JOP20200253A1 - تركيبات وطرق لعلاج الحثل البقعي - Google Patents

تركيبات وطرق لعلاج الحثل البقعي

Info

Publication number
JOP20200253A1
JOP20200253A1 JOP/2020/0253A JOP20200253A JOP20200253A1 JO P20200253 A1 JOP20200253 A1 JO P20200253A1 JO P20200253 A JOP20200253 A JO P20200253A JO P20200253 A1 JOP20200253 A1 JO P20200253A1
Authority
JO
Jordan
Prior art keywords
macular dystrophy
compositions
methods
treating macular
sequence encoding
Prior art date
Application number
JOP/2020/0253A
Other languages
English (en)
Inventor
Gregory S Robinson
De La Camara Cristina Martinez-Fernandez
Robert Maclaren
Original Assignee
Univ Oxford Innovation Ltd
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 Univ Oxford Innovation Ltd, Nightstarx Ltd filed Critical Univ Oxford Innovation Ltd
Publication of JOP20200253A1 publication Critical patent/JOP20200253A1/ar

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Classifications

    • 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
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • 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
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

Landscapes

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

Abstract

يوفر الكشف تركيبة تشتمل على متوالية حمض نووي تشتمل على (أ) متوالية تُرمِّز معزز حثل بقعي محي الشكل-2 (VMD2)، و(ب) متوالية تُرمِّز بروتين بيستروفين -1 (BEST1) إلى جانب استخدام تلك المركبات لعلاج الحثل البقعي في خاضع للعلاج تشتمل على إعطاء التركيبة إلى عين خاضع للعلاج من خلال نمط تحت الشبكية أو فوق المشيمية.
JOP/2020/0253A 2018-04-05 2019-04-05 تركيبات وطرق لعلاج الحثل البقعي JOP20200253A1 (ar)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862653131P 2018-04-05 2018-04-05
PCT/US2019/026062 WO2019195727A1 (en) 2018-04-05 2019-04-05 Compositions and methods for treating macular dystrophy

Publications (1)

Publication Number Publication Date
JOP20200253A1 true JOP20200253A1 (ar) 2020-10-04

Family

ID=66223872

Family Applications (1)

Application Number Title Priority Date Filing Date
JOP/2020/0253A JOP20200253A1 (ar) 2018-04-05 2019-04-05 تركيبات وطرق لعلاج الحثل البقعي

Country Status (21)

Country Link
US (2) US20190307900A1 (ar)
EP (1) EP3775233A1 (ar)
JP (1) JP2021520232A (ar)
KR (1) KR20210005040A (ar)
CN (1) CN113056561A (ar)
AU (1) AU2019247864A1 (ar)
BR (1) BR112020020204A2 (ar)
CA (1) CA3096088A1 (ar)
CL (1) CL2020002561A1 (ar)
CO (1) CO2020013690A2 (ar)
EA (1) EA202092069A1 (ar)
IL (1) IL277779A (ar)
JO (1) JOP20200253A1 (ar)
MA (1) MA52199A (ar)
MX (1) MX2020010477A (ar)
PE (1) PE20210918A1 (ar)
PH (1) PH12020551641A1 (ar)
RU (1) RU2020132890A (ar)
SG (1) SG11202009759SA (ar)
TW (1) TW202003052A (ar)
WO (1) WO2019195727A1 (ar)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022511348A (ja) * 2018-10-25 2022-01-31 バクスアルタ インコーポレイテッド Ａａｖトリプルプラスミドシステム
US20220089670A1 (en) * 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
WO2021174173A1 (en) * 2020-02-28 2021-09-02 The Trustees Of The University Of Pennsylvania Treating autosomal dominant bestrophinopathies and methods for evaluating same
CN111849998A (zh) * 2020-07-29 2020-10-30 武汉纽福斯生物科技有限公司 编码人卵黄状黄斑病蛋白1的核酸分子及其应用

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5615271B2 (ja) * 2008-06-18 2014-10-29 オックスフォード バイオメディカ（ユーケー）リミテッド ウイルス精製法
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
EP3071592B1 (en) * 2013-11-20 2021-01-06 Fondazione Telethon Artificial dna-binding proteins and uses thereof
AU2014359136B2 (en) * 2013-12-06 2020-06-25 Centre Hospitalier Universitaire De Montpellier Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
GB201322798D0 (en) * 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
BR112017018728A2 (pt) * 2015-03-03 2018-04-17 Fond Telethon sistema de vetor para expressar a sequência de codificação de um gene de interesse em uma célula, célula hospedeira, composição farmacêutica, método para tratar e/ou prevenir uma patologia ou doença, uso de uma sequência de nucleotídeo de um sinal de degradação em um sistema de vetor e método para diminuir a expressão de uma proteína na forma truncada
WO2017083722A1 (en) * 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy

Also Published As

Publication number Publication date
WO2019195727A1 (en) 2019-10-10
PH12020551641A1 (en) 2021-07-26
EA202092069A1 (ru) 2021-03-12
JP2021520232A (ja) 2021-08-19
IL277779A (en) 2020-11-30
SG11202009759SA (en) 2020-10-29
TW202003052A (zh) 2020-01-16
EP3775233A1 (en) 2021-02-17
BR112020020204A2 (pt) 2021-01-19
CN113056561A (zh) 2021-06-29
CA3096088A1 (en) 2019-10-10
AU2019247864A1 (en) 2020-10-22
MX2020010477A (es) 2021-03-02
US20230149566A1 (en) 2023-05-18
KR20210005040A (ko) 2021-01-13
RU2020132890A (ru) 2022-05-06
US20190307900A1 (en) 2019-10-10
MA52199A (fr) 2021-02-17
PE20210918A1 (es) 2021-05-19
CO2020013690A2 (es) 2021-04-19
CL2020002561A1 (es) 2021-04-23

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