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

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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
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Prior art keywords
rdcvf
rdcvf2
treat
exon
neuronal viability
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ES08760606.7T
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Spanish (es)
Inventor
Thierry Leveillard
José-Alain Sahel
Céline JAILLARD
Olivier Poch
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Centre National de la Recherche Scientifique
Institut National de la Santé et de la Recherche Médicale
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Centre National de la Recherche Scientifique
Institut National de la Santé et de la Recherche Médicale
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    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical 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
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • 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
    • C07K14/46—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
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/118—Prognosis of disease development
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2814—Dementia; Cognitive disorders
    • G01N2800/2821—Alzheimer
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2835—Movement disorders, e.g. Parkinson, Huntington, Tourette

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  • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • General Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Food Science & Technology (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
EP07109652 2007-06-05
EP07109652A EP2027889A1 (en) 2007-06-05 2007-06-05 New neuronal viability factor and use thereof
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 (enExample)
EP (2) EP2027889A1 (enExample)
JP (1) JP5671678B2 (enExample)
ES (1) ES2623464T3 (enExample)
WO (1) WO2008148860A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
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
WO2009146183A1 (en) 2008-04-15 2009-12-03 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
CN110777124A (zh) 2011-10-27 2020-02-11 威尔斯达眼科制剂公司 编码视杆细胞来源的视锥细胞活力因子的载体
US9265786B1 (en) * 2012-03-30 2016-02-23 University Of Wyoming Method for reducing protein misfolding and accumulation in Huntington'S disease cells
JP6684343B2 (ja) * 2015-05-21 2020-04-22 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ニューロン生存因子の相乗組み合わせ及びその使用
KR20260085174A (ko) 2016-10-11 2026-06-08 파마 싱크, 엘엘씨 짧은 형태의 간상체 유래 원추체 생존능 인자와 친수성 펩타이드 사이의 융합 단백질
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
KR102763312B1 (ko) 2017-10-20 2025-02-07 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔) 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법
WO2019122403A1 (en) 2017-12-22 2019-06-27 Sparingvision Constructs comprising neuronal viability factors and uses thereof
ES3034370T3 (en) 2021-04-20 2025-08-18 Inst Nat Sante Rech Med Compositions and methods for treating retinal degenerative disorders
EP4366757A1 (en) * 2021-07-07 2024-05-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Synergistic combination of rdcfv2 and rdcvf2l for the treatment of tauopathies
KR20250115381A (ko) 2022-10-20 2025-07-30 스페어링비젼 망막 변성 질환 치료를 위한 조성물 및 방법
CN121285568A (zh) 2023-03-30 2026-01-06 制药五有限责任公司 编码视杆细胞源性视锥细胞活力因子和人IgK信号序列的载体

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2402563A1 (en) * 1999-12-23 2001-07-26 Hyseq, Inc. Novel 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
JP5710265B2 (ja) * 2007-12-06 2015-04-30 アンセルム（アンスチチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル） 網膜変性疾患の治療のための栄養因子
JP5748223B2 (ja) * 2008-09-10 2015-07-15 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ニューロン生存因子およびその使用
CN110777124A (zh) * 2011-10-27 2020-02-11 威尔斯达眼科制剂公司 编码视杆细胞来源的视锥细胞活力因子的载体
JP6684343B2 (ja) * 2015-05-21 2020-04-22 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ニューロン生存因子の相乗組み合わせ及びその使用
KR102763312B1 (ko) * 2017-10-20 2025-02-07 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔) 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법
WO2019122403A1 (en) * 2017-12-22 2019-06-27 Sparingvision Constructs comprising neuronal viability factors and uses thereof

Also Published As

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

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