PT2470656E - Composição para inibir expressão de gene e suas utilizações - Google Patents

Composição para inibir expressão de gene e suas utilizações Download PDF

Info

Publication number
PT2470656E
PT2470656E PT107495707T PT10749570T PT2470656E PT 2470656 E PT2470656 E PT 2470656E PT 107495707 T PT107495707 T PT 107495707T PT 10749570 T PT10749570 T PT 10749570T PT 2470656 E PT2470656 E PT 2470656E
Authority
PT
Portugal
Prior art keywords
composition
gene expression
inhibiting gene
inhibiting
expression
Prior art date
Application number
PT107495707T
Other languages
English (en)
Inventor
Sudhir Agrawal
Ekambar Kandimalla
Millikarjuna Putta
Tao Lan
Lakshmi Bhagat
Daqing Wang
Dong Yu
Original Assignee
Idera Pharmaceuticals Inc
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 Idera Pharmaceuticals Inc filed Critical Idera Pharmaceuticals Inc
Publication of PT2470656E publication Critical patent/PT2470656E/pt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • A61P11/06Antiasthmatics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/10Antimycotics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00Antiparasitic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/08Antiallergic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/15Nucleic acids forming more than 2 strands, e.g. TFOs
    • C12N2310/152Nucleic acids forming more than 2 strands, e.g. TFOs on a single-stranded target, e.g. fold-back TFOs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Communicable Diseases (AREA)
  • Pulmonology (AREA)
  • Zoology (AREA)
  • Oncology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Rheumatology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Pain & Pain Management (AREA)
  • Transplantation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
PT107495707T 2009-08-27 2010-08-26 Composição para inibir expressão de gene e suas utilizações PT2470656E (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US27525209P 2009-08-27 2009-08-27
US24055309P 2009-09-08 2009-09-08

Publications (1)

Publication Number Publication Date
PT2470656E true PT2470656E (pt) 2015-07-16

Family

ID=43446697

Family Applications (1)

Application Number Title Priority Date Filing Date
PT107495707T PT2470656E (pt) 2009-08-27 2010-08-26 Composição para inibir expressão de gene e suas utilizações

Country Status (15)

Country Link
US (3) US8431544B1 (pt)
EP (3) EP2960333B1 (pt)
JP (1) JP5805088B2 (pt)
CN (2) CN102712926B (pt)
CA (1) CA2772352A1 (pt)
DK (1) DK2470656T3 (pt)
ES (2) ES2538347T3 (pt)
HK (1) HK1210223A1 (pt)
HR (1) HRP20150566T1 (pt)
HU (1) HUE026020T2 (pt)
PL (1) PL2470656T3 (pt)
PT (1) PT2470656E (pt)
SI (1) SI2470656T1 (pt)
SM (1) SMT201500140B (pt)
WO (1) WO2011031520A1 (pt)

Families Citing this family (143)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
WO2011117353A1 (en) * 2010-03-24 2011-09-29 Mirrx Therapeutics A/S Bivalent antisense oligonucleotides
EP2609198B8 (en) 2010-08-24 2018-03-28 Sirna Therapeutics, Inc. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
US8877722B2 (en) * 2011-03-25 2014-11-04 Idera Pharmaceuticals, Inc. Compositions for inhibiting gene expression and uses thereof
DK2756080T3 (da) 2011-09-14 2019-05-20 Translate Bio Ma Inc Multimeriske oligonukleotidforbindelser
KR102095699B1 (ko) 2011-11-18 2020-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) 관련 질병을 치료하기 위한 RNAi 제제, 조성 및 그의 사용방법
CN104105790A (zh) 2011-11-18 2014-10-15 阿尔尼拉姆医药品有限公司 修饰的RNAi试剂
WO2013169382A1 (en) * 2012-05-07 2013-11-14 DePuy Synthes Products, LLC Methods and devices for treating intervertebral disc disease
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
ITNA20120046A1 (it) 2012-08-02 2014-02-03 No Self S R L Uso di acidi nucleici di sistemi biologici parassiti, patogeni e infestanti per l'inibizione e/o controllo degli stessi sistemi
CA2879683C (en) 2012-08-03 2023-02-14 Alnylam Pharmaceuticals, Inc. Modified rnai agents
US20150247141A1 (en) * 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
HUE035887T2 (en) 2012-12-05 2018-05-28 Alnylam Pharmaceuticals Inc PCSK9 iRNA preparations and methods for their use
EP2970970B1 (en) * 2013-03-14 2018-12-12 Andes Biotechnologies Global, Inc. Antisense oligonucleotides for treatment of cancer stem cells
SI2970974T1 (sl) 2013-03-14 2017-12-29 Alnylam Pharmaceuticals, Inc. Komplementarna komponenta C5 sestavkov IRNA in postopki njene uporabe
IL293657A (en) 2013-05-22 2022-08-01 Alnylam Pharmaceuticals Inc Preparations of tmprss6 irna and methods of using them
CN113018307A (zh) 2013-05-22 2021-06-25 阿尔尼拉姆医药品有限公司 SERPINA1 iRNA组合物及其使用方法
WO2015042564A1 (en) 2013-09-23 2015-03-26 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing transthyretin (ttr) associated diseases
WO2015089368A2 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
US10119136B2 (en) 2014-01-09 2018-11-06 Alnylam Pharmaceuticals, Inc. RNAi agents modified at the 4′-C position
JP6594902B2 (ja) 2014-02-11 2019-10-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド ケトヘキソキナーゼ(KHK)iRNA組成物及びその使用方法
WO2015175510A1 (en) 2014-05-12 2015-11-19 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating a serpinc1-associated disorder
MX2016015126A (es) 2014-05-22 2017-02-23 Alnylam Pharmaceuticals Inc Composiciones de angiotensinogeno (agt) arni y metodos de uso de las mismas.
EP3164113B1 (en) 2014-06-04 2019-03-27 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
SG10201913791QA (en) 2014-08-20 2020-03-30 Alnylam Pharmaceuticals Inc Modified double-stranded rna agents
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
EP3221451A1 (en) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
JP2017537619A (ja) 2014-11-21 2017-12-21 ノースウェスタン ユニバーシティ 球状核酸ナノ粒子複合体の配列特異的細胞内取込
EP3234141A4 (en) 2014-12-18 2018-06-20 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
US10704043B2 (en) 2015-01-14 2020-07-07 Exicure, Inc. Nucleic acid nanostructures with core motifs
EP3256587A2 (en) 2015-02-13 2017-12-20 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2016138278A2 (en) 2015-02-27 2016-09-01 Idera Pharmaceuticals, Inc. Compositions for inhibiting checkpoint gene expression and uses thereof
LT3277814T (lt) 2015-04-03 2020-10-26 University Of Massachusetts Oligonukleotidų junginiai, skirti nukreipimui į hantingtino mrnr
HUE057431T2 (hu) 2015-04-03 2022-05-28 Univ Massachusetts Oligonukleotid vegyületek preeklampszia és más angiogén rendellenességek kezelésére
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
KR20180039621A (ko) 2015-06-15 2018-04-18 엠펙 엘에이, 엘엘씨 정의된 다중 접합체 올리고뉴클레오티드
DE102015008536A1 (de) * 2015-07-02 2017-01-05 Rheinische Friedrich-Wilhelms-Universität Bonn Diskontinuierliche Oligonukleotid-Liganden
WO2017007886A2 (en) * 2015-07-08 2017-01-12 Idera Pharmaceuticals, Inc. Compositions for inhibiting dux4 gene expression and uses thereof
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
EP3334499A4 (en) 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
JP7028764B2 (ja) 2015-09-02 2022-03-02 アルナイラム ファーマシューティカルズ, インコーポレイテッド プログラム細胞死1リガンド1(PD-L1)iRNA組成物およびその使用方法
SK500652015A3 (sk) 2015-10-15 2017-05-03 Ústav Polymérov Sav Spôsob úpravy funkčného stavu ľubovoľnej mRNA umožňujúci jej selektívne a špecifické rozpoznanie
WO2017079352A2 (en) * 2015-11-04 2017-05-11 Idera Pharmaceuticals, Inc. Compositions for inhibiting nlrp3 gene expression and uses thereof
AU2017210726B2 (en) 2016-01-31 2023-08-03 University Of Massachusetts Branched oligonucleotides
JP7186094B2 (ja) 2016-05-06 2022-12-08 イグジキュア オペレーティング カンパニー インターロイキン17受容体mRNAの特異的ノックダウンのためのアンチセンスオリゴヌクレオチド(ASO)を提示するリポソーム系球状核酸(SNA)構築物
WO2017205384A1 (en) * 2016-05-23 2017-11-30 Idera Pharmaceuticals, Inc. Compositions and methods for inhibiting target rna expression
WO2017214518A1 (en) 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
US11364304B2 (en) 2016-08-25 2022-06-21 Northwestern University Crosslinked micellar spherical nucleic acids
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
WO2018112320A1 (en) 2016-12-16 2018-06-21 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
US11696954B2 (en) 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
WO2018209270A1 (en) 2017-05-11 2018-11-15 Northwestern University Adoptive cell therapy using spherical nucleic acids (snas)
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
US20200385719A1 (en) 2017-11-16 2020-12-10 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
EP3714054A1 (en) 2017-11-20 2020-09-30 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
BR112020012088A2 (pt) 2017-12-18 2020-11-17 Alnylam Pharmaceuticals, Inc. composições de irna do grupo de elevada mobilidade box-1 (hmgb1) e métodos de uso dos mesmos
KR20210018267A (ko) 2018-05-07 2021-02-17 알닐람 파마슈티칼스 인코포레이티드 간외 전달
TW202016304A (zh) 2018-05-14 2020-05-01 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
KR20210018192A (ko) * 2018-05-31 2021-02-17 다이이찌 산쿄 가부시키가이샤 항인간 tlr7 항체
SG11202101288TA (en) 2018-08-10 2021-03-30 Univ Massachusetts Modified oligonucleotides targeting snps
JP2022500003A (ja) 2018-09-18 2022-01-04 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法
JP7470107B2 (ja) 2018-09-28 2024-04-17 アルナイラム ファーマシューティカルズ, インコーポレイテッド トランスサイレチン(TTR)iRNA組成物及びTTR関連眼疾患を治療又は予防するためのその使用方法
CA3133241A1 (en) * 2019-03-15 2020-09-24 University Of Massachusetts Oligonucleotides for tissue specific apoe modulation
US11629347B2 (en) 2019-05-06 2023-04-18 University Of Massachusetts Anti-C9ORF72 oligonucleotides and related methods
WO2020236600A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
EP4007811A2 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase b2 (cpb2) irna compositions and methods of use thereof
WO2021022109A1 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
EP4045652A1 (en) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
WO2021081026A1 (en) 2019-10-22 2021-04-29 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
EP4051796A1 (en) 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
AU2020376840A1 (en) 2019-11-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Huntingtin (HTT) iRNA agent compositions and methods of use thereof
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
AU2020378414A1 (en) 2019-11-06 2022-05-26 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
KR20220115946A (ko) 2019-11-13 2022-08-19 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐 (agt) 관련 장애를 치료하기 위한 방법 및 조성물
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
MX2022006433A (es) 2019-12-13 2022-06-23 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) del marco de lectura abierto 72 del cromosoma 9 humano (c9orf72) y metodos de uso de los mismos.
WO2021126734A1 (en) 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
IL295445A (en) 2020-02-10 2022-10-01 Alnylam Pharmaceuticals Inc Preparations and methods for silencing vegf-a expression
IL295496A (en) 2020-02-18 2022-10-01 Alnylam Pharmaceuticals Inc Apolipoprotein c3 (apoc3) irna preparations and methods of using them
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
CN115485383A (zh) 2020-03-06 2022-12-16 阿尔尼拉姆医药品有限公司 己酮糖激酶(KHK)iRNA组合物及其使用方法
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
BR112022020145A2 (pt) 2020-04-06 2023-01-03 Alnylam Pharmaceuticals Inc Composições e métodos para silenciamento da expressão de myoc
EP4133079A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
AU2021263554A1 (en) 2020-04-27 2022-12-08 Alnylam Pharmaceuticals, Inc. Apolipoprotein E (APOE) iRNA agent compositions and methods of use thereof
BR112022021136A2 (pt) 2020-04-30 2022-11-29 Alnylam Pharmaceuticals Inc Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas
EP4153746A1 (en) 2020-05-21 2023-03-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
EP4168546A1 (en) 2020-06-18 2023-04-26 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
WO2022011214A1 (en) 2020-07-10 2022-01-13 Alnylam Pharmaceuticals, Inc. Circular sirnas
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
EP4225917A1 (en) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
AU2021365822A1 (en) 2020-10-21 2023-06-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc Coagulation factor iRNA compositions (F5) and methods of using them
TW202237150A (zh) 2020-12-01 2022-10-01 美商艾拉倫製藥股份有限公司 用於抑制hao1(羥基酸氧化酶1(乙醇酸氧化酶))基因表現的方法及組成物
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
JP2024501857A (ja) 2020-12-31 2024-01-16 アルナイラム ファーマシューティカルズ, インコーポレイテッド 環状ジスルフィド修飾リン酸ベースのオリゴヌクレオチドプロドラッグ
WO2022147223A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
WO2022150260A1 (en) 2021-01-05 2022-07-14 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
IL304880A (en) 2021-02-12 2023-10-01 Alnylam Pharmaceuticals Inc Superoxide dismutase 1 (SOD1) IRNA compositions and methods of using them to treat or prevent superoxide dismutase 1- (SOD1-) associated neurodegenerative diseases
EP4298220A1 (en) 2021-02-25 2024-01-03 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods of use thereof
IL305153A (en) 2021-02-26 2023-10-01 Alnylam Pharmaceuticals Inc Ketohexokinase (KHK) iRNA compositions and methods of using them
WO2022187435A1 (en) 2021-03-04 2022-09-09 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
IL307239A (en) 2021-03-29 2023-11-01 Alnylam Pharmaceuticals Inc Preparations containing Huntingtin IRNA factor (HTT) and methods of using them
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
JP2024517686A (ja) 2021-04-26 2024-04-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド 膜貫通プロテアーゼ、セリン6(TMPRSS6)iRNA組成物およびその使用方法
JP2024519293A (ja) 2021-04-29 2024-05-10 アルナイラム ファーマシューティカルズ, インコーポレイテッド シグナル伝達兼転写活性化因子6(STAT6)iRNA組成物およびその使用方法
JP2024522068A (ja) 2021-05-18 2024-06-11 アルナイラム ファーマシューティカルズ, インコーポレイテッド ナトリウム-グルコース共輸送体2(sglt2)irna組成物およびその使用方法
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
BR112023025224A2 (pt) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos
WO2022260939A2 (en) 2021-06-08 2022-12-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
CA3174095A1 (en) 2021-06-23 2022-12-29 Vignesh Narayan HARIHARAN Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
KR20240026203A (ko) 2021-06-30 2024-02-27 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐(agt)-관련 장애를 치료하는 방법 및 조성물
EP4367237A2 (en) 2021-07-09 2024-05-15 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
WO2023003805A1 (en) 2021-07-19 2023-01-26 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder
IL309897A (en) 2021-07-21 2024-03-01 Alnylam Pharmaceuticals Inc Target gene IRNA compositions associated with a metabolic disorder and methods of using them
AU2022316139A1 (en) 2021-07-23 2024-01-18 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
CN117795074A (zh) 2021-08-03 2024-03-29 阿尔尼拉姆医药品有限公司 转甲状腺素蛋白(TTR)iRNA组合物和其使用方法
IL310295A (en) 2021-08-04 2024-03-01 Alnylam Pharmaceuticals Inc iRNA compositions and methods for silencing angiotensinogen (AGT)
IL310407A (en) 2021-08-13 2024-03-01 Alnylam Pharmaceuticals Inc Factor XII (F12) IRNA compositions and methods of using them
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
IL311864A (en) 2021-10-15 2024-06-01 Alnylam Pharmaceuticals Inc IRNA compositions for extrahepatic administration and methods of using them
CA3234636A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
TW202334418A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 杭丁頓(HTT)iRNA劑組成物及其使用方法
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024073732A1 (en) 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5721218A (en) 1989-10-23 1998-02-24 Gilead Sciences, Inc. Oligonucleotides with inverted polarity
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US6204027B1 (en) 1992-02-26 2001-03-20 University Of Massachusetts Worcester Ribozymes having 2′-O substituted nucleotides in the flanking sequences
TW244371B (pt) 1992-07-23 1995-04-01 Tri Clover Inc
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
US5652355A (en) 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
JPH10504184A (ja) 1994-06-01 1998-04-28 ハイブライドン インコーポレイテッド 病原抑制剤としての分枝オリゴヌクレオチド
US5652356A (en) 1995-08-17 1997-07-29 Hybridon, Inc. Inverted chimeric and hybrid oligonucleotides
US5912332A (en) 1996-07-26 1999-06-15 Hybridon, Inc. Affinity-based purification of oligonucleotides using soluble multimeric oligonucleotides
US20010049436A1 (en) 1998-04-01 2001-12-06 Wen-Qiang Zhou Mixed-backbone oligonucleotides containing pops blocks to obtain reduced phosphorothioate content
EP1086216B1 (en) * 1999-03-31 2003-05-28 Hybridon, Inc. Pseudo-cyclic oligonucleobases
WO2003035836A2 (en) * 2001-10-24 2003-05-01 Hybridon Inc. Modulation of immunostimulatory properties of oligonucleotide-based compounds by optimal presentation of 5' ends
CA2480775C (en) * 2002-04-04 2016-05-31 Coley Pharmaceutical Gmbh Immunostimulatory g,u-containing oligoribonucleotides
WO2003100099A1 (en) * 2002-05-24 2003-12-04 Cygene, Inc. Parallel stranded duplexes of deoxyribonucleic acid and methods of use
CA2512484A1 (en) * 2003-01-16 2004-05-08 Hybridon, Inc. Modulation of immunostimulatory properties of oligonucleotide-based compounds by utilizing modified immunostimulatory dinucleotides
JP2007531699A (ja) * 2003-07-15 2007-11-08 イデラ ファーマシューティカルズ インコーポレイテッド 免疫刺激性オリゴヌクレオチドおよび/またはイムノマー化合物をサイトカインおよび/または化学療法剤または放射線療法と組み合わせて用いる、免疫系の相乗的刺激
AU2004304770B2 (en) * 2003-12-08 2010-05-06 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory properties by small oligonucleotide-based compounds
WO2005083084A1 (en) * 2004-02-25 2005-09-09 Syddansk Universitet Intercalating triplex forming oligonucleotide derivatives and process for the preparation thereof
ES2542989T3 (es) * 2005-10-12 2015-08-13 Idera Pharmaceuticals, Inc. Compuestos oligonucleótidos inmuno reguladores (IRO) para modular la respuesta inmune basada en receptor semejante a Toll
CN101460178B (zh) * 2006-04-07 2016-12-07 艾德拉药物股份有限公司 用于tlr7和tlr8的稳定化的免疫调节性rna(simra)化合物
WO2009023819A2 (en) * 2007-08-15 2009-02-19 Idera Pharmaceuticals, Inc. Toll like receptor modulators
CA2752694A1 (en) * 2009-02-10 2010-08-19 Idera Pharmaceuticals, Inc. Synthetic rna-based agonists of tlr7

Also Published As

Publication number Publication date
PL2470656T3 (pl) 2015-08-31
CN102712926A (zh) 2012-10-03
HRP20150566T1 (hr) 2015-07-17
EP3272870A1 (en) 2018-01-24
CN104673795A (zh) 2015-06-03
HK1210223A1 (en) 2016-04-15
EP2470656B1 (en) 2015-05-06
CA2772352A1 (en) 2011-03-17
ES2538347T3 (es) 2015-06-19
CN102712926B (zh) 2015-04-29
EP2470656A1 (en) 2012-07-04
SMT201500140B (it) 2015-09-07
JP5805088B2 (ja) 2015-11-04
EP2960333B1 (en) 2017-10-04
SI2470656T1 (sl) 2015-07-31
ES2646097T3 (es) 2017-12-12
HUE026020T2 (en) 2016-05-30
US20110082186A1 (en) 2011-04-07
DK2470656T3 (da) 2015-06-22
WO2011031520A1 (en) 2011-03-17
US8431544B1 (en) 2013-04-30
EP2960333A1 (en) 2015-12-30
US20120016004A1 (en) 2012-01-19
JP2013507905A (ja) 2013-03-07

Similar Documents

Publication Publication Date Title
HK1210223A1 (en) Compositions for inhibiting gene expression and uses thereof
EP2462230A4 (en) METHOD AND COMPOSITIONS FOR TARGETED GENE MODIFICATION
GB0908129D0 (en) Composition
ZA200908481B (en) Compositions and methods for altering gene expression
GB0918450D0 (en) Composition
EP2445067A4 (en) COMPOSITION FOR FILLING THE DISCHARGE GAP AND ELECTROSTATIC DISCHARGE PROTECTION
GB0911428D0 (en) Composition
EP2235033A4 (en) METHOD AND COMPOSITIONS FOR INCREASING GENE EXPRESSION
GB0909362D0 (en) Composition
GB0915796D0 (en) Polymerase compositions and uses
GB0903299D0 (en) Composition and methods
GB0908642D0 (en) Composition
HK1169306A1 (en) Composition having lipolysis-promoting effect
GB0901966D0 (en) Composition
EP2490536A4 (en) COMPOSITION AND METHOD
EP2394634A4 (en) HESPERIDO-CONTAINING COMPOSITION
GB0906779D0 (en) Composition
EP2451982A4 (en) COMPOSITIONS AND METHODS FOR MAMMALS AND USE THEREOF
GB0904941D0 (en) Composition
GB0911294D0 (en) Composition
EP2444061A4 (en) COMPOSITION CONTAINING APIGENIN
GB0906548D0 (en) Composition
GB0904942D0 (en) Composition
GB0903688D0 (en) Composition
GB0902959D0 (en) Composition