AR125267A1 - Administración de ácido nucleico al sistema nervioso central - Google Patents

Administración de ácido nucleico al sistema nervioso central

Info

Publication number
AR125267A1
AR125267A1 ARP220100805A ARP220100805A AR125267A1 AR 125267 A1 AR125267 A1 AR 125267A1 AR P220100805 A ARP220100805 A AR P220100805A AR P220100805 A ARP220100805 A AR P220100805A AR 125267 A1 AR125267 A1 AR 125267A1
Authority
AR
Argentina
Prior art keywords
poly
antisense oligonucleotide
lactide
delivery composition
glycolide
Prior art date
Application number
ARP220100805A
Other languages
English (en)
Inventor
Hasan M Yildiz
Mandana Bornapour
Paul Peng
Vishwesh A Patil
Brian R Simler
William F Kiesman
Original Assignee
Biogen Ma 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 Biogen Ma Inc filed Critical Biogen Ma Inc
Publication of AR125267A1 publication Critical patent/AR125267A1/es

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/56—Medicinal 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
    • A61K47/59—Medicinal 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
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A61K9/5153—Polyesters, e.g. poly(lactide-co-glycolide)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Biophysics (AREA)
  • Optics & Photonics (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Psychology (AREA)
  • Nanotechnology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

Se presentan nanoportadores poliméricos (p. ej., nanopartículas de PLGA) con ácido nucleico encapsulado (p. ej., un oligonucleótido antisentido) para la administración (p. ej., por vía intratecal) al sistema nervioso central. Estos nanoportadores poliméricos son útiles en el tratamiento de trastornos del sistema nervioso central. Son capaces de administrar su carga (p. ej., un oligonucleótido antisentido) en cantidades mayores, durante un período de tiempo más largo y en regiones más profundas del cerebro que un oligonucleótido antisentido libre o no formulado. La administración y distribución eficientes del oligonucleótido antisentido resulta en la reducción del número de administraciones y la mejora de la experiencia y el cumplimiento del paciente. Reivindicación 1: Una composición de administración al sistema nervioso central (SNC) que comprende un nanoportador polimérico y un oligonucleótido antisentido, donde el oligonucleótido antisentido está encapsulado dentro del nanoportador polimérico, y donde el oligonucleótido antisentido está precomplejado directamente con una molécula catiónica. Reivindicación 2: La composición de administración al SNC de la reivindicación 1, donde el nanoportador polimérico se selecciona del grupo que consiste en poli(l-lactida), poli(glicólida), poli(d, l-lactida) (PLA), poli(dioxanona), poli(d, l-lactida-co-l-lactida), poli(d, l-lactida-co-glicólida), poli(glicólida-co-trimetileno carbonato), poli(caprolactona) (“policaprolactona”), poli(d, l-lactida-co-glicólida) (PLGA), poli(dioxanona) poli(glicólida-co-trimetileno carbonato), y mezclas de estos. Reivindicación 5: La composición de administración al SNC de una cualquiera de las reivindicaciones 1 a 4, donde la molécula catiónica es un péptido catiónico. Reivindicación 6: La composición de administración al SNC de una cualquiera de las reivindicaciones 1 a 4, donde la molécula catiónica es un quitosano, hexadecilamina o arginato láurico. Reivindicación 10: La composición de administración al SNC de la reivindicación 9, donde el agente terapéutico se selecciona del grupo que consiste en una molécula pequeña, un ADNc, un ARNm, un ARNip, un miARN, un aptámero y una ribozima. Reivindicación 17: Un método para tratar la atrofia muscular espinal (AME), aumentando la inclusión del exón 7 en las transcripciones del ácido ribonucleico mensajero (ARNm) SMN2 en un sujeto humano que tiene pérdida de ambas copias funcionales del gen SMN1, o aumentando la inclusión del exón 7 en el ácido ribonucleico mensajero (ARNm) SMN2 en un sujeto humano que tiene mutaciones en el gen SMN1 que conducen a una deficiencia funcional de la proteína SMN, en un sujeto humano que lo necesita, el método que comprende administrar mediante una inyección en el espacio intratecal del sujeto humano la composición de administración al SNC de la reivindicación 14.
ARP220100805A 2021-04-01 2022-03-31 Administración de ácido nucleico al sistema nervioso central AR125267A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163169539P 2021-04-01 2021-04-01

Publications (1)

Publication Number Publication Date
AR125267A1 true AR125267A1 (es) 2023-06-28

Family

ID=81384621

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220100805A AR125267A1 (es) 2021-04-01 2022-03-31 Administración de ácido nucleico al sistema nervioso central

Country Status (8)

Country Link
US (1) US20240167031A1 (es)
EP (1) EP4312977A1 (es)
JP (1) JP2024513403A (es)
CN (1) CN117337168A (es)
AR (1) AR125267A1 (es)
TW (1) TW202304473A (es)
UY (1) UY39713A (es)
WO (1) WO2022212648A1 (es)

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3937272A1 (de) 1989-11-09 1991-05-16 Boehringer Ingelheim Kg Neue copolymere aus trimethylencarbonat und optisch inaktiven laktiden
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5599797A (en) 1991-10-15 1997-02-04 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
TW244371B (es) 1992-07-23 1995-04-01 Tri Clover Inc
DE10200738A1 (de) 2002-01-11 2003-08-07 Boehringer Ingelheim Pharma Verfahren zur Herstellung von resorbierbaren Polyestern durch Massepolymerisation
US6706854B2 (en) 2002-01-11 2004-03-16 Boehringer Ingelheim Pharma Gmbh & Co. Kg Process for preparing reabsorbable polyesters by mass polymerization
DE102005033101A1 (de) 2005-07-15 2007-01-25 Boehringer Ingelheim Pharma Gmbh & Co. Kg Resorbierbare Polyetherester und ihre Verwendung zur Herstellung von medizinischen Implantaten
DE102007020951A1 (de) 2007-05-04 2008-11-06 Boehringer Ingelheim Pharma Gmbh & Co. Kg Verfahren und Vorrichtung zur Reinigung eines resorbierbaren Polyesters
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
EP3033113B1 (en) * 2013-08-13 2023-10-04 Baylor College of Medicine A novel plga-modified polyethylenimine self-assembly nanotechnology for nucleic acid and drug delivery
US10385341B2 (en) 2014-04-01 2019-08-20 Biogen Ma Inc. Compositions for modulating SOD-1 expression
US20180126014A1 (en) * 2015-04-15 2018-05-10 Yale University Compositions for enhancing delivery of agents across the blood brain barrier and methods of use thereof
BR112018003110A2 (pt) * 2015-08-21 2018-09-25 Pfizer nanopartículas terapêuticas compreendendo um agente terapêutico e métodos de fabricação e uso dos mesmos
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
US20220031630A1 (en) * 2018-09-13 2022-02-03 The Brigham And Women's Hospital, Inc. Nanoparticle formulations and methods of their use

Also Published As

Publication number Publication date
US20240167031A1 (en) 2024-05-23
EP4312977A1 (en) 2024-02-07
TW202304473A (zh) 2023-02-01
WO2022212648A1 (en) 2022-10-06
UY39713A (es) 2022-10-31
CN117337168A (zh) 2024-01-02
JP2024513403A (ja) 2024-03-25

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