CL2021001034A1 - Métodos y composiciones para terapia con células oculares - Google Patents
Métodos y composiciones para terapia con células ocularesInfo
- 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
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- C—CHEMISTRY; METALLURGY
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- 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
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- 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
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- 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
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- 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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- A—HUMAN NECESSITIES
- 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
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- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- 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
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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
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- 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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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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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
- C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/999—Small molecules not provided for elsewhere
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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.
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)
Publication Number | Publication Date |
---|---|
CL2021001034A1 true CL2021001034A1 (es) | 2021-11-19 |
Family
ID=68393038
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2021001034A CL2021001034A1 (es) | 2018-10-26 | 2021-04-22 | Métodos y composiciones para terapia con células oculares |
Country Status (22)
Country | Link |
---|---|
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) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
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EA201992232A1 (ru) | 2017-03-22 | 2020-05-14 | Новартис Аг | Композиции и способы для иммуноонкологии |
JOP20190257A1 (ar) * | 2017-04-28 | 2019-10-28 | Novartis Ag | مركبات أريل غير متجانسة ثنائية الحلقة مندمجة 6-6 واستخدامها كمثبطات lats |
US20230000762A1 (en) * | 2019-12-03 | 2023-01-05 | The Schepens Eye Research Institute, Inc. | Injectable hydrogels for cell delivery to the vitreous |
JP2023522784A (ja) * | 2020-04-27 | 2023-05-31 | ノバルティス アーゲー | 眼細胞療法のための方法及び組成物 |
TW202309271A (zh) * | 2021-04-20 | 2023-03-01 | 美商步行魚治療公司 | 細胞群、工程化b細胞及其產生方法與用途、基因體編輯系統以及核酸構築體 |
CA3225082A1 (en) * | 2021-08-27 | 2023-03-02 | Brian C. Thomas | Enzymes with ruvc domains |
CN116286905B (zh) * | 2023-05-11 | 2023-08-15 | 内蒙古大学 | 牛源化CRISPR/boCas9基因编辑系统、方法及应用 |
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2019
- 2019-10-23 UY UY0001038427A patent/UY38427A/es unknown
- 2019-10-24 TW TW108138491A patent/TW202030324A/zh unknown
- 2019-10-25 US US16/663,722 patent/US20200131474A1/en not_active Abandoned
- 2019-10-25 CN CN201980069687.5A patent/CN112969472A/zh active Pending
- 2019-10-25 AU AU2019365590A patent/AU2019365590A1/en not_active Abandoned
- 2019-10-25 CR CR20210196A patent/CR20210196A/es unknown
- 2019-10-25 CU CU2021000033A patent/CU20210033A7/es unknown
- 2019-10-25 EP EP19795669.1A patent/EP3870289A1/en not_active Withdrawn
- 2019-10-25 CA CA3116512A patent/CA3116512A1/en active Pending
- 2019-10-25 BR BR112021007628-0A patent/BR112021007628A2/pt unknown
- 2019-10-25 MX MX2021004532A patent/MX2021004532A/es unknown
- 2019-10-25 KR KR1020217012812A patent/KR20210069075A/ko not_active Application Discontinuation
- 2019-10-25 PE PE2021000558A patent/PE20211114A1/es unknown
- 2019-10-25 JP JP2021522072A patent/JP2022505658A/ja active Pending
- 2019-10-25 WO PCT/IB2019/059162 patent/WO2020084580A1/en unknown
- 2019-10-25 SG SG11202102615RA patent/SG11202102615RA/en unknown
- 2019-10-25 JO JOP/2021/0080A patent/JOP20210080A1/ar unknown
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2021
- 2021-04-06 PH PH12021550761A patent/PH12021550761A1/en unknown
- 2021-04-19 IL IL282447A patent/IL282447A/en unknown
- 2021-04-22 EC ECSENADI202128556A patent/ECSP21028556A/es unknown
- 2021-04-22 CL CL2021001034A patent/CL2021001034A1/es unknown
- 2021-04-23 CO CONC2021/0005289A patent/CO2021005289A2/es unknown
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SG11202102615RA (en) | 2021-05-28 |
ECSP21028556A (es) | 2021-05-31 |
EP3870289A1 (en) | 2021-09-01 |
CO2021005289A2 (es) | 2021-05-10 |
US20200131474A1 (en) | 2020-04-30 |
IL282447A (en) | 2021-06-30 |
UY38427A (es) | 2020-05-29 |
KR20210069075A (ko) | 2021-06-10 |
WO2020084580A1 (en) | 2020-04-30 |
CA3116512A1 (en) | 2020-04-30 |
AU2019365590A1 (en) | 2021-04-22 |
MX2021004532A (es) | 2021-07-16 |
CR20210196A (es) | 2021-07-27 |
JP2022505658A (ja) | 2022-01-14 |
CU20210033A7 (es) | 2021-12-08 |
PH12021550761A1 (en) | 2021-12-13 |
PE20211114A1 (es) | 2021-06-22 |
CN112969472A (zh) | 2021-06-15 |
JOP20210080A1 (ar) | 2023-01-30 |
BR112021007628A2 (pt) | 2021-10-13 |
TW202030324A (zh) | 2020-08-16 |
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