MX2019007110A - Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos. - Google Patents

Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos.

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Publication number
MX2019007110A
MX2019007110A MX2019007110A MX2019007110A MX2019007110A MX 2019007110 A MX2019007110 A MX 2019007110A MX 2019007110 A MX2019007110 A MX 2019007110A MX 2019007110 A MX2019007110 A MX 2019007110A MX 2019007110 A MX2019007110 A MX 2019007110A
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MX
Mexico
Prior art keywords
opmd
reagents
treatment
muscular dystrophy
oculopharyngeal muscular
Prior art date
Application number
MX2019007110A
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English (en)
Inventor
Suhy David
STRINGS-UFOMBAH Vanessa
Original Assignee
Benitec Biopharma Ltd
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Publication date
Application filed by Benitec Biopharma Ltd filed Critical Benitec Biopharma Ltd
Publication of MX2019007110A publication Critical patent/MX2019007110A/es

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K48/005—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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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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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
    • 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
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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
    • 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
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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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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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
    • 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
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    • C12N2310/50—Physical structure
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    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)

Abstract

La presente descripción se refiere a reactivos de interferencia de ARN (iARN), tales como microARN de horquilla pequeña (shmiR) y ARN de horquilla pequeña (ARNhp), para el tratamiento de la distrofia muscular oculofaríngea (OPMD), composiciones que los comprenden y uso de estos para tratar individuos que sufren OPMD o que son propensos a sufrirla. La presente descripción también se refiere al uso de los reactivos de iARN combinados con reactivos de reemplazo de PABPN1, tales como construcciones que codifican la proteína PABPN1 funcional, para el tratamiento de la OPMD, composiciones que los comprenden y uso de estos para tratar individuos que sufren OPMD o que son propensos a sufrirla.
MX2019007110A 2016-12-14 2017-12-14 Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos. MX2019007110A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662434312P 2016-12-14 2016-12-14
PCT/AU2017/051385 WO2018107228A1 (en) 2016-12-14 2017-12-14 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

Publications (1)

Publication Number Publication Date
MX2019007110A true MX2019007110A (es) 2019-12-05

Family

ID=62557655

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019007110A MX2019007110A (es) 2016-12-14 2017-12-14 Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos.
MX2024003643A MX2024003643A (es) 2016-12-14 2019-06-14 Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y usos de estos.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2024003643A MX2024003643A (es) 2016-12-14 2019-06-14 Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y usos de estos.

Country Status (13)

Country Link
US (2) US11142765B2 (es)
EP (1) EP3555294A4 (es)
JP (2) JP7233149B2 (es)
KR (2) KR102495222B1 (es)
CN (2) CN110382698B (es)
AU (2) AU2017377661B2 (es)
BR (1) BR112019012312A8 (es)
CA (1) CA3047154A1 (es)
IL (2) IL304674B2 (es)
MX (2) MX2019007110A (es)
RU (1) RU2763319C2 (es)
WO (1) WO2018107228A1 (es)
ZA (1) ZA202106827B (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019007110A (es) * 2016-12-14 2019-12-05 Benitec Biopharma Ltd Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos.
CN112867511A (zh) * 2018-10-17 2021-05-28 贝尼泰克生物制药有限公司 用于治疗眼咽肌营养不良(opmd)的方法
KR20210138030A (ko) 2019-02-28 2021-11-18 베니텍 아이피 홀딩스 아이엔씨. 안구인두 근이영양증 (opmd) 치료용 조성물 및 방법

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003006477A1 (en) * 2001-07-12 2003-01-23 University Of Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
EP1783645A1 (en) * 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
AR065121A1 (es) * 2007-01-31 2009-05-20 Basf Plant Science Gmbh Plantas con rasgos aumentados relacionados con el rendimiento y/o resistencia incrementada al estres abiotico y un metodo para desarrollar las mismas
PT2164967E (pt) * 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
WO2010105372A1 (en) 2009-03-20 2010-09-23 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis c virus expression
EP2814514B1 (en) * 2012-02-16 2017-09-13 Atyr Pharma, Inc. Histidyl-trna synthetases for treating autoimmune and inflammatory diseases
WO2014077693A1 (en) * 2012-11-16 2014-05-22 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Means and methods for reducing an effect of aging in a mammalian cell
MX2015008697A (es) * 2013-01-08 2016-08-04 Benitec Biopharma Ltd Tratamiento contra la degeneración macular relacionada con la edad.
WO2017177277A1 (en) 2016-04-14 2017-10-19 Benitec Biopharma Limited Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
MX2019007110A (es) * 2016-12-14 2019-12-05 Benitec Biopharma Ltd Reactivos para el tratamiento de la distrofia muscular oculofaringea (opmd) y uso de estos.

Also Published As

Publication number Publication date
EP3555294A4 (en) 2020-08-26
IL304674B1 (en) 2024-07-01
RU2763319C2 (ru) 2021-12-28
IL267353A (en) 2019-08-29
US20190330629A1 (en) 2019-10-31
IL267353B1 (en) 2023-08-01
US11932852B2 (en) 2024-03-19
EP3555294A1 (en) 2019-10-23
KR20190104151A (ko) 2019-09-06
MX2024003643A (es) 2024-04-15
KR102667867B1 (ko) 2024-05-29
IL304674A (en) 2023-09-01
JP7431359B2 (ja) 2024-02-14
KR102495222B1 (ko) 2023-02-09
AU2017377661B2 (en) 2023-07-27
KR20230020586A (ko) 2023-02-10
BR112019012312A2 (pt) 2020-02-11
CN110382698B (zh) 2024-05-10
AU2017377661A1 (en) 2019-07-18
JP7233149B2 (ja) 2023-03-06
CA3047154A1 (en) 2018-06-21
US11142765B2 (en) 2021-10-12
IL304674B2 (en) 2024-11-01
IL267353B2 (en) 2023-12-01
CN110382698A (zh) 2019-10-25
RU2019121950A3 (es) 2021-01-18
CN118421624A (zh) 2024-08-02
JP2023062092A (ja) 2023-05-02
JP2020503014A (ja) 2020-01-30
AU2023222827A1 (en) 2023-09-14
RU2019121950A (ru) 2021-01-18
WO2018107228A1 (en) 2018-06-21
BR112019012312A8 (pt) 2023-03-28
US20220056446A1 (en) 2022-02-24
ZA202106827B (en) 2022-07-27

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