CO2021013967A2 - Vector y método para tratar el síndrome de angelman - Google Patents

Vector y método para tratar el síndrome de angelman

Info

Publication number
CO2021013967A2
CO2021013967A2 CONC2021/0013967A CO2021013967A CO2021013967A2 CO 2021013967 A2 CO2021013967 A2 CO 2021013967A2 CO 2021013967 A CO2021013967 A CO 2021013967A CO 2021013967 A2 CO2021013967 A2 CO 2021013967A2
Authority
CO
Colombia
Prior art keywords
vector
angelman syndrome
treat
treat angelman
ube3a
Prior art date
Application number
CONC2021/0013967A
Other languages
English (en)
Inventor
Antonio Arulanandam
Kevin Nash
Edwin Weeber
Liangxian Cao
Min Jung Kim
Original Assignee
Ptc Therapeutics Inc
Univ South Florida
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 Ptc Therapeutics Inc, Univ South Florida filed Critical Ptc Therapeutics Inc
Publication of CO2021013967A2 publication Critical patent/CO2021013967A2/es

Links

Classifications

    • 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

Un aspecto descrito en la presente descripción se refiere a un vector de virus adenoasociado recombinante (rAAV) y un método para su uso para tratar el síndrome de Angelman. Otro aspecto descrito en la presente descripción es un vector rAAV de UBE3A y un método para su uso para tratar una deficiencia de UBE3A, por ejemplo, el síndrome de Angelman, en humanos.
CONC2021/0013967A 2019-03-21 2021-10-19 Vector y método para tratar el síndrome de angelman CO2021013967A2 (es)

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
CO2021013967A2 true CO2021013967A2 (es) 2022-02-28

Family

ID=72521277

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0013967A CO2021013967A2 (es) 2019-03-21 2021-10-19 Vector y método para tratar el síndrome de angelman

Country Status (17)

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

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20210158859A (ko) * 2019-05-22 2021-12-31 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 Ube3a 유전자 및 발현 카세트 및 이들의 용도
WO2022272171A2 (en) * 2021-06-25 2022-12-29 University Of South Florida Secreted ube3a for treatment of neurological disorders

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001092582A1 (en) * 2000-06-01 2001-12-06 Genaissance Pharmaceuticals, Inc. Haplotypes of the ube3a gene
WO2010017515A2 (en) * 2008-08-08 2010-02-11 Integrated Diagnostics Inc. Breast cancer specific markers and methods of use
US9546369B2 (en) * 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
JP5704361B2 (ja) * 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2015060722A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
SI3119437T1 (sl) * 2014-03-21 2020-01-31 Genzyme Corporation Genska terapija za retinitis pigmentoza
WO2016071911A1 (en) * 2014-11-06 2016-05-12 Yeda Research And Development Co. Ltd. Treatment of cns inflammatory disorders
WO2016179584A1 (en) * 2015-05-07 2016-11-10 University Of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
HRP20231451T1 (hr) * 2016-02-05 2024-03-01 Emory University Ubrizgavanje jednolančanog ili samo-komplementarnog adeno-povezanog virusa 9 u cerebrospinalnu tekućinu
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
CN110869031A (zh) * 2017-06-28 2020-03-06 南佛罗里达大学 用于天使人综合征的基因治疗方法的修饰的ube3a基因

Also Published As

Publication number Publication date
EA202192543A1 (ru) 2021-12-27
CA3133455A1 (en) 2020-09-24
IL286476A (en) 2021-12-01
WO2020191366A1 (en) 2020-09-24
KR20210145180A (ko) 2021-12-01
EP3941530A1 (en) 2022-01-26
US20220152223A1 (en) 2022-05-19
AR118481A1 (es) 2021-10-06
CN114206393A (zh) 2022-03-18
CL2021002427A1 (es) 2022-07-01
EP3941530A4 (en) 2022-12-14
TW202102672A (zh) 2021-01-16
JP2022525564A (ja) 2022-05-17
MX2021011198A (es) 2022-03-04
AU2020240136A1 (en) 2021-09-30
SG11202109736RA (en) 2021-10-28
BR112021018354A2 (pt) 2021-11-23

Similar Documents

Publication Publication Date Title
CO2021013967A2 (es) Vector y método para tratar el síndrome de angelman
CL2022003763A1 (es) Anticuerpos humanos contra glicoproteína del virus ébola
BR112019001737A2 (pt) expressão de pten-long com vírus oncolíticos
CO2017006580A2 (es) Anticuerpos humanos para hemaglutinina de influenza
BR112019000278A2 (pt) neurotoxina botulínica e seus derivados
CL2017002731A1 (es) Adenovirus oncolítico que codifica una proteína b7
BR112016028066A2 (pt) composição de vacina; idessuis; ridessuis; anticorpo; composição parenteral; uso de uma proteína idessuis ou ridessuis; e célula hospedeira
AR057586A1 (es) Vacuna combinada que comprende un virus atenuado de la diarrea viral bovina
BRPI1013780A2 (pt) composições para imunização contra staphylococcus aureus.
BR112022007677A2 (pt) L-asparaginase recombinante
AR093578A1 (es) Metodos para la elaboracion de polipeptidos procesados proteoliticamente
CO2021010786A2 (es) Toxinas apxia, apxiia y apxiiia inactivadas
EA202190708A1 (ru) Модуляторы экспрессии pnpla3
UY36820A (es) Anticuerpos monoclonales específicos para el antígeno p del virus respiratorio sincicial humano (vrsh), producidos y secretados por hibridomas celulares, útiles para la detección y el diagnóstico de la infección causada por vrsh
EA201992170A1 (ru) Модуляторы экспрессии pcsk9
CL2021002727A1 (es) Vacuna con subunidades del vfpc
BR112019003816A2 (pt) composição de tratamento de enxágue dos cabelos, método para tratamento de cabelos quimicamente danificados e uso de uma composição
EA201890753A1 (ru) Применение триентина для доставки меди в ишемизированную ткань
EP3956439A4 (en) RECOMBINANT CLASSICAL SWINE FEVER VIRUS
AR104271A1 (es) Vacuna contra las paperas en base al virus jeryl lynn 2 recombinante
PE20150369A1 (es) Composicion y metodo para el control de nematodos fitoparasitos
BR112021024855A2 (pt) Composições de vírus adenoassociados para transferência de gene arsa e métodos de uso das mesmas
BR112017016205A2 (pt) thymosin alpha 1 para uso no tratamento de fibrose cística
BR112021026616A2 (pt) Composições de exenatidas para administração pulmonar e uso das mesmas
BR112022009587A2 (pt) Anticorpo anti-pcsk9 e uso do mesmo