HRP20201544T1 - Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) - Google Patents
Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) Download PDFInfo
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- HRP20201544T1 HRP20201544T1 HRP20201544TT HRP20201544T HRP20201544T1 HR P20201544 T1 HRP20201544 T1 HR P20201544T1 HR P20201544T T HRP20201544T T HR P20201544TT HR P20201544 T HRP20201544 T HR P20201544T HR P20201544 T1 HRP20201544 T1 HR P20201544T1
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- hotc
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- acid sequence
- viral vector
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- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 title claims 6
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 title 1
- 230000007812 deficiency Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 150000007523 nucleic acids Chemical group 0.000 claims 18
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 15
- 239000013603 viral vector Substances 0.000 claims 13
- 102100028200 Ornithine transcarbamylase, mitochondrial Human genes 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 5
- 239000013598 vector Substances 0.000 claims 5
- 210000000234 capsid Anatomy 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108091026890 Coding region Proteins 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 101000986595 Homo sapiens Ornithine transcarbamylase, mitochondrial Proteins 0.000 claims 2
- 102000002248 Thyroxine-Binding Globulin Human genes 0.000 claims 2
- 108010000259 Thyroxine-Binding Globulin Proteins 0.000 claims 2
- 210000005229 liver cell Anatomy 0.000 claims 2
- 201000011278 ornithine carbamoyltransferase deficiency Diseases 0.000 claims 2
- XUIIKFGFIJCVMT-UHFFFAOYSA-N thyroxine-binding globulin Natural products IC1=CC(CC([NH3+])C([O-])=O)=CC(I)=C1OC1=CC(I)=C(O)C(I)=C1 XUIIKFGFIJCVMT-UHFFFAOYSA-N 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 1
- 108091092195 Intron Proteins 0.000 claims 1
- 208000000599 Ornithine Carbamoyltransferase Deficiency Disease Diseases 0.000 claims 1
- 206010052450 Ornithine transcarbamoylase deficiency Diseases 0.000 claims 1
- 208000035903 Ornithine transcarbamylase deficiency Diseases 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 238000004806 packaging method and process Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
- C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
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- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- 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
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- C12Y201/00—Transferases transferring one-carbon groups (2.1)
- C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
- C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
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- C12N2710/10011—Adenoviridae
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- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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Claims (17)
1. Rekombinantni virusni vektor koji sadrži sekvencu nukleinske kiseline koja kodira protein ljudske ornitinske transkarbamilaze (hOTC) i kontrolne sekvence ekspresije koje usmjeravaju ekspresiju hOTC u stanici jetre, pri čemu je sekvenca hOTC nukleinske kiseline manje od 80% identična sekvenci hOTC divljeg tipa SEQ ID NO: 1 koja kodira zreli ili pune duljine hOTC protein, i izražava funkcionalni hOTC, pri čemu je navedena sekvenca nukleinske kiseline hOTC odabrana između sekvence nukleinske kiseline koja sadrži SEQ ID NO: 5 ili sekvence nukleinske kiseline najmanje 96% do 99% identičnu tome.
2. Rekombinantni virusni vektor u skladu s patentnim zahtjevom 1, naznačen time što hOTC sekvenca nukleinske kiseline ima sekvencu SEQ ID NO: 5.
3. Rekombinantni virusni vektor u skladu s patentnim zahtjevom 1, naznačen time što hOTC sekvenca nukleinske kiseline ima sekvencu SEQ ID NO: 4 ili SEQ ID NO: 3.
4. Rekombinantni virusni vektor u skladu s bilo kojim od patentnih zahtjeva 1 do 3, naznačen time što hOTC je himerni OTC koji sadrži heterolognu tranzitnu sekvencu supstituiranu za prirodnu tranzitnu sekvencu SEQ ID NO: 5.
5. Rekombinantni virusni vektor u skladu s bilo kojim od patentnih zahtjeva 1 do 4, naznačen time što je virusni vektor odabran od vektora adeno-povezanog virusa (AAV), adenovirusnog vektora i lentivirusnog vektora.
6. Rekombinantni virusni vektor u skladu s bilo kojim od patentnih zahtjeva 1 do 5, naznačen time što sekvence kontrole ekspresije sadrže promotor specifičan za jetru, izborno gdje promotor specifičan za jetru je promotor globulina koji veže tiroksin (TBG).
7. Rekombinantni virusni vektor u skladu s bilo kojim od patentnih zahtjeva 1 do 6, naznačen time što kazeta ekspresije nadalje sadrži jedan ili više introna, Kozakovu sekvencu, poli A, i regulatorne elemente nakon transkripcije.
8. Rekombinantni virusni vektor u skladu s patentnim zahtjevom 1, naznačen time što je rekombinantni virusni vektor rekombinantni AAV vektor koji sadrži kapsid AAV koji je u njega zapakirao sekvencu nukleinske kiseline koja sadrži najmanje jednu ITR sekvencu i nukleinsku kiselinu koja kodira hOTC, izborno gdje je kapsid AAV odabran između AAV8, AAV9 i / ili AAVrh10.
9. Rekombinantni adeno-povezani virus (rAAV) koji ima kapsid AAV i u njega je zapakirao kazetu ekspresije koja sadrži najmanje jednu AAV sekvencu obrnutog terminalnog ponavljanja (ITR), sekvencu nukleinske kiseline koja kodira barem zrelu ljudsku ornitinsku transkarbamilazu (hOTC), i kontrolnu sekvencu ekspresiju koje usmjeravaju ekspresiju hOTC u stanici jetre, navedene kontrolne sekvence ekspresije sadrže promotor specifičan za jetru, pri čemu je hOTC sekvenca nukleinske kiseline manje od 80% identična hOTC sekvenci divljeg tipa SEQ ID NO: 1 koja kodira zreli hOTC protein i sadrži barem sekvencu nukleinske kiseline koja kodira zreli hOTC protein SEQ ID NO: 5 ili sekvencu nukleinske kiseline najmanje 96% do 99.9% identičan tome.
10. RAAV u skladu s patentnim zahtjevom 9, naznačen time što hOTC sekvenca nukleinske kiseline ima sekvencu SEQ ID NO: 5.
11. RAAV u skladu s bilo kojim od patentnih zahtjeva 8 do 10, naznačen time što je kapsid AAV odabran između AAV8, AAV9 ili AAVrh10.
12. RAAV u skladu s patentnim zahtjevom 11, naznačen time što kazeta ekspresije nadalje sadrži 5' AAV sekvencu invertiranog terminala ponovljanja (ITR) i 3' ITR sekvencu ili gdje barem jedan AAV ITR sadrži 5' ITR u kojem D-sekvenca i mjesto razlučivosti terminala se briše ili gdje su 5' i 3' ITR-ovi iz AAV2.
13. RAAV u skladu s bilo kojim od patentnih zahtjeva 9 do 12, naznačen time što nukleinska kiselina koja kodira hOTC ima kodirajuću sekvencu SEQ ID NO: 3 ili, gdje nukleinska kiselina koja kodira hOTC ima kodirajuću sekvencu SEQ ID NO: 4.
14. Virusni vektor koji sadrži hOTC gen koji kodira himernu ornitinsku transkarbamilazu koja sadrži barem zrelu ljudsku ornitinsku transkarbamilazu s heterolognom tranzitnom sekvencom, pri čemu je sekvenca nukleinske kiseline koja kodira zrelu ljudsku ornitinsku transkarbamilazu odabranu između sekvence nukleinske kiseline SEQ ID NO : 3, 4 ili 5.
15. Virusni vektor u skladu s patentnim zahtjevom 14, naznačen time što je kodirajuća sekvenca hOTC sekvenca SEQ ID NO: 5.
16. Farmaceutski pripravak koji sadrži nosač i učinkovitu količinu vektora u skladu sa bilo kojim od patentnih zahtjeva 1 do 8, rAAV u skladu sa bilo kojim od patentnih zahtjeva 9 do 13, i / ili u skladu sa patentnim zahtjevom 14 ili 15.
17. Virusni vektor u skladu sa bilo kojim od patentnih zahtjeva 1 do 8, rAAV u skladu sa bilo kojim od patentnih zahtjeva 9 do 13, i / ili vektor u skladu sa patentnim zahtjevom 14 ili 15 za uporabu u postupku liječenja nedostatka ornitinske transkarbamilaze (OTCD) kod ljudskog pacijenta.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 | |
| PCT/US2015/019513 WO2015138348A1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
| EP15712226.8A EP3116900B1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20201544T1 true HRP20201544T1 (hr) | 2021-02-19 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20201544TT HRP20201544T1 (hr) | 2014-03-09 | 2020-09-28 | Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) |
Country Status (22)
| Country | Link |
|---|---|
| US (6) | US10167454B2 (hr) |
| EP (2) | EP3778627A1 (hr) |
| JP (2) | JP6822841B2 (hr) |
| KR (1) | KR102390075B1 (hr) |
| AU (2) | AU2015229750A1 (hr) |
| BR (2) | BR112016020688B1 (hr) |
| CA (1) | CA2939950C (hr) |
| CL (2) | CL2016002235A1 (hr) |
| CY (1) | CY1123378T1 (hr) |
| DK (1) | DK3116900T3 (hr) |
| ES (1) | ES2821938T3 (hr) |
| HR (1) | HRP20201544T1 (hr) |
| HU (1) | HUE051311T2 (hr) |
| IL (2) | IL247329B (hr) |
| LT (1) | LT3116900T (hr) |
| MX (1) | MX2016011686A (hr) |
| PL (1) | PL3116900T3 (hr) |
| PT (1) | PT3116900T (hr) |
| RS (1) | RS60902B1 (hr) |
| SI (1) | SI3116900T1 (hr) |
| SM (1) | SMT202000531T1 (hr) |
| WO (2) | WO2015138357A2 (hr) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20140029468A (ko) | 2011-04-29 | 2014-03-10 | 셀렉타 바이오사이언시즈, 인크. | 항체 반응을 감소시키기 위한 관용원성 합성 나노운반체 |
| IL284924B2 (en) | 2013-05-03 | 2023-04-01 | Selecta Biosciences Inc | Adjacent, local administration of tolerogenic synthetic nanocarriers to reduce type I and type IV hypersensitivity |
| CA2919828C (en) | 2013-07-30 | 2022-07-19 | Phaserx, Inc. | Block copolymers and their conjugates or complexes with oligonucleotides |
| WO2015138357A2 (en) | 2014-03-09 | 2015-09-17 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of otc deficency |
| CA2957800A1 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector immune responses |
| AU2015343037B2 (en) | 2014-11-05 | 2019-01-17 | Voyager Therapeutics, Inc. | AADC polynucleotides for the treatment of parkinson's disease |
| WO2016118697A1 (en) | 2015-01-21 | 2016-07-28 | Phaserx, Inc. | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
| WO2017059902A1 (en) | 2015-10-07 | 2017-04-13 | Biontech Rna Pharmaceuticals Gmbh | 3' utr sequences for stabilization of rna |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
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