JP2018506304A - バリアントＲＮＡｉ - Google Patents

バリアントＲＮＡｉ Download PDF

Info

Publication number
JP2018506304A
JP2018506304A JP2017560478A JP2017560478A JP2018506304A JP 2018506304 A JP2018506304 A JP 2018506304A JP 2017560478 A JP2017560478 A JP 2017560478A JP 2017560478 A JP2017560478 A JP 2017560478A JP 2018506304 A JP2018506304 A JP 2018506304A
Authority
JP
Japan
Prior art keywords
vector
rnai
strand
promoter
nucleic acid
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2017560478A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018506304A5 (enExample
Inventor
エム・リサ・スタネック
アダム・パレーモ
ブレンダ・リチャーズ
パブロ・セルジオ・サルディ
キャサリン・オリオーダン
アントニウス・ソング
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
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 JP2018506304A publication Critical patent/JP2018506304A/ja
Publication of JP2018506304A5 publication Critical patent/JP2018506304A5/ja
Priority to JP2021078464A priority Critical patent/JP7526710B2/ja
Pending legal-status Critical Current

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 nucleic acids [NA]
    • 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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychiatry (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
JP2017560478A 2015-02-10 2016-02-09 バリアントＲＮＡｉ Pending JP2018506304A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2021078464A JP7526710B2 (ja) 2015-02-10 2021-05-06 バリアントＲＮＡｉ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
US62/114,578 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021078464A Division JP7526710B2 (ja) 2015-02-10 2021-05-06 バリアントＲＮＡｉ

Publications (2)

Publication Number Publication Date
JP2018506304A true JP2018506304A (ja) 2018-03-08
JP2018506304A5 JP2018506304A5 (enExample) 2019-03-22

Family

ID=56614905

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2017560478A Pending JP2018506304A (ja) 2015-02-10 2016-02-09 バリアントＲＮＡｉ
JP2021078464A Active JP7526710B2 (ja) 2015-02-10 2021-05-06 バリアントＲＮＡｉ

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2021078464A Active JP7526710B2 (ja) 2015-02-10 2021-05-06 バリアントＲＮＡｉ

Country Status (25)

Country Link
US (4) US10450563B2 (enExample)
EP (1) EP3256588A2 (enExample)
JP (2) JP2018506304A (enExample)
KR (1) KR102670852B1 (enExample)
CN (1) CN107438671B (enExample)
AR (1) AR103646A1 (enExample)
AU (3) AU2016219396B2 (enExample)
CA (2) CA3255657A1 (enExample)
CL (1) CL2017002027A1 (enExample)
CO (1) CO2017009083A2 (enExample)
CR (1) CR20170406A (enExample)
EA (1) EA201791805A1 (enExample)
EC (1) ECSP17059343A (enExample)
HK (1) HK1247641A1 (enExample)
IL (1) IL253893B (enExample)
MA (1) MA40819B1 (enExample)
MX (2) MX2017010369A (enExample)
MY (2) MY181458A (enExample)
PE (1) PE20171382A1 (enExample)
PH (1) PH12017501432A1 (enExample)
SG (3) SG11201706444TA (enExample)
TN (1) TN2017000354A1 (enExample)
TW (1) TWI781079B (enExample)
UY (1) UY36554A (enExample)
WO (1) WO2016130589A2 (enExample)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021519065A (ja) * 2018-03-23 2021-08-10 ユニバーシティ オブ マサチューセッツ 骨障害を処置するための遺伝子治療法
JP2021534814A (ja) * 2018-08-03 2021-12-16 ジェンザイム・コーポレーション アルファ−シヌクレインに対するバリアントＲＮＡｉ
JP2022516779A (ja) * 2019-01-09 2022-03-02 ウニベルシダージ デ コインブラ 二本鎖ｒｎａ及びその使用
JP2024123125A (ja) * 2018-10-12 2024-09-10 ジェンザイム・コーポレーション 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成
JP2025102783A (ja) * 2018-11-21 2025-07-08 武田薬品工業株式会社 組換えウイルスベクター及びそれの産生のための核酸

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
WO2015108047A1 (ja) 2014-01-15 2015-07-23 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
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
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
SG11201706444TA (en) 2015-02-10 2017-09-28 Genzyme Corp VARIANT RNAi
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
CN108025089A (zh) 2015-07-22 2018-05-11 波涛生命科学有限公司 寡核苷酸组合物及其方法
EP3334499A4 (en) 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
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
CN109477108A (zh) 2016-05-04 2019-03-15 波涛生命科学有限公司 寡核苷酸组合物和其方法
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics, Inc. composições e métodos de tratamento da doença de huntington
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CN110799647A (zh) 2017-06-23 2020-02-14 马萨诸塞大学 双尾自递送sirna及相关的方法
MY205041A (en) * 2017-09-22 2024-09-29 Genzyme Corp Variant rnai
US20200377887A1 (en) * 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
US10870855B2 (en) * 2017-12-06 2020-12-22 Ovid Therapeutics Inc. Use of MIR101 or MIR128 in the treatment of seizure disorders
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.
EP3840759A4 (en) 2018-08-23 2022-06-01 University Of Massachusetts O-METHYL RICH FULLY STABILIZED OLIGONUCLEOTIDES
AU2020207935A1 (en) 2019-01-18 2021-08-26 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
US20210355491A1 (en) * 2020-04-20 2021-11-18 University Of Massachusetts Oligonucleotides for msh3 modulation
US12534724B2 (en) 2020-05-26 2026-01-27 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
EP4442830A4 (en) 2021-11-29 2025-04-30 Shanghai Regenelead Therapies Co., Ltd. AADC/GDNF polynucleotide and use thereof in the treatment of Parkinson's disease
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
EP4677091A2 (en) * 2023-03-09 2026-01-14 Leal Therapeutics, Inc. Compositions and methods for modulating grin2a
WO2025178406A1 (ko) * 2024-02-21 2025-08-28 서울대학교산학협력단 Drosha 절단을 촉진시키는 구조적 모티프를 포함하는 rna 간섭 유도 핵산 분자의 변형 전구체

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004101787A1 (ja) * 2003-05-14 2004-11-25 Japan Science And Technology Agency ハンチンチン遺伝子の発現抑制
JP2010500025A (ja) * 2006-08-09 2010-01-07 メドトロニック，インコーポレイテッド 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列
WO2011034811A1 (en) * 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics
JP2011517339A (ja) * 2008-02-04 2011-06-02 ガラパゴス・ナムローゼ・フェンノートシャップ 神経変性疾患の治療に有用な分子標的及び化合物並びにこれらの同定方法

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6989264B2 (en) 1997-09-05 2006-01-24 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
ATE403715T1 (de) 1999-08-09 2008-08-15 Targeted Genetics Corp Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
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
CN103555677B (zh) 2001-11-13 2018-01-30 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
AU2003282877B9 (en) * 2002-09-25 2011-05-12 University Of Massachusetts In Vivo gene silencing by chemically modified and stable siRNA
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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2008134646A2 (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
AU2009214837A1 (en) * 2008-02-14 2009-08-20 Michael Paul Marie Gantier Immunostimulatory siRNA molecules
FR2929292A1 (fr) 2008-03-28 2009-10-02 Exonhit Therapeutics S A Sa Procede et methodes de diagnostic de la maladie d'alzheimer
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
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
ES2724800T3 (es) 2011-02-17 2019-09-16 Univ Pennsylvania Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9
NZ629427A (en) 2012-02-29 2016-04-29 Sangamo Biosciences 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
EP3628334B1 (en) 2014-03-21 2023-06-28 Genzyme Corporation Gene therapy for retinitis pigmentosa
WO2015168666A2 (en) 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
SMT201900347T1 (it) 2014-12-24 2019-07-11 Uniqure Ip Bv Soppressione del gene huntingtina indotta da rnai
SG11201706444TA (en) 2015-02-10 2017-09-28 Genzyme Corp VARIANT RNAi
MY205041A (en) 2017-09-22 2024-09-29 Genzyme Corp Variant rnai

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004101787A1 (ja) * 2003-05-14 2004-11-25 Japan Science And Technology Agency ハンチンチン遺伝子の発現抑制
JP2010500025A (ja) * 2006-08-09 2010-01-07 メドトロニック，インコーポレイテッド 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列
JP2011517339A (ja) * 2008-02-04 2011-06-02 ガラパゴス・ナムローゼ・フェンノートシャップ 神経変性疾患の治療に有用な分子標的及び化合物並びにこれらの同定方法
WO2011034811A1 (en) * 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
CELL, vol. 151, JPN6020013319, 2012, pages 900 - 911, ISSN: 0004249514 *
MOLECULAR CELL, vol. 10, JPN6020013320, 2002, pages 549 - 561, ISSN: 0004249515 *
MOLECULAR THERAPY, vol. 18, no. 4, JPN6020013323, 2010, pages 785 - 795, ISSN: 0004249516 *
THE JOURNAL OF GENE MEDICINE, vol. 6, JPN6020013324, 2004, pages 715 - 723, ISSN: 0004249517 *

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021519065A (ja) * 2018-03-23 2021-08-10 ユニバーシティ オブ マサチューセッツ 骨障害を処置するための遺伝子治療法
US12194108B2 (en) 2018-03-23 2025-01-14 University Of Massachusetts Gene therapeutics for treating bone disorders
JP2021534814A (ja) * 2018-08-03 2021-12-16 ジェンザイム・コーポレーション アルファ−シヌクレインに対するバリアントＲＮＡｉ
US12365895B2 (en) 2018-08-03 2025-07-22 Genzyme Corporation Variant RNAi against alpha-synuclein
JP2024123125A (ja) * 2018-10-12 2024-09-10 ジェンザイム・コーポレーション 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成
JP2025102783A (ja) * 2018-11-21 2025-07-08 武田薬品工業株式会社 組換えウイルスベクター及びそれの産生のための核酸
JP2022516779A (ja) * 2019-01-09 2022-03-02 ウニベルシダージ デ コインブラ 二本鎖ｒｎａ及びその使用
JP2025016506A (ja) * 2019-01-09 2025-02-04 ウニベルシダージ デ コインブラ 二本鎖ｒｎａ及びその使用

Also Published As

Publication number Publication date
PH12017501432A1 (en) 2018-01-15
JP7526710B2 (ja) 2024-08-01
WO2016130589A2 (en) 2016-08-18
US20200109401A1 (en) 2020-04-09
NZ772770A (en) 2024-10-25
MY181458A (en) 2020-12-22
BR112017017028A2 (pt) 2018-04-10
TWI781079B (zh) 2022-10-21
US10450563B2 (en) 2019-10-22
US20240182896A1 (en) 2024-06-06
TW201704470A (zh) 2017-02-01
US11781137B2 (en) 2023-10-10
PE20171382A1 (es) 2017-09-15
CN107438671A (zh) 2017-12-05
US10760079B2 (en) 2020-09-01
WO2016130589A3 (en) 2016-09-15
CA2976075A1 (en) 2016-08-18
US20210047641A1 (en) 2021-02-18
AU2016219396A1 (en) 2017-09-28
US20180023082A1 (en) 2018-01-25
MY194175A (en) 2022-11-17
SG10201912942YA (en) 2020-02-27
CN107438671B (zh) 2021-05-25
CA3255657A1 (en) 2025-12-01
AR103646A1 (es) 2017-05-24
AU2025205501A1 (en) 2025-08-07
MX2022013005A (es) 2022-11-09
IL253893B (en) 2021-08-31
TN2017000354A1 (en) 2019-01-16
JP2021118740A (ja) 2021-08-12
CO2017009083A2 (es) 2018-01-31
MX2017010369A (es) 2017-12-14
EA201791805A1 (ru) 2018-05-31
SG11201706444TA (en) 2017-09-28
MA40819B1 (fr) 2020-04-30
CR20170406A (es) 2017-11-14
CA2976075C (en) 2025-08-12
KR20170110149A (ko) 2017-10-10
CL2017002027A1 (es) 2018-04-20
HK1247641A1 (zh) 2018-09-28
KR102670852B1 (ko) 2024-05-29
UY36554A (es) 2016-09-30
AU2022204274A1 (en) 2022-07-07
IL253893A0 (en) 2017-10-31
ECSP17059343A (es) 2019-03-29
AU2016219396B2 (en) 2022-03-17
MA40819A1 (fr) 2018-07-31
SG10201907396XA (en) 2019-09-27
AU2022204274B2 (en) 2025-04-17
NZ735289A (en) 2024-07-26
EP3256588A2 (en) 2017-12-20

Similar Documents

Publication Publication Date Title
JP7526710B2 (ja) バリアントＲＮＡｉ
US12331296B2 (en) Variant RNAi
JP2018516978A (ja) 深部イントロン突然変異の遺伝子編集
JP2021534814A (ja) アルファ−シヌクレインに対するバリアントＲＮＡｉ
RU2789647C2 (ru) ВАРИАНТ СРЕДСТВА ДЛЯ RNAi
BR112017017028B1 (pt) Rnai variante

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190205

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190205

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200407

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200707

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20210105

C60 Trial request (containing other claim documents, opposition documents)

Free format text: JAPANESE INTERMEDIATE CODE: C60

Effective date: 20210506

C116 Written invitation by the chief administrative judge to file amendments

Free format text: JAPANESE INTERMEDIATE CODE: C116

Effective date: 20210518

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20210518

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20220215

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20220412

C13 Notice of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: C13

Effective date: 20220913

C28A Non-patent document cited

Free format text: JAPANESE INTERMEDIATE CODE: C2838

Effective date: 20220913

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221213

C22 Notice of designation (change) of administrative judge

Free format text: JAPANESE INTERMEDIATE CODE: C22

Effective date: 20230411

C23 Notice of termination of proceedings

Free format text: JAPANESE INTERMEDIATE CODE: C23

Effective date: 20230418