LT2560687T - Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams - Google Patents

Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams

Info

Publication number
LT2560687T
LT2560687T LTEP11718678.3T LT11718678T LT2560687T LT 2560687 T LT2560687 T LT 2560687T LT 11718678 T LT11718678 T LT 11718678T LT 2560687 T LT2560687 T LT 2560687T
Authority
LT
Lithuania
Prior art keywords
compositions
methods
oligonucleotide molecules
selective delivery
specific neuron
Prior art date
Application number
LTEP11718678.3T
Other languages
English (en)
Lithuanian (lt)
Inventor
Andrés Pablo Montefeltro
Gabriel Alvarado Urbina
Analia Bortolozzi Biassoni
Francesc ARTIGAS PÉREZ
Miquel Vila Bover
Original Assignee
Nlife Therapeutics S.L.
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
Priority claimed from EP10382087A external-priority patent/EP2380595A1/en
Application filed by Nlife Therapeutics S.L. filed Critical Nlife Therapeutics S.L.
Publication of LT2560687T publication Critical patent/LT2560687T/lt

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/13Amines
    • A61K31/135Amines having aromatic rings, e.g. ketamine, nortriptyline
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/13Amines
    • A61K31/135Amines having aromatic rings, e.g. ketamine, nortriptyline
    • A61K31/138Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/13Amines
    • A61K31/15Oximes (>C=N—O—); Hydrazines (>N—N<); Hydrazones (>N—N=) ; Imines (C—N=C)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/4523Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4525Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47Quinolines; Isoquinolines
    • A61K31/472Non-condensed isoquinolines, e.g. papaverine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/543Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/55Medicinal 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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/0002General or multifunctional contrast agents, e.g. chelated agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/24Antidepressants
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D207/00Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/46Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with hetero atoms directly attached to the ring nitrogen atom
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D217/00Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems
    • C07D217/02Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems with only hydrogen atoms or radicals containing only carbon and hydrogen atoms, directly attached to carbon atoms of the nitrogen-containing ring; Alkylene-bis-isoquinolines
    • C07D217/04Heterocyclic compounds containing isoquinoline or hydrogenated isoquinoline ring systems with only hydrogen atoms or radicals containing only carbon and hydrogen atoms, directly attached to carbon atoms of the nitrogen-containing ring; Alkylene-bis-isoquinolines with hydrocarbon or substituted hydrocarbon radicals attached to the ring nitrogen atom
    • 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
    • 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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • 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
    • C12N2310/11Antisense
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Pain & Pain Management (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Other In-Based Heterocyclic Compounds (AREA)
LTEP11718678.3T 2010-04-19 2011-04-19 Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams LT2560687T (lt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US32551510P 2010-04-19 2010-04-19
EP10382087A EP2380595A1 (en) 2010-04-19 2010-04-19 Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
EP11382031 2011-02-09
PCT/EP2011/056270 WO2011131693A2 (en) 2010-04-19 2011-04-19 Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types

Publications (1)

Publication Number Publication Date
LT2560687T true LT2560687T (lt) 2017-09-25

Family

ID=44063382

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP11718678.3T LT2560687T (lt) 2010-04-19 2011-04-19 Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams

Country Status (14)

Country Link
US (2) US9193969B2 (enExample)
EP (2) EP3231446A1 (enExample)
JP (1) JP5819401B2 (enExample)
KR (1) KR20130103662A (enExample)
AR (1) AR083715A1 (enExample)
AU (4) AU2011244321A1 (enExample)
BR (1) BR112012026471A2 (enExample)
CA (1) CA2796722C (enExample)
DK (1) DK2560687T3 (enExample)
ES (1) ES2638309T3 (enExample)
LT (1) LT2560687T (enExample)
MX (1) MX342764B (enExample)
RU (1) RU2664452C2 (enExample)
WO (1) WO2011131693A2 (enExample)

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1463512B1 (en) 2002-01-11 2014-05-28 biOasis Technologies Inc. Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
DK2560687T3 (en) 2010-04-19 2017-09-18 Nlife Therapeutics S L Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types
CA2817960C (en) 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
PT2717917T (pt) 2011-07-05 2016-07-27 Bioasis Technologies Inc Conjugados de anticorpos p97
ES2632212T3 (es) 2011-08-04 2017-09-11 Yeda Research And Development Co. Ltd. miR-135 y composiciones que lo comprenden para el tratamiento de afecciones médicas asociadas con la serotonina
CA2858576A1 (en) * 2011-12-06 2013-06-13 Research Institute At Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
WO2013148260A1 (en) 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
AU2013296557B2 (en) 2012-07-31 2019-04-18 Bioasis Technologies Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
AU2013336582A1 (en) * 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to cell types
CA2890112A1 (en) 2012-10-26 2014-05-01 Nlife Therapeutics, S.L. Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
EP3406718A1 (en) 2012-11-15 2018-11-28 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
EP2743695A1 (en) 2012-12-12 2014-06-18 Nanogap Sub NM Powder, S.A. Methods and reagents for the detection of biomolecules using luminescence
CN105263958B (zh) 2013-03-13 2019-09-27 比奥阿赛斯技术有限公司 p97片段及其应用
EP3766974A1 (en) 2013-03-14 2021-01-20 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
JP6262848B2 (ja) * 2013-05-21 2018-01-17 成都先導薬物開発有限公司 薬物標的の捕獲方法
CN104178515B (zh) * 2013-05-21 2018-08-31 成都先导药物开发有限公司 一种化合物的细胞透膜的方法
CN104177465B (zh) * 2013-05-21 2017-09-29 成都先导药物开发有限公司 一种化合物给药前体及药物载体制剂
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ma公司 用於調節τ蛋白表現之組合物
BR112016017933A2 (pt) 2014-02-03 2017-10-10 Bioasis Technologies Inc ?proteínas de fusão p97?
CA2936158C (en) 2014-02-05 2023-06-13 Yeda Research And Development Co. Ltd. Use of mir-135 or precursor thereof for the treatment and diagnosis of a bipolar disease
WO2015126729A1 (en) 2014-02-19 2015-08-27 Bioasis Technologies, Inc. P97-ids fusion proteins
CA2943890A1 (en) * 2014-05-01 2015-11-05 Bioasis Technologies, Inc. P97-polynucleotide conjugates
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
JP6867945B2 (ja) 2014-10-03 2021-05-12 コールド スプリング ハーバー ラボラトリー 核内遺伝子出力の標的とされた増強
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US10733023B1 (en) * 2015-08-06 2020-08-04 D2Iq, Inc. Oversubscription scheduling
DK3133166T4 (da) * 2015-08-21 2023-03-20 42 Life Sciences Gmbh & Co Kg Sammensætning og fremgangsmåde til hybridisering
WO2017060731A1 (en) 2015-10-09 2017-04-13 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
AU2016370653A1 (en) 2015-12-14 2018-06-21 Cold Spring Harbor Laboratory Antisense oligomers for treatment of Autosomal Dominant Mental Retardation-5 and Dravet Syndrome
GB2574525B (en) * 2015-12-21 2020-09-02 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
GB2545898B (en) 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
KR101916652B1 (ko) * 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
US10208098B2 (en) * 2016-09-15 2019-02-19 Council Of Scientific & Industrial Research Recombinant protein-based method for the delivery of silencer RNA to target the brain
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
LT3673080T (lt) 2017-08-25 2023-12-27 Stoke Therapeutics, Inc. Priešprasminiai oligomerai, skirti būklėms ir ligoms gydyti
CA3076612A1 (en) 2017-09-22 2019-03-28 John Mansell Compositions and methods for treatment of sepsis-related disorders
EP3461487A1 (en) * 2017-09-29 2019-04-03 Nlife Therapeutics S.L. Compositions and methods for the delivery of mrna to hepatic cells
US20210268667A1 (en) 2017-10-23 2021-09-02 Stoke Therapeutics, Inc. Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
IL302108A (en) 2017-10-25 2023-06-01 Ac Immune Sa Preparations of phosphorylated tau peptides and uses thereof
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
BR112020014011A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso dirigidos à alfa-sinucleína e usos dos mesmos
IL275902B2 (en) 2018-01-12 2025-10-01 Bristol Myers Squibb Co Antisense oligonucleotides directed against alpha-synuclein and their uses
BR112020012921A2 (pt) 2018-01-12 2020-12-08 Roche Innovation Center Copenhagen A/S Oligonucleotídeos antissenso de alfa-sinucleína e usos dos mesmos
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
CN112218877B (zh) 2018-08-27 2025-07-25 瑞泽恩制药公司 拉曼光谱在下游纯化中的应用
ES2698565B2 (es) * 2018-12-12 2019-07-16 Univ Madrid Complutense Procedimiento para la elaboración de nanopartículas lipídicas, y nanopartículas lipídicas con los macrófagos cerebrales como células diana
CA3129252A1 (en) 2019-02-08 2020-08-13 Ac Immune S.A. Method of safe administration of phosphorylated tau peptide vaccine
CN114364404A (zh) 2019-04-11 2022-04-15 东北大学 寡核苷酸-聚合物多手臂偶联物以及使用方法
WO2020257194A1 (en) * 2019-06-17 2020-12-24 Alnylam Pharmaceuticals, Inc. Delivery of oligonucleotides to the striatum
EP4150092A4 (en) 2020-05-11 2024-11-06 Stoke Therapeutics, Inc. OPA1 ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASES AND CONDITIONS
CA3204415A1 (en) * 2021-01-18 2022-07-21 Alon Chen Modified mir-135, conjugated form thereof, and uses of same
WO2024069229A2 (en) 2022-08-03 2024-04-04 Sutura Therapeutics Ltd Biologically active compounds
US12396981B2 (en) 2023-03-09 2025-08-26 William Shulman Methods of using DMT

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003042162A1 (en) * 2001-11-17 2003-05-22 Neurosearch A/S Prodrugs of antidepressants and their use for treating depressions
EP1336406A1 (en) * 2002-02-14 2003-08-20 Solvay Pharmaceuticals B.V. Partial dopamine-D2 receptor agonist plus serotonin and/or noradrenaline inhibitory activity
EP1711632A4 (en) * 2004-01-19 2009-03-11 Technion Res & Dev Foundation DIAGNOSTIC TEST FOR PARKINSON
WO2007050789A2 (en) * 2005-10-25 2007-05-03 Nëdken Corporation Glycosyl-oligonucleotide conjugates and methods
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
WO2008033285A2 (en) * 2006-09-15 2008-03-20 The Trustees Of Culumbia University In The City Of New York Delivery of double-stranded rna into the central nervous system
EP2235175A4 (en) * 2007-12-20 2012-04-25 Angiochem Inc POLYPEPTIDE-NUCLEIC ACID CONJUGATES AND USES THEREOF
GB0809476D0 (en) 2008-05-23 2008-07-02 Cambridge Entpr Ltd Treatment of neurodegenerative disorders
DE102009058769A1 (de) * 2009-12-16 2011-06-22 MagForce Nanotechnologies AG, 10589 Temperaturabhängige Aktivierung von katalytischen Nukleinsäuren zur kontrollierten Wirkstofffreisetzung
DK2560687T3 (en) 2010-04-19 2017-09-18 Nlife Therapeutics S L Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types
CA2817960C (en) * 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
AU2013336582A1 (en) 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to cell types
CA2890112A1 (en) 2012-10-26 2014-05-01 Nlife Therapeutics, S.L. Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types

Also Published As

Publication number Publication date
BR112012026471A2 (pt) 2016-11-29
EP2560687B1 (en) 2017-05-24
ES2638309T3 (es) 2017-10-19
AU2017204529A1 (en) 2017-07-20
AR083715A1 (es) 2013-03-20
AU2020202533B2 (en) 2022-01-20
CA2796722A1 (en) 2011-10-27
CN103025357A (zh) 2013-04-03
JP2013525330A (ja) 2013-06-20
US10071163B2 (en) 2018-09-11
WO2011131693A3 (en) 2012-02-16
JP5819401B2 (ja) 2015-11-24
CA2796722C (en) 2021-06-01
AU2019203887A1 (en) 2019-07-18
RU2664452C2 (ru) 2018-08-17
WO2011131693A2 (en) 2011-10-27
EP3231446A1 (en) 2017-10-18
KR20130103662A (ko) 2013-09-24
US20160235856A1 (en) 2016-08-18
MX2012012214A (es) 2013-02-07
US20120322991A1 (en) 2012-12-20
DK2560687T3 (en) 2017-09-18
MX342764B (es) 2016-10-12
US9193969B2 (en) 2015-11-24
AU2011244321A1 (en) 2012-11-15
AU2019203887B2 (en) 2020-01-23
AU2020202533A1 (en) 2020-05-07
RU2012149042A (ru) 2014-05-27
EP2560687A2 (en) 2013-02-27

Similar Documents

Publication Publication Date Title
LT2560687T (lt) Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams
EP2736533A4 (en) SELECTIVE DELIVERY MOLECULES AND METHODS OF USE
IL227442A0 (en) Methods and reagents for targeted and efficient delivery of therapeutic molecules to cxcr4 cells
EP2521553A4 (en) METHODS AND COMPOSITIONS FOR THE DEVELOPMENT OF TARGETED MEDICAMENTS
EP2416652A4 (en) METHODS OF ADMINISTERING OLIGONUCLEOTIDES INTO DIRECTION OF IMMUNE CELLS
ZA201106262B (en) Nanoparticle mediated delivery of sequence specific nucleases
EP2655334A4 (en) COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISEASES
PT3623481T (pt) Composições para sequenciação de ácidos nucleicos
GB201010557D0 (en) RNA molecules and uses thereof
EP2648706A4 (en) COMPOSITIONS AND METHODS RELATING TO PROLIFERATIVE DISEASES
PH12013501969A1 (en) Compositions and methods for inhibiting expression of tmprss6 gene
IL221476A0 (en) Methods and compositions to treat hemorrhagic conditions of the brain
SG10201500048SA (en) Compositions and methods for treating glioblastoma gbm
ZA201304546B (en) Oligonucleotide probe set and methods of microbiota profiling
IL227624A0 (en) Empty nano-particle compounds and their use for the treatment of skin conditions
GB2478849A8 (en) Improved pharmaceutical compositions and methods of delivery
PT2782565T (pt) Utilização de aril- e hetaril-carboxamidas como endoparasiticidas
ZA201209643B (en) Method for hydrogenation of iso-alpha-acids and tetrahydro-iso-alpha-acids to hexahydro-iso-alpha-acids
EP2648763A4 (en) COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A
EP2535410A4 (en) APTAMER FOR FGF2 AND USE THEREOF
ZA201404281B (en) Methods and products to diagnose and treat heatiness
IL226170A0 (en) Compositions and methods for delivery of nucleic acids based on nanopolymers
IL226536A (en) DNA position-oriented change
IL226537A (en) Position-directed change of DNA with oligonucleotides
EP2751071A4 (en) Fatty Acid Amide, Compositions Thereof and Method of Use Therefor