AR123420A1 - Inhibidores de dux4 y métodos de uso de estos - Google Patents
Inhibidores de dux4 y métodos de uso de estosInfo
- 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
Links
Classifications
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- 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
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- 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
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- 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
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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
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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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
- 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.
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)
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)
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 |
-
2021
- 2021-09-01 AR ARP210102453A patent/AR123420A1/es unknown
- 2021-09-01 AU AU2021337595A patent/AU2021337595A1/en active Pending
- 2021-09-01 WO PCT/US2021/048611 patent/WO2022051332A1/en unknown
- 2021-09-01 US US18/043,520 patent/US20230348906A1/en active Pending
- 2021-09-01 JP JP2023514067A patent/JP2023539341A/ja active Pending
- 2021-09-01 CA CA3189861A patent/CA3189861A1/en active Pending
- 2021-09-01 TW TW110132422A patent/TW202227626A/zh unknown
- 2021-09-01 EP EP21865016.6A patent/EP4208548A1/en active Pending
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 |
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