CL2021001034A1 - Métodos y composiciones para terapia con células oculares - Google Patents

Métodos y composiciones para terapia con células oculares

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Publication number
CL2021001034A1
CL2021001034A1 CL2021001034A CL2021001034A CL2021001034A1 CL 2021001034 A1 CL2021001034 A1 CL 2021001034A1 CL 2021001034 A CL2021001034 A CL 2021001034A CL 2021001034 A CL2021001034 A CL 2021001034A CL 2021001034 A1 CL2021001034 A1 CL 2021001034A1
Authority
CL
Chile
Prior art keywords
cells
methods
ocular
cell therapy
compositions
Prior art date
Application number
CL2021001034A
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
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.)
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Publication date
Application filed by Novartis Ag, Intellia Therapeutics Inc filed Critical Novartis Ag
Publication of CL2021001034A1 publication Critical patent/CL2021001034A1/es

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    • 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
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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
    • A61K31/4375Heterocyclic 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
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    • A61K31/435Heterocyclic 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
    • A61K31/47Quinolines; Isoquinolines
    • A61K31/472Non-condensed isoquinolines, e.g. papaverine
    • A61K31/4725Non-condensed isoquinolines, e.g. papaverine containing further heterocyclic rings
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    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
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    • C12N2310/00Structure or type of the nucleic acid
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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.
CL2021001034A 2018-10-26 2021-04-22 Métodos y composiciones para terapia con células oculares CL2021001034A1 (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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CL2021001034A1 true CL2021001034A1 (es) 2021-11-19

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CL2021001034A CL2021001034A1 (es) 2018-10-26 2021-04-22 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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JOP20190257A1 (ar) * 2017-04-28 2019-10-28 Novartis Ag مركبات أريل غير متجانسة ثنائية الحلقة مندمجة 6-6 واستخدامها كمثبطات lats
CN115671398B (zh) * 2022-11-22 2024-03-01 首都医科大学附属北京同仁医院 一种3d打印仿生角膜缘移植物及其制备方法和用途

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