CU20210033A7 - Métodos y composiciones para terapia con células oculares - Google Patents

Métodos y composiciones para terapia con células oculares

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Publication number
CU20210033A7
CU20210033A7 CU2021000033A CU20210033A CU20210033A7 CU 20210033 A7 CU20210033 A7 CU 20210033A7 CU 2021000033 A CU2021000033 A CU 2021000033A CU 20210033 A CU20210033 A CU 20210033A CU 20210033 A7 CU20210033 A7 CU 20210033A7
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CU
Cuba
Prior art keywords
cells
methods
cell therapy
ocular
compositions
Prior art date
Application number
CU2021000033A
Other languages
English (en)
Inventor
Frada Berenshteyn
Bo Han
Xueshi Hao
Jessica Heyder
Timothy Z Hoffman
Qihui Jin
Arnaud Lacoste
Jun Liu
Yahu Liu
Tingting Mo
Bradley Andrew Murray
Daniel Joseph O'connell
Jianfeng Pan
Yun Feng Xie
Shanshan Yan
Yefen Zou
Original Assignee
Intellia Therapeutics Inc
Novartis Ag
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 Intellia Therapeutics Inc, Novartis Ag filed Critical Intellia Therapeutics Inc
Publication of CU20210033A7 publication Critical patent/CU20210033A7/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
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    • 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
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    • 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
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    • 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
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    • C12N5/0602—Vertebrate 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 [RNase]; Deoxyribonucleases [DNase]
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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 [CRISPR]
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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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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Abstract

<p>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 madres 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.</p>
CU2021000033A 2018-10-26 2019-10-25 Métodos y composiciones para terapia con células oculares CU20210033A7 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862750962P 2018-10-26 2018-10-26
US201962902639P 2019-09-19 2019-09-19
PCT/IB2019/059162 WO2020084580A1 (en) 2018-10-26 2019-10-25 Methods and compositions for ocular cell therapy

Publications (1)

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CU20210033A7 true CU20210033A7 (es) 2021-12-08

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CU2021000033A CU20210033A7 (es) 2018-10-26 2019-10-25 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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