MX2021014868A - Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas. - Google Patents

Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas.

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Publication number
MX2021014868A
MX2021014868A MX2021014868A MX2021014868A MX2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A
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MX
Mexico
Prior art keywords
neurological diseases
methods
oligonucleotides
treating neurological
ftd
Prior art date
Application number
MX2021014868A
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English (en)
Inventor
Sudhir Agrawal
Sandra Hinckley
Duncan Brown
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Quralis Corp
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Publication date
Application filed by Quralis Corp filed Critical Quralis Corp
Publication of MX2021014868A publication Critical patent/MX2021014868A/es

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • 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
    • A61K31/713—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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

En el presente documento se describen secuencias de oligonucleótidos antisentido y métodos de uso para tratar enfermedades neurológicas.
MX2021014868A 2019-06-03 2020-06-03 Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas. MX2021014868A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962856264P 2019-06-03 2019-06-03
US201962914252P 2019-10-11 2019-10-11
US201962949817P 2019-12-18 2019-12-18
PCT/US2020/035811 WO2020247419A2 (en) 2019-06-03 2020-06-03 Oligonucleotides and methods of use for treating neurological diseases

Publications (1)

Publication Number Publication Date
MX2021014868A true MX2021014868A (es) 2022-03-25

Family

ID=73653367

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021014868A MX2021014868A (es) 2019-06-03 2020-06-03 Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas.

Country Status (11)

Country Link
US (1) US20220333105A1 (es)
EP (1) EP3976010A4 (es)
JP (1) JP2022536085A (es)
KR (1) KR20220033472A (es)
CN (1) CN114555069A (es)
AU (1) AU2020288555A1 (es)
BR (1) BR112021024463A2 (es)
CA (1) CA3142526A1 (es)
IL (1) IL288574A (es)
MX (1) MX2021014868A (es)
WO (1) WO2020247419A2 (es)

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JP2022517117A (ja) * 2019-01-14 2022-03-04 プレジデント アンド フェローズ オブ ハーバード カレッジ Ｓｔｍｎ２レベルを回復させるための方法及び組成物
WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
JP2023534557A (ja) 2020-07-23 2023-08-09 エフ． ホフマン－ラ ロシュ アーゲー Ｒｎａ結合タンパク質部位を標的とするオリゴヌクレオチド
JP2024513237A (ja) * 2021-04-06 2024-03-22 メイズ セラピューティクス， インコーポレイテッド Ｔｄｐ－４３プロテイノパチーを処置するための組成物及び方法
AU2022328401A1 (en) * 2021-08-11 2024-02-22 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting stathmin 2 (stmn2) and uses thereof
AU2022402929A1 (en) * 2021-12-03 2024-06-13 Quralis Corporation Splice switcher antisense oligonucleotides with modified backbone chemistries
GB202208384D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
WO2024124203A2 (en) * 2022-12-09 2024-06-13 Auttx, Llc Targeted treatment of spliceopathy-induced neurological disorders
US20240301447A1 (en) * 2023-02-15 2024-09-12 Arbor Biotechnologies, Inc. Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript

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EP2134863B1 (en) * 2007-03-13 2014-05-21 The Children's Hospital Of Philadelphia Genetic alterations on chromosome 16 and methods of use thereof for the diagnosis of type 1 diabetes
KR101703695B1 (ko) * 2009-05-22 2017-02-08 큐알엔에이, 인크. 전사 인자 ｅ3(ｔｆｅ3)에 대한 천연 안티센스 전사체의 억제에 의해 ｔｆｅ3 및 인슐린 수용체 기질 2(ｉｒｓ2)의 치료
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CN116034161A (zh) * 2020-03-25 2023-04-28 哈佛学院校长同事会 用于恢复stmn2水平的方法和组合物

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Publication number Publication date
WO2020247419A3 (en) 2021-01-14
US20220333105A1 (en) 2022-10-20
JP2022536085A (ja) 2022-08-12
CN114555069A (zh) 2022-05-27
KR20220033472A (ko) 2022-03-16
IL288574A (en) 2022-02-01
BR112021024463A2 (pt) 2022-03-08
EP3976010A2 (en) 2022-04-06
AU2020288555A1 (en) 2022-01-20
EP3976010A4 (en) 2023-08-02
WO2020247419A2 (en) 2020-12-10
CA3142526A1 (en) 2020-12-10

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