ES2623464T3 - Nuevo factor de viabilidad neuronal y su uso para tratar la distrofia de conos - Google Patents

Nuevo factor de viabilidad neuronal y su uso para tratar la distrofia de conos Download PDF

Info

Publication number
ES2623464T3
ES2623464T3 ES08760606.7T ES08760606T ES2623464T3 ES 2623464 T3 ES2623464 T3 ES 2623464T3 ES 08760606 T ES08760606 T ES 08760606T ES 2623464 T3 ES2623464 T3 ES 2623464T3
Authority
ES
Spain
Prior art keywords
rdcvf
rdcvf2
treat
exon
neuronal viability
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
ES08760606.7T
Other languages
English (en)
Spanish (es)
Inventor
Thierry Leveillard
José-Alain Sahel
Céline JAILLARD
Olivier Poch
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
Original Assignee
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
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 Centre National de la Recherche Scientifique CNRS, Institut National de la Sante et de la Recherche Medicale INSERM filed Critical Centre National de la Recherche Scientifique CNRS
Application granted granted Critical
Publication of ES2623464T3 publication Critical patent/ES2623464T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896Neurological disorders, e.g. Alzheimer's disease
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/118Prognosis of disease development
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/158Expression markers
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • G01N2800/2814Dementia; Cognitive disorders
    • G01N2800/2821Alzheimer
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • G01N2800/2835Movement disorders, e.g. Parkinson, Huntington, Tourette

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Food Science & Technology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • General Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
ES08760606.7T 2007-06-05 2008-06-05 Nuevo factor de viabilidad neuronal y su uso para tratar la distrofia de conos Active ES2623464T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP07109652A EP2027889A1 (en) 2007-06-05 2007-06-05 New neuronal viability factor and use thereof
EP07109652 2007-06-05
PCT/EP2008/057031 WO2008148860A1 (en) 2007-06-05 2008-06-05 New neuronal viability factor and use thereof

Publications (1)

Publication Number Publication Date
ES2623464T3 true ES2623464T3 (es) 2017-07-11

Family

ID=38573348

Family Applications (1)

Application Number Title Priority Date Filing Date
ES08760606.7T Active ES2623464T3 (es) 2007-06-05 2008-06-05 Nuevo factor de viabilidad neuronal y su uso para tratar la distrofia de conos

Country Status (5)

Country Link
US (5) US9575075B2 (OSRAM)
EP (2) EP2027889A1 (OSRAM)
JP (1) JP5671678B2 (OSRAM)
ES (1) ES2623464T3 (OSRAM)
WO (1) WO2008148860A1 (OSRAM)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1625210B1 (en) 2003-05-21 2010-12-01 Genzyme Corporation Methods for producing preparations of recombinant aav virions substantially free of empty capsids
EP2027889A1 (en) * 2007-06-05 2009-02-25 Institut National De La Sante Et De La Recherche Medicale (Inserm) New neuronal viability factor and use thereof
US9567382B2 (en) 2008-04-15 2017-02-14 Genzyme Corporation Methods to produce rod-derived cone viability factor (RdCVF)
EP2383286A1 (en) 2010-04-30 2011-11-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for treatment of retinal degenerative diseases
JP6293664B2 (ja) * 2011-10-27 2018-03-14 ウェルスタット オフサルミクス コーポレイション 桿体由来錐体生存因子をコードするベクター
US9265786B1 (en) * 2012-03-30 2016-02-23 University Of Wyoming Method for reducing protein misfolding and accumulation in Huntington'S disease cells
WO2016185242A1 (en) 2015-05-21 2016-11-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Synergistic combination of neuronal viability factors and uses thereof
MX2018015596A (es) * 2016-10-11 2019-03-14 Wellstat Ophthalmics Corp Proteina de fusion entre el factor de viabilidad de los conos derivado de bastones de forma corta y un peptido hidrofilo.
WO2018134168A1 (en) 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
US11723988B2 (en) 2017-10-20 2023-08-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
DK3728610T5 (da) 2017-12-22 2024-08-26 Sparingvision Konstrukter omfattende neuronale viabilitetsfaktorer og anvendelser heraf
EP4326339B1 (en) 2021-04-20 2025-04-02 Institut National de la Santé et de la Recherche Médicale (INSERM) Compositions and methods for treating retinal degenerative disorders
US20250276088A1 (en) * 2021-07-07 2025-09-04 Institut National de la Santé et de la Recherche Médicale Synergistic combination of rdcfv2 and rdcvf2l for the treatment of tauopathies
CN120303405A (zh) 2022-10-20 2025-07-11 节约视觉公司 用于治疗视网膜退行性疾病的组合物和方法
WO2024206928A1 (en) 2023-03-30 2024-10-03 Pharma Cinq, Llc VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IgK SIGNAL SEQUENCE

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1242443A4 (en) 1999-12-23 2005-06-22 Nuvelo Inc NEW NUCLEIC ACIDS AND POLYPEPTIDES
FR2823221B1 (fr) * 2001-04-06 2004-04-02 Univ Pasteur Sequences associees a la degenerescence retinienne et applications
FR2870241B1 (fr) * 2004-05-13 2015-02-27 Novartis Ag Facteur de viabilite des cones derive des batonnets ou rdcvf et applications
US20090011040A1 (en) * 2007-05-02 2009-01-08 Naash Muna I Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases
EP2027889A1 (en) * 2007-06-05 2009-02-25 Institut National De La Sante Et De La Recherche Medicale (Inserm) New neuronal viability factor and use thereof
ES2582559T3 (es) * 2007-12-06 2016-09-13 Inserm - Institut National De La Santé Et De La Recherche Médicale Factor trófico para el tratamiento de las enfermedades degenerativas de la retina
WO2010029130A1 (en) * 2008-09-10 2010-03-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Neuronal viability factor and use thereof
JP6293664B2 (ja) * 2011-10-27 2018-03-14 ウェルスタット オフサルミクス コーポレイション 桿体由来錐体生存因子をコードするベクター
WO2016185242A1 (en) * 2015-05-21 2016-11-24 INSERM (Institut National de la Santé et de la Recherche Médicale) Synergistic combination of neuronal viability factors and uses thereof
US11723988B2 (en) * 2017-10-20 2023-08-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
DK3728610T5 (da) * 2017-12-22 2024-08-26 Sparingvision Konstrukter omfattende neuronale viabilitetsfaktorer og anvendelser heraf

Also Published As

Publication number Publication date
WO2008148860A1 (en) 2008-12-11
EP2175932B1 (en) 2017-01-25
US20170014478A1 (en) 2017-01-19
US20200174023A1 (en) 2020-06-04
EP2175932A1 (en) 2010-04-21
EP2027889A1 (en) 2009-02-25
JP5671678B2 (ja) 2015-02-18
US11428698B2 (en) 2022-08-30
US20220404376A1 (en) 2022-12-22
US20100247496A1 (en) 2010-09-30
US20190064189A1 (en) 2019-02-28
US9575075B2 (en) 2017-02-21
JP2010529958A (ja) 2010-09-02

Similar Documents

Publication Publication Date Title
ES2623464T3 (es) Nuevo factor de viabilidad neuronal y su uso para tratar la distrofia de conos
Chakraborty et al. Evolution of genome structure in the Drosophila simulans species complex
ES2670531T3 (es) Un método para producir una escisión de ADN precisa utilizando la actividad nickasa de Cas9
AU2023278107A1 (en) 3' UTR sequences for stabilization of RNA
TWI747808B (zh) 新穎之cho整合位點及其用途
ES2887852T3 (es) Animales con IL-4 e IL-4R alfa humanizadas
ES2877275T3 (es) Animales con dipeptidil peptidasa IV (DPP4) humanizada
KR102151065B1 (ko) 동물 배아의 염기 교정용 조성물 및 염기 교정 방법
Schurko et al. Meiosis genes in Daphnia pulex and the role of parthenogenesis in genome evolution
PT1979474E (pt) Transcriptase inversa da telomerase aviária
WO2021011929A9 (en) Use of frataxin for treating leigh syndrome, french canadian type
KR102271414B1 (ko) Fam50a 유전자를 녹아웃시킨 동물모델 및 이의 용도
Huang et al. NXT2 is required for embryonic heart development in zebrafish
CN111100877B (zh) 肥厚型心肌病小鼠模型的制备方法及其应用
CN111718933A (zh) 一种rrbp1基因敲除热带爪蛙模型的制备方法与应用
CN104946621A (zh) 基因敲入组合物及其使用方法和应用
WO2022148253A1 (zh) 一种人klf7基因启动子及其构建方法与应用
Burgoyne et al. The roles of mouse Y chromosome genes in spermatogenesis
Rentsendorj et al. A genomic imprinting defect in mice traced to a single gene
JP7055469B2 (ja) ホモ接合型細胞の作製方法
CA3189755A1 (en) Modulation of endoplasmic reticulum aminopeptidase 2 (erap2)-mediated immune response
Shimoda et al. Imprinting of a small nucleolar RNA gene on mouse chromosome 12
Street et al. A DNA variant within the MYO7A promoter regulates YY1 transcription factor binding and gene expression serving as a potential dominant DFNA11 auditory genetic modifier
WO2022072458A1 (en) Crispr/cas9 targeted excision of the intronic ctg18.1 trinucleotide repeat expansion of tcf4 as a therapy in fuchs' endothelial corneal dystrophy
Daish et al. Location, location, location! Monotremes provide unique insights into the evolution of sex chromosome silencing in mammals