DK2066791T3 - Genterapi for amyotrofisk lateralsklerose og andre rygmarvssygdomme - Google Patents
Genterapi for amyotrofisk lateralsklerose og andre rygmarvssygdommeInfo
- Publication number
- DK2066791T3 DK2066791T3 DK07839216.4T DK07839216T DK2066791T3 DK 2066791 T3 DK2066791 T3 DK 2066791T3 DK 07839216 T DK07839216 T DK 07839216T DK 2066791 T3 DK2066791 T3 DK 2066791T3
- Authority
- DK
- Denmark
- Prior art keywords
- gene therapy
- spinal cord
- lateral sclerosis
- amyotrophic lateral
- cord diseases
- 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
-
- 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
-
- 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
- A61K48/0075—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
- 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
- A61P25/28—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
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- 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
- 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
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Marine Sciences & Fisheries (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US82797706P | 2006-10-03 | 2006-10-03 | |
| PCT/US2007/021272 WO2008042420A2 (en) | 2006-10-03 | 2007-10-03 | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2066791T3 true DK2066791T3 (da) | 2012-10-15 |
Family
ID=39269037
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12157957.7T DK2497500T3 (da) | 2006-10-03 | 2007-10-03 | Genterapi mod spinal muskelatrofi |
| DK07839216.4T DK2066791T3 (da) | 2006-10-03 | 2007-10-03 | Genterapi for amyotrofisk lateralsklerose og andre rygmarvssygdomme |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK12157957.7T DK2497500T3 (da) | 2006-10-03 | 2007-10-03 | Genterapi mod spinal muskelatrofi |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US9890394B2 (da) |
| EP (4) | EP2066791B1 (da) |
| CN (3) | CN108853517A (da) |
| AR (1) | AR063113A1 (da) |
| CY (1) | CY1118477T1 (da) |
| DK (2) | DK2497500T3 (da) |
| ES (3) | ES2757723T3 (da) |
| HU (2) | HUE031182T2 (da) |
| LT (2) | LT2497500T (da) |
| PL (2) | PL2066791T3 (da) |
| PT (3) | PT2497500T (da) |
| SI (3) | SI2066791T1 (da) |
| WO (1) | WO2008042420A2 (da) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2066791T3 (pl) | 2006-10-03 | 2013-02-28 | Genzyme Corp | Terapia Genowa do leczenia stwardnienia zanikowego bocznego i innych zaburzeń rdzenia kręgowego |
| WO2009090933A1 (ja) * | 2008-01-16 | 2009-07-23 | Japan Science And Technology Agency | 低酸素状態にある細胞で増殖するウイルスまたは遺伝子を発現するウイルスベクター |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| WO2013025963A2 (en) | 2011-08-17 | 2013-02-21 | President And Fellows Of Harvard College | Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof |
| CN119876138A (zh) | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CA2975447C (en) * | 2015-01-30 | 2021-02-23 | The Regents Of The University Of California | Spinal subpial gene delivery system |
| 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. |
| AU2017241534A1 (en) | 2016-03-28 | 2018-10-04 | The Regents Of The University Of California | Method and composition for treating neuronal hyper-excitability |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| BR112019009834B1 (pt) * | 2016-11-17 | 2022-05-17 | Ohio State Innovation Foundation | Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| EP3668891B1 (en) * | 2017-08-16 | 2023-07-26 | Lgv1 S.R.L. | Vtft isoform of a bpifb4 protein for use in neuronal deseases and injuries |
| JP7291125B2 (ja) | 2017-09-08 | 2023-06-14 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 神経障害性疼痛を処置するための方法および組成物 |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| EP3908198B1 (en) | 2019-01-10 | 2024-12-11 | The Regents of the University of California | Subpial delivery system |
| USD963164S1 (en) | 2020-01-09 | 2022-09-06 | The Regents Of The University Of California | Surgical needle |
| WO2023288086A2 (en) * | 2021-07-16 | 2023-01-19 | President And Fellows Of Harvard College | Enhancers driving expression in motor neurons |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SE8303626D0 (sv) | 1983-06-23 | 1983-06-23 | Kabigen Ab | A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced |
| US5672344A (en) | 1987-12-30 | 1997-09-30 | The Regents Of The University Of Michigan | Viral-mediated gene transfer system |
| ES2249761T3 (es) | 1993-06-24 | 2006-04-01 | Advec Inc. | Vectores de adenovirus para terapia genica. |
| EP1637608B1 (en) | 1993-10-25 | 2009-07-22 | CANJI, Inc. | Recombinant adenoviral vector and methods of use |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| CA2187626C (en) | 1994-04-13 | 2009-11-03 | Michael G. Kaplitt | Aav-mediated delivery of dna to cells of the nervous system |
| US5882914A (en) | 1995-06-06 | 1999-03-16 | The Johns Hopkins University School Of Medicine | Nucleic acids encoding the hypoxia inducible factor-1 |
| AU1525797A (en) | 1996-04-22 | 1997-11-12 | Medtronic, Inc. | Two-stage angled venous cannula |
| US6432947B1 (en) | 1997-02-19 | 2002-08-13 | Berlex Laboratories, Inc. | N-heterocyclic derivatives as NOS inhibitors |
| US20030104973A1 (en) * | 1997-08-21 | 2003-06-05 | Quark Biotech, Inc. | Hypoxia-regulated genes |
| IL136454A0 (en) | 1997-12-04 | 2001-06-14 | Genzyme Corp | Compositions and methods for inducing gene expression |
| US6124131A (en) | 1998-08-25 | 2000-09-26 | The Johns Hopkins University School Of Medicine | Mutant hypoxia inducible factor-1 HIF-1 |
| US6544785B1 (en) | 1998-09-14 | 2003-04-08 | Mount Sinai School Of Medicine Of New York University | Helper-free rescue of recombinant negative strand RNA viruses |
| AU2001290984A1 (en) | 2000-09-18 | 2002-04-02 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
| US6838430B2 (en) * | 2001-09-28 | 2005-01-04 | The Regents Of The University Of California | Use of HIF-1a variants to accelerate wound healing |
| NZ533833A (en) * | 2001-12-21 | 2008-01-31 | Salk Inst For Biological Studi | Targeted retrograde gene delivery to motor neurons |
| US7193053B2 (en) * | 2003-04-25 | 2007-03-20 | United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Hypoxia-inducible factor 1alpha variants and methods of use |
| GB2414934A (en) * | 2004-06-11 | 2005-12-14 | Gill Steven Streatfield | Treatment of Parkinson's disease with GDNF |
| PL2066791T3 (pl) | 2006-10-03 | 2013-02-28 | Genzyme Corp | Terapia Genowa do leczenia stwardnienia zanikowego bocznego i innych zaburzeń rdzenia kręgowego |
| RU2640115C2 (ru) | 2011-11-01 | 2017-12-26 | Ресверлоджикс Корп. | Фармацевтические композиции замещенных хиназолинонов |
-
2007
- 2007-10-03 PL PL07839216T patent/PL2066791T3/pl unknown
- 2007-10-03 WO PCT/US2007/021272 patent/WO2008042420A2/en not_active Ceased
- 2007-10-03 PL PL12157957T patent/PL2497500T3/pl unknown
- 2007-10-03 EP EP07839216A patent/EP2066791B1/en active Active
- 2007-10-03 PT PT121579577T patent/PT2497500T/pt unknown
- 2007-10-03 DK DK12157957.7T patent/DK2497500T3/da active
- 2007-10-03 DK DK07839216.4T patent/DK2066791T3/da active
- 2007-10-03 PT PT78392164T patent/PT2066791E/pt unknown
- 2007-10-03 ES ES16184966T patent/ES2757723T3/es active Active
- 2007-10-03 ES ES07839216T patent/ES2391789T3/es active Active
- 2007-10-03 SI SI200731083T patent/SI2066791T1/sl unknown
- 2007-10-03 EP EP16184966.6A patent/EP3146982B1/en active Active
- 2007-10-03 HU HUE12157957A patent/HUE031182T2/en unknown
- 2007-10-03 EP EP19192274.9A patent/EP3632472A1/en active Pending
- 2007-10-03 AR ARP070104379A patent/AR063113A1/es active IP Right Grant
- 2007-10-03 ES ES12157957.7T patent/ES2607060T3/es active Active
- 2007-10-03 LT LTEP12157957.7T patent/LT2497500T/lt unknown
- 2007-10-03 CN CN201810575782.XA patent/CN108853517A/zh active Pending
- 2007-10-03 PT PT161849666T patent/PT3146982T/pt unknown
- 2007-10-03 LT LT16184966T patent/LT3146982T/lt unknown
- 2007-10-03 CN CNA200780038545XA patent/CN101528922A/zh active Pending
- 2007-10-03 SI SI200731873A patent/SI2497500T1/sl unknown
- 2007-10-03 EP EP12157957.7A patent/EP2497500B1/en active Active
- 2007-10-03 CN CN201610214446.3A patent/CN105770910A/zh active Pending
- 2007-10-03 SI SI200732128T patent/SI3146982T1/sl unknown
- 2007-10-03 HU HUE16184966A patent/HUE046709T2/hu unknown
-
2009
- 2009-04-03 US US12/417,910 patent/US9890394B2/en active Active
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2016
- 2016-12-20 CY CY20161101325T patent/CY1118477T1/el unknown
-
2017
- 2017-12-29 US US15/858,303 patent/US10920245B2/en active Active
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