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1. WO2017132530 - EXPANSION AND DIFFERENTIATION OF INNER EAR SUPPORTING CELLS AND METHODS OF USE THEREOF

Publication Number WO/2017/132530
Publication Date 03.08.2017
International Application No. PCT/US2017/015379
International Filing Date 27.01.2017
IPC
A61K 35/12 2015.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
A61P 27/16 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27Drugs for disorders of the senses
16Otologicals
C12N 5/071 2010.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07Animal cells or tissues
071Vertebrate cells or tissues, e.g. human cells or tissues
CPC
A61K 31/506
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
495having six-membered rings with two ; or more; nitrogen atoms as the only ring heteroatoms, e.g. piperazine ; or tetrazines
505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
506not condensed and containing further heterocyclic rings
A61K 31/519
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
495having six-membered rings with two ; or more; nitrogen atoms as the only ring heteroatoms, e.g. piperazine ; or tetrazines
505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
519ortho- or peri-condensed with heterocyclic rings
A61K 31/55
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
55having seven-membered rings, e.g. azelastine, pentylenetetrazole
A61K 35/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
A61P 27/16
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27Drugs for disorders of the senses
16Otologicals
A61P 43/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
Applicants
  • MASSACHUSETTS EYE AND EAR INFIRMARY [US]/[US]
  • DECIBEL THERAPEUTICS, INC. [US]/[US]
Inventors
  • EDGE, Albert
  • VENUTI, Michael
  • CZECHOWICZ, Agnieszka
Agents
  • DEYOUNG, Janice Kugler
  • ALPHA-COBB, Cambria J.
Priority Data
62/288,95829.01.2016US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) EXPANSION AND DIFFERENTIATION OF INNER EAR SUPPORTING CELLS AND METHODS OF USE THEREOF
(FR) EXPANSION ET DIFFÉRENCIATION DES CELLULES DE SUPPORT DE L'OREILLE INTERNE, ET MÉTHODES D'UTILISATION DE CES DERNIÈRES
Abstract
(EN) This disclosure relates to methods for expanding inner ear supporting cells (e.g., Lgr5+ inner ear supporting cells) and differentiating inner ear supporting cells (e.g., Lgr5+ inner ear supporting cells) to inner ear hair cells (e.g., atonal homolog 1 (Atoh1)+ inner ear hair cells) and the use of the inner hear supporting cells and hair cells, e.g., for identifying candidate therapeutic compounds for the treatment of hearing loss and balance loss. Additionally, the methods described herein can be used in the treatment of a subject having hearing loss and balance loss that would benefit from increased proliferation and differentiation of inner ear supporting cells (e.g., Lgr5+ inner ear supporting cells).
(FR) L'invention concerne des méthodes pour réaliser l'expansion de cellules de support de l'oreille interne (par exemple les cellules de support de l'oreille interne Lgr5+) et la différentiation de cellules de soutien de l'oreille interne (par exemple les cellules de support de l'oreille interne Lgr5+) en cellules ciliées de l'oreille interne (par exemple des cellules ciliées positives pour homolog atonal 1 (Atoh1+)), ainsi que l'utilisation des cellules de support et des cellules ciliées de l'oreille interne, par exemple pour identifier des composés thérapeutiques potentiels pour le traitement de la perte de l'audition et de la perte de l'équilibre. De plus, les méthodes de l'invention peuvent être utilisées dans le traitement de patients atteints de perte de l'audition et de perte de l'équilibre qui bénéficieraient d'une augmentation de la prolifération et la différenciation des cellules de soutien de l'oreille interne (par exemple des cellules de soutien de l'oreille interne Lgr5+).
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