AR097738A1 - Métodos para tratar o prevenir enfermedades asociadas con la transtiretina (ttr) - Google Patents

Métodos para tratar o prevenir enfermedades asociadas con la transtiretina (ttr)

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Publication number
AR097738A1
AR097738A1 ARP140103519A ARP140103519A AR097738A1 AR 097738 A1 AR097738 A1 AR 097738A1 AR P140103519 A ARP140103519 A AR P140103519A AR P140103519 A ARP140103519 A AR P140103519A AR 097738 A1 AR097738 A1 AR 097738A1
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AR
Argentina
Prior art keywords
ttr
nucleotides
yyy
zzz
xxx
Prior art date
Application number
ARP140103519A
Other languages
English (en)
Inventor
G Rajeev Kallanthottathil
Zimmermann Trady
Manoharan Muthiah
Maier Martin
Kuchimanchi Satyanarayana
Charisse Klaus
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Alnylam Pharmaceuticals Inc
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AR097738A1 publication Critical patent/AR097738A1/es

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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Métodos para tratar o prevenir enfermedades asociadas con la transtiretina (TTR) usando agentes de ARNi, por ejemplo, agentes de ARNi de doble hebra, que se dirigen contra el gen de transtiretina (TTR). Reivindicación 1: Un método para tratar o prevenir una enfermedad relacionada con la TTR en un sujeto, caracterizado porque comprende administrar a dicho sujeto un agente de ARNi a una dosis de aproximadamente 7,5 mg/kg, por día durante cinco días, seguido por una dosis de aproximadamente 7,5 mg/kg por semana durante aproximadamente cinco semanas, en donde el agente de ARNi es un agente de ARNi de doble hebra, que comprende una cadena orientada en el sentido del marco de lectura que es complementaria de una hebra antisentido, en donde dicha hebra antisentido comprende una región que es la parte complementaria de un ARNm que codifica transtiretina (TTR), en donde cada cadena tiene entre aproximadamente 14 y aproximadamente 30 nucleótidos, en donde dicho agente de ARNi de doble hebra está representado por la fórmula: hebra sentido: 5 nₚ-Na-(XXX)ⁱ-Nb-YYY-Nb-(ZZZ)ʲ-Na-nq 3 hebra antisentido: 3 nₚ-Na-(XXX)ₖ-Nb-YYY-Nb-(ZZZ)ₗ-Na-nq 5 en donde: i, j, k y I son cada uno de manera independiente 0 ó 1; p, p, q y q son cada uno de manera independiente 0 - 6; cada Na y Na representa de manera independiente una secuencia de oligonucleótidos que comprende 0 - 25 nucleótidos que están modificados o no modificados o combinaciones de los mismos, donde cada secuencia comprende al menos dos nucleótidos modificados de manera diferente; cada Nb y Nb representa de manera independiente una secuencia de oligonucleótidos que comprende 0 - 10 nucleótidos que están modificados o no modificados o combinaciones de los mismos; cada nₚ, nₚ, nq y nq representa de manera independiente un nucleótido saliente; XXX, YYY, ZZZ, XXX, YYY, y ZZZ representan cada uno de manera independiente un motivo de tres modificaciones idénticas en tres nucleótidos consecutivos; las modificaciones de Nb difieren de la modificación de Y; y las modificaciones de Nb difieren de la modificación de Y; y en donde la hebra sentido se conjuga a por lo menos un ligando, con lo que se trata o previene dicha enfermedad asociada con TTR en dicho sujeto.
ARP140103519A 2013-09-23 2014-09-23 Métodos para tratar o prevenir enfermedades asociadas con la transtiretina (ttr) AR097738A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201361881257P 2013-09-23 2013-09-23

Publications (1)

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AR097738A1 true AR097738A1 (es) 2016-04-13

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ARP140103519A AR097738A1 (es) 2013-09-23 2014-09-23 Métodos para tratar o prevenir enfermedades asociadas con la transtiretina (ttr)

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AR (1) AR097738A1 (es)
TW (1) TW201610151A (es)
WO (1) WO2015042564A1 (es)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2555778A4 (en) 2010-04-06 2014-05-21 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING CD274 / PD-L1 GENE EXPRESSION
JP6771387B2 (ja) * 2014-03-25 2020-10-21 アークトゥラス・セラピューティクス・インコーポレイテッドＡｒｃｔｕｒｕｓ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的ｕｎａオリゴマー
US9856475B2 (en) * 2014-03-25 2018-01-02 Arcturus Therapeutics, Inc. Formulations for treating amyloidosis
AU2015236215B2 (en) 2014-03-25 2020-03-19 Arcturus Therapeutics, Inc. UNA oligomers having reduced off-target effects in gene silencing
EP3812462A1 (en) 2014-08-20 2021-04-28 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
US10519447B2 (en) * 2015-04-01 2019-12-31 Arcturus Therapeutics, Inc. Therapeutic UNA oligomers and uses thereof
WO2017015671A1 (en) * 2015-07-23 2017-01-26 Arcturus Therapeutics, Inc. Compositions for treating amyloidosis
JP6896703B2 (ja) 2015-07-31 2021-06-30 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． ＴＴＲ関連疾患を治療または予防するためのトランスサイレチン（ＴＴＲ）ｉＲＮＡ組成物およびその使用方法
KR20180051550A (ko) 2015-09-02 2018-05-16 알닐람 파마슈티칼스 인코포레이티드 프로그램된 세포사 1 리간드 1 (PD-L1) iRNA 조성물 및 그의 사용 방법
MA45478A (fr) 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
JP2019535839A (ja) 2016-11-29 2019-12-12 ピュアテック ヘルス エルエルシー 治療剤の送達のためのエクソソーム
CN118460536A (zh) 2017-04-11 2024-08-09 阿布特斯生物制药公司 靶向组合物
AU2018336806A1 (en) 2017-09-19 2020-05-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
CA3114396A1 (en) * 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
WO2020093053A1 (en) * 2018-11-02 2020-05-07 Arbutus Biopharma Corporation Bivalent targeted conjugates
WO2021092145A1 (en) * 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna composition and methods of use thereof for treating or preventing ttr-associated ocular diseases
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
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

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1861608A (en) 1929-12-21 1932-06-07 Emerson Electric Mfg Co Fan and means for directing the air current therethrough
US1861108A (en) 1930-01-24 1932-05-31 Eugene O Brace Integral clutch and transmission control
US3974808A (en) 1975-07-02 1976-08-17 Ford Motor Company Air intake duct assembly
US4708708A (en) 1982-12-06 1987-11-24 International Paper Company Method and apparatus for skiving and hemming
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5143854A (en) 1989-06-07 1992-09-01 Affymax Technologies N.V. Large scale photolithographic solid phase synthesis of polypeptides and receptor binding screening thereof
US5744101A (en) 1989-06-07 1998-04-28 Affymax Technologies N.V. Photolabile nucleoside protecting groups
NL8901881A (nl) 1989-07-20 1991-02-18 Rockwool Grodan Bv Drainagekoppelelement.
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5677195A (en) 1991-11-22 1997-10-14 Affymax Technologies N.V. Combinatorial strategies for polymer synthesis
AU4528493A (en) 1992-06-04 1994-01-04 Regents Of The University Of California, The In vivo gene therapy with intron-free sequence of interest
JPH07508410A (ja) 1992-06-18 1995-09-21 ジェンファーム　インターナショナル　インコーポレイテッド 酵母人工染色体を有するトランスジェニック非ヒト動物の製造方法
US5556752A (en) 1994-10-24 1996-09-17 Affymetrix, Inc. Surface-bound, unimolecular, double-stranded DNA
EP0833613A1 (en) 1995-05-26 1998-04-08 Somatix Therapy Corporation Delivery vehicles comprising stable lipid/nucleic acid complexes
US5545531A (en) 1995-06-07 1996-08-13 Affymax Technologies N.V. Methods for making a device for concurrently processing multiple biological chip assays
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US6034135A (en) 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
EP1520022B1 (en) 2002-07-10 2015-07-22 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Rna-interference by single-stranded rna molecules
US7858769B2 (en) 2004-02-10 2010-12-28 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using multifunctional short interfering nucleic acid (multifunctional siNA)
WO2007091269A2 (en) 2006-02-08 2007-08-16 Quark Pharmaceuticals, Inc. NOVEL TANDEM siRNAS
WO2007117686A2 (en) 2006-04-07 2007-10-18 Idera Pharmaceuticals, Inc. Stabilized immune modulatory rna (simra) compounds for tlr7 and tlr8
EP2357231A2 (en) 2007-07-09 2011-08-17 Idera Pharmaceuticals, Inc. Stabilized immune modulatory RNA (SIMRA) compounds
EP4074344A1 (en) 2007-12-04 2022-10-19 Arbutus Biopharma Corporation Targeting lipids
CN103937793B (zh) * 2008-10-20 2017-08-11 阿尔尼拉姆医药品有限公司 抑制运甲状腺素蛋白表达的组合物和方法
US9200276B2 (en) 2009-06-01 2015-12-01 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent RNA interference, compositions and methods of use thereof
HUE026020T2 (en) 2009-08-27 2016-05-30 Idera Pharmaceuticals Inc Preparation for inhibiting gene expression and its uses
EP2496238A4 (en) * 2009-11-03 2013-10-02 Alnylam Pharmaceuticals Inc FORMULATED LIPID COMPOSITIONS AND METHODS FOR INHIBITING TRANSTHYRETIN EXPRESSION (TTR)
CA2792291A1 (en) * 2010-03-29 2011-10-06 Kumamoto University Sirna therapy for transthyretin (ttr) related ocular amyloidosis
WO2012177906A1 (en) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
DK3301177T3 (da) 2011-11-18 2020-06-15 Alnylam Pharmaceuticals Inc Rnai-midler, sammensætninger og fremgangsmåder til anvendelse deraf til behandling af transthyretin (ttr)-forbundne sygdomme
KR102318555B1 (ko) 2020-03-19 2021-10-29 한국과학기술연구원 광소자용 역나노콘과 그 제조방법

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Publication number Publication date
WO2015042564A1 (en) 2015-03-26
TW201610151A (zh) 2016-03-16
WO2015042564A8 (en) 2015-05-28

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