CL2016003282A1 - Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington - Google Patents

Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington

Info

Publication number
CL2016003282A1
CL2016003282A1 CL2016003282A CL2016003282A CL2016003282A1 CL 2016003282 A1 CL2016003282 A1 CL 2016003282A1 CL 2016003282 A CL2016003282 A CL 2016003282A CL 2016003282 A CL2016003282 A CL 2016003282A CL 2016003282 A1 CL2016003282 A1 CL 2016003282A1
Authority
CL
Chile
Prior art keywords
igf2
aav
huntington
disease
diseases related
Prior art date
Application number
CL2016003282A
Other languages
English (en)
Inventor
Flores Claudio Hetz
Garcia Paula Huerta
Original Assignee
Univ Chile
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 Univ Chile filed Critical Univ Chile
Priority to CL2016003282A priority Critical patent/CL2016003282A1/es
Publication of CL2016003282A1 publication Critical patent/CL2016003282A1/es
Priority to JP2019555522A priority patent/JP2020501613A/ja
Priority to CN201780087076.4A priority patent/CN110741079A/zh
Priority to EP17883718.3A priority patent/EP3560519A4/en
Priority to PCT/CL2017/000040 priority patent/WO2018112672A1/es
Priority to US16/472,794 priority patent/US20200030391A1/en
Priority to JP2023061439A priority patent/JP2023098943A/ja

Links

Classifications

    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61P25/28—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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Abstract

<p>La presente invención incluye la expresión de dos moléculas en vectores virales AAV/IGF2-HA y AAV/IGF2, su método y su uso en el mejoramiento de enfermedades relacionadas con mal plegamiento de proteínas, tal como la enfermedad de Huntington, como se presenta en los modelos in vivo en la figura 11/19</p>
CL2016003282A 2016-12-21 2016-12-21 Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington CL2016003282A1 (es)

Priority Applications (7)

Application Number Priority Date Filing Date Title
CL2016003282A CL2016003282A1 (es) 2016-12-21 2016-12-21 Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington
JP2019555522A JP2020501613A (ja) 2016-12-21 2017-12-21 ウイルスａａｖ／ｉｇｆ２、遺伝子治療方法およびハンチントン病のようなタンパク質ミスフォールディング関連疾患におけるその使用
CN201780087076.4A CN110741079A (zh) 2016-12-21 2017-12-21 病毒aav/igf2、基因治疗方法及其在亨廷顿氏病等蛋白质折叠错误相关疾病中的应用
EP17883718.3A EP3560519A4 (en) 2016-12-21 2017-12-21 VIRUS AAV / IGF2, METHOD OF GENETIC TREATMENT AND USE OF IT IN PROTEIN-RELATED DISEASES SUCH AS HUNTINGTON'S MORBUS
PCT/CL2017/000040 WO2018112672A1 (es) 2016-12-21 2017-12-21 Virus aav/igf2, metodo de tratamiento genetico y su uso en enfermedades relacionadas con mal plegamiento de proteinas tal como la enfermedad de huntington
US16/472,794 US20200030391A1 (en) 2016-12-21 2017-12-21 Virus aav/igf2, genetic treatment method and use thereof in protein misfolding-related diseases, such as huntington's disease
JP2023061439A JP2023098943A (ja) 2016-12-21 2023-04-05 ウイルスａａｖ／ｉｇｆ２、遺伝子治療方法およびハンチントン病のようなタンパク質ミスフォールディング関連疾患におけるその使用

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CL2016003282A CL2016003282A1 (es) 2016-12-21 2016-12-21 Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington

Publications (1)

Publication Number Publication Date
CL2016003282A1 true CL2016003282A1 (es) 2017-08-18

Family

ID=60331262

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2016003282A CL2016003282A1 (es) 2016-12-21 2016-12-21 Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington

Country Status (6)

Country Link
US (1) US20200030391A1 (es)
EP (1) EP3560519A4 (es)
JP (2) JP2020501613A (es)
CN (1) CN110741079A (es)
CL (1) CL2016003282A1 (es)
WO (1) WO2018112672A1 (es)

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8198257B2 (en) * 2007-09-12 2012-06-12 Institut National De La Sante Et De La Recherche Medicale (Inserm) CYP46A1 gene for the treatment of alzheimer's disease
WO2011047204A1 (en) * 2009-10-14 2011-04-21 Mount Sinai School Of Medicine Method of treating memory disorders and enhancing memory using igf-ii compounds
ES2442242B1 (es) 2012-08-09 2014-11-25 Biotechnology Institute, I Mas D, S.L. Composición con factores de crecimiento destinada al tratamiento intranasal de una enfermedad neurodegenerativa u otra patología del sistema nervioso central, y su método de fabricación.
KR102262882B1 (ko) 2012-11-27 2021-06-10 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
WO2015060722A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
BR112017013674A2 (pt) * 2014-12-30 2018-02-06 Univ Iowa Res Found métodos e composições para tratamento de doenças cerebrais.
CL2014003590A1 (es) * 2014-12-30 2015-07-10 Univ Chile Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimizacion y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje.

Also Published As

Publication number Publication date
EP3560519A1 (en) 2019-10-30
CN110741079A (zh) 2020-01-31
WO2018112672A1 (es) 2018-06-28
JP2023098943A (ja) 2023-07-11
JP2020501613A (ja) 2020-01-23
US20200030391A1 (en) 2020-01-30
EP3560519A4 (en) 2020-09-02

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