CA3066107A1 - Combination therapies for inner ear sensory hair cell regeneration/replacement - Google Patents

Combination therapies for inner ear sensory hair cell regeneration/replacement Download PDF

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Publication number
CA3066107A1
CA3066107A1 CA3066107A CA3066107A CA3066107A1 CA 3066107 A1 CA3066107 A1 CA 3066107A1 CA 3066107 A CA3066107 A CA 3066107A CA 3066107 A CA3066107 A CA 3066107A CA 3066107 A1 CA3066107 A1 CA 3066107A1
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Prior art keywords
inner ear
tissue
expression
gene
plga
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Pending
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CA3066107A
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English (en)
French (fr)
Inventor
Matthew B. WEST
Richard D. Kopke
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Hough Ear Institute
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Hough Ear Institute
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/513Organic macromolecular compounds; Dendrimers
    • A61K9/5146Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A61K9/5153Polyesters, e.g. poly(lactide-co-glycolide)
    • 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
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/403Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with carbocyclic rings, e.g. carbazole
    • A61K31/404Indoles, e.g. pindolol
    • 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
    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/433Thidiazoles
    • 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
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/1825Fibroblast growth factor [FGF]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0002Galenical forms characterised by the drug release technique; Application systems commanded by energy
    • A61K9/0009Galenical forms characterised by the drug release technique; Application systems commanded by energy involving or responsive to electricity, magnetism or acoustic waves; Galenical aspects of sonophoresis, iontophoresis, electroporation or electroosmosis
    • 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
    • A61K9/0046Ear
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605Excipients; Inactive ingredients
    • A61K9/1629Organic macromolecular compounds
    • A61K9/1641Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poloxamers
    • A61K9/1647Polyesters, e.g. poly(lactide-co-glycolide)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/16Otologicals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • 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
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    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3517Marker; Tag
    • 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
    • C12N2320/31Combination therapy
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CA3066107A 2016-06-03 2017-06-02 Combination therapies for inner ear sensory hair cell regeneration/replacement Pending CA3066107A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662345740P 2016-06-03 2016-06-03
US62/345,740 2016-06-03
PCT/US2017/035673 WO2017210553A1 (en) 2016-06-03 2017-06-02 Combination therapies for inner ear sensory hair cell regeneration/replacement

Publications (1)

Publication Number Publication Date
CA3066107A1 true CA3066107A1 (en) 2017-12-07

Family

ID=60477923

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3066107A Pending CA3066107A1 (en) 2016-06-03 2017-06-02 Combination therapies for inner ear sensory hair cell regeneration/replacement

Country Status (6)

Country Link
US (2) US11013752B2 (cg-RX-API-DMAC7.html)
EP (1) EP3463417A4 (cg-RX-API-DMAC7.html)
JP (2) JP7523209B2 (cg-RX-API-DMAC7.html)
CN (2) CN109562140B (cg-RX-API-DMAC7.html)
CA (1) CA3066107A1 (cg-RX-API-DMAC7.html)
WO (1) WO2017210553A1 (cg-RX-API-DMAC7.html)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020254249A1 (en) * 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
JP2023106639A (ja) * 2020-06-09 2023-08-02 株式会社オトリンク 内耳前駆細胞の製造方法、内耳有毛細胞の製造方法、薬剤の評価方法、及び内耳細胞分化誘導用組成物
CN114736924B (zh) * 2021-01-07 2024-11-22 中国科学院脑科学与智能技术卓越创新中心 异位联合过表达Atoh1和Ikzf2再生耳蜗外毛细胞及其应用
CN116440249A (zh) * 2022-01-05 2023-07-18 中国科学院脑科学与智能技术卓越创新中心 异位联合表达Atoh1和Tbx2基因促进内毛细胞再生及其应用

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US6593290B1 (en) * 1996-11-01 2003-07-15 Genentech, Inc. Treatment of inner ear hair cells
US6156728A (en) 1996-11-01 2000-12-05 Genentech, Inc. Treatment of inner ear hair cells
CA2493289C (en) 2002-07-31 2014-12-16 Phafag Ag A pharmaceutical formulation and its use in the treatment of inner ear diseases
US20060030042A1 (en) * 2003-12-19 2006-02-09 Ali Brivanlou Maintenance of embryonic stem cells by the GSK-3 inhibitor 6-bromoindirubin-3'-oxime
EP1838852A2 (en) * 2004-11-12 2007-10-03 Asuragen, Inc. Methods and compositions involving mirna and mirna inhibitor molecules
CA2725953A1 (en) * 2007-05-29 2008-12-11 Christopher B. Reid Methods for production and uses of multipotent cell populations, pluripotent cell populations, neuronal cell populations, and muscle cell populations
WO2010060088A2 (en) * 2008-11-24 2010-05-27 Massachusetts Eye & Ear Infirmary Pathways to generate hair cells
CN101732723B (zh) * 2009-12-30 2012-07-25 上海市肿瘤研究所 聚乙二醇-聚乳酸羟基乙酸-聚赖氨酸纳米递送系统、制备方法及其应用
US9101647B2 (en) * 2010-06-04 2015-08-11 Hough Ear Institute Composition and method for inner ear sensory hair cell regeneration and replacement
WO2012069428A1 (en) 2010-11-22 2012-05-31 Noscira, S.A. Bipyridine sulfonamide derivatives for the treatment of neurodegenerative diseases or conditions
US20150018404A1 (en) * 2011-08-03 2015-01-15 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide compounds for treating hearing and balance disorders
EP2609918A1 (en) * 2011-12-27 2013-07-03 Zytoprotec GmbH Peritoneal dialysis fluid comprising a GSK-3 inhibitor
US20150209406A1 (en) * 2012-09-07 2015-07-30 Massachusetts Eye And Ear Infirmary Methods and compositions for regenerating hair cells and/or supporting cells
PT3702443T (pt) 2013-03-14 2022-02-17 Massachusetts Inst Technology Composições e métodos para expansão e cultura de células estaminais epiteliais
US20150126507A1 (en) * 2013-09-05 2015-05-07 Fate Therapeutics, Inc. Compounds to treat hearing loss
CA2959404A1 (en) * 2014-09-03 2016-03-10 The Brigham And Women's Hospital, Inc. Compositions, systems, and methods for generating inner ear hair cells for treatment of hearing loss
EP3212773B1 (en) 2014-10-29 2021-09-15 Massachusetts Eye and Ear Infirmary Efficient delivery of therapeutic molecules to cells of the inner ear
WO2017132530A1 (en) * 2016-01-29 2017-08-03 Massachusetts Eye And Ear Infirmary Expansion and differentiation of inner ear supporting cells and methods of use thereof
US20190142969A1 (en) 2016-04-26 2019-05-16 Massachusetts Eye And Ear Infirmary ISL1-Based Gene Therapy to Treat Hearing Loss
CN111655228B (zh) 2017-05-03 2024-02-09 听治疗有限责任公司 预防和治疗听力损失的组合物和方法
WO2019053727A1 (en) 2017-09-18 2019-03-21 Ramot At Tel-Aviv University Ltd. REDIFFERENCING IN VITRO DEVELOPED CELLS FROM HUMAN PANCREATIC ISLAND BEA CELLS ADULPTED BY SMALL MOLECULE INHIBITORS OF THE SIGNALING PATH
EP3462349A1 (en) 2017-10-02 2019-04-03 Koninklijke Philips N.V. Assessment of notch cellular signaling pathway activity using mathematical modelling of target gene expression

Also Published As

Publication number Publication date
US20210252038A1 (en) 2021-08-19
EP3463417A4 (en) 2020-04-01
JP2022180476A (ja) 2022-12-06
JP7523209B2 (ja) 2024-07-26
CN109562140A (zh) 2019-04-02
CN109562140B (zh) 2023-01-03
WO2017210553A1 (en) 2017-12-07
US11013752B2 (en) 2021-05-25
US20170348346A1 (en) 2017-12-07
EP3463417A1 (en) 2019-04-10
JP2019521101A (ja) 2019-07-25
CN115814097A (zh) 2023-03-21

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