BR112017017028A2 - rnai variante - Google Patents

rnai variante

Info

Publication number
BR112017017028A2
BR112017017028A2 BR112017017028A BR112017017028A BR112017017028A2 BR 112017017028 A2 BR112017017028 A2 BR 112017017028A2 BR 112017017028 A BR112017017028 A BR 112017017028A BR 112017017028 A BR112017017028 A BR 112017017028A BR 112017017028 A2 BR112017017028 A2 BR 112017017028A2
Authority
BR
Brazil
Prior art keywords
variant
guide sequence
recombinant
disease
filament
Prior art date
Application number
BR112017017028A
Other languages
English (en)
Inventor
Palermo Adam
Song Antonius
Richards Brenda
O'riordan Catherine
M Stanek Lisa
Pablo Sardi Sergio
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of BR112017017028A2 publication Critical patent/BR112017017028A2/pt

Links

Classifications

    • 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
    • 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
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Vascular Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Psychiatry (AREA)
  • Urology & Nephrology (AREA)
  • Pain & Pain Management (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)

Abstract

a presente invenção refere-se às moléculas de rnai incluindo um primeiro filamento contendo uma sequência guia e um segundo filamento que compreende uma sequência não guia, onde a sequência não guia contém uma protuberância oposta à região semente das sequências guia; por exemplo, oposta à sequência de clivagem. em alguns aspectos, a invenção fornece rnai para tratamento de doença de huntington. fornecidos aqui também são os cassetes de expressão, vetores (por exemplo, raav, vetores adenovirais recombinantes, lentivirais recombinantes, e de hsv recombinante), células, partículas virais, e composições farmacêuticas contendo o rnai. ainda também fornecidos aqui são métodos e kits relacionados ao uso do rnai, por exemplo, para tratar doença de huntington.
BR112017017028A 2015-02-10 2016-02-09 rnai variante BR112017017028A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

Publications (1)

Publication Number Publication Date
BR112017017028A2 true BR112017017028A2 (pt) 2018-04-10

Family

ID=56614905

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112017017028A BR112017017028A2 (pt) 2015-02-10 2016-02-09 rnai variante

Country Status (26)

Country Link
US (4) US10450563B2 (pt)
EP (1) EP3256588A2 (pt)
JP (2) JP2018506304A (pt)
KR (1) KR102670852B1 (pt)
CN (1) CN107438671B (pt)
AR (1) AR103646A1 (pt)
AU (2) AU2016219396B2 (pt)
BR (1) BR112017017028A2 (pt)
CA (1) CA2976075A1 (pt)
CL (1) CL2017002027A1 (pt)
CO (1) CO2017009083A2 (pt)
CR (1) CR20170406A (pt)
EA (1) EA201791805A1 (pt)
EC (1) ECSP17059343A (pt)
HK (1) HK1247641A1 (pt)
IL (1) IL253893B (pt)
MA (1) MA40819B1 (pt)
MX (2) MX2017010369A (pt)
MY (2) MY181458A (pt)
PE (1) PE20171382A1 (pt)
PH (1) PH12017501432A1 (pt)
SG (3) SG11201706444TA (pt)
TN (1) TN2017000354A1 (pt)
TW (1) TWI781079B (pt)
UY (1) UY36554A (pt)
WO (1) WO2016130589A2 (pt)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2744987C (en) 2008-12-02 2018-01-16 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
BR112012000828A8 (pt) 2009-07-06 2017-10-10 Ontorii Inc Novas pró-drogas de ácido nucleico e métodos de uso das mesmas
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
KR20220139425A (ko) 2012-07-13 2022-10-14 웨이브 라이프 사이언시스 리미티드 키랄 제어
KR101835401B1 (ko) 2012-07-13 2018-03-08 신 니뽄 바이오메디칼 라보라토리즈, 엘티디. 키랄 핵산 어쥬번트
AU2013288048A1 (en) 2012-07-13 2015-01-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
PT3094728T (pt) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Desenho quiral
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017068791A1 (ja) * 2015-10-23 2017-04-27 レナセラピューティクス株式会社 少なくとも１つのバルジ構造を有する核酸複合体
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc modulatory polynucleotides
CN109831916B (zh) 2016-05-18 2023-07-21 沃雅戈治疗公司 治疗亨廷顿氏舞蹈病的组合物和方法
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
WO2019060726A1 (en) * 2017-09-22 2019-03-28 Genzyme Corporation VARIANT OF ARNI
CA3084985A1 (en) * 2017-12-06 2019-06-13 Ovid Therapeutics Inc. Use of mir101 or mir128 in the treatment of seizure disorders
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020028816A1 (en) * 2018-08-03 2020-02-06 Genzyme Corporation Variant rnai against alpha-synuclein
EP3908658A1 (en) * 2019-01-09 2021-11-17 Universidade de Coimbra Double stranded rna and uses thereof
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
JP2023522701A (ja) * 2020-04-20 2023-05-31 ユニバーシティー オブ マサチューセッツ Ｍｓｈ３調節のためのオリゴヌクレオチド

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2001500015A (ja) 1996-09-06 2001-01-09 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
CA2410828C (en) 2000-06-01 2012-01-24 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
ATE520707T1 (de) 2001-11-13 2011-09-15 Univ Pennsylvania Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
CA2881743A1 (en) 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
US7589189B2 (en) * 2003-05-14 2009-09-15 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US7902352B2 (en) * 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
CA2688514A1 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
PT2164967E (pt) 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
US20110077283A1 (en) 2008-02-04 2011-03-31 David Frederik Fischer Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
EP2250183A4 (en) * 2008-02-14 2012-07-25 Michael Paul Marie Gantier SMALL IMMUNOSTIMULATORY RNAI MOLECULES
FR2929292A1 (fr) 2008-03-28 2009-10-02 Exonhit Therapeutics S A Sa Procede et methodes de diagnostic de la maladie d'alzheimer
WO2011034811A1 (en) * 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics
CA3066596A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
PL2675902T3 (pl) 2011-02-17 2019-08-30 The Trustees Of The University Of Pennsylvania Kompozycje i sposoby do zmieniania specyficzności względem tkanki i poprawy transferu genu z udziałem aav9
CN108285491B (zh) 2012-02-29 2021-08-10 桑格摩生物科学股份有限公司 治疗亨廷顿氏病的方法和组合物
US9550989B2 (en) * 2013-10-03 2017-01-24 Washington University Rational design of microRNA-siRNA chimeras for multi-functional target suppression
FI3628334T3 (fi) 2014-03-21 2023-09-15 Genzyme Corp Geenihoito verkkokalvon pigmenttirappeuma
MX2016014220A (es) 2014-05-02 2017-02-06 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc.
CA2971920C (en) 2014-12-24 2024-05-07 Uniqure Ip B.V. Rnai induced huntingtin gene suppression
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
WO2019060726A1 (en) 2017-09-22 2019-03-28 Genzyme Corporation VARIANT OF ARNI

Also Published As

Publication number Publication date
MY181458A (en) 2020-12-22
US11781137B2 (en) 2023-10-10
US20200109401A1 (en) 2020-04-09
PH12017501432A1 (en) 2018-01-15
AU2022204274A1 (en) 2022-07-07
SG10201907396XA (en) 2019-09-27
SG10201912942YA (en) 2020-02-27
US20210047641A1 (en) 2021-02-18
US20180023082A1 (en) 2018-01-25
AU2016219396B2 (en) 2022-03-17
IL253893B (en) 2021-08-31
WO2016130589A2 (en) 2016-08-18
TWI781079B (zh) 2022-10-21
JP2021118740A (ja) 2021-08-12
AU2016219396A1 (en) 2017-09-28
MY194175A (en) 2022-11-17
EA201791805A1 (ru) 2018-05-31
KR20170110149A (ko) 2017-10-10
JP2018506304A (ja) 2018-03-08
US10450563B2 (en) 2019-10-22
AR103646A1 (es) 2017-05-24
IL253893A0 (en) 2017-10-31
CN107438671B (zh) 2021-05-25
UY36554A (es) 2016-09-30
MA40819B1 (fr) 2020-04-30
HK1247641A1 (zh) 2018-09-28
CA2976075A1 (en) 2016-08-18
KR102670852B1 (ko) 2024-05-29
MX2022013005A (es) 2022-11-09
TW201704470A (zh) 2017-02-01
US10760079B2 (en) 2020-09-01
US20240182896A1 (en) 2024-06-06
ECSP17059343A (es) 2019-03-29
PE20171382A1 (es) 2017-09-15
MA40819A1 (fr) 2018-07-31
CL2017002027A1 (es) 2018-04-20
SG11201706444TA (en) 2017-09-28
CR20170406A (es) 2017-11-14
TN2017000354A1 (en) 2019-01-16
CO2017009083A2 (es) 2018-01-31
MX2017010369A (es) 2017-12-14
WO2016130589A3 (en) 2016-09-15
EP3256588A2 (en) 2017-12-20
CN107438671A (zh) 2017-12-05

Similar Documents

Publication Publication Date Title
BR112017017028A2 (pt) rnai variante
BR112017005892A2 (pt) variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
BR112019001887A2 (pt) composições e métodos para o tratamento de doença associada a cep290
PH12020550117A1 (en) Variant rnai
BR112019010699A2 (pt) receptores de células t e imunoterapia empregando os mesmos
BR112018072849A2 (pt) capsídeos variantes de vírus adeno associado e métodos de uso
BR112018011975A2 (pt) composições úteis no tratamento de atrofia muscular espinhal
BR112018003665A2 (pt) métodos e composições para vetores virais de evasão de anticorpo
BR112018011711A2 (pt) peptídeos de direcionamento para direcionar virus adeno-associados (aav)
BR112015020235A2 (pt) fragmento biologicamente ativo de proteína csf-1 ou um seu homólogo, ácido nucléico, proteína de fusão, ácido nucléico isolado, vetor, célula hospedeira, método de realização da proteína de fusão do primeiro aspeto da invenção, composição, utilização de uma proteína de fusão, método de tratamento para um indivíduo sofrendo de cancro do fígado e que irá ser submetido a cirurgia, método de tratamento para um indivíduo que irá ser submetido a cirurgia de transplante de fígado, e, kit
BR112018007046A2 (pt) anticorpo isolado ou fragmento de ligação a antígeno, composição farmacêutica, molécula de polinucleotídeo isolado, vetor, célula, e, métodos para inibir o crescimento de um tumor ou de uma célula tumoral e para tratar uma doença ou distúrbio.
BR112017004349A2 (pt) terapia com gene de globina para o tratamento de hemoglobinopatias
BR112018070676A2 (pt) construtos de ligação a antígeno multiespecífico que têm como alvo agentes imunoterapêuticos
BR112018071180A2 (pt) métodos de reforço potência biológica de baculovirus system-produzido vírus adeno-associado recombinante
BR112014018331A8 (pt) Biomarcadores e terapias combinadas usando vírus oncolítico e imunomodulação
BRPI0821034A8 (pt) Vírus influenza b recombinante, seu uso, metodo de preparação, composição de vacina o contendo e seu gene m, bem como ácido nucléico isolado, molécula do vírus influenza m e seu uso e método para aumentar a taxa de crescimento de vírus influenza b deficiente de replicação
BR112018075692A2 (pt) genes cln1 otimizados e cassetes de expressão e seu uso
BR112018003238A2 (pt) seleção de pacientes para terapia de combinação
BR112019008810A2 (pt) micrornas modificadas com 5-halouracila e seu uso no tratamento de câncer
BR112022006476A2 (pt) Oligonucleotídeos com análogos de nucleosídeos
BR112015015488A2 (pt) composição para cuidado de fibras de queratina e uso da mesma para limpeza e condicionamento das fibras de queratina
BR112016010342A2 (pt) sequência de rna isolada ou sintética, sequência de dna, vetor que permite a expressão de uma sequência de rna, célula que compreende uma sequência de rna, composição que compreende uma sequência de rna, utilização de uma sequência de rna, camundongo transgênico, método que permite avaliar a eficácia cosmética de um composto de teste, e, método que permite avaliar a eficácia terapêutica de um composto de teste
BR112019010034A2 (pt) fator viii alvejado de hemácia e método de uso do mesmo
BR112021019032A2 (pt) Vetores de fator h e usos dos mesmos
BR112017008152A2 (pt) promotor recombinante com expressão específica de fibra aumentada

Legal Events

Date Code Title Description
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]