UY38427A - Métodos y composiciones para terapia con células oculares - Google Patents

Métodos y composiciones para terapia con células oculares

Info

Publication number
UY38427A
UY38427A UY0001038427A UY38427A UY38427A UY 38427 A UY38427 A UY 38427A UY 0001038427 A UY0001038427 A UY 0001038427A UY 38427 A UY38427 A UY 38427A UY 38427 A UY38427 A UY 38427A
Authority
UY
Uruguay
Prior art keywords
cells
methods
cell therapy
ocular
compositions
Prior art date
Application number
UY0001038427A
Other languages
English (en)
Inventor
Jessica Heyder
Arnaud Lacoste
Xueshi Hao
Timothy Z Hoffman
Qihui Jin
Jun Liu
Yahu A Liu
Tingting Mo
Jianfeng Pan
Yun Feng Xie
Shanshan Yan
Yefen Zou
Frada Berenshteyn
Bo Han
Bradley Andrew Murray
Daniel Joseph O'connell
Original Assignee
Novartis Ag
Intellia 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 Novartis Ag, Intellia Therapeutics Inc filed Critical Novartis Ag
Publication of UY38427A publication Critical patent/UY38427A/es

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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0623—Stem cells
    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4375—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having nitrogen as a ring heteroatom, e.g. quinolizines, naphthyridines, berberine, vincamine
    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/472—Non-condensed isoquinolines, e.g. papaverine
    • A61K31/4725—Non-condensed isoquinolines, e.g. papaverine containing further 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
    • A61K31/33—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
    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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    • 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
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    • C12N15/09—Recombinant DNA-technology
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70—Enzymes
    • C12N2501/72—Transferases [EC 2.]
    • C12N2501/727—Kinases (EC 2.7.)
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    • C12N2502/00—Coculture with; Conditioned medium produced by
    • C12N2502/08—Coculture with; Conditioned medium produced by cells of the nervous system
    • C12N2502/085—Coculture with; Conditioned medium produced by cells of the nervous system eye cells
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente invención provee células oculares, genéticamente modificadas por un sistema CRISPR dirigido a la expresión de B2M para terapia celular ocular. La invención provee además métodos para generar una población expandida de células oculares genéticamente modificadas, por ejemplo, células madre limbares (LSC) o células endoteliales corneales (CEC), en donde las células se expanden con el uso de un inhibidor de LATS y la expresión de B2M en las células se ha reducido o eliminado. La presente invención además provee poblaciones celulares, preparaciones, usos y métodos de terapia que comprenden dichas células.
UY0001038427A 2018-10-26 2019-10-23 Métodos y composiciones para terapia con células oculares UY38427A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862750962P 2018-10-26 2018-10-26
US201962902639P 2019-09-19 2019-09-19

Publications (1)

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UY38427A true UY38427A (es) 2020-05-29

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UY0001038427A UY38427A (es) 2018-10-26 2019-10-23 Métodos y composiciones para terapia con células oculares

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US (1) US20200131474A1 (es)
EP (1) EP3870289A1 (es)
JP (1) JP2022505658A (es)
KR (1) KR20210069075A (es)
CN (1) CN112969472A (es)
AU (1) AU2019365590A1 (es)
BR (1) BR112021007628A2 (es)
CA (1) CA3116512A1 (es)
CL (1) CL2021001034A1 (es)
CO (1) CO2021005289A2 (es)
CR (1) CR20210196A (es)
CU (1) CU20210033A7 (es)
EC (1) ECSP21028556A (es)
IL (1) IL282447A (es)
JO (1) JOP20210080A1 (es)
MX (1) MX2021004532A (es)
PE (1) PE20211114A1 (es)
PH (1) PH12021550761A1 (es)
SG (1) SG11202102615RA (es)
TW (1) TW202030324A (es)
UY (1) UY38427A (es)
WO (1) WO2020084580A1 (es)

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CO2021005289A2 (es) 2021-05-10
CR20210196A (es) 2021-07-27
ECSP21028556A (es) 2021-05-31
BR112021007628A2 (pt) 2021-10-13
PH12021550761A1 (en) 2021-12-13
JOP20210080A1 (ar) 2023-01-30
SG11202102615RA (en) 2021-05-28
US20200131474A1 (en) 2020-04-30
CL2021001034A1 (es) 2021-11-19
IL282447A (en) 2021-06-30
CN112969472A (zh) 2021-06-15
TW202030324A (zh) 2020-08-16
JP2022505658A (ja) 2022-01-14
AU2019365590A1 (en) 2021-04-22
MX2021004532A (es) 2021-07-16
EP3870289A1 (en) 2021-09-01
CU20210033A7 (es) 2021-12-08
WO2020084580A1 (en) 2020-04-30
CA3116512A1 (en) 2020-04-30
PE20211114A1 (es) 2021-06-22

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