MX2018002899A - Tratamiento de retinitis pigmentosa. - Google Patents
Tratamiento de retinitis pigmentosa.Info
- Publication number
- MX2018002899A MX2018002899A MX2018002899A MX2018002899A MX2018002899A MX 2018002899 A MX2018002899 A MX 2018002899A MX 2018002899 A MX2018002899 A MX 2018002899A MX 2018002899 A MX2018002899 A MX 2018002899A MX 2018002899 A MX2018002899 A MX 2018002899A
- Authority
- MX
- Mexico
- Prior art keywords
- retinitis pigmentosa
- treatment
- rpgrorf15
- nucleotide sequence
- encoding
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- 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
- C12N15/86—Viral vectors
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11014—Rhodopsin kinase (2.7.11.14)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
-
- 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
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Ophthalmology & Optometry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
- Pigments, Carbon Blacks, Or Wood Stains (AREA)
- Compounds Of Unknown Constitution (AREA)
Abstract
Un polinucleótido que comprende una secuencia de nucleótidos que codifica la isoforma de regulador ORF15 de GTPasa de retinitis pigmentosa (RPGRORF15), en donde la secuencia de nucleótidos que codifica RPGRORF15 ha sido optimizada en codones para aumentar la fidelidad de replicación de la secuencia.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
GBGB1516066.6A GB201516066D0 (en) | 2015-09-10 | 2015-09-10 | Treatment of retinitis pigmentosa |
PCT/GB2016/052802 WO2017042584A1 (en) | 2015-09-10 | 2016-09-09 | Treatment of retinitis pigmentosa |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2018002899A true MX2018002899A (es) | 2018-09-12 |
Family
ID=54362987
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2018002899A MX2018002899A (es) | 2015-09-10 | 2016-09-09 | Tratamiento de retinitis pigmentosa. |
MX2022003859A MX2022003859A (es) | 2015-09-10 | 2018-03-07 | Compuestos para usarse en el tratamiento o prevencion de retinitis pigmentosa. |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022003859A MX2022003859A (es) | 2015-09-10 | 2018-03-07 | Compuestos para usarse en el tratamiento o prevencion de retinitis pigmentosa. |
Country Status (14)
Country | Link |
---|---|
US (3) | US20180273594A1 (es) |
EP (1) | EP3347371B1 (es) |
JP (1) | JP7011580B2 (es) |
KR (1) | KR20180043373A (es) |
CN (1) | CN108699118A (es) |
AU (2) | AU2016320551C1 (es) |
CA (1) | CA2995656A1 (es) |
GB (1) | GB201516066D0 (es) |
HK (1) | HK1250727A1 (es) |
IL (1) | IL257881B (es) |
MX (2) | MX2018002899A (es) |
RU (2) | RU2020137429A (es) |
SG (1) | SG10202013246VA (es) |
WO (1) | WO2017042584A1 (es) |
Families Citing this family (19)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
BR122020011529B1 (pt) | 2014-02-12 | 2022-11-29 | Gyroscope Therapeutics Limited | Aparelho para a liberação de agente terapêutico em um olho |
US9925088B2 (en) | 2014-06-06 | 2018-03-27 | Janssen Biotech, Inc. | Sub-retinal tangential needle catheter guide and introducer |
US9949874B2 (en) | 2014-06-06 | 2018-04-24 | Janssen Biotech, Inc. | Therapeutic agent delivery device with convergent lumen |
US10219936B2 (en) | 2014-09-11 | 2019-03-05 | Orbit Biomedical Limited | Therapeutic agent delivery device with advanceable cannula and needle |
US10258502B2 (en) | 2014-09-18 | 2019-04-16 | Orbit Biomedical Limited | Therapeutic agent delivery device |
GB201516066D0 (en) * | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
US10478553B2 (en) | 2016-03-09 | 2019-11-19 | Orbit Biomedical Limited | Apparatus for subretinal administration of therapeutic agent via a curved needle |
US10646374B2 (en) | 2016-06-17 | 2020-05-12 | Orbit Biomedical Limited | Apparatus and method to form entry bleb for subretinal delivery of therapeutic agent |
US10806629B2 (en) | 2016-06-17 | 2020-10-20 | Gyroscope Therapeutics Limited | Injection device for subretinal delivery of therapeutic agent |
US11000410B2 (en) | 2016-06-17 | 2021-05-11 | Gyroscope Therapeutics Limited | Guide apparatus for tangential entry into suprachoroidal space |
US11273072B2 (en) | 2017-01-13 | 2022-03-15 | Gyroscope Therapeutics Limited | Suprachoroidal injection device |
US11076984B2 (en) | 2017-03-13 | 2021-08-03 | Gyroscope Therapeutics Limited | Method of performing subretinal drainage and agent delivery |
JOP20210054A1 (ar) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | تركيبات وطرق لعلاج التهاب الشبكية الصباغي |
JP2020174859A (ja) * | 2019-04-17 | 2020-10-29 | 学校法人日本医科大学 | 網膜剥離が生じるリスクが低い網膜変性症(rd)モデルマウスを取得する方法 |
GB202005641D0 (en) * | 2020-04-17 | 2020-06-03 | Ucl Business Plc | Gene therapy for bardet-biedl syndrome |
CN113952472A (zh) * | 2020-07-21 | 2022-01-21 | 英斯培瑞有限公司 | 用于治疗眼部疾病的组合物和方法 |
WO2022021149A1 (zh) * | 2020-07-29 | 2022-02-03 | 北京中因科技有限公司 | Aav介导的rpgr x连锁视网膜变性的基因编辑治疗 |
US20230338580A1 (en) * | 2020-08-14 | 2023-10-26 | Case Western Reserve University | Plasmid vectors and nanoparticles for treating ocular disorders |
AU2021337530B2 (en) | 2020-09-02 | 2023-08-24 | 4D Molecular Therapeutics Inc. | Codon Optimized RPGRorf 15 Genes And Uses Thereof |
Family Cites Families (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB0008801D0 (en) | 2000-04-10 | 2000-05-31 | Medical Res Council | Sequences |
US7799904B2 (en) * | 2003-06-13 | 2010-09-21 | University Of Kentucky Research Foundation | Gilvocarcin gene cluster, recombinant production and use thereof |
US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
FR2949115B1 (fr) * | 2009-08-14 | 2012-11-02 | Sanofi Aventis | OLIGOSACCHARIDES N-SULFATES ACTIVATEURS DES RECEPTEURS DES FGFs, LEUR PREPARATION ET LEUR APPLICATION EN THERAPEUTIQUE |
ES2362604B1 (es) | 2009-12-22 | 2012-06-28 | Bcn Peptides, S.A. | Formulación tópica oftálmica de péptidos. |
CN110777124A (zh) * | 2011-10-27 | 2020-02-11 | 威尔斯达眼科制剂公司 | 编码视杆细胞来源的视锥细胞活力因子的载体 |
DE18200782T1 (de) | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
EP2872183B1 (en) | 2012-07-11 | 2018-09-26 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
US9534225B2 (en) | 2014-04-15 | 2017-01-03 | Applied Genetic Technologies Corporation | Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (RPGR) |
GB201412011D0 (en) | 2014-07-04 | 2014-08-20 | Ucl Business Plc | Treatments |
HUE052781T2 (hu) | 2014-07-24 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | RPGR génterápia a retinitis pigmentosa terápiájára |
GB201516066D0 (en) * | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
-
2015
- 2015-09-10 GB GBGB1516066.6A patent/GB201516066D0/en not_active Ceased
-
2016
- 2016-09-09 WO PCT/GB2016/052802 patent/WO2017042584A1/en active Application Filing
- 2016-09-09 IL IL257881A patent/IL257881B/en unknown
- 2016-09-09 US US15/549,627 patent/US20180273594A1/en not_active Abandoned
- 2016-09-09 CN CN201680052007.5A patent/CN108699118A/zh active Pending
- 2016-09-09 EP EP16766067.9A patent/EP3347371B1/en active Active
- 2016-09-09 JP JP2018512918A patent/JP7011580B2/ja active Active
- 2016-09-09 AU AU2016320551A patent/AU2016320551C1/en active Active
- 2016-09-09 RU RU2020137429A patent/RU2020137429A/ru unknown
- 2016-09-09 KR KR1020187009860A patent/KR20180043373A/ko not_active Application Discontinuation
- 2016-09-09 CA CA2995656A patent/CA2995656A1/en active Pending
- 2016-09-09 SG SG10202013246VA patent/SG10202013246VA/en unknown
- 2016-09-09 MX MX2018002899A patent/MX2018002899A/es unknown
- 2016-09-09 RU RU2018112506A patent/RU2737049C2/ru active
-
2018
- 2018-03-07 MX MX2022003859A patent/MX2022003859A/es unknown
- 2018-08-03 HK HK18110029.4A patent/HK1250727A1/zh unknown
-
2019
- 2019-07-08 US US16/505,117 patent/US10836803B2/en active Active
-
2020
- 2020-10-07 US US17/065,003 patent/US20210130425A1/en active Pending
-
2022
- 2022-01-18 AU AU2022200306A patent/AU2022200306A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
RU2020137429A (ru) | 2022-01-17 |
GB201516066D0 (en) | 2015-10-28 |
US20210130425A1 (en) | 2021-05-06 |
RU2018112506A (ru) | 2019-10-10 |
KR20180043373A (ko) | 2018-04-27 |
US20180273594A1 (en) | 2018-09-27 |
RU2018112506A3 (es) | 2020-01-27 |
IL257881A (en) | 2018-05-31 |
CN108699118A (zh) | 2018-10-23 |
HK1250727A1 (zh) | 2019-01-11 |
EP3347371A1 (en) | 2018-07-18 |
WO2017042584A1 (en) | 2017-03-16 |
IL257881B (en) | 2022-12-01 |
AU2016320551B2 (en) | 2021-11-25 |
SG10202013246VA (en) | 2021-02-25 |
JP2018529336A (ja) | 2018-10-11 |
RU2737049C2 (ru) | 2020-11-24 |
EP3347371B1 (en) | 2023-05-10 |
US10836803B2 (en) | 2020-11-17 |
JP7011580B2 (ja) | 2022-01-26 |
US20200002392A1 (en) | 2020-01-02 |
MX2022003859A (es) | 2022-06-09 |
AU2022200306A1 (en) | 2022-02-10 |
CA2995656A1 (en) | 2017-03-16 |
AU2016320551A1 (en) | 2018-03-08 |
AU2016320551C1 (en) | 2023-12-14 |
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