HRP20200862T1 - Proizvodi i metode liječenja amiotrofične lateralne skleroze - Google Patents
Proizvodi i metode liječenja amiotrofične lateralne skleroze Download PDFInfo
- Publication number
- HRP20200862T1 HRP20200862T1 HRP20200862TT HRP20200862T HRP20200862T1 HR P20200862 T1 HRP20200862 T1 HR P20200862T1 HR P20200862T T HRP20200862T T HR P20200862TT HR P20200862 T HRP20200862 T HR P20200862T HR P20200862 T1 HRP20200862 T1 HR P20200862T1
- Authority
- HR
- Croatia
- Prior art keywords
- recombinant adeno
- associated virus
- treatment
- lateral sclerosis
- amyotrophic lateral
- Prior art date
Links
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 title claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 10
- 102000008221 Superoxide Dismutase-1 Human genes 0.000 claims 4
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- 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
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- 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
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0089—Oxidoreductases (1.) acting on superoxide as acceptor (1.15)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
- C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
- C12Y115/01001—Superoxide dismutase (1.15.1.1)
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- General Chemical & Material Sciences (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Claims (6)
1. Rekombinantni adeno-pridruženi virus koji sadrži superoksid dismutazu 1 (SOD1) shRNA-kodirajuću DNA CATGGATTCCATGTTCATGA (SEQ ID NO: 4), naznačen time da rekombinantnom adeno-pridruženom virusnom genomu nedostaju rep i kapica gena.
2. Pripravak naznačen time da sadrži rekombinantni adeno-povezani virus prema zahtjevu 1.
3. Rekombinantni adeno-pridruženi virus za upotrebu u terapiji koji sadrži SOD1 shRNA-kodirajuću DNA CATGGATTCCATGTTCATGA (SEQ ID NO: 4), naznačen time da rekombinantnom adeno-pridruženom genomu virusa nedostaje rep i kapica gena.
4. Rekombinantni adeno-pridruženi virus za upotrebu u liječenju amiotrofične lateralne skleroze (ALS), naznačen time da sadrži SOD1 shRNA-enkodirajuću DNA CATGGATTCCATGTTCATGA (SEQ ID NO: 4), pri čemu rekombinantno adeno-pridruženom virusnom genomu nedostaje rep i kapica gena.
5. Rekombinantni adeno-pridruženi virus za upotrebu u skladu s patentnim zahtjevima 3 ili 4, naznačen time da je rekombinantni adeno-pridruženi virus za sistemsku primjenu.
6. Rekombinantni adeno-pridruženi virus za upotrebu u skladu s patentnim zahtjevima 3 ili 4, naznačen time da je rekombinantni adeno-pridruženi virus za intratekalnu primjenu.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361870585P | 2013-08-27 | 2013-08-27 | |
EP14761771.6A EP3039146B1 (en) | 2013-08-27 | 2014-08-26 | Products and methods for treatment of amyotrophic lateral sclerosis |
PCT/US2014/052753 WO2015031392A1 (en) | 2013-08-27 | 2014-08-26 | Products and methods for treatment of amyotrophic lateral sclerosis |
Publications (1)
Publication Number | Publication Date |
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HRP20200862T1 true HRP20200862T1 (hr) | 2020-08-21 |
Family
ID=51494538
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20230015TT HRP20230015T1 (hr) | 2013-08-27 | 2014-08-26 | Proizvodi i postupci liječenja amiotrofične lateralne skleroze |
HRP20200862TT HRP20200862T1 (hr) | 2013-08-27 | 2020-05-29 | Proizvodi i metode liječenja amiotrofične lateralne skleroze |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20230015TT HRP20230015T1 (hr) | 2013-08-27 | 2014-08-26 | Proizvodi i postupci liječenja amiotrofične lateralne skleroze |
Country Status (15)
Country | Link |
---|---|
US (3) | US20160272976A1 (hr) |
EP (3) | EP4219727A3 (hr) |
CA (1) | CA2922500A1 (hr) |
CY (1) | CY1123272T1 (hr) |
DK (2) | DK3039146T3 (hr) |
ES (2) | ES2936220T3 (hr) |
FI (1) | FI3702466T3 (hr) |
HR (2) | HRP20230015T1 (hr) |
HU (2) | HUE061629T2 (hr) |
LT (2) | LT3039146T (hr) |
PL (1) | PL3702466T3 (hr) |
PT (2) | PT3702466T (hr) |
RS (2) | RS60434B1 (hr) |
SI (2) | SI3702466T1 (hr) |
WO (1) | WO2015031392A1 (hr) |
Families Citing this family (47)
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US8664189B2 (en) | 2008-09-22 | 2014-03-04 | Rxi Pharmaceuticals Corporation | RNA interference in skin indications |
WO2010090762A1 (en) | 2009-02-04 | 2010-08-12 | Rxi Pharmaceuticals Corporation | Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
US9226976B2 (en) | 2011-04-21 | 2016-01-05 | University Of Massachusetts | RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
KR20220119187A (ko) * | 2013-05-15 | 2022-08-26 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
HUE061629T2 (hu) * | 2013-08-27 | 2023-07-28 | Res Inst Nationwide Childrens Hospital | Termékek és módszerek amyotrophiás lateralis sclerosis kezelésére |
EP3065784A4 (en) | 2013-11-05 | 2017-05-10 | The Research Institute at Nationwide Children's Hospital | COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS |
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US10280418B2 (en) * | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
HRP20220798T1 (hr) | 2014-04-01 | 2022-10-14 | Biogen Ma Inc. | Pripravci za modulaciju ekspresije sod-1 |
US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
RU2020140209A (ru) | 2014-10-21 | 2021-01-25 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
WO2016172008A1 (en) | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
WO2017007825A1 (en) * | 2015-07-06 | 2017-01-12 | Rxi Pharmaceuticals Corporation | Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach |
CN108135923B (zh) | 2015-07-06 | 2021-03-02 | 菲奥医药公司 | 靶向超氧化物歧化酶1(sod1)的核酸分子 |
WO2017005806A1 (en) | 2015-07-07 | 2017-01-12 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject |
EP3121284A1 (en) * | 2015-07-22 | 2017-01-25 | Ecole Polytechnique Federale de Lausanne (EPFL) | Bicistronic aav vector for rna interference in als |
CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
CA3002982A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
US11826433B2 (en) | 2016-02-02 | 2023-11-28 | University Of Massachusetts | Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system |
US11060088B2 (en) | 2016-02-12 | 2021-07-13 | University Of Massachusetts | Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization |
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US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
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2014
- 2014-08-26 HU HUE20165345A patent/HUE061629T2/hu unknown
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- 2014-08-26 DK DK14761771.6T patent/DK3039146T3/da active
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- 2014-08-26 HR HRP20230015TT patent/HRP20230015T1/hr unknown
- 2014-08-26 FI FIEP20165345.8T patent/FI3702466T3/fi not_active Application Discontinuation
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CA2922500A1 (en) | 2015-03-05 |
PT3702466T (pt) | 2023-01-24 |
RS63942B1 (sr) | 2023-02-28 |
ES2936220T3 (es) | 2023-03-15 |
LT3702466T (lt) | 2023-02-27 |
DK3039146T3 (da) | 2020-08-10 |
EP3702466A1 (en) | 2020-09-02 |
RS60434B1 (sr) | 2020-07-31 |
US20190144868A1 (en) | 2019-05-16 |
CY1123272T1 (el) | 2021-12-31 |
EP4219727A2 (en) | 2023-08-02 |
HUE061629T2 (hu) | 2023-07-28 |
EP3702466B1 (en) | 2022-12-28 |
PT3039146T (pt) | 2020-08-11 |
HRP20230015T1 (hr) | 2023-02-17 |
ES2799437T3 (es) | 2020-12-17 |
HUE050546T2 (hu) | 2020-12-28 |
EP3039146B1 (en) | 2020-05-27 |
EP4219727A3 (en) | 2023-09-06 |
WO2015031392A1 (en) | 2015-03-05 |
SI3039146T1 (sl) | 2020-10-30 |
US10793861B2 (en) | 2020-10-06 |
US20160272976A1 (en) | 2016-09-22 |
LT3039146T (lt) | 2020-07-27 |
DK3702466T3 (da) | 2023-03-20 |
SI3702466T1 (sl) | 2023-04-28 |
FI3702466T3 (fi) | 2023-03-23 |
US20210108209A1 (en) | 2021-04-15 |
WO2015031392A9 (en) | 2015-06-18 |
EP3039146A1 (en) | 2016-07-06 |
PL3702466T3 (pl) | 2023-04-24 |
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