EA201791805A1 - ВАРИАНТ СРЕДСТВА ДЛЯ RNAi - Google Patents

ВАРИАНТ СРЕДСТВА ДЛЯ RNAi

Info

Publication number
EA201791805A1
EA201791805A1 EA201791805A EA201791805A EA201791805A1 EA 201791805 A1 EA201791805 A1 EA 201791805A1 EA 201791805 A EA201791805 A EA 201791805A EA 201791805 A EA201791805 A EA 201791805A EA 201791805 A1 EA201791805 A1 EA 201791805A1
Authority
EA
Eurasian Patent Office
Prior art keywords
rnai
sequence
guide
huntington
disease
Prior art date
Application number
EA201791805A
Other languages
English (en)
Inventor
Лайза М. Стенек
Адам Палермо
Бренда Ричардс
Серджио Пабло Сарди
Кэтрин О'риордан
Антониус Сонг
Original Assignee
Джензим Корпорейшн
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 Джензим Корпорейшн filed Critical Джензим Корпорейшн
Publication of EA201791805A1 publication Critical patent/EA201791805A1/ru

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

Abstract

В данном документе предусматриваются молекулы для RNAi, включающие в себя первую цепь, содержащую направляющую последовательность, и вторую цепь, содержащую последовательность, не являющуюся направляющей, которая содержит выпячивание напротив затравочной области направляющей последовательности; например напротив последовательности расщепления. В некоторых аспектах настоящее изобретение обеспечивает RNAi для лечения болезни Хантингтона. В данном документе предусматриваются кассеты экспрессии, векторы (например, векторы на основе rAAV, рекомбинантного аденовируса, рекомбинантного лентивируса и рекомбинантного HSV), клетки, вирусные частицы и фармацевтические композиции, содержащие RNAi. Кроме того, в данном документе дополнительно предусматриваются способы и наборы, связанные с применением RNAi, например, для лечения болезни Хантингтона.
EA201791805A 2015-02-10 2016-02-09 ВАРИАНТ СРЕДСТВА ДЛЯ RNAi EA201791805A1 (ru)

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
EA201791805A1 true EA201791805A1 (ru) 2018-05-31

Family

ID=56614905

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201791805A EA201791805A1 (ru) 2015-02-10 2016-02-09 ВАРИАНТ СРЕДСТВА ДЛЯ RNAi

Country Status (26)

Country Link
US (4) US10450563B2 (ru)
EP (1) EP3256588A2 (ru)
JP (2) JP2018506304A (ru)
KR (1) KR102670852B1 (ru)
CN (1) CN107438671B (ru)
AR (1) AR103646A1 (ru)
AU (2) AU2016219396B2 (ru)
BR (1) BR112017017028A2 (ru)
CA (1) CA2976075A1 (ru)
CL (1) CL2017002027A1 (ru)
CO (1) CO2017009083A2 (ru)
CR (1) CR20170406A (ru)
EA (1) EA201791805A1 (ru)
EC (1) ECSP17059343A (ru)
HK (1) HK1247641A1 (ru)
IL (1) IL253893B (ru)
MA (1) MA40819B1 (ru)
MX (2) MX2017010369A (ru)
MY (2) MY181458A (ru)
PE (1) PE20171382A1 (ru)
PH (1) PH12017501432A1 (ru)
SG (3) SG10201907396XA (ru)
TN (1) TN2017000354A1 (ru)
TW (1) TWI781079B (ru)
UY (1) UY36554A (ru)
WO (1) WO2016130589A2 (ru)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2009323766B2 (en) 2008-12-02 2016-10-06 Wave Life Sciences Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
BR112014001244A2 (pt) 2011-07-19 2017-02-21 Wave Life Sciences Pte Ltd métodos para a síntese de ácidos nucléicos funcionalizados
KR102450907B1 (ko) 2012-07-13 2022-10-04 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
EP3812370A1 (en) 2012-07-13 2021-04-28 Wave Life Sciences Ltd. Asymmetric auxiliary group
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
EP4137572A1 (en) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Chiral design
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
CA2976075A1 (en) 2015-02-10 2016-08-18 Genzyme Corporation Variant rnai
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
JPWO2017068791A1 (ja) * 2015-10-23 2018-08-09 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
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
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
JP7248658B2 (ja) 2017-09-22 2023-03-29 ジェンザイム・コーポレーション バリアントＲＮＡｉ
MX2020005952A (es) * 2017-12-06 2022-01-03 Ovid Therapeutics Inc Uso de mir101 o mir128 en el tratamiento de trastornos convulsivos.
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
MX2021000810A (es) 2018-07-24 2021-04-28 Voyager Therapeutics Inc Sistemas y metodos para producir formulaciones de terapia genetica.
WO2020028816A1 (en) * 2018-08-03 2020-02-06 Genzyme Corporation Variant rnai against alpha-synuclein
BR112021013109A2 (pt) * 2019-01-09 2021-10-13 Universidade De Coimbra Rna de fita dupla e seus usos.
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
US12024706B2 (en) 2019-08-09 2024-07-02 University Of Massachusetts Modified oligonucleotides targeting SNPs
CA3176204A1 (en) * 2020-04-20 2021-10-28 Anastasia Khvorova Oligonucleotides for msh3 modulation
WO2023093905A1 (zh) 2021-11-29 2023-06-01 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
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
SG10201912509RA (en) 2001-11-13 2020-02-27 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
WO2004029212A2 (en) * 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
CA2526893C (en) * 2003-05-14 2010-10-26 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
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US20090036395A1 (en) 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
CA2691617C (en) * 2007-05-31 2020-07-21 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
CA2711585A1 (en) 2008-02-04 2009-08-13 Galapagos Nv 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
US8796238B2 (en) * 2009-09-17 2014-08-05 Sigma-Aldrich Co. Llc Short RNA mimetics
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system 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
JP6224459B2 (ja) 2011-02-17 2017-11-01 ザ・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 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
EP2820159B1 (en) 2012-02-29 2019-10-23 Sangamo Therapeutics, Inc. Methods and compositions for treating huntington's disease
US9550989B2 (en) * 2013-10-03 2017-01-24 Washington University Rational design of microRNA-siRNA chimeras for multi-functional target suppression
RS64611B1 (sr) 2014-03-21 2023-10-31 Genzyme Corp Genska terapija za retinitis pigmentozu
KR20160147974A (ko) 2014-05-02 2016-12-23 젠자임 코포레이션 망막 및 cns 유전자 요법을 위한 aav 벡터
CN108064292B (zh) 2014-12-24 2021-05-04 尤尼克尔生物制药股份有限公司 RNAi诱导的亨廷顿蛋白基因抑制
CA2976075A1 (en) 2015-02-10 2016-08-18 Genzyme Corporation Variant rnai
JP7248658B2 (ja) 2017-09-22 2023-03-29 ジェンザイム・コーポレーション バリアントＲＮＡｉ

Also Published As

Publication number Publication date
PH12017501432A1 (en) 2018-01-15
JP2018506304A (ja) 2018-03-08
US10760079B2 (en) 2020-09-01
US10450563B2 (en) 2019-10-22
AR103646A1 (es) 2017-05-24
CO2017009083A2 (es) 2018-01-31
TN2017000354A1 (en) 2019-01-16
SG10201912942YA (en) 2020-02-27
CL2017002027A1 (es) 2018-04-20
JP2021118740A (ja) 2021-08-12
KR102670852B1 (ko) 2024-05-29
IL253893B (en) 2021-08-31
SG10201907396XA (en) 2019-09-27
CR20170406A (es) 2017-11-14
KR20170110149A (ko) 2017-10-10
UY36554A (es) 2016-09-30
AU2022204274A1 (en) 2022-07-07
MX2017010369A (es) 2017-12-14
AU2016219396B2 (en) 2022-03-17
US20210047641A1 (en) 2021-02-18
US11781137B2 (en) 2023-10-10
MX2022013005A (es) 2022-11-09
WO2016130589A2 (en) 2016-08-18
SG11201706444TA (en) 2017-09-28
MY181458A (en) 2020-12-22
EP3256588A2 (en) 2017-12-20
IL253893A0 (en) 2017-10-31
WO2016130589A3 (en) 2016-09-15
MA40819A1 (fr) 2018-07-31
TWI781079B (zh) 2022-10-21
US20180023082A1 (en) 2018-01-25
US20240182896A1 (en) 2024-06-06
MY194175A (en) 2022-11-17
HK1247641A1 (zh) 2018-09-28
CA2976075A1 (en) 2016-08-18
CN107438671B (zh) 2021-05-25
ECSP17059343A (es) 2019-03-29
CN107438671A (zh) 2017-12-05
TW201704470A (zh) 2017-02-01
BR112017017028A2 (pt) 2018-04-10
US20200109401A1 (en) 2020-04-09
PE20171382A1 (es) 2017-09-15
MA40819B1 (fr) 2020-04-30
AU2016219396A1 (en) 2017-09-28

Similar Documents

Publication Publication Date Title
EA201791805A1 (ru) ВАРИАНТ СРЕДСТВА ДЛЯ RNAi
PH12020550117A1 (en) Variant rnai
CL2018001611A1 (es) Adenovirus del grupo b que codifica un anticuerpo o fragmento anti-tcr-complejo
CL2020000990A1 (es) Proteínas b de unión a antígenos de maduración celular
EA201990346A1 (ru) Антитела к gprc5d, биспецифические антигенсвязывающие молекулы, которые связывают gprc5d и cd3, и их применение
EA201791971A1 (ru) Онколитический аденовирус, кодирующий белок b7
EA201690969A1 (ru) Способы и композиции для лечения связанных со старением состояний
BR112017014551A2 (pt) ?anticorpo, construção de ácido nucleico, vetor de expressão, célula hospedeira, composição, métodos para tratamento de uma doença, para produção de um anticorpo biespecífico e para detecção da possível ocorrência de reticulação entre células com expressão de cd3 e cd20 em uma amostra derivada de um paciente, uso de um anticorpo biespecífico, e, kit?
EA201890725A1 (ru) Агонисты фарнезоидного х-рецептора и их применение
BR112017003104A2 (pt) tratamento de câncer usando um receptor antigênico quimérico anti-cd123
BR112019001737A2 (pt) expressão de pten-long com vírus oncolíticos
BR112017005517A2 (pt) anticorpos antiage para o tratamento de inflama-ção e de transtornos autoimunes
EA202090124A1 (ru) Аденовирус, вооруженный биспецифическим рекрутером t-клеток (bite)
EA202190073A1 (ru) Композиции и способы лечения видов гемоглобинопатии и талассемии
EA201790192A1 (ru) СПОСОБ СНИЖЕНИЯ Fc-РЕЦЕПТОРНОГО СВЯЗЫВАНИЯ МОЛЕКУЛ СЫВОРОТОЧНОГО IgG У СУБЪЕКТА
EA201891399A1 (ru) Бипиразолильные производные, пригодные для лечения аутоиммунных заболеваний
EA201892225A1 (ru) АНТИТЕЛА К ФАКТОРУ Bb КОМПЛЕМЕНТА И ИХ ПРИМЕНЕНИЕ
EA202190708A1 (ru) Модуляторы экспрессии pnpla3
EA202091712A1 (ru) МОДИФИЦИРОВАННЫЙ КАПСИДНЫЙ БЕЛОК rAAV ДЛЯ ГЕННОЙ ТЕРАПИИ
EA202191067A1 (ru) Композиции и способы для лечения дефицита альфа-1-антитрипсина
MX369414B (es) Peptido.
EA202191058A1 (ru) Антитела против муцина-16 и способы их применения
EA202092265A1 (ru) Антитела против cd137 для комбинации с антителами против pd-l1
EA202190045A1 (ru) Составы/композиции, содержащие ибрутиниб
DOP2017000185A (es) Arni variante