HRP20240099T1 - Sve-u-jedan aav vektori za liječenje koronavirus-induciranih bolesti - Google Patents
Sve-u-jedan aav vektori za liječenje koronavirus-induciranih bolesti Download PDFInfo
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- HRP20240099T1 HRP20240099T1 HRP20240099TT HRP20240099T HRP20240099T1 HR P20240099 T1 HRP20240099 T1 HR P20240099T1 HR P20240099T T HRP20240099T T HR P20240099TT HR P20240099 T HRP20240099 T HR P20240099T HR P20240099 T1 HRP20240099 T1 HR P20240099T1
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- Prior art keywords
- seq
- guide rna
- cas13d
- nucleic acid
- acid molecule
- Prior art date
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- 239000013598 vector Substances 0.000 title claims 11
- 201000010099 disease Diseases 0.000 title claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title claims 4
- 241000711573 Coronaviridae Species 0.000 title claims 2
- 239000013607 AAV vector Substances 0.000 claims 11
- 108020005004 Guide RNA Proteins 0.000 claims 10
- 108020004707 nucleic acids Proteins 0.000 claims 9
- 102000039446 nucleic acids Human genes 0.000 claims 9
- 150000007523 nucleic acids Chemical class 0.000 claims 9
- 239000013612 plasmid Substances 0.000 claims 7
- 239000000825 pharmaceutical preparation Substances 0.000 claims 6
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 102000004169 proteins and genes Human genes 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 3
- 108091006197 SARS-CoV-2 Nucleocapsid Protein Proteins 0.000 claims 2
- 241000700605 Viruses Species 0.000 claims 2
- 108020001507 fusion proteins Proteins 0.000 claims 2
- 102000037865 fusion proteins Human genes 0.000 claims 2
- 125000006850 spacer group Chemical group 0.000 claims 2
- 208000011580 syndromic disease Diseases 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 208000025721 COVID-19 Diseases 0.000 claims 1
- 241001678559 COVID-19 virus Species 0.000 claims 1
- 241000494545 Cordyline virus 2 Species 0.000 claims 1
- 241000127282 Middle East respiratory syndrome-related coronavirus Species 0.000 claims 1
- 241000315672 SARS coronavirus Species 0.000 claims 1
- 239000000443 aerosol Substances 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 210000004165 myocardium Anatomy 0.000 claims 1
- 239000006199 nebulizer Substances 0.000 claims 1
- 210000002345 respiratory system Anatomy 0.000 claims 1
Classifications
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- 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
- C12N15/1131—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 viruses
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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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
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- 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
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- 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
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
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- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10041—Use of virus, viral particle or viral elements as a vector
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Claims (16)
1. RNK vodič za uporabu sa Cas13 proteinom, naznačeno time što ciljno mjesto RNK vodiča je sekvenca koja je sačinjena od SARS-CoV-2 genoma, pri čemu sekvenca RNK vodiča sadrži sekvencu koja je odabrana iz grupe koja se sastoji od SEQ ID NO: 4, 7, 11, 15, 23, 27, 31.
2. Molekula nukleinske kiseline naznačena time što sadrži sekvencu koja kodira Cas13d protein i ekspresijsku kazetu RNK vodiča koja kodira RNK vodič u skladu sa patentnim zahtjevom 1 ili više od jednog RNK vodiča u skladu sa patentnim zahtjevom 1 i koja sadrži U6 promotor.
3. Molekula nukleinske kiseline u skladu sa patentnim zahtjevom 2, koja kodira RNK vodič naznačen time što sadrži sekvence SEQ ID NO: 4, 7, i 15; SEQ ID NO: 15, 23, i 31; SEQ ID NO: 15, 27, i 31; SEQ ID NO: 23, 27, i 31; SEQ ID NO: 4, 15, 23, 27, i 31; SEQ ID NO: 4, 7, 27, i 31; SEQ ID NO: 4, 23, i 31; SEQ ID NO: 7, 27, i 31; ili SEQ ID NO: 4, 7, 15, 23, 27, i 31, poželjno SEQ ID NO: 4, 7, 27, i 31.
4. Molekula nukleinske kiseline u skladu sa bilo kojim od patentnih zahtjeva 2 ili 3, naznačena time što je molekula nukleinske kiseline plazmid, poželjno jedan plazmid.
5. Molekula nukleinske kiseline u skladu sa bilo kojim od patentnih zahtjeva 2-4, naznačena time što navedeni Cas13d protein kodiran navedenom sekvencom ne sadrži nukleus-lokalizirajući signal (NLS) i/ili je fuzijski protein koji sadrži N-terminalnu vezujuću domenu (N-NTD) nukleokapsidnog proteina SARS-CoV-2.
6. Molekula nukleinske kiseline u skladu sa bilo kojim od patentnih zahtjeva 4-5, naznačena time što se molekula nukleinske kiseline može dobiti insertiranjem razmaknice sekvence RNK vodiča u skladu sa bilo kojim od patentnih zahtjeva 1-2 u plazmid pAAV2-U6-gRNA-CMV-Cas13d SEQ ID NO:40; ili insertiranjem najmanje jedne razmaknice sekvence najmanje jednog RNK vodiča u skladu sa bilo kojim od patentnih zahtjeva 1-2 u plazmid pAAV2-U6-gRNA-CMV-Cas13d-niz-triguide SEQ ID NO:41, plazmid pAAV-U6-gRNA-quadguide-CMV-Cas13d-V3-osnovni SEQ ID NO:42, plazmid pAAV-U6-gRNA-CMV-Cas13d-Sapl SEQ ID NO:43, ili plazmid pAAV-U6-gRNA-CMV-Cas13d-NTD-Aarl SEQ ID NO:44.
7. AAV vektor naznačen time što sadrži molekulu nukleinske kiseline prema bilo kojem od patentnih zahtjeva 2-6, poželjno pri čemu je AAV vektor AAV2 ili AAV9 vektor i/ili pri čemu je okosnica AAV vektora reducirane veličine.
8. Adenovirusni vektor naznačen time što sadrži molekula nukleinske kiseline prema bilo kojem od patentnih zahtjeva 2-6.
9. Farmaceutski pripravak naznačen time što sadrži AAV vektor prema patentnom zahtjevu 7 ili adenovirusni vektor prema patentnom zahtjevu 8.
10. Farmaceutski pripravak naznačen time što sadrži najmanje jedan RNK vodič prema patentnom zahtjevu 1 i najmanje jednu iRNK koja kodira Cas13 protein, poželjno pri čemu navedeni Cas13 protein kodiran navedenom iRNK je Cas13d i ne sadrži nukleus-lokalizirajući signal (NLS).
11. Farmaceutski pripravak u skladu sa bilo kojim od patentnih zahtjeva 9-10, naznačen time što je navedeni Cas13d protein kodiran navedenom iRNK fuzijski protein koji sadrži N- terminalnu vezujuću domenu (N-NTD) nukleokapsidnog proteina SARS-CoV-2.
12. AAV vektor u skladu sa patentnim zahtjevom 7, adenovirusni vektor u skladu sa patentnim zahtjevom 8, ili farmaceutski pripravak u skladu sa bilo kojim od patentnih zahtjeva 9-11 za uporabu u liječenju humano-povezanim virusom uzrokovane bolesti ili sindroma kod pacijenta kojem je to potrebno.
13. AAV vektor, adenovirusni vektor, ili farmaceutski pripravak za uporabu u skladu sa patentnim zahtjevom 12, naznačen time što je bolest ili sindrom rezultat infekcije koronavirusom koji je genetički srodan sa grupom koja se sastoji od MERS-CoV, SARS-CoV i SARS-CoV-2, poželjno pri čemu je bolest COVID-19.
14. AAV vektor, adenovirusni vektor, ili farmaceutski pripravak za uporabu u skladu sa bilo kojim od patentnih zahtjeva 12-13, naznačeno time što Cas13 nakon ekspresije cijepa humano-povezani virus.
15. AAV vektor, adenovirusni vektor, ili farmaceutski pripravak za uporabu u skladu sa bilo kojim od patentnih zahtjeva 12-14, naznačen time što se AAV vektor, adenovirusni vektor, ili farmaceutski pripravak primjenjuju putem gornjeg respiratornog trakta, poželjno intranazalno ili intratrahealno ili u aerosolnom pripravku kroz inhalator ili nebulizator, poželjno kroz respirator.
16. AAV vektor, adenovirusni vektor, ili farmaceutski pripravak za uporabu u skladu sa bilo kojim od patentnih zahtjeva 12-15, naznačen time što se AAV vektor, adenovirusni vektor, ili farmaceutski pripravak primjenjuju u miokard.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063006996P | 2020-04-08 | 2020-04-08 | |
| PCT/EP2021/056221 WO2021204492A1 (en) | 2020-04-08 | 2021-03-11 | All-in-one aav vectors for treating coronavirus-induced diseases |
| EP21715779.1A EP4034661B1 (en) | 2020-04-08 | 2021-03-11 | All-in-one aav vectors for treating coronavirus-induced diseases |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| HRP20240099T1 true HRP20240099T1 (hr) | 2024-05-24 |
| HRP20240099T8 HRP20240099T8 (hr) | 2024-06-21 |
Family
ID=75339666
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20240099TT HRP20240099T8 (hr) | 2020-04-08 | 2021-03-11 | Sve-u-jedan aav vektori za liječenje koronavirus-induciranih bolesti |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230242914A1 (hr) |
| EP (1) | EP4034661B1 (hr) |
| JP (1) | JP2023520814A (hr) |
| CN (1) | CN115667512A (hr) |
| AU (1) | AU2021253344A1 (hr) |
| CA (1) | CA3174832A1 (hr) |
| ES (1) | ES2967509T3 (hr) |
| HR (1) | HRP20240099T8 (hr) |
| HU (1) | HUE065161T2 (hr) |
| PL (1) | PL4034661T3 (hr) |
| RS (1) | RS65372B1 (hr) |
| WO (1) | WO2021204492A1 (hr) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023158379A2 (en) * | 2022-02-16 | 2023-08-24 | Agency For Science, Technology And Research | Methods of preventing or treating an rna viral infection in a subject |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE102014207153A1 (de) | 2014-04-14 | 2015-10-15 | Rabea Hinkel | AAV-Vektoren für vaskuläre Gentherapie in koronarer Herzerkrankung und peripherer Ischämie |
| US10476825B2 (en) * | 2017-08-22 | 2019-11-12 | Salk Institue for Biological Studies | RNA targeting methods and compositions |
| WO2021183887A1 (en) * | 2020-03-13 | 2021-09-16 | The Board Of Trustees Of The Leland Stanford Junior University | Systems and methods for viral genome targeting |
-
2021
- 2021-03-11 US US17/917,958 patent/US20230242914A1/en active Pending
- 2021-03-11 CN CN202180026993.8A patent/CN115667512A/zh active Pending
- 2021-03-11 HR HRP20240099TT patent/HRP20240099T8/hr unknown
- 2021-03-11 HU HUE21715779A patent/HUE065161T2/hu unknown
- 2021-03-11 EP EP21715779.1A patent/EP4034661B1/en active Active
- 2021-03-11 CA CA3174832A patent/CA3174832A1/en active Pending
- 2021-03-11 RS RS20231159A patent/RS65372B1/sr unknown
- 2021-03-11 ES ES21715779T patent/ES2967509T3/es active Active
- 2021-03-11 AU AU2021253344A patent/AU2021253344A1/en active Pending
- 2021-03-11 JP JP2022562056A patent/JP2023520814A/ja active Pending
- 2021-03-11 PL PL21715779.1T patent/PL4034661T3/pl unknown
- 2021-03-11 WO PCT/EP2021/056221 patent/WO2021204492A1/en active Application Filing
Also Published As
| Publication number | Publication date |
|---|---|
| HRP20240099T8 (hr) | 2024-06-21 |
| EP4034661C0 (en) | 2023-09-06 |
| ES2967509T3 (es) | 2024-04-30 |
| HUE065161T2 (hu) | 2024-05-28 |
| RS65372B1 (sr) | 2024-04-30 |
| EP4034661A1 (en) | 2022-08-03 |
| CA3174832A1 (en) | 2021-10-14 |
| EP4034661B1 (en) | 2023-09-06 |
| CN115667512A (zh) | 2023-01-31 |
| JP2023520814A (ja) | 2023-05-19 |
| AU2021253344A1 (en) | 2022-12-01 |
| US20230242914A1 (en) | 2023-08-03 |
| PL4034661T3 (pl) | 2024-03-18 |
| WO2021204492A1 (en) | 2021-10-14 |
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