MA39082B2 - Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet - Google Patents

Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet

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Publication number
MA39082B2
MA39082B2 MA39082A MA39082A MA39082B2 MA 39082 B2 MA39082 B2 MA 39082B2 MA 39082 A MA39082 A MA 39082A MA 39082 A MA39082 A MA 39082A MA 39082 B2 MA39082 B2 MA 39082B2
Authority
MA
Morocco
Prior art keywords
polynucleotide
interest
retinal pigment
subject
pigment epithelium
Prior art date
Application number
MA39082A
Other languages
English (en)
Other versions
MA39082A1 (fr
Inventor
Christian Hamel
Vasiliki Kalatzis
Marie Pequignot
Nicolas Cereso
Original Assignee
Inst Nat Sante Rech Med
Ct Hospitalier Universitaire Montpellier
Univ Montpellier
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 Inst Nat Sante Rech Med, Ct Hospitalier Universitaire Montpellier, Univ Montpellier filed Critical Inst Nat Sante Rech Med
Publication of MA39082A1 publication Critical patent/MA39082A1/fr
Publication of MA39082B2 publication Critical patent/MA39082B2/fr

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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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61K48/005—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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
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    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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Abstract

L'invention concerne des méthodes et des compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet. L'invention concerne, en particulier, une méthode pour exprimer sélectivement un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien dans l'œil d'un sujet qui en a besoin, comprenant l'étape consistant à transduire l'épithélium pigmentaire rétinien avec une quantité de vecteur raav2/5 contenant le polynucléotide d'intérêt.
MA39082A 2013-12-06 2014-12-05 Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet MA39082B2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP13306676 2013-12-06
PCT/EP2014/076740 WO2015082690A1 (fr) 2013-12-06 2014-12-05 Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet

Publications (2)

Publication Number Publication Date
MA39082A1 MA39082A1 (fr) 2017-10-31
MA39082B2 true MA39082B2 (fr) 2021-11-30

Family

ID=49885103

Family Applications (1)

Application Number Title Priority Date Filing Date
MA39082A MA39082B2 (fr) 2013-12-06 2014-12-05 Méthodes et compositions pharmaceutiques pour exprimer un polynucléotide d'intérêt dans l'épithélium pigmentaire rétinien d'un sujet

Country Status (14)

Country Link
US (1) US10105452B2 (fr)
EP (1) EP3077520A1 (fr)
JP (2) JP6875856B2 (fr)
KR (1) KR102281881B1 (fr)
CN (1) CN105940109A (fr)
AU (1) AU2014359136B2 (fr)
BR (1) BR112016012716A2 (fr)
CA (1) CA2932495A1 (fr)
IL (1) IL245989A0 (fr)
MA (1) MA39082B2 (fr)
MX (1) MX2016007278A (fr)
RU (1) RU2723101C2 (fr)
TN (1) TN2016000220A1 (fr)
WO (1) WO2015082690A1 (fr)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190017096A1 (en) * 2016-03-10 2019-01-17 Nightstarx Limited Prenylation assay
CN107287238B (zh) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物
JP7028802B2 (ja) 2016-10-11 2022-03-02 ウェルスタット オフサルミックス コーポレーション 短鎖型桿体由来錐体生存因子及び親水性ペプチド間の融合タンパク質
JP2019534018A (ja) * 2016-11-07 2019-11-28 スパーク セラピューティクス インコーポレイテッドＳｐａｒｋ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． Ｒａｂエスコートタンパク質の力価アッセイ
AU2018311504A1 (en) * 2017-07-31 2020-03-12 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
AU2018350990A1 (en) * 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
MA52199A (fr) * 2018-04-05 2021-02-17 Nightstarx Ltd Compositions et procédés de traitement de la dystrophie maculaire
CN109136266B (zh) * 2018-08-10 2022-02-18 深圳泓熙生物科技发展有限公司 用于治疗或预防结晶样视网膜色素变性的基因载体及其用途
CN109112134B (zh) * 2018-09-28 2020-06-02 中国人民解放军陆军军医大学第一附属医院 一种视网膜变性疾病的best1新突变致病基因及其试剂盒
RU2732479C2 (ru) * 2018-12-21 2020-09-17 Селл энд Джин Терапи Лтд Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов BDNF, VEGFA, BFGF, NGF, GDNF, NT3, CNTF, IGF1, для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-BDNF, или Escherichia coli SCS110-AF/VTvaf17-VEGFA, или Escherichia coli SCS110-AF/VTvaf17-BFGF, или Escherichia coli SCS110-AF/VTvaf17-NGF, или Escherichia coli SCS110-AF/VTvaf17-GDNF, или Escherichia coli SCS110-AF/VTvaf17-NT3, или Escherichia coli SCS110-AF/VTvaf17-CNTF, или Escherichia coli SCS110-AF/VTvaf17-IGF1, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора
US20220143217A1 (en) * 2019-03-04 2022-05-12 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the akt pathway
CN110455655B (zh) * 2019-08-23 2024-05-28 水利部杭州机械设计研究所 一种热喷涂涂层高通量检测装置及测试方法
CN110791502B (zh) * 2019-12-04 2022-07-08 陕西理工大学 人源rbp4启动子片段及其应用
WO2023213817A1 (fr) * 2022-05-02 2023-11-09 Fondazione Telethon Ets Thérapie génique pour l'atrophie gyrate de la choroïde et de la rétine

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101589259B1 (ko) * 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
WO2009100253A1 (fr) * 2008-02-07 2009-08-13 Ceregene, Inc. Préservation de photorécepteurs par administration intravitréenne d'un vecteur d'expression codant une protéine thérapeutique
WO2011034947A2 (fr) * 2009-09-15 2011-03-24 University Of Washington Réactifs et procédés pour moduler l'activité des photorécepteurs à cônes
CA2798988C (fr) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Polypeptides liant l'adn des tale et leurs utilisations
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method

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Publication number Publication date
MX2016007278A (es) 2016-09-16
WO2015082690A1 (fr) 2015-06-11
IL245989A0 (en) 2016-07-31
RU2723101C2 (ru) 2020-06-08
KR102281881B1 (ko) 2021-07-27
CN105940109A (zh) 2016-09-14
AU2014359136B2 (en) 2020-06-25
JP2017500060A (ja) 2017-01-05
TN2016000220A1 (en) 2017-10-06
EP3077520A1 (fr) 2016-10-12
BR112016012716A2 (pt) 2020-11-24
US10105452B2 (en) 2018-10-23
KR20170009812A (ko) 2017-01-25
JP6875856B2 (ja) 2021-05-26
MA39082A1 (fr) 2017-10-31
AU2014359136A1 (en) 2016-06-16
US20160310618A1 (en) 2016-10-27
CA2932495A1 (fr) 2015-06-11
JP2020023568A (ja) 2020-02-13

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