BR112018071186A2 - rna, pluralidade de rnas, construto de interferência por rna dirigida por dna, composição, método para inibir a expressão de uma proteína, método para tratar distrofia muscular oculofaríngea e kit - Google Patents

rna, pluralidade de rnas, construto de interferência por rna dirigida por dna, composição, método para inibir a expressão de uma proteína, método para tratar distrofia muscular oculofaríngea e kit

Info

Publication number
BR112018071186A2
BR112018071186A2 BR112018071186-1A BR112018071186A BR112018071186A2 BR 112018071186 A2 BR112018071186 A2 BR 112018071186A2 BR 112018071186 A BR112018071186 A BR 112018071186A BR 112018071186 A2 BR112018071186 A2 BR 112018071186A2
Authority
BR
Brazil
Prior art keywords
muscular dystrophy
rna
rnas
dna
kit
Prior art date
Application number
BR112018071186-1A
Other languages
English (en)
Other versions
BR112018071186A8 (pt
Inventor
Suhy David
Graham Michael
Trollet Capucine
Malerba Alberto
J. Dickson George
Original Assignee
Benitec Biopharma Limited
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 Benitec Biopharma Limited filed Critical Benitec Biopharma Limited
Publication of BR112018071186A2 publication Critical patent/BR112018071186A2/pt
Publication of BR112018071186A8 publication Critical patent/BR112018071186A8/pt

Links

Classifications

    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/04—Drugs for disorders of the respiratory system for throat disorders
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/11—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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • 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
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pulmonology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Otolaryngology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

a presente revelação refere-se a reagentes de interferência por rna (rnai) que têm como alvo pabpn1 para tratamento de distrofia muscular oculofaríngea (opmd), composições que compreendem os mesmos e uso dos mesmos para tratar indivíduos que sofrem de opmd ou que são predispostos à mesma.
BR112018071186A 2016-04-14 2017-04-13 Rna, pluralidade de rnas, construto de interferência por rna dirigida por dna, composição, método para inibir a expressão de uma proteína, método para tratar distrofia muscular oculofaríngea e kit BR112018071186A8 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662322745P 2016-04-14 2016-04-14
US62/322,745 2016-04-14
PCT/AU2017/050330 WO2017177277A1 (en) 2016-04-14 2017-04-13 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

Publications (2)

Publication Number Publication Date
BR112018071186A2 true BR112018071186A2 (pt) 2019-02-12
BR112018071186A8 BR112018071186A8 (pt) 2023-03-28

Family

ID=60041243

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018071186A BR112018071186A8 (pt) 2016-04-14 2017-04-13 Rna, pluralidade de rnas, construto de interferência por rna dirigida por dna, composição, método para inibir a expressão de uma proteína, método para tratar distrofia muscular oculofaríngea e kit

Country Status (15)

Country Link
US (1) US11234994B2 (pt)
EP (1) EP3443091A4 (pt)
JP (1) JP7236195B2 (pt)
KR (1) KR102353847B1 (pt)
CN (1) CN109219659B (pt)
AU (1) AU2017250017B2 (pt)
BR (1) BR112018071186A8 (pt)
CA (1) CA3020754C (pt)
IL (1) IL262337B2 (pt)
MX (1) MX2018012545A (pt)
NZ (1) NZ747314A (pt)
RU (1) RU2755544C2 (pt)
SG (1) SG11201808981XA (pt)
WO (1) WO2017177277A1 (pt)
ZA (1) ZA201807589B (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2755544C2 (ru) 2016-04-14 2021-09-17 Бенитек Биофарма Лимитед Реагенты для лечения окулофарингеальной мышечной дистрофии (OPMD) и их применение
EP3555294A4 (en) 2016-12-14 2020-08-26 Benitec Biopharma Limited REAGENTS FOR TREATMENT OF OKULOPHARYNGEAL MUSCLE DYSTROPHY (OPMD) AND USES THEREOF
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

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
CA2088673A1 (en) 1990-08-03 1992-02-04 Alexander L. Weis Compounds and methods for inhibiting gene expression
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
EP0642589A4 (en) 1992-05-11 1997-05-21 Ribozyme Pharm Inc METHOD AND REAGENT TO INHIBIT VIRAL REPLICATION.
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
AU6412794A (en) 1993-03-31 1994-10-24 Sterling Winthrop Inc. Oligonucleotides with amide linkages replacing phosphodiester linkages
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US6001311A (en) 1997-02-05 1999-12-14 Protogene Laboratories, Inc. Apparatus for diverse chemical synthesis using two-dimensional array
US6054576A (en) 1997-10-02 2000-04-25 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA
CA2317549C (en) 1998-01-05 2006-04-11 University Of Washington Composition for enhancing transport through lipid-containing membranes, and uses thereof
US7273933B1 (en) 1998-02-26 2007-09-25 Isis Pharmaceuticals, Inc. Methods for synthesis of oligonucleotides
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US6995259B1 (en) 1998-10-23 2006-02-07 Sirna Therapeutics, Inc. Method for the chemical synthesis of oligonucleotides
US6465628B1 (en) 1999-02-04 2002-10-15 Isis Pharmaceuticals, Inc. Process for the synthesis of oligomeric compounds
WO2001053307A1 (en) 2000-01-21 2001-07-26 Geron Corporation 2'-arabino-fluorooligonucleotide n3'→p5'phosphoramidates: their synthesis and use
WO2002018405A2 (en) 2000-09-01 2002-03-07 Ribozyme Pharmaceuticals, Incorporated Methods for synthesizing nucleosides, nucleoside derivatives and non-nucleoside derivatives
ES2215494T5 (es) 2000-12-01 2017-12-28 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Moléculas de RNA pequeñas que median la interferencia de RNA
US20020130430A1 (en) 2000-12-29 2002-09-19 Castor Trevor Percival Methods for making polymer microspheres/nanospheres and encapsulating therapeutic proteins and other products
EP2017338A1 (en) 2001-05-24 2009-01-21 Genzyme Corporation Muscle-specific expression vectors
US7060498B1 (en) 2001-11-28 2006-06-13 Genta Salus Llc Polycationic water soluble copolymer and method for transferring polyanionic macromolecules across biological barriers
US20030215395A1 (en) 2002-05-14 2003-11-20 Lei Yu Controllably degradable polymeric biomolecule or drug carrier and method of synthesizing said carrier
US6989442B2 (en) 2002-07-12 2006-01-24 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
RU2005106999A (ru) 2002-08-29 2005-08-27 Дзе Борд Оф Трастиз Оф Дзе Лелэнд Стэнфорд Джуниор Юниверсити (Us) Кольцевые векторы нуклеиновой кислоты и способы их создания и использования
EP1560931B1 (en) * 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
US7358223B2 (en) 2004-10-04 2008-04-15 Nitto Denko Corporation Biodegradable cationic polymers
EP1838853A2 (en) * 2005-01-06 2007-10-03 Benitec, Inc. Rnai agents for maintenance of stem cells
WO2007086881A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
US8110184B2 (en) * 2005-09-30 2012-02-07 University Of Kentucky Research Foundation Ex vivo and in vivo methods and related compositions for generating hematopoietic stem cell populations
EP2014281A1 (en) * 2007-06-19 2009-01-14 Institut National De La Sante Et De La Recherche Medicale (Inserm) Use of inhibitors of sirtuins and/or ampk for the preparation of a medicament for the treatment of polyalanine diseases.
EP2326331A4 (en) 2008-08-18 2013-05-15 Merck Sharp & Dohme NOVEL LIPID NANOPARTICLES AND NEW COMPONENTS FOR NUCLEIC ACID ADMINISTRATION
EP2350043B9 (en) 2008-10-09 2014-08-20 TEKMIRA Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
WO2010080724A1 (en) 2009-01-12 2010-07-15 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
JP6032724B2 (ja) 2009-03-12 2016-11-30 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 脂質製剤組成物およびＥｇ５遺伝子とＶＥＧＦ遺伝子の発現を阻害する方法
WO2011022460A1 (en) 2009-08-20 2011-02-24 Merck Sharp & Dohme Corp. Novel cationic lipids with various head groups for oligonucleotide delivery
WO2011098449A1 (en) * 2010-02-10 2011-08-18 Novartis Ag Methods and compounds for muscle growth
UY33851A (es) 2010-12-30 2012-07-31 Dow Agrosciences Llc Moléculas de ácido nucleico que confieren resistencia a las plagas de coleópteros
KR101454425B1 (ko) 2012-10-11 2014-11-03 포항공과대학교 산학협력단 미리세틴을 유효성분으로 포함하는 운동수행능력 증강용 조성물
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
KR101683964B1 (ko) 2014-05-29 2016-12-09 연세대학교 산학협력단 Daxx의 발현을 억제하는 핵산 분자, TRAIL 유전자 및 Bcl-xL 의 발현을 억제하는 핵산 분자를 포함하는 재조합 발현 벡터
RU2755544C2 (ru) 2016-04-14 2021-09-17 Бенитек Биофарма Лимитед Реагенты для лечения окулофарингеальной мышечной дистрофии (OPMD) и их применение

Also Published As

Publication number Publication date
WO2017177277A1 (en) 2017-10-19
AU2017250017A1 (en) 2018-11-08
AU2017250017B2 (en) 2022-12-22
JP2019513395A (ja) 2019-05-30
CA3020754A1 (en) 2017-10-19
BR112018071186A8 (pt) 2023-03-28
RU2018138271A3 (pt) 2020-09-22
RU2018138271A (ru) 2020-05-14
CN109219659B (zh) 2022-09-09
US11234994B2 (en) 2022-02-01
CN109219659A (zh) 2019-01-15
NZ747314A (en) 2022-07-29
EP3443091A1 (en) 2019-02-20
EP3443091A4 (en) 2019-11-27
JP7236195B2 (ja) 2023-03-09
IL262337A (en) 2018-11-29
ZA201807589B (en) 2023-02-22
KR20180133500A (ko) 2018-12-14
US20200138849A1 (en) 2020-05-07
CA3020754C (en) 2023-07-25
KR102353847B1 (ko) 2022-01-21
MX2018012545A (es) 2019-06-10
IL262337B2 (en) 2023-06-01
RU2755544C2 (ru) 2021-09-17
SG11201808981XA (en) 2018-11-29

Similar Documents

Publication Publication Date Title
BR112018011450A2 (pt) métodos e composições para o tratamento de um transtorno associado à serpinc1
BR112019005548A2 (pt) tratamento da doença de huntington com aav
BR112019001887A2 (pt) composições e métodos para o tratamento de doença associada a cep290
AU2018253578A1 (en) RNA interference mediated inhibition of catenin (cadherin-associated protein), beta 1 (CTNNB1) gene expression using short interfering nucleic acid (siNA)
CO2020001354A2 (es) Métodos y composiciones para tratar un evento de sangrado en un sujeto que padece hemofilia un arni, por ejemplo, un ácido ribonucleico de hebra doble (arnhd), composiciones dirigidas al gen serpinc1 y métodos para usar dicho arni, por ejemplo, arnhd, composiciones para tratar un evento de sangrado en un sujeto que tiene una hemofilia (por ejemplo, con o sin inhibidores).
WO2016106401A3 (en) Rna agents for p21 gene modulation
BR112021019793A2 (pt) Composições e métodos para inibir expressão de gene no sistema nervoso central
MX2018014152A (es) Composiciones y métodos para tratar la enfermedad de huntington.
BR112018005862A2 (pt) anticorpos, anticorpos isolados, polinucleotídeo, vetor, célula hospedeira, método de produção do anticorpo, imunoconjugado, composição, usos dos anticorpos, métodos para tratar ou retardar a progressão de um câncer e de uma doença e para aumentar, potencializar ou estimular uma resposta ou função imune e kit
WO2017201102A8 (en) Method of improved sequencing by strand identification
BR112017016068A2 (pt) composições para modulação de expressão de c9orf72
BR112014028644A2 (pt) Composições e métodos para modulação da expressão de atp2a2
EA201790420A1 (ru) Средства, представляющие собой модифицированную двухнитевую рнк
EA201490993A1 (ru) МОДИФИЦИРОВАННЫЕ СРЕДСТВА РНКи
BR112016016916A8 (pt) uso de um anticorpo que se liga à calicreína plasmática ativa para tratamento de angiodema hereditário (hae)
BR112019010342A2 (pt) composições do irna de serpina1 e métodos de uso destas
BR112013027774A2 (pt) novos compostos como moduladores de proteína quinases
ATE548454T1 (de) Nukleinsäurewirkstoffe zur stilllegung von h19 zwecks behandlung rheumatoider arthritis
BR112018071186A2 (pt) rna, pluralidade de rnas, construto de interferência por rna dirigida por dna, composição, método para inibir a expressão de uma proteína, método para tratar distrofia muscular oculofaríngea e kit
BR112021024080A2 (pt) Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos
WO2010111503A3 (en) RNA INTERFERENCE MEDIATED INHIBITION OF THE HIGH AFFINITY IGE RECEPTOR ALPHA CHAIN (FCεRLα) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
BR112018010669A8 (pt) anticorpo anti-pcsk9 e utilização do mesmo
BR112021021686A2 (pt) Moléculas inibidoras de ácido nucleico de fita dupla com fitas senso curtas
IL292053A (en) Preparations, methods and kits for stabilizing a biological sample and RNA
BR112019012312A8 (pt) Reagentes para tratamento da distrofia muscular oculofaríngea (opmd) e uso dos mesmos

Legal Events

Date Code Title Description
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]
B25D Requested change of name of applicant approved

Owner name: BENITEC BIOPHARMA PTY LTD (AU)

B25G Requested change of headquarter approved

Owner name: BENITEC BIOPHARMA PTY LTD (AU)

B25A Requested transfer of rights approved

Owner name: BENITEC IP HOLDINGS INC. (US)

B06A Patent application procedure suspended [chapter 6.1 patent gazette]