруланандам
Кевин Нэш
Эдвин Вибер
Лянсянь Цао
Мин Чжон Ким
Original Assignee
Птс Терапьютикс, Инк.
Юниверсити Оф Сауз Флорида
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 Птс Терапьютикс, Инк., Юниверсити Оф Сауз Флорида filed Critical Птс Терапьютикс, Инк.
Publication of EA202192543A1 publication Critical patent/EA202192543A1/ru

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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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • 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
    • C12N9/93—Ligases (6)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Один описанный в данном документе аспект относится к вектору на основе рекомбинантного аденоассоциированного вируса (rAAV) и способу его применения для лечения синдрома Ангельмана. Другой описанный в данном документе аспект относится к вектору rAAV UBE3A и способу его применения для лечения дефицита UBE3A, например синдрома Ангельмана, у людей.
EA202192543A 2019-03-21 2020-03-20 Вектор и способ для лечения синдрома ангельмана EA202192543A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962821442P 2019-03-21 2019-03-21
PCT/US2020/024030 WO2020191366A1 (en) 2019-03-21 2020-03-20 Vector and method for treating angelman syndrome

Publications (1)

Publication Number Publication Date
EA202192543A1 true EA202192543A1 (ru) 2021-12-27

Family

ID=72521277

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192543A EA202192543A1 (ru) 2019-03-21 2020-03-20 Вектор и способ для лечения синдрома ангельмана

Country Status (17)

Country Link
US (1) US20220152223A1 (ru)
EP (1) EP3941530A4 (ru)
JP (1) JP2022525564A (ru)
KR (1) KR20210145180A (ru)
CN (1) CN114206393A (ru)
AR (1) AR118481A1 (ru)
AU (1) AU2020240136A1 (ru)
BR (1) BR112021018354A2 (ru)
CA (1) CA3133455A1 (ru)
CL (1) CL2021002427A1 (ru)
CO (1) CO2021013967A2 (ru)
EA (1) EA202192543A1 (ru)
IL (1) IL286476A (ru)
MX (1) MX2021011198A (ru)
SG (1) SG11202109736RA (ru)
TW (1) TW202102672A (ru)
WO (1) WO2020191366A1 (ru)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020237130A1 (en) 2019-05-22 2020-11-26 The University Of North Carolina At Chapel Hill Ube3a genes and expression cassettes and their use
KR20230128001A (ko) * 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도
WO2022272171A2 (en) * 2021-06-25 2022-12-29 University Of South Florida Secreted ube3a for treatment of neurological disorders
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001268163A1 (en) * 2000-06-01 2001-12-11 Genaissance Pharmaceuticals, Inc. Haplotypes of the ube3a gene
US20100190656A1 (en) * 2008-08-08 2010-07-29 Integrated Diagnostics, Inc. Breast Cancer Specific Markers and Methods of Use
WO2011133874A1 (en) * 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
US9254311B2 (en) * 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9447433B2 (en) * 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
CN120174012A (zh) * 2013-10-24 2025-06-20 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
FI3628334T3 (fi) * 2014-03-21 2023-09-15 Genzyme Corp Geenihoito verkkokalvon pigmenttirappeuma
EP3215178A4 (en) * 2014-11-06 2018-07-25 Yeda Research and Development Co. Ltd Treatment of cns inflammatory disorders
HUE062186T2 (hu) * 2015-05-07 2023-09-28 Univ South Florida Módosított UBE3A gén az Angelman-szindróma génterápiás megközelítésére
PL3411484T3 (pl) * 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
US20200138921A1 (en) * 2017-06-23 2020-05-07 The Trustees Of Columbia University In The City Of New York Methods of preventing and treating diseases characterized by synaptic dysfunction and neurodegeneration including alzheimer's disease
EP3645012A4 (en) * 2017-06-28 2021-06-30 University Of South Florida MODIFIED UBE3A GENE FOR A GENE THERAPY APPROACH FOR ANGELMAN SYNDROME
WO2019155833A1 (ja) * 2018-02-07 2019-08-15 学校法人日本医科大学 改良型アデノ随伴ウイルスベクター

Also Published As

Publication number Publication date
TW202102672A (zh) 2021-01-16
IL286476A (en) 2021-12-01
KR20210145180A (ko) 2021-12-01
EP3941530A1 (en) 2022-01-26
BR112021018354A2 (pt) 2021-11-23
WO2020191366A1 (en) 2020-09-24
SG11202109736RA (en) 2021-10-28
CA3133455A1 (en) 2020-09-24
CO2021013967A2 (es) 2022-02-28
US20220152223A1 (en) 2022-05-19
EP3941530A4 (en) 2022-12-14
AR118481A1 (es) 2021-10-06
CL2021002427A1 (es) 2022-07-01
JP2022525564A (ja) 2022-05-17
AU2020240136A1 (en) 2021-09-30
MX2021011198A (es) 2022-03-04
CN114206393A (zh) 2022-03-18

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