HRP20210265T1 - Terapije na temelju reverzne transkriptaze telomeraze - Google Patents
Terapije na temelju reverzne transkriptaze telomeraze Download PDFInfo
- Publication number
- HRP20210265T1 HRP20210265T1 HRP20210265TT HRP20210265T HRP20210265T1 HR P20210265 T1 HRP20210265 T1 HR P20210265T1 HR P20210265T T HRP20210265T T HR P20210265TT HR P20210265 T HRP20210265 T HR P20210265T HR P20210265 T1 HRP20210265 T1 HR P20210265T1
- Authority
- HR
- Croatia
- Prior art keywords
- vector
- nucleic acid
- use according
- adeno
- sequence
- Prior art date
Links
- 108010017842 Telomerase Proteins 0.000 title claims 7
- 102100032938 Telomerase reverse transcriptase Human genes 0.000 title claims 7
- 238000002560 therapeutic procedure Methods 0.000 title 1
- 150000007523 nucleic acids Chemical class 0.000 claims 15
- 108020004707 nucleic acids Proteins 0.000 claims 12
- 102000039446 nucleic acids Human genes 0.000 claims 12
- 241000702421 Dependoparvovirus Species 0.000 claims 6
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 210000000234 capsid Anatomy 0.000 claims 3
- 230000001105 regulatory effect Effects 0.000 claims 3
- 108091026890 Coding region Proteins 0.000 claims 2
- 241000701022 Cytomegalovirus Species 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 208000005069 pulmonary fibrosis Diseases 0.000 claims 1
Classifications
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- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/45—Transferases (2)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0271—Chimeric animals, e.g. comprising exogenous cells
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0276—Knockout animals
-
- 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
-
- 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
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
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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
- C12N9/10—Transferases (2.)
- C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
- C12N9/1241—Nucleotidyltransferases (2.7.7)
- C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/07—Nucleotidyltransferases (2.7.7)
- C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2207/00—Modified animals
- A01K2207/12—Animals modified by administration of exogenous cells
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/20—Animal model comprising regulated expression system
- A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0381—Animal model for diseases of the hematopoietic system
-
- 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
Claims (11)
1. Vektor nukleinske kiseline koji sadrži kodirajuću sekvencu za reverznu transkriptazu telomeraze (TERT) za uporabu u liječenju plućne fibroze.
2. Vektor nukleinske kiseline za uporabu prema zahtjevu 1, naznačen time što je TERT kodiran sekvencom nukleinske kiseline koja sadrži sekvencu SEQ ID NO: 1 ili SEQ ID NO: 3.
3. Vektor nukleinske kiseline za uporabu prema bilo kojem od zahtjeva 1-2, naznačen time što TERT sadrži aminokiselinsku sekvencu SEQ ID NO: 2 ili SEQ ID NO: 4.
4. Vektor nukleinske kiseline za uporabu prema bilo kojem od zahtjeva 1-3, naznačen time što je sekvenca nukleinske kiseline koja kodira TERT operativno povezana s regulatornom sekvencom koja pokreće ekspresiju kodirajuće sekvence.
5. Vektor nukleinske kiseline za uporabu prema bilo kojem od zahtjeva 1-4, naznačen time što je vektor ne-integrativni vektor.
6. Vektor nukleinske kiseline za uporabu prema bilo kojem od zahtjeva 1-5, naznačen time što je vektor ne-integrativni vektor zasnovan na adeno-povezanom virusu.
7. Vektor nukleinske kiseline za uporabu prema bilo kojem od zahtjeva 1-6, naznačen time što vektor je adeno-povezani vektor zasnovan na virusu izveden iz adeno-povezanog virusa serotipa 9 (AAV9).
8. Vektor nukleinske kiseline za uporabu prema zahtjevu 7, naznačen time što je kapsida adeno-povezanog vektora zasnovanog na virusu izrađena od kapsidnih proteina adeno-povezanog virusa serotipa 9 (AAV9), i sekvenca nukleinske kiseline sadržana u kapsidi je s oba kraja završena unutarnjim terminalnim ponavljanjima koja odgovaraju adeno-povezanom virusu serotipa 2.
9. Vektor nukleinske kiseline za uporabu prema zahtjevu 8, naznačen time što nukleinska kiselina sadržana u kapsidi sadrži fragment koji kodira aminokiselinsku sekvencu koja kodira za TERT.
10. Vektor nukleinske kiseline za uporabu prema bilo kojem od zahtjeva 1-9, naznačen time što vektor sadrži regulatornu sekvencu koja je konstitutivni promotor.
11. Vektor nukleinske kiseline za uporabu prema zahtjevu 10, naznačen time što je regulatorna sekvenca promotor citomegalovirusa (CMV).
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP14382312 | 2014-08-08 | ||
EP18215767.7A EP3485914B1 (en) | 2014-08-08 | 2015-08-04 | Telomerase reverse transcriptase-based therapies |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20210265T1 true HRP20210265T1 (hr) | 2021-04-02 |
Family
ID=51454634
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20210265TT HRP20210265T1 (hr) | 2014-08-08 | 2021-02-16 | Terapije na temelju reverzne transkriptaze telomeraze |
Country Status (13)
Country | Link |
---|---|
US (3) | US11529396B2 (hr) |
EP (4) | EP3485914B1 (hr) |
JP (1) | JP6676262B2 (hr) |
CY (1) | CY1124012T1 (hr) |
DK (2) | DK3485914T3 (hr) |
ES (2) | ES2864731T3 (hr) |
HR (1) | HRP20210265T1 (hr) |
HU (1) | HUE053526T2 (hr) |
LT (1) | LT3485914T (hr) |
PL (1) | PL3485914T3 (hr) |
PT (1) | PT3485914T (hr) |
SI (1) | SI3485914T1 (hr) |
WO (1) | WO2016020345A1 (hr) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN116870195A (zh) * | 2020-09-22 | 2023-10-13 | 浙江愈方生物科技有限公司 | 一种灭活性端粒酶、具有其的腺病毒和人造mRNA及应用 |
EP4089171A1 (en) | 2021-05-12 | 2022-11-16 | Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) | Recombinant tert-encoding viral genomes and vectors |
WO2023138791A1 (en) * | 2022-01-24 | 2023-07-27 | Fundación Del Sector Público Estatal Centro Nacional De Investigaciones Oncológicas Carlos III (F.S.P. CNIO) | Telomerase reverse transcriptase therapy for kidney fibrosis and non-human animals thereof |
Family Cites Families (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US7569385B2 (en) * | 2003-08-14 | 2009-08-04 | The Regents Of The University Of California | Multipotent amniotic fetal stem cells |
US20110151447A1 (en) * | 2007-11-06 | 2011-06-23 | Children's Medical Center Corporation | Method to produce induced pluripotent stem (ips) cells from non-embryonic human cells |
US20110243910A1 (en) * | 2008-08-12 | 2011-10-06 | Japan Health Sciences Foundation | mammalian rna dependent rna polymerase |
EP2402038B1 (en) * | 2010-07-02 | 2016-05-25 | Fundación Centro Nacional De Investigaciones Oncológicas Carlos III | Telomerase reverse transcriptase for protection against ageing |
US9453209B2 (en) * | 2012-12-27 | 2016-09-27 | Sierra Sciences, Llc | Enhancing health in mammals using telomerase reverse transcriptase gene therapy |
-
2015
- 2015-08-04 ES ES18215767T patent/ES2864731T3/es active Active
- 2015-08-04 LT LTEP18215767.7T patent/LT3485914T/lt unknown
- 2015-08-04 HU HUE18215767A patent/HUE053526T2/hu unknown
- 2015-08-04 EP EP18215767.7A patent/EP3485914B1/en active Active
- 2015-08-04 PT PT182157677T patent/PT3485914T/pt unknown
- 2015-08-04 PL PL18215767T patent/PL3485914T3/pl unknown
- 2015-08-04 WO PCT/EP2015/067874 patent/WO2016020345A1/en active Application Filing
- 2015-08-04 EP EP15744254.2A patent/EP3193943B1/en active Active
- 2015-08-04 ES ES15744254T patent/ES2729872T3/es active Active
- 2015-08-04 DK DK18215767.7T patent/DK3485914T3/da active
- 2015-08-04 EP EP21151155.5A patent/EP3848056B1/en active Active
- 2015-08-04 JP JP2017508100A patent/JP6676262B2/ja active Active
- 2015-08-04 US US15/502,522 patent/US11529396B2/en active Active
- 2015-08-04 DK DK15744254.2T patent/DK3193943T3/da active
- 2015-08-04 SI SI201531509T patent/SI3485914T1/sl unknown
- 2015-08-04 EP EP21198809.2A patent/EP3978031A1/en active Pending
-
2021
- 2021-02-16 HR HRP20210265TT patent/HRP20210265T1/hr unknown
- 2021-03-02 US US17/189,368 patent/US20210260169A1/en active Pending
- 2021-04-12 CY CY20211100310T patent/CY1124012T1/el unknown
-
2023
- 2023-09-07 US US18/463,155 patent/US20240075109A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
ES2729872T3 (es) | 2019-11-06 |
EP3485914B1 (en) | 2021-01-13 |
US20210260169A1 (en) | 2021-08-26 |
US20240075109A1 (en) | 2024-03-07 |
EP3485914A1 (en) | 2019-05-22 |
LT3485914T (lt) | 2021-03-25 |
ES2864731T3 (es) | 2021-10-14 |
US11529396B2 (en) | 2022-12-20 |
DK3193943T3 (da) | 2019-06-11 |
EP3193943B1 (en) | 2019-03-06 |
EP3848056A1 (en) | 2021-07-14 |
EP3193943A1 (en) | 2017-07-26 |
PL3485914T3 (pl) | 2021-08-02 |
WO2016020345A1 (en) | 2016-02-11 |
EP3978031A1 (en) | 2022-04-06 |
PT3485914T (pt) | 2021-03-16 |
JP6676262B2 (ja) | 2020-04-08 |
EP3848056B1 (en) | 2024-02-14 |
SI3485914T1 (sl) | 2021-04-30 |
US20170232075A1 (en) | 2017-08-17 |
HUE053526T2 (hu) | 2021-07-28 |
DK3485914T3 (da) | 2021-01-25 |
JP2017524369A (ja) | 2017-08-31 |
CY1124012T1 (el) | 2022-05-27 |
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