PL2718437T3 - Sposoby leczenia wrodzonej ślepoty Lebera - Google Patents

Sposoby leczenia wrodzonej ślepoty Lebera

Info

Publication number
PL2718437T3
PL2718437T3 PL12729421T PL12729421T PL2718437T3 PL 2718437 T3 PL2718437 T3 PL 2718437T3 PL 12729421 T PL12729421 T PL 12729421T PL 12729421 T PL12729421 T PL 12729421T PL 2718437 T3 PL2718437 T3 PL 2718437T3
Authority
PL
Poland
Prior art keywords
treatment
methods
congenital amaurosis
leber congenital
leber
Prior art date
Application number
PL12729421T
Other languages
English (en)
Inventor
Jean-Michel Rozet
Antoine Kichler
Isabelle Perrault
Josseline Kaplan
Xavier GERARD
Daniel Scherman
Arnold Munnich
Original Assignee
Institut National De La Santé Et De La Recherche Médicale (Inserm)
Centre National De La Recherche Scientifique
Genethon
Université Paris Descartes
ENSCP - Chimie ParisTech École Nationale Supérieure de Chimie de Paris
Assistance Publique Hôpitaux De Paris
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 Institut National De La Santé Et De La Recherche Médicale (Inserm), Centre National De La Recherche Scientifique, Genethon, Université Paris Descartes, ENSCP - Chimie ParisTech École Nationale Supérieure de Chimie de Paris, Assistance Publique Hôpitaux De Paris filed Critical Institut National De La Santé Et De La Recherche Médicale (Inserm)
Publication of PL2718437T3 publication Critical patent/PL2718437T3/pl

Links

Classifications

    • 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
    • C12N15/11—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
    • 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
    • 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
    • C12N15/11—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
    • C12N15/1137—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 enzymes
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3517—Marker; Tag
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL12729421T 2011-06-10 2012-06-08 Sposoby leczenia wrodzonej ślepoty Lebera PL2718437T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP11305735 2011-06-10
EP12729421.3A EP2718437B1 (en) 2011-06-10 2012-06-08 Methods for the treatment of leber congenital amaurosis
PCT/EP2012/060906 WO2012168435A1 (en) 2011-06-10 2012-06-08 Methods for the treatment of leber congenital amaurosis

Publications (1)

Publication Number Publication Date
PL2718437T3 true PL2718437T3 (pl) 2019-01-31

Family

ID=44720213

Family Applications (1)

Application Number Title Priority Date Filing Date
PL12729421T PL2718437T3 (pl) 2011-06-10 2012-06-08 Sposoby leczenia wrodzonej ślepoty Lebera

Country Status (6)

Country Link
US (5) US9012425B2 (pl)
EP (2) EP3378938A1 (pl)
DK (1) DK2718437T3 (pl)
ES (1) ES2683161T3 (pl)
PL (1) PL2718437T3 (pl)
WO (1) WO2012168435A1 (pl)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2718437T3 (en) * 2011-06-10 2018-08-06 Inst Nat Sante Rech Med Methods for the Treatment of Liver's Congenital Amaurosis
BR112014004895B1 (pt) 2011-09-05 2022-07-05 Stichting Radboud Universitair Medisch Centrum Oligonucleotídeos antissentido para o tratamento de amaurose congênita de leber
EP3019610B1 (en) 2013-07-08 2020-05-06 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof
US9834791B2 (en) 2013-11-07 2017-12-05 Editas Medicine, Inc. CRISPR-related methods and compositions with governing gRNAS
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US9938521B2 (en) 2014-03-10 2018-04-10 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
WO2015190922A1 (en) 2014-06-10 2015-12-17 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of pompe disease
WO2016034680A1 (en) * 2014-09-05 2016-03-10 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis
GB201503408D0 (en) 2015-02-27 2015-04-15 Proqr Therapeutics N V Oligonucleotides
SG11201802870RA (en) 2015-10-09 2018-05-30 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
US10993995B2 (en) 2015-12-07 2021-05-04 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for pompe disease
SG11201804443UA (en) 2015-12-14 2018-06-28 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
JP7186094B2 (ja) 2016-05-06 2022-12-08 イグジキュア オペレーティング カンパニー インターロイキン１７受容体ｍＲＮＡの特異的ノックダウンのためのアンチセンスオリゴヌクレオチド（ＡＳＯ）を提示するリポソーム系球状核酸（ＳＮＡ）構築物
US20180010117A1 (en) * 2016-07-07 2018-01-11 Sangamo Therapeutics, Inc. Targeted treatment of leber congenital amourosis
BR112019001887A2 (pt) 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
CN110234545B (zh) * 2017-03-10 2022-01-25 金泰克斯公司 用于车辆乘员识别和监测的成像系统和方法
JP7048574B2 (ja) * 2017-03-10 2022-04-05 国立研究開発法人国立成育医療研究センター アンチセンスオリゴヌクレオチドおよび糖原病Ｉａ型予防または治療用組成物
RS65031B1 (sr) 2017-08-25 2024-02-29 Stoke Therapeutics Inc Antisens oligomeri za lečenje stanja i bolesti
JP7261482B2 (ja) * 2018-01-25 2023-04-20 国立大学法人大阪大学 ペリオスチンの発現量増加またはスプライシングバリアントの変化を伴う疾患の治療用医薬組成物
CN111511915A (zh) 2018-03-09 2020-08-07 第一三共株式会社 糖原病Ia型治疗药
WO2019213525A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
AU2020214704A1 (en) 2019-01-28 2021-08-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of leber's congenital amaurosis
WO2020157008A1 (en) 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
AU2020259856A1 (en) 2019-04-18 2021-11-18 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
WO2021130313A1 (en) 2019-12-23 2021-07-01 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease
WO2021175904A1 (en) 2020-03-04 2021-09-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of usher syndrome
MX2022014151A (es) 2020-05-11 2022-11-30 Stoke Therapeutics Inc Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades.
WO2022090256A1 (en) 2020-10-26 2022-05-05 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of stargardt disease
EP4301856A2 (en) 2021-03-05 2024-01-10 ProQR Therapeutics II B.V. Antisense oligonucleotides for use in the treatment of corneal dystrophies
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia

Family Cites Families (7)

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Publication number Priority date Publication date Assignee Title
US7517644B1 (en) 1991-08-23 2009-04-14 Larry J. Smith Method and compositions for cellular reprogramming
US20040102398A1 (en) * 2002-11-23 2004-05-27 Isis Pharmaceuticals Inc. Modulation of B7H expression
WO2004028458A2 (en) 2002-09-25 2004-04-08 Pharmacia Corporation Antisense modulation of microsomal prostaglandin e2 synthase expression
US7838231B2 (en) 2006-04-07 2010-11-23 The Regents Of The University Of Michigan NPH6 nucleic acids and proteins
US20110117058A1 (en) * 2008-04-02 2011-05-19 Fondazione Telethon Method of treating genetic disorders
DK2718437T3 (en) * 2011-06-10 2018-08-06 Inst Nat Sante Rech Med Methods for the Treatment of Liver's Congenital Amaurosis
BR112014004895B1 (pt) 2011-09-05 2022-07-05 Stichting Radboud Universitair Medisch Centrum Oligonucleotídeos antissentido para o tratamento de amaurose congênita de leber

Also Published As

Publication number Publication date
US10597654B2 (en) 2020-03-24
DK2718437T3 (en) 2018-08-06
EP2718437B1 (en) 2018-05-09
US9012425B2 (en) 2015-04-21
US9487782B2 (en) 2016-11-08
US20200291402A1 (en) 2020-09-17
EP2718437A1 (en) 2014-04-16
ES2683161T3 (es) 2018-09-25
US20180016579A1 (en) 2018-01-18
WO2012168435A1 (en) 2012-12-13
US11492621B2 (en) 2022-11-08
US20170044533A1 (en) 2017-02-16
EP3378938A1 (en) 2018-09-26
US20150111951A1 (en) 2015-04-23
US9777272B2 (en) 2017-10-03
US20140179767A1 (en) 2014-06-26

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