MX2021002982A - Composiciones y metodos para tratar retinitis pigmentosa. - Google Patents

Composiciones y metodos para tratar retinitis pigmentosa.

Info

Publication number
MX2021002982A
MX2021002982A MX2021002982A MX2021002982A MX2021002982A MX 2021002982 A MX2021002982 A MX 2021002982A MX 2021002982 A MX2021002982 A MX 2021002982A MX 2021002982 A MX2021002982 A MX 2021002982A MX 2021002982 A MX2021002982 A MX 2021002982A
Authority
MX
Mexico
Prior art keywords
compositions
methods
retinitis pigmentosa
treating retinitis
treating
Prior art date
Application number
MX2021002982A
Other languages
English (en)
Inventor
Gregory S Robinson
Tuyen Ong
Original Assignee
Nightstarx Ltd
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 Nightstarx Ltd filed Critical Nightstarx Ltd
Publication of MX2021002982A publication Critical patent/MX2021002982A/es

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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
    • C12N15/86—Viral vectors
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • 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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays, needleless injectors
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    • A61K9/0048—Eye, e.g. artificial tears
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    • 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
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Mycology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Dermatology (AREA)
  • Inorganic Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invención se refiere a composiciones y métodos para el tratamiento de Retinitis Pigmentosa a través de la administración de un vector de rAAV que comprende una secuencia de RPGRORF15.
MX2021002982A 2018-09-21 2019-09-23 Composiciones y metodos para tratar retinitis pigmentosa. MX2021002982A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862734746P 2018-09-21 2018-09-21
US201962830106P 2019-04-05 2019-04-05
PCT/US2019/052471 WO2020061574A1 (en) 2018-09-21 2019-09-23 Compositions and methods for treating retinitis pigmentosa

Publications (1)

Publication Number Publication Date
MX2021002982A true MX2021002982A (es) 2021-08-11

Family

ID=69887995

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021002982A MX2021002982A (es) 2018-09-21 2019-09-23 Composiciones y metodos para tratar retinitis pigmentosa.

Country Status (19)

Country Link
US (1) US20210348193A1 (es)
EP (1) EP3852877A4 (es)
JP (1) JP2022501376A (es)
KR (1) KR20210094518A (es)
CN (1) CN113382770A (es)
AU (1) AU2019345330A1 (es)
BR (1) BR112021004830A2 (es)
CA (1) CA3112821A1 (es)
CL (1) CL2021000689A1 (es)
CO (1) CO2021004934A2 (es)
CR (1) CR20210187A (es)
IL (1) IL281577A (es)
JO (1) JOP20210054A1 (es)
MX (1) MX2021002982A (es)
PE (1) PE20220747A1 (es)
PH (1) PH12021550594A1 (es)
SG (1) SG11202102800YA (es)
TW (1) TW202028225A (es)
WO (1) WO2020061574A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2021337530B2 (en) 2020-09-02 2023-08-24 4D Molecular Therapeutics Inc. Codon Optimized RPGRorf 15 Genes And Uses Thereof
WO2023199230A1 (en) * 2022-04-13 2023-10-19 Notal Vision Ltd. Oct guided therapy
CN117625619A (zh) * 2023-12-05 2024-03-01 云舟生物科技(广州)股份有限公司 核酸分子及其作为特异启动子的应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016145345A1 (en) * 2015-03-11 2016-09-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 and rpgr vectors for treating x-linked retinitis pigmentosa
GB201516066D0 (en) * 2015-09-10 2015-10-28 Young & Co Llp D Treatment of retinitis pigmentosa
CA3019425A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia a
GB201704192D0 (en) * 2017-03-16 2017-05-03 Nightstarx Ltd Treatment of Retinitis Pigmentosa

Also Published As

Publication number Publication date
PE20220747A1 (es) 2022-05-10
PH12021550594A1 (en) 2021-11-22
SG11202102800YA (en) 2021-04-29
JOP20210054A1 (ar) 2020-03-21
US20210348193A1 (en) 2021-11-11
CR20210187A (es) 2021-09-02
AU2019345330A1 (en) 2021-04-29
CA3112821A1 (en) 2020-03-26
EP3852877A4 (en) 2022-07-13
WO2020061574A1 (en) 2020-03-26
EP3852877A1 (en) 2021-07-28
BR112021004830A2 (pt) 2021-06-08
CN113382770A (zh) 2021-09-10
TW202028225A (zh) 2020-08-01
CO2021004934A2 (es) 2021-07-30
KR20210094518A (ko) 2021-07-29
JP2022501376A (ja) 2022-01-06
IL281577A (en) 2021-05-31
CL2021000689A1 (es) 2021-10-08

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