PL2489733T3 - Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego - Google Patents
Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowegoInfo
- Publication number
- PL2489733T3 PL2489733T3 PL12152550T PL12152550T PL2489733T3 PL 2489733 T3 PL2489733 T3 PL 2489733T3 PL 12152550 T PL12152550 T PL 12152550T PL 12152550 T PL12152550 T PL 12152550T PL 2489733 T3 PL2489733 T3 PL 2489733T3
- Authority
- PL
- Poland
- Prior art keywords
- gene therapy
- spinal cord
- lateral sclerosis
- amyotrophic lateral
- cord disorders
- Prior art date
Links
- 208000029033 Spinal Cord disease Diseases 0.000 title 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
Classifications
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- 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
- A61K38/22—Hormones
- A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
- Zoology (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Pain & Pain Management (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US81141906P | 2006-06-07 | 2006-06-07 | |
| PCT/US2007/013391 WO2007146046A2 (en) | 2006-06-07 | 2007-06-07 | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| EP12152550.5A EP2489733B1 (en) | 2006-06-07 | 2007-06-07 | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| EP07795835.3A EP2029742B1 (en) | 2006-06-07 | 2007-06-07 | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL2489733T3 true PL2489733T3 (pl) | 2019-08-30 |
Family
ID=38832369
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL07795835T PL2029742T3 (pl) | 2006-06-07 | 2007-06-07 | Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego |
| PL12152550T PL2489733T3 (pl) | 2006-06-07 | 2007-06-07 | Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL07795835T PL2029742T3 (pl) | 2006-06-07 | 2007-06-07 | Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US9034836B2 (pl) |
| EP (3) | EP3540054A3 (pl) |
| JP (6) | JP2009539847A (pl) |
| CN (1) | CN101460621B (pl) |
| AR (2) | AR061282A1 (pl) |
| BR (1) | BRPI0711965A2 (pl) |
| CA (2) | CA3139726A1 (pl) |
| CY (1) | CY1118555T1 (pl) |
| ES (2) | ES2725552T3 (pl) |
| HU (2) | HUE044182T2 (pl) |
| IL (2) | IL195647A (pl) |
| LT (2) | LT2489733T (pl) |
| MX (4) | MX364444B (pl) |
| PL (2) | PL2029742T3 (pl) |
| PT (2) | PT2029742T (pl) |
| RU (1) | RU2008152821A (pl) |
| SI (2) | SI2029742T1 (pl) |
| TR (1) | TR201906106T4 (pl) |
| WO (1) | WO2007146046A2 (pl) |
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|---|---|---|---|---|
| MX364444B (es) | 2006-06-07 | 2019-04-26 | Genzyme Corp | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal. |
| ES2596360T3 (es) * | 2008-01-16 | 2017-01-09 | Neurodyn Life Sciences Inc. | Progranulina para su uso en el tratamiento de enfermedad de Parkinson o enfermedad de Alzheimer |
| CN102272321A (zh) | 2008-10-31 | 2011-12-07 | 神经动力公司 | 神经毒性甾醇糖苷 |
| EP3988660B1 (en) | 2009-05-02 | 2025-05-14 | Genzyme Corporation | Gene therapy for neurodegenerative disorders |
| EP2640407A4 (en) * | 2010-11-16 | 2014-07-09 | Denis G Kay | PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN |
| CN103083667A (zh) * | 2011-11-01 | 2013-05-08 | 北京清美联创干细胞科技有限公司 | 嗅鞘细胞增殖试剂的连续给药方案 |
| EP2850093B1 (en) | 2012-01-27 | 2019-01-09 | The Board of Trustees of the Leland Stanford Junior University | Therapeutic il-13 polypeptides |
| CN104619334A (zh) | 2012-08-09 | 2015-05-13 | 利兰斯坦福初级大学董事会 | 超级因子和合成因子:具有新的和增强的信号传导活性的经改造细胞因子 |
| US20140065110A1 (en) | 2012-08-31 | 2014-03-06 | The Regents Of The University Of California | Genetically modified msc and therapeutic methods |
| US9663564B2 (en) | 2013-03-15 | 2017-05-30 | The Regents Of The University Of California | Vectors and methods to treat ischemia |
| CN104853778A (zh) | 2012-10-24 | 2015-08-19 | 第一三共株式会社 | 用于肌萎缩性侧索硬化的治疗剂 |
| WO2014084085A1 (ja) * | 2012-11-28 | 2014-06-05 | 国立大学法人名古屋大学 | 聴覚障害又は小脳性運動失調症の予防・治療剤 |
| AU2013388083B2 (en) * | 2013-05-01 | 2019-08-22 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| RU2018128780A (ru) * | 2013-07-26 | 2018-12-05 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
| KR101548989B1 (ko) | 2013-07-30 | 2015-09-01 | 광주과학기술원 | SirT1을 이용한 관절 질환의 예방 또는 치료용 물질의 스크리닝 방법 |
| US9119832B2 (en) | 2014-02-05 | 2015-09-01 | The Regents Of The University Of California | Methods of treating mild brain injury |
| DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| KR20170096998A (ko) | 2014-11-05 | 2017-08-25 | 보이저 테라퓨틱스, 인크. | 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드 |
| CA2987103A1 (en) * | 2015-05-28 | 2016-12-01 | Cornell University | Adeno-associated virus mediated delivery of c1ei as a therapy for angioedema |
| CL2015003024A1 (es) | 2015-10-09 | 2016-05-13 | Univ Chile | Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica. |
| WO2017087486A1 (en) * | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
| JP7436089B2 (ja) * | 2016-03-02 | 2024-02-21 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | 前頭側頭型認知症の治療 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| KR102709597B1 (ko) * | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| IL273776B2 (en) | 2017-10-03 | 2025-06-01 | Prevail Therapeutics Inc | Gene therapies for lysosomal disorders |
| AU2018346102B2 (en) | 2017-10-03 | 2023-05-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| WO2019073299A1 (en) * | 2017-10-10 | 2019-04-18 | Medicenna Therapeutics, Inc. | IL-4 FUSION FORMULATIONS FOR THE TREATMENT OF CENTRAL NERVOUS SYSTEM (CNS) TUMORS |
| JP2021523914A (ja) | 2018-05-15 | 2021-09-09 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | パーキンソン病を治療するための組成物および方法 |
| WO2019239213A2 (en) | 2018-06-01 | 2019-12-19 | Medicenna Therapeutics Inc. | Uses and methods for oncolytic virus targeting of il-4/il-13 and fusions thereof |
| JP7528066B2 (ja) | 2018-09-28 | 2024-08-05 | ボイジャー セラピューティクス インコーポレイテッド | 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 |
| RU2731514C2 (ru) * | 2018-12-21 | 2020-09-03 | Селл энд Джин Терапи Лтд | Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов SHH, CTNNB1, NOG, WNT7A для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-SHH, или Escherichia coli SCS110-AF/VTvaf17-CTNNB1, или Escherichia coli SCS110-AF/VTvaf17-NOG, или Escherichia coli SCS110-AF/VTvaf17-WNT7A, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора |
| WO2020160458A1 (en) * | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| KR20210131370A (ko) | 2019-02-22 | 2021-11-02 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스 |
| CA3136004A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| CN114430684A (zh) * | 2019-04-12 | 2022-05-03 | 编码治疗公司 | 用于治疗剂投予的组成物及方法 |
| US20220235373A1 (en) * | 2019-10-12 | 2022-07-28 | Shenzhen University | Agent that enables sirt7 gene expression and the use thereof |
| AU2020411480B2 (en) | 2019-12-23 | 2023-12-21 | Denali Therapeutics Inc. | Progranulin variants |
| WO2023242844A1 (en) * | 2022-06-15 | 2023-12-21 | Ramot At Tel-Aviv University Ltd. | Method and therapeutic agent for treatment of disease or disorder associated with impaired firing rate and/or mitochondrial calcium homeostasis |
| CN115323002A (zh) * | 2022-07-28 | 2022-11-11 | 深圳先进技术研究院 | 一种基因递送系统在从脑部逆行递送基因到脊髓神经元中的应用 |
| CN120590487A (zh) * | 2025-06-17 | 2025-09-05 | 河北医科大学第二医院 | 一种信号肽、重组载体及其应用 |
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| MX364444B (es) | 2006-06-07 | 2019-04-26 | Genzyme Corp | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal. |
| AU2012330885B2 (en) | 2011-11-01 | 2016-10-06 | Resverlogix Corp. | Oral immediate release formulations for substituted quinazolinones |
| US20150182637A1 (en) | 2012-06-21 | 2015-07-02 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
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2007
- 2007-06-07 MX MX2015001403A patent/MX364444B/es unknown
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- 2007-06-07 LT LTEP07795835.3T patent/LT2029742T/lt unknown
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- 2007-06-07 CA CA3139726A patent/CA3139726A1/en active Pending
- 2007-06-07 EP EP19156264.4A patent/EP3540054A3/en active Pending
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- 2007-06-07 PT PT12152550T patent/PT2489733T/pt unknown
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- 2007-06-07 AR ARP070102460A patent/AR061282A1/es not_active Application Discontinuation
- 2007-06-07 RU RU2008152821/10A patent/RU2008152821A/ru not_active Application Discontinuation
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- 2007-06-07 MX MX2008015530A patent/MX2008015530A/es active IP Right Grant
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2008
- 2008-12-02 IL IL195647A patent/IL195647A/en active IP Right Grant
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2013
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2015
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2016
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2017
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2018
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2019
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2022
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