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AR125230A1 - COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO - Google Patents

COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO

Info

Publication number
AR125230A1
AR125230A1 ARP220100733A ARP220100733A AR125230A1 AR 125230 A1 AR125230 A1 AR 125230A1 AR P220100733 A ARP220100733 A AR P220100733A AR P220100733 A ARP220100733 A AR P220100733A AR 125230 A1 AR125230 A1 AR 125230A1
Authority
AR
Argentina
Prior art keywords
htt
huntingtin
methods
compositions
double
Prior art date
Application number
ARP220100733A
Other languages
English (en)
Inventor
Mangala Meenakshi Soundarapandian
James D Mininch
Mark K Schlegel
Adam Castoreno
Original Assignee
Alnylam 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AR125230A1 publication Critical patent/AR125230A1/es

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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
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    • 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
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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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
    • C12N2310/351—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)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Agentes de ácido ribonucleico de hebra doble (ARNihd) y composiciones contra un gen de Huntingtina (HTT), así como métodos para inhibir la expresión de un gen de HTT y métodos para tratar sujetos que tienen una enfermedad o trastorno asociado con HTT, por ejemplo, la enfermedad de Huntington, usando dichos agentes y composiciones de ARNihd. Reivindicación 1: Un agente de ácido ribonucleico de hebra doble (ARNhd), que inhibe la expresión de la Huntingtina (HTT) en una célula, caracterizado porque el ARNhd comprende una hebra sentido y una hebra antisentido que forman una región de hebra doble, en donde la hebra antisentido comprende una región de complementariedad con un ARNm que codifica HTT, y en donde la región de complementariedad comprende por lo menos 15 nucleótidos contiguos que difieren en no más de 3 nucleótidos de cualquiera de las secuencias de nucleótidos antisentido de cualquiera de las Tablas 2 - 5, excepto por los dúplex AD-1019465 y AD-1019476.
ARP220100733A 2021-03-29 2022-03-28 COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO AR125230A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163167140P 2021-03-29 2021-03-29

Publications (1)

Publication Number Publication Date
AR125230A1 true AR125230A1 (es) 2023-06-28

Family

ID=81388848

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220100733A AR125230A1 (es) 2021-03-29 2022-03-28 COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO

Country Status (13)

Country Link
US (2) US11993774B2 (es)
EP (1) EP4314296A2 (es)
JP (1) JP2024512635A (es)
KR (1) KR20230162024A (es)
CN (1) CN117203336A (es)
AR (1) AR125230A1 (es)
AU (1) AU2022246786A1 (es)
BR (1) BR112023019981A2 (es)
CA (1) CA3214499A1 (es)
IL (1) IL307239A (es)
MX (1) MX2023011466A (es)
TW (1) TW202305134A (es)
WO (1) WO2022212231A2 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR120341A1 (es) * 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
WO2024156083A1 (en) * 2023-01-27 2024-08-02 Beijing Jitai Pharmaceutical Technology Co., Ltd. Ai-assisted design of lnps for effective cns delivery of rna
WO2025168022A1 (en) * 2024-02-07 2025-08-14 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of huntingtin (htt)
WO2025265013A2 (en) 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use

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CA3214499A1 (en) 2022-10-06
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IL307239A (en) 2023-11-01
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