HRP20230015T1 - Proizvodi i postupci liječenja amiotrofične lateralne skleroze - Google Patents
Proizvodi i postupci liječenja amiotrofične lateralne skleroze Download PDFInfo
- Publication number
- HRP20230015T1 HRP20230015T1 HRP20230015TT HRP20230015T HRP20230015T1 HR P20230015 T1 HRP20230015 T1 HR P20230015T1 HR P20230015T T HRP20230015T T HR P20230015TT HR P20230015 T HRP20230015 T HR P20230015T HR P20230015 T1 HRP20230015 T1 HR P20230015T1
- Authority
- HR
- Croatia
- Prior art keywords
- associated virus
- recombinant adeno
- aav
- preparation
- virus according
- Prior art date
Links
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 title claims 5
- 241000702421 Dependoparvovirus Species 0.000 claims 18
- 238000002360 preparation method Methods 0.000 claims 10
- 102000008221 Superoxide Dismutase-1 Human genes 0.000 claims 6
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 6
- 210000000234 capsid Anatomy 0.000 claims 3
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 239000002872 contrast media Substances 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- AMDBBAQNWSUWGN-UHFFFAOYSA-N Ioversol Chemical compound OCCN(C(=O)CO)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I AMDBBAQNWSUWGN-UHFFFAOYSA-N 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 229960001025 iohexol Drugs 0.000 claims 1
- NTHXOOBQLCIOLC-UHFFFAOYSA-N iohexol Chemical compound OCC(O)CN(C(=O)C)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I NTHXOOBQLCIOLC-UHFFFAOYSA-N 0.000 claims 1
- 229960004647 iopamidol Drugs 0.000 claims 1
- XQZXYNRDCRIARQ-LURJTMIESA-N iopamidol Chemical compound C[C@H](O)C(=O)NC1=C(I)C(C(=O)NC(CO)CO)=C(I)C(C(=O)NC(CO)CO)=C1I XQZXYNRDCRIARQ-LURJTMIESA-N 0.000 claims 1
- 229960000824 iopentol Drugs 0.000 claims 1
- IUNJANQVIJDFTQ-UHFFFAOYSA-N iopentol Chemical compound COCC(O)CN(C(C)=O)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I IUNJANQVIJDFTQ-UHFFFAOYSA-N 0.000 claims 1
- 229960002603 iopromide Drugs 0.000 claims 1
- DGAIEPBNLOQYER-UHFFFAOYSA-N iopromide Chemical compound COCC(=O)NC1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)N(C)CC(O)CO)=C1I DGAIEPBNLOQYER-UHFFFAOYSA-N 0.000 claims 1
- 229960004537 ioversol Drugs 0.000 claims 1
- -1 jomeprole Chemical compound 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 101150066583 rep gene Proteins 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 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
- C12N15/11—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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- 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)
-
- C—CHEMISTRY; METALLURGY
- 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)
-
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
- 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
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2750/14041—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
-
- 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
Landscapes
- 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)
- Wood Science & Technology (AREA)
- Zoology (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)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (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 (18)
1. Rekombinantni adeno-povezani virus koji sadrži DNA koja kodira shRNA za superoksid dismutazu 1 (SOD1) ima sekvencu SEQ ID NO: 4, sekvencu ukosnice i sekvencu komplementarnu sa SEQ ID NO: 4, naznačen time što virusni genom nema rep gen i nedostaje mu cap gen.
2. Rekombinantni adeno-povezani virus prema zahtjevu 1, naznačen time što nadalje sadrži promotor operativno vezan na DNA koja kodira shRNA za superoksid dismutazu 1 (SOD1).
3. Rekombinantni adeno-povezani virus prema zahtjevu 2, naznačen time što je promotor H1 promotor.
4. Rekombinantni adeno-povezani virus prema zahtjevu 3, naznačen time što H1 promotor sadrži nukleotide 966 do 1064 iz SEQ ID NO: 20.
5. Rekombinantni adeno-povezani virus prema bilo kojem od zahtjeva 1-4, naznačen time što nadalje sadrži sekvencu za punjenje.
6. Rekombinantni adeno-povezani virus prema zahtjevu 5, naznačen time što sekvenca za punjenje sadrži SEQ ID NO: 22.
7. Rekombinantni adeno-povezani virus prema bilo kojem od zahtjeva 1-6, naznačen time što je virusni genom samokomplementarni genom.
8. Rekombinantni adeno-povezani virus prema bilo kojem od zahtjeva 1-7, naznačen time što sadrži kapsidu izvedenu iz AAV-1, AAV-2, AAV-3, AAV-4, AAV-5, AAV-6, AAV-7, AAV-8, AAV-9, AAV-10, AAV-11, AAV-rh74.
9. Rekombinantni adeno-povezani virus prema zahtjevu 8, naznačen time što je kapsida AAV9 kapsida.
10. Pripravak koji sadrži rekombinantni adeno-povezani virus prema bilo kojem od zahtjeva 1-9 i farmaceutski prihvatljiv nosač.
11. Pripravak prema zahtjevu 10, za upotrebu u kombinaciji s kontrastnim sredstvom.
12. Pripravak prema zahtjevu 11, naznačen time što je kontrastno sredstvo jobitridol, joheksol, jomeprol, jopamidol, jopentol, jopromid, joversol ili joksilan.
13. Rekombinantni adeno-povezani virus prema bilo kojem od zahtjeva 1-9 ili pripravak prema bilo kojem od zahtjeva 10-12, naznačen time što se koristi u liječenju amiotrofične lateralne skleroze (ALS).
14. Rekombinantni adeno-povezani virus ili pripravak za upotrebu prema zahtjevu 13, naznačen time što je ALS obiteljski.
15. Rekombinantni adeno-povezani virus ili pripravak za upotrebu prema zahtjevu 14, naznačen time što je ALS uzrokovan jednom ili više mutacija u superoksid dismutazi 1 (SOD1).
16. Rekombinantni adeno-povezani virus ili pripravak za upotrebu prema bilo kojem od zahtjeva 13-15, naznačen time što je rekombinantni adeno-povezani virus ili pripravak za sistemsku primjenu.
17. Rekombinantni adeno-povezani virus ili pripravak za upotrebu prema bilo kojem od zahtjeva 13-15, naznačen time što je rekombinantni adeno-povezani virus ili pripravak za intratekalnu primjenu.
18. Rekombinantni adeno-povezani virus prema bilo kojem od zahtjeva 1 do 9, naznačen time što se koristi u terapiji.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361870585P | 2013-08-27 | 2013-08-27 | |
| EP20165345.8A EP3702466B1 (en) | 2013-08-27 | 2014-08-26 | Products and methods for treatment of amyotrophic lateral sclerosis |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20230015T1 true HRP20230015T1 (hr) | 2023-02-17 |
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 After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20200862TT HRP20200862T1 (hr) | 2013-08-27 | 2020-05-29 | Proizvodi i metode 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3643782A1 (en) | 2008-02-11 | 2020-04-29 | Phio Pharmaceuticals Corp. | Modified rnai polynucleotides and uses thereof |
| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| 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 |
| US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
| 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 |
| WO2017176929A1 (en) | 2016-04-05 | 2017-10-12 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| IL305449A (en) | 2016-04-15 | 2023-10-01 | Univ Pennsylvania | Gene therapy for the treatment of type II mucositis |
| KR102392236B1 (ko) | 2016-05-18 | 2022-05-03 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| KR20190055086A (ko) | 2016-08-19 | 2019-05-22 | 칼리뮨, 인코포레이티드 | 재조합 자기-상보적인 아데노-부속 바이러스를 이용한 병태의 치료 방법 및 조성물 |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| WO2018106956A2 (en) | 2016-12-07 | 2018-06-14 | University Of Florida Research Foundation, Incorporated | IL-1RA CDNAs |
| US20210108197A1 (en) * | 2017-03-31 | 2021-04-15 | Staidson (Beijing) Biopharmaceuticals Co., Ltd. | Shrna expression cassette, polynucleotide sequence carrying same, and application thereof |
| CN110913866A (zh) | 2017-05-05 | 2020-03-24 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| EP3684937A4 (en) | 2017-09-22 | 2021-06-02 | University of Massachusetts | DUAL EXPRESSION VECTORS OF SOD1 AND THEIR USES |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| JP7502991B2 (ja) | 2017-10-16 | 2024-06-19 | ボイジャー セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症（ａｌｓ）の治療 |
| TWI827560B (zh) | 2017-11-08 | 2024-01-01 | 瑞士商諾華股份有限公司 | 用於製備病毒載體之手段及方法與其用途 |
| CN118043039A (zh) | 2021-10-25 | 2024-05-14 | 诺华股份有限公司 | 用于改善腺相关病毒（aav）递送的方法 |
| WO2024015794A2 (en) * | 2022-07-12 | 2024-01-18 | The Research Institute At Nationwide Children's Hospital | Adeno-associated virus gene therapy products and methods |
Family Cites Families (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5448517A (en) | 1987-06-29 | 1995-09-05 | Kabushiki Kaisha Toshiba | Electrically programmable nonvolatile semiconductor memory device with NAND cell structure |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5837484A (en) | 1993-11-09 | 1998-11-17 | Medical College Of Ohio | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| CA2176117C (en) | 1993-11-09 | 2006-01-03 | Terence R. Flotte | Generation of high titers of recombinant aav vectors |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| WO1996017947A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| US6143548A (en) | 1995-08-30 | 2000-11-07 | Genzyme Corporation | Chromatographic purification of adeno-associated virus (AAV) |
| EP0850313B8 (en) | 1995-09-08 | 2009-07-29 | Genzyme Corporation | Improved aav vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| AU723497C (en) | 1996-09-06 | 2001-10-11 | Trustees Of The University Of Pennsylvania, The | Method for recombinant adeno-associated virus-directed gene therapy |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| WO2001083692A2 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2005096781A2 (en) * | 2004-04-06 | 2005-10-20 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using rna interference |
| WO2006066203A2 (en) * | 2004-12-16 | 2006-06-22 | Alsgen, Llc | Small interfering rna (sirna) molecules for modulating superoxide dismutase (sod) |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| EP3643782A1 (en) * | 2008-02-11 | 2020-04-29 | Phio Pharmaceuticals Corp. | Modified rnai polynucleotides and uses thereof |
| WO2010071832A1 (en) | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| EP2826860B1 (en) | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
| WO2012058220A2 (en) * | 2010-10-26 | 2012-05-03 | University Of Massachusetts | Anti-sod1 antibodies and uses thereof |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| AU2013337651B2 (en) * | 2012-11-01 | 2018-12-13 | Factor Bioscience Inc. | Methods and products for expressing proteins in cells |
| HUE061629T2 (hu) * | 2013-08-27 | 2023-07-28 | Res Inst Nationwide Childrens Hospital | Termékek és módszerek amyotrophiás lateralis sclerosis kezelésére |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
-
2014
- 2014-08-26 HU HUE20165345A patent/HUE061629T2/hu unknown
- 2014-08-26 PL PL20165345.8T patent/PL3702466T3/pl unknown
- 2014-08-26 DK DK14761771.6T patent/DK3039146T3/da active
- 2014-08-26 PT PT201653458T patent/PT3702466T/pt unknown
- 2014-08-26 HR HRP20230015TT patent/HRP20230015T1/hr unknown
- 2014-08-26 FI FIEP20165345.8T patent/FI3702466T3/fi not_active Application Discontinuation
- 2014-08-26 ES ES20165345T patent/ES2936220T3/es active Active
- 2014-08-26 HU HUE14761771A patent/HUE050546T2/hu unknown
- 2014-08-26 EP EP22215568.1A patent/EP4219727A3/en active Pending
- 2014-08-26 SI SI201432021T patent/SI3702466T1/sl unknown
- 2014-08-26 WO PCT/US2014/052753 patent/WO2015031392A1/en active Application Filing
- 2014-08-26 RS RS20200638A patent/RS60434B1/sr unknown
- 2014-08-26 CA CA2922500A patent/CA2922500A1/en active Pending
- 2014-08-26 ES ES14761771T patent/ES2799437T3/es active Active
- 2014-08-26 RS RS20230086A patent/RS63942B1/sr unknown
- 2014-08-26 US US14/914,861 patent/US20160272976A1/en not_active Abandoned
- 2014-08-26 DK DK20165345.8T patent/DK3702466T3/da active
- 2014-08-26 EP EP20165345.8A patent/EP3702466B1/en active Active
- 2014-08-26 LT LTEP14761771.6T patent/LT3039146T/lt unknown
- 2014-08-26 PT PT147617716T patent/PT3039146T/pt unknown
- 2014-08-26 EP EP14761771.6A patent/EP3039146B1/en active Active
- 2014-08-26 SI SI201431633T patent/SI3039146T1/sl unknown
- 2014-08-26 LT LTEP20165345.8T patent/LT3702466T/lt unknown
-
2018
- 2018-07-20 US US16/041,381 patent/US10793861B2/en active Active
-
2020
- 2020-05-29 HR HRP20200862TT patent/HRP20200862T1/hr unknown
- 2020-08-04 CY CY20201100717T patent/CY1123272T1/el unknown
- 2020-08-19 US US16/997,304 patent/US20210108209A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| 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 |
| HRP20200862T1 (hr) | 2020-08-21 |
| PT3039146T (pt) | 2020-08-11 |
| 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 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| HRP20230015T1 (hr) | Proizvodi i postupci liječenja amiotrofične lateralne skleroze | |
| HRP20201324T1 (hr) | Vektori aav koji sadrže faktor viii koji kodira gen | |
| JP2020019772A5 (hr) | ||
| WO2016077687A4 (en) | Compositions and methods of treating amyotrophic lateral sclerosis (als) | |
| JP2021500049A5 (hr) | ||
| JP6366581B2 (ja) | 改変されたアデノ随伴ウイルスベクター組成物 | |
| JP2019513393A5 (hr) | ||
| FI3684423T3 (fi) | Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä | |
| HRP20201544T1 (hr) | Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) | |
| WO2016137949A4 (en) | Regulatable expression using adeno-associated virus (aav) | |
| JP2019513779A5 (hr) | ||
| JP2020522269A5 (hr) | ||
| JP2018148927A5 (hr) | ||
| FI3596222T3 (fi) | Lihasspesifisen mikrodystrofiinin adenoassosioitu virusvektori -kuljetus lihasdystrofian hoitamiseksi | |
| JP2014012022A5 (hr) | ||
| RU2017125234A (ru) | Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний | |
| JP2020513811A5 (hr) | ||
| JP2020532981A5 (hr) | ||
| JP2016503405A5 (hr) | ||
| EP2660325A3 (en) | AAV vectors and corresponding nucleotide sequences and methods | |
| JP2019533439A5 (hr) | ||
| JP2019503649A5 (hr) | ||
| JP2017536116A5 (hr) | ||
| JP2015523379A5 (hr) | ||
| JP2014512171A5 (hr) |