CA3225436A1 - Methode de regulation de l'expression genique - Google Patents

Methode de regulation de l'expression genique Download PDF

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Publication number
CA3225436A1
CA3225436A1 CA3225436A CA3225436A CA3225436A1 CA 3225436 A1 CA3225436 A1 CA 3225436A1 CA 3225436 A CA3225436 A CA 3225436A CA 3225436 A CA3225436 A CA 3225436A CA 3225436 A1 CA3225436 A1 CA 3225436A1
Authority
CA
Canada
Prior art keywords
csf
catheter
pump
ventricle
patient
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3225436A
Other languages
English (en)
Inventor
Gianna RICCARDI
William X. Siopes, Jr.
Marcie GLICKMAN
Anthony DEPASQUA
Kevin KALISH
Joshua G. VOSE
Rajan Patel
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Enclear Therapies Inc
Original Assignee
Enclear Therapies 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 Enclear Therapies Inc filed Critical Enclear Therapies Inc
Publication of CA3225436A1 publication Critical patent/CA3225436A1/fr
Pending legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M27/00—Drainage appliance for wounds or the like, i.e. wound drains, implanted drains
    • A61M27/002—Implant devices for drainage of body fluids from one part of the body to another
    • A61M27/006—Cerebrospinal drainage; Accessories therefor, e.g. valves
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/14—Infusion devices, e.g. infusing by gravity; Blood infusion; Accessories therefor
    • A61M5/168—Means for controlling media flow to the body or for metering media to the body, e.g. drip meters, counters ; Monitoring media flow to the body
    • A61M5/172—Means for controlling media flow to the body or for metering media to the body, e.g. drip meters, counters ; Monitoring media flow to the body electrical or electronic
    • A61M5/1723—Means for controlling media flow to the body or for metering media to the body, e.g. drip meters, counters ; Monitoring media flow to the body electrical or electronic using feedback of body parameters, e.g. blood-sugar, pressure
    • 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
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2202/00—Special media to be introduced, removed or treated
    • A61M2202/04—Liquids
    • A61M2202/0464—Cerebrospinal fluid
    • 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
    • A61P25/16—Anti-Parkinson drugs
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Anesthesiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Vascular Medicine (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Otolaryngology (AREA)
  • Infusion, Injection, And Reservoir Apparatuses (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

L'invention concerne une méthode de régulation de l'expression génique d'un patient par la circulation contrôlée d'une matière d'oligonucléotide antisens (ASO) à travers un circuit de fluide fermé formé entre le ventricule du patient et les régions lombaires. À cet effet, après le couplage d'un canal de fluide entre ces régions, de tels modes de réalisation ajoutent de l'ASO au canal de fluide (de préférence après que le canal a été amorcé avec du CSF) et activent une pompe pour faire circuler de manière contrôlée le CSF et l'ASO mélangé au CSF. Le flux de fluide CSF/ASO peut être géré pour localiser un traitement (par exemple, fournir une distribution cérébrale profonde) tout en réduisant au minimum la toxicité potentiellement provoquée par l'ASO vers certains nerfs (par exemple, le nerf périphérique).
CA3225436A 2021-06-23 2022-06-23 Methode de regulation de l'expression genique Pending CA3225436A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163214239P 2021-06-23 2021-06-23
US63/214,239 2021-06-23
PCT/US2022/034706 WO2022271938A1 (fr) 2021-06-23 2022-06-23 Méthode de régulation de l'expression génique

Publications (1)

Publication Number Publication Date
CA3225436A1 true CA3225436A1 (fr) 2022-12-29

Family

ID=84544860

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3225436A Pending CA3225436A1 (fr) 2021-06-23 2022-06-23 Methode de regulation de l'expression genique

Country Status (4)

Country Link
EP (1) EP4359054A4 (fr)
AU (1) AU2022297513A1 (fr)
CA (1) CA3225436A1 (fr)
WO (1) WO2022271938A1 (fr)

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6197300B1 (en) * 1997-08-12 2001-03-06 Smithkline Beecham Corporation ftsZ
WO1999013886A1 (fr) * 1997-09-17 1999-03-25 East Carolina University Acides nucleiques s'hybridant a des cibles multiples, leur preparation, compositions, formulation, trousses et applications
EP1135113B1 (fr) * 1998-11-05 2008-10-01 Thomas Jefferson University Traitement de la maladie de parkinson avec des oligonucleotides
CA2985347A1 (fr) 2015-05-11 2016-11-17 Alcyone Lifesciences, Inc. Systemes et procedes d'administration de medicaments
WO2017117138A1 (fr) * 2015-12-28 2017-07-06 Cognost Therapeutics Inc. Appareil et procédé de micro-dialyse cérébrale pour le traitement de maladie neurologique, notamment la maladie d'alzheimer, la maladie de parkinson ou la sclérose en plaques
EP3411479A4 (fr) * 2016-02-04 2019-09-18 Zhenglun Zhu Traitement et diagnostic de troubles inflammatoires
JP2022526671A (ja) 2019-04-11 2022-05-25 エンクリアー セラピーズ， インク． 脳脊髄液の改善の方法ならびにそのためのデバイスおよびシステム
JP2023543868A (ja) * 2020-09-29 2023-10-18 エンクリアー セラピーズ， インク． くも膜下液管理方法およびシステム

Also Published As

Publication number Publication date
WO2022271938A1 (fr) 2022-12-29
EP4359054A1 (fr) 2024-05-01
AU2022297513A1 (en) 2024-01-18
WO2022271938A9 (fr) 2023-12-07
EP4359054A4 (fr) 2025-06-25

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