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)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Psychiatry (AREA)
  • Pain & Pain Management (AREA)
  • Gastroenterology & Hepatology (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) CA2976075C (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 (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
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
JP6246121B2 (ja) 2012-07-13 2017-12-13 株式会社新日本科学 キラル核酸アジュバント
CN112007045B (zh) 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
DK2872485T3 (da) 2012-07-13 2021-03-08 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
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
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
KR102423317B1 (ko) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
WO2017015575A1 (en) 2015-07-22 2017-01-26 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US20180228830A1 (en) * 2015-10-23 2018-08-16 Rena Therapeutics Inc. Nucleic acid complex having at least one bulge structure
JP2019516680A (ja) 2016-05-04 2019-06-20 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物およびその方法
KR20220108216A (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
CA3064590A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
IL273394B2 (en) 2017-09-22 2025-04-01 Genzyme Corp RNAI variant
EP3684388A4 (en) * 2017-09-22 2021-09-22 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF HUNTINGTON'S DISEASE
KR20200119782A (ko) * 2017-12-06 2020-10-20 오비드 테라퓨틱스 인크. 발작 장애 치료 시 mir101 또는 mir128의 용도
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
JP2021530548A (ja) 2018-07-24 2021-11-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. 遺伝子治療製剤を生産するための系および方法
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
WO2020150636A1 (en) 2019-01-18 2020-07-23 University Of Massachusetts Dynamic pharmacokinetic-modifying anchors
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
CA3149835A1 (en) 2019-08-09 2021-02-18 University Of Massachusetts Chemically modified oligonucleotides targeting snps
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
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
EP4157289A4 (en) 2020-05-26 2024-06-26 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
CA3238867A1 (en) 2021-11-29 2023-06-01 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
CN118660913A (zh) 2022-02-21 2024-09-17 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
WO2024187106A2 (en) * 2023-03-09 2024-09-12 Leal Therapeutics, Inc. Compositions and methods for modulating grin2a
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
KR20250129583A (ko) * 2024-02-21 2025-08-29 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
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
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
EP2369002A1 (en) 1999-08-09 2011-09-28 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 intrastrand base pairs
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
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
SG10202108118RA (en) 2001-11-13 2021-08-30 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
EP1556402B1 (en) 2002-09-25 2011-06-22 University of Massachusetts In vivo gene silencing by chemically modified and stable sirna
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
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
WO2008150897A2 (en) * 2007-05-31 2008-12-11 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
US8318924B2 (en) * 2008-02-14 2012-11-27 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
EP3567106A1 (en) 2010-04-23 2019-11-13 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
SG10202007803QA (en) 2011-02-17 2020-09-29 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
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
DK3628334T5 (da) 2014-03-21 2024-09-02 Genzyme Corp Genterapi til behandling af retinitis pigmentosa
EP3137497B2 (en) 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
EP3237618B1 (en) 2014-12-24 2019-05-22 uniQure IP B.V. Rnai induced huntingtin gene suppression
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
IL273394B2 (en) 2017-09-22 2025-04-01 Genzyme Corp RNAI variant

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

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