MA47015A - Conjugués oligomères de sauts d'exons pour la dystrophie musculaire - Google Patents

Conjugués oligomères de sauts d'exons pour la dystrophie musculaire

Info

Publication number
MA47015A
MA47015A MA047015A MA47015A MA47015A MA 47015 A MA47015 A MA 47015A MA 047015 A MA047015 A MA 047015A MA 47015 A MA47015 A MA 47015A MA 47015 A MA47015 A MA 47015A
Authority
MA
Morocco
Prior art keywords
muscle dystrophy
oligomeric conjugates
exon
jumping
exon jumping
Prior art date
Application number
MA047015A
Other languages
English (en)
Other versions
MA47015B1 (fr
Inventor
Gunnar J Hanson
Marco A Passini
Original Assignee
Sarepta Therapeutics 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 Sarepta Therapeutics Inc filed Critical Sarepta Therapeutics Inc
Priority claimed from PCT/US2017/066351 external-priority patent/WO2018118627A1/fr
Publication of MA47015A publication Critical patent/MA47015A/fr
Publication of MA47015B1 publication Critical patent/MA47015B1/fr

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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • 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
    • A61K47/62Medicinal 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 a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N2310/11Antisense
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • 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
    • C12N2310/3513Protein; Peptide
    • 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
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA47015A 2016-12-19 2017-12-14 Conjugués oligomères de sauts d'exons pour la dystrophie musculaire MA47015B1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662436199P 2016-12-19 2016-12-19
PCT/US2017/066351 WO2018118627A1 (fr) 2016-12-19 2017-12-14 Conjugués oligomères de sauts d'exons pour la dystrophie musculaire

Publications (2)

Publication Number Publication Date
MA47015A true MA47015A (fr) 2019-10-23
MA47015B1 MA47015B1 (fr) 2022-11-30

Family

ID=66326249

Family Applications (1)

Application Number Title Priority Date Filing Date
MA47015A MA47015B1 (fr) 2016-12-19 2017-12-14 Conjugués oligomères de sauts d'exons pour la dystrophie musculaire

Country Status (2)

Country Link
AR (1) AR110537A1 (fr)
MA (1) MA47015B1 (fr)

Also Published As

Publication number Publication date
AR110537A1 (es) 2019-04-10
MA47015B1 (fr) 2022-11-30

Similar Documents

Publication Publication Date Title
FR25C1001I2 (fr) Compositions d'anticorps pour le traitement de tumeurs
EP3347026A4 (fr) Modification génétique de macrophages pour l'immunothérapie
EP3353278A4 (fr) Préparation d'échantillons pour des types d'échantillons compliqués
MA71266A (fr) Procédés d'intervention précoce pour prévenir ou améliorer la toxicité
IL255125A0 (en) Evaluation of cas9 molecule/guide rna molecule complexs
EP3423044A4 (fr) Nanovaccin activant sting pour l'immunothérapie
EP3356612A4 (fr) Détail facilité pour membranes d'imperméabilisation pré-appliquées
MA46652A (fr) Polythérapie pour l'inhibition de c3
EP3334499A4 (fr) Conjugués bioactifs pour l'administration d'oligonucléotides
EP3340786A4 (fr) Production améliorée d'immunoglobulines
MA52801A (fr) Conjugués oligomères à saut d'exon pour dystrophie musculaire
EP3383418A4 (fr) Peptides slc45a2 pour l'immunothérapie
EP3409108A4 (fr) Couvre-lit pour lit d'animaux de compagnie
EP3464636A4 (fr) Extension d'amorce conditionnelle pour la détection de molécule unique
MA45845A (fr) Traitement d'association pour cancers hématologiques
EP3627918C0 (fr) Radiorecherche d'extension de couverture pour ec-gsm
MA46048A (fr) Kit de débutant pour le titrage de l'insuline de base
EP3476478A4 (fr) Composition pour l'élimination de composés soufrés
EP3397360A4 (fr) Ensemble de pédale pour machine d'exercice
FR3034840B3 (fr) Ensemble d'eclairage flexible pour voutes courbees
EP3541379A4 (fr) Compositions pour le traitement de l'hypertension
EP3443969A4 (fr) Composition d'amélioration de la fonction cérébrale pour nouveau-nés
EP3633372A4 (fr) Biomarqueur pour la maladie d'alzheimer
EP3344650A4 (fr) Aav-epo pour le traitement d'animaux de compagnie
EP3538087A4 (fr) Réactifs basés sur une oxydo-réduction pour la bioconjugaison d'une méthionine