AR123420A1 - Inhibidores de dux4 y métodos de uso de estos - Google Patents

Inhibidores de dux4 y métodos de uso de estos

Info

Publication number
AR123420A1
AR123420A1 ARP210102453A ARP210102453A AR123420A1 AR 123420 A1 AR123420 A1 AR 123420A1 AR P210102453 A ARP210102453 A AR P210102453A AR P210102453 A ARP210102453 A AR P210102453A AR 123420 A1 AR123420 A1 AR 123420A1
Authority
AR
Argentina
Prior art keywords
double
stranded
small interfering
dux4
sirna
Prior art date
Application number
ARP210102453A
Other languages
English (en)
Inventor
Sean Christopher Daugherty
Lishan Chen
Francis Michael Sverdrup
Original Assignee
Ultragenyx Pharmaceutical Inc
Univ Saint Louis
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 Ultragenyx Pharmaceutical Inc, Univ Saint Louis filed Critical Ultragenyx Pharmaceutical Inc
Publication of AR123420A1 publication Critical patent/AR123420A1/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Steroid Compounds (AREA)

Abstract

ARN interferentes pequeños bicatenarios que modulan la expresión del gen DUX4 y describe métodos para inhibir la expresión del gen DUX4 al poner en contacto una célula con dichos ARN interferentes pequeños bicatenarios. Además composiciones que comprenden dichos ARN interferentes pequeños bicatenarios y su uso en métodos para prevenir o tratar una enfermedad o trastorno asociado con la expresión aberrante de DUX4, como distrofia muscular facioescapulohumeral (FSHD) o cáncer en un sujeto. Reivindicación 1: Un ARN interferente pequeño bicatenario (ARNip) que comprende una cadena con sentido y una cadena antisentido, caracterizado porque la cadena antisentido del ARNip bicatenario comprende una secuencia de nucleobase de al menos 12 nucleótidos contiguos de una secuencia seleccionada del grupo que consiste en SEQ ID Nº 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, 26, 28, 30, 32, 34, 36, 38, 40, 42, 44, 46, 48, 50, 52, 54, 56, 58, 60, 62, 64, 66, 68, 70, 72, 74, 76, 78, 80, 82, 84, 86, 88, 90, 92, 94, 96, 98, 100, 102, 104, 106, 108, 110, 112, 114, 116, 118, 120, 122, 124, 126, 128, 130, 132, 134, 136, 138, 140, 142, 144, 146, 148, 150, 152, 154, 156, 158, 160, 162, 164, 166, 168, 170, 172, 174, 176, 178, 180, 182, 184, 186, 188, 190, 192, 194, 196, 198, 200, 202, 204, 206, 208, 210, 212, 214, 216, 218, 220, 222, 224, 226, 228, 230, 232, 234, 236, 238, 240, 242, 244, 246, 248, 250, 252, 254, 256, 258, 260, 262, 264, 266, 268, 270, 272, 274, 276, 278, 280, 282, 284, 286, 288, 290, 292, 294, 296, 298, 300, 302, 304, 306, 308, 310, 312, 314, 316, 318, 320, 322, 324, 326, 328, 330, 332, 334, 336, 338, 340, 342, 344, 346, 348, 350, 352, 354, 356, 358, 360, 362, 364, 366, 368, 370, 372, 374, 376, 378, 380, 382, 384, 386, 388, 390, 392, 394, 396, 398, 400, 402, 404, 406, 408, 410, 412, 414, 416, 418, 420, 422, 424, 426, 428, 430, 432, 434, 436, 438, 440, 442, 444, 446, 448, 450, 452, 454, 456, 458, 460, 462, 464, 466, 468, 470, 472, 474, 476, 478, 480, 482, 484, 486, 488, 490, 492, 494, 496, 498, 500, 502, 504, 506, 508, 510, 512, 514, 516, 518, 520, 522, 524, 526, 528, 530, 532, 534, 536, 538, 540, 542, 544, 546, 548, 550, 552, 554, 556, 558, 560, 562, 564, 566, 568, 570, 572, 574, 576, 578, 580, 582, 584, 586, 588, 590 y 592, y en donde el ARNip bicatenario comprende al menos un nucleósido modificado. Reivindicación 17: Una composición farmacéutica caracterizada porque comprende el ARNip bicatenario de acuerdo con cualquiera de las reivindicaciones anteriores y un portador farmacéuticamente aceptable. Reivindicación 36: Un ARNip bicatenario que comprende una cadena con sentido y una cadena antisentido, caracterizado porque la cadena con sentido comprende al menos 8 nucleótidos contiguos de una secuencia seleccionada del grupo que consiste en SEQ ID Nº 597, 599, 601, 603, 605, 607, 609, 611, 613, 615, 617, 619, 621, 623, 625, 627, 629, 631, 633, 635, 637, 639, 641, 643, 645, 647, 649, 651, 653, 655, 657, 659, 661, 663, 665, 667, 669, 671, 673, 675, 677, 679, 681, 683, 685 y 687. Reivindicación 39: Una composición farmacéutica caracterizada porque comprende el ARNip bicatenario de acuerdo con cualquiera de las reivindicaciones 36 - 38 y un portador farmacéuticamente aceptable.
ARP210102453A 2020-09-01 2021-09-01 Inhibidores de dux4 y métodos de uso de estos AR123420A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063073304P 2020-09-01 2020-09-01

Publications (1)

Publication Number Publication Date
AR123420A1 true AR123420A1 (es) 2022-11-30

Family

ID=80492176

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210102453A AR123420A1 (es) 2020-09-01 2021-09-01 Inhibidores de dux4 y métodos de uso de estos

Country Status (8)

Country Link
US (1) US20230348906A1 (es)
EP (1) EP4208548A1 (es)
JP (1) JP2023539341A (es)
AR (1) AR123420A1 (es)
AU (1) AU2021337595A1 (es)
CA (1) CA3189861A1 (es)
TW (1) TW202227626A (es)
WO (1) WO2022051332A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA202190416A1 (ru) 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. Мышечно-специфические комлексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии
CA3174286A1 (en) 2020-04-02 2021-10-07 Robert PLACE Targeted inhibition using engineered oligonucleotides
JP2023541404A (ja) 2020-09-11 2023-10-02 アローヘッド ファーマシューティカルズ インコーポレイテッド ＤＵＸ４の発現を阻害するためのＲＮＡｉ剤、その組成物、及び使用方法
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
WO2023185946A1 (zh) * 2022-03-30 2023-10-05 苏州瑞博生物技术股份有限公司 一种寡核苷酸缀合物、含有该寡核苷酸缀合物的组合物及制备方法和用途

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3636778B1 (en) * 2010-08-18 2022-01-12 Fred Hutchinson Cancer Research Center Agents for use in treating facioscapulohumeral dystrophy (fshd)
US10538763B2 (en) * 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4

Also Published As

Publication number Publication date
JP2023539341A (ja) 2023-09-13
WO2022051332A1 (en) 2022-03-10
AU2021337595A1 (en) 2023-04-06
US20230348906A1 (en) 2023-11-02
CA3189861A1 (en) 2022-03-10
EP4208548A1 (en) 2023-07-12
TW202227626A (zh) 2022-07-16

Similar Documents

Publication Publication Date Title
AR123420A1 (es) Inhibidores de dux4 y métodos de uso de estos
DK2756845T3 (en) Methods and compositions for specific inhibition of KRAS with asymmetric double stranded RNA
Le et al. TERRA, hnRNP A1, and DNA-PKcs interactions at human telomeres
EP2756845B1 (en) Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
CL2021002585A1 (es) Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central
AU2011276365B2 (en) Methods and compositions for the specific inhibition of beta-catenin by double-stranded RNA
US10301624B2 (en) Targeting human satellite II (HSATII)
US10612020B2 (en) Artificial mimic miRNA for controlling gene expression, and use of same
PE20212248A1 (es) Molecula oligomerica de acido nucleico y uso de esta
US10844376B2 (en) Structurally-enhanced miRNA inhibitor S-TuD
AR079649A1 (es) Composiciones y metodos para inhibir la expresion de genes il-18
US20220298512A1 (en) Sirna sequences targeting the expression of human genes jak1 or jak3 for a therapeutic use
EP2453017A1 (en) Oligonucleotides for use in prophylaxis and/or treatment of TGF-beta1 and TGF-beta2, TGF-beta2 and TGF-beta3, TGF-beta1 and TGF-beta3, or TGF-beta1, TGF-beta2, and TGF-beta3 mRNA overexpressing diseases
AR125230A1 (es) COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
PE20242176A1 (es) Compuestos dirigidos a pmp22 para el tratamiento de la enfermedad de charcot-marie-tooth
WO2021262919A3 (en) 5-halouracil-modified micrornas and their use in the treatment of cancer
WO2014112144A1 (ja) 染色体セントロメア由来のサテライトノンコーディングｒｎａを標的とした核酸抗癌剤、及び該抗癌剤を用いる方法
Bonnet et al. Post-transcriptional gene regulation: From mechanisms to RNA chemistry and therapeutics
Xu et al. Identification of genes associated to 2′, 2′-difluorodeoxycytidine resistance in HeLa cells with a lentiviral short-hairpin RNA library
US11453879B2 (en) Method for screening splicing variants or events
WO2024263791A1 (en) Antisense oligonucleotides targeting mir-34a-5p and methods of use thereof
AR126691A1 (es) COMPOSICIONES DE ARNi Y MÉTODOS PARA SILENCIAR EL GEN DE ANGIOTENSINÓGENO (AGT)
WO2023168202A3 (en) Certain dux4 inhibitors and methods of use thereof
AR124052A1 (es) COMPOSICIONES DE ARNi CONTRA EL FACTOR DE COAGULACIÓN V (F5) Y MÉTODOS DE USO DE LAS MISMAS
AR114435A1 (es) COMPOSICIONES DE ARNi DE 17b-HIDROXIESTEROIDE DESHIDROGENASAS TIPO 13 (HSD17B13) Y MÉTODOS DE USO DE LAS MISMAS

Legal Events

Date Code Title Description
FB Suspension of granting procedure