AR127588A1 - LIPID CONJUGATION TO DIRECT ASTROCYTES OF THE CENTRAL NERVOUS SYSTEM - Google Patents

LIPID CONJUGATION TO DIRECT ASTROCYTES OF THE CENTRAL NERVOUS SYSTEM

Info

Publication number
AR127588A1
AR127588A1 ARP220103040A ARP220103040A AR127588A1 AR 127588 A1 AR127588 A1 AR 127588A1 AR P220103040 A ARP220103040 A AR P220103040A AR P220103040 A ARP220103040 A AR P220103040A AR 127588 A1 AR127588 A1 AR 127588A1
Authority
AR
Argentina
Prior art keywords
astrocytes
nervous system
central nervous
direct
lipid conjugation
Prior art date
Application number
ARP220103040A
Other languages
Spanish (es)
Inventor
Bob Dale Brown
Maire Jung
Travis Grim
Matthew Costales
Original Assignee
Dicerna 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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of AR127588A1 publication Critical patent/AR127588A1/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • C12N2310/3125Methylphosphonates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/345Spatial arrangement of the modifications having at least two different backbone modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

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

Abstract

La presente invención proporciona conjugados de oligonucleótidos que inhiben o reducen la expresión de genes diana en los astrocitos del sistema nervioso central. Además se proporcionan composiciones que incluyen los mismos y usos de los mismos, en particular usos relativos al tratamiento de enfermedades, trastornos y/o afecciones asociadas con la expresión de un gen diana de astrocito en el SNC.The present invention provides oligonucleotide conjugates that inhibit or reduce the expression of target genes in astrocytes of the central nervous system. Additionally provided are compositions including the same and uses thereof, in particular uses relating to the treatment of diseases, disorders and/or conditions associated with the expression of an astrocyte target gene in the CNS.

ARP220103040A 2021-11-05 2022-11-04 LIPID CONJUGATION TO DIRECT ASTROCYTES OF THE CENTRAL NERVOUS SYSTEM AR127588A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163276406P 2021-11-05 2021-11-05

Publications (1)

Publication Number Publication Date
AR127588A1 true AR127588A1 (en) 2024-02-07

Family

ID=86242054

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220103040A AR127588A1 (en) 2021-11-05 2022-11-04 LIPID CONJUGATION TO DIRECT ASTROCYTES OF THE CENTRAL NERVOUS SYSTEM

Country Status (3)

Country Link
AR (1) AR127588A1 (en)
TW (1) TW202335674A (en)
WO (1) WO2023081823A1 (en)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113748208A (en) * 2019-04-04 2021-12-03 迪克纳制药公司 Compositions and methods for inhibiting gene expression in the central nervous system
WO2021178607A1 (en) * 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases

Also Published As

Publication number Publication date
TW202335674A (en) 2023-09-16
WO2023081823A1 (en) 2023-05-11

Similar Documents

Publication Publication Date Title
ECSP20018305A (en) IRNA AGENTS AND COMPOSITIONS TO INHIBIT THE EXPRESSION OF ANGIOPOYETIN TYPE 3 (ANGPTL3) AND METHODS OF USE
CL2022002837A1 (en) Antibody-drug conjugates comprising sting agonists (divisional 202202711)
CL2020002053A1 (en) Methods and compositions for macrophage polarization.
BR112018074185A2 (en) 3-oxo-2,6-diphenyl-2,3-dihydropyridazine-4-carboxamides
BR112021019880A2 (en) Gene therapies for lysosomal disorders
EA201591281A1 (en) RIBONUCLEIC ACIDS WITH 4'-THIOMODIFIED NUCLEOTIDES AND RELATED METHODS
CL2023000371A1 (en) Compositions and methods for inhibiting plp1 expression.
BR112022016382A2 (en) SPECIFIC TRIPTAMINES FOR USE IN THE TREATMENT OF MOOD DISEASES
DOP2007000015A (en) THERAPEUTIC USES OF RTP801 INHIBITORS
BR112023001957A2 (en) COMPOSITIONS AND METHODS TO INHIBIT LPA EXPRESSION
BR112018013084A2 (en) combination treatments comprising administration of imidazopyrazinones
CY1117499T1 (en) 5'-TRIPHOSPHORIC OLIGONUCLEOTIDE WITH HELD TYPE AND USES
MX2020004600A (en) Oligonucleotide constructs and uses thereof.
ECSP23096498A (en) NOVEL THERAPEUTIC ADMINISTRATION REMAINS AND USES OF THESE
CL2021001490A1 (en) Chemically Modified Iarn Constructs and Uses of These
PA8446301A1 (en) 2-AMINO-6- (4-PHENOXI-2-SUBSTITUTED) -PIRIDINES SUBSTITUTED
CL2022002521A1 (en) Compositions and methods to inhibit the expression of angptl3
CY1118297T1 (en) THERAPEUTIC USES OF ESLICARVAZEPIN
CL2022003369A1 (en) Rnai constructs for inhibiting hsd17b13 expression and methods of use thereof
ECSP23076906A (en) COMPOSITIONS AND METHODS FOR INHIBITING KETOHEXOKINASE (KHK)
CL2022001503A1 (en) Human chromosome 9 open reading frame 72 (c9orf72) arni agents, compositions
BR112022019609A2 (en) GENETICLY MODIFIED CELLS ENRICHED WITH MITOCHONDRIA AND USES THEREOF
BR112022009710A2 (en) COMBINATION THERAPY COMPRISING AN ALK2 INHIBITOR AND A JAK2 INHIBITOR
CL2020002038A1 (en) Oligonucleotides to modulate the expression of tmem106b.
AR127588A1 (en) LIPID CONJUGATION TO DIRECT ASTROCYTES OF THE CENTRAL NERVOUS SYSTEM

Legal Events

Date Code Title Description
FB Suspension of granting procedure