BR112021007256A2 - métodos para o tratamento da distrofia muscular oculofaríngea (opmd) - Google Patents

métodos para o tratamento da distrofia muscular oculofaríngea (opmd) Download PDF

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Publication number
BR112021007256A2
BR112021007256A2 BR112021007256-0A BR112021007256A BR112021007256A2 BR 112021007256 A2 BR112021007256 A2 BR 112021007256A2 BR 112021007256 A BR112021007256 A BR 112021007256A BR 112021007256 A2 BR112021007256 A2 BR 112021007256A2
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seq
shmir
sequence shown
nucleic acid
effector
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BR112021007256-0A
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English (en)
Portuguese (pt)
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Vanessa STRINGS-UFOMBAH
David Suhy
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Benitec Biopharma Limited
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Publication of BR112021007256A2 publication Critical patent/BR112021007256A2/pt

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Wood Science & Technology (AREA)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Marine Sciences & Fisheries (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
BR112021007256-0A 2018-10-17 2019-10-17 métodos para o tratamento da distrofia muscular oculofaríngea (opmd) BR112021007256A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862747089P 2018-10-17 2018-10-17
US62/747,089 2018-10-17
PCT/AU2019/051134 WO2020077412A1 (en) 2018-10-17 2019-10-17 Methods for treating oculopharyngeal muscular dystrophy (opmd)

Publications (1)

Publication Number Publication Date
BR112021007256A2 true BR112021007256A2 (pt) 2021-08-10

Family

ID=70282838

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021007256-0A BR112021007256A2 (pt) 2018-10-17 2019-10-17 métodos para o tratamento da distrofia muscular oculofaríngea (opmd)

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Country Link
US (1) US20220106594A1 (https=)
EP (1) EP3866859A4 (https=)
JP (1) JP2022508835A (https=)
KR (1) KR20210081361A (https=)
CN (1) CN112867511A (https=)
AU (1) AU2019362925A1 (https=)
BR (1) BR112021007256A2 (https=)
CA (1) CA3114945A1 (https=)
IL (1) IL282323A (https=)
MX (1) MX2021004225A (https=)
SG (1) SG11202102185RA (https=)
WO (1) WO2020077412A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014117050A2 (en) * 2013-01-26 2014-07-31 Mirimus, Inc. Modified mirna as a scaffold for shrna
CA3020754C (en) * 2016-04-14 2023-07-25 Benitec Biopharma Limited Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
EP3555294A4 (en) * 2016-12-14 2020-08-26 Benitec Biopharma Limited REAGENTS FOR THE TREATMENT OF OCULOPHARYNGEAL MUSCLE DYSTROPHY (PDD) AND THEIR USE
US20220098614A1 (en) * 2019-02-28 2022-03-31 Benitec IP Holdings Inc. Compositions and Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD)

Also Published As

Publication number Publication date
EP3866859A4 (en) 2023-08-09
IL282323A (en) 2021-05-31
JP2022508835A (ja) 2022-01-19
SG11202102185RA (en) 2021-05-28
US20220106594A1 (en) 2022-04-07
CA3114945A1 (en) 2020-04-23
AU2019362925A1 (en) 2021-05-06
CN112867511A (zh) 2021-05-28
WO2020077412A1 (en) 2020-04-23
MX2021004225A (es) 2021-05-27
KR20210081361A (ko) 2021-07-01
EP3866859A1 (en) 2021-08-25

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B25C Requirement related to requested transfer of rights

Owner name: BENITEC BIOPHARMA LIMITED (US)

Free format text: A FIM DE ATENDER A ALTERACAO DE NOME E POSTERIOR TRANSFERENCIA DE TITULAR, REQUERIDAS ATRAVES DA PETICAO NO 870230009580 DE 02/02/2023, E NECESSARIO APRESENTAR A TRADUCAO JURAMENTADA DO DOCUMENTO DE ALTERACAO DE NOME. ALEM DISSO, E PRECISO APRESENTAR A GUIA DE CUMPRIMENTO DE EXIGENCIA.

B25D Requested change of name of applicant approved

Owner name: BENITEC BIOPHARMA PTY LTD (AU)

B25A Requested transfer of rights approved

Owner name: BENITEC IP HOLDINGS INC. (US)

B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

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B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: EM VIRTUDE DO ARQUIVAMENTO PUBLICADO NA RPI 2797 DE 13-08-2024 E CONSIDERANDO AUSENCIA DE MANIFESTACAO DENTRO DOS PRAZOS LEGAIS, INFORMO QUE CABE SER MANTIDO O ARQUIVAMENTO DO PEDIDO DE PATENTE, CONFORME O DISPOSTO NO ARTIGO 12, DA RESOLUCAO 113/2013.