SG11202104634UA - Codon-optimized abcb11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (pfic2) - Google Patents
Codon-optimized abcb11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (pfic2)Info
- Publication number
- SG11202104634UA SG11202104634UA SG11202104634UA SG11202104634UA SG11202104634UA SG 11202104634U A SG11202104634U A SG 11202104634UA SG 11202104634U A SG11202104634U A SG 11202104634UA SG 11202104634U A SG11202104634U A SG 11202104634UA SG 11202104634U A SG11202104634U A SG 11202104634UA
- Authority
- SG
- Singapore
- Prior art keywords
- pfic2
- abcb11
- transgene
- codon
- optimized
- Prior art date
Links
- 108700019146 Transgenes Proteins 0.000 title 1
- 208000020709 progressive familial intrahepatic cholestasis type 2 Diseases 0.000 title 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
- C12N15/63—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
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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
- A01K2267/0306—Animal model for genetic diseases
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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
- 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
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/14123—Virus like particles [VLP]
-
- 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
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biophysics (AREA)
- Animal Behavior & Ethology (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP18306458 | 2018-11-07 | ||
PCT/EP2019/080341 WO2020094693A1 (en) | 2018-11-07 | 2019-11-06 | Codon-optimized abcb11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (pfic2) |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11202104634UA true SG11202104634UA (en) | 2021-06-29 |
Family
ID=65496678
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11202104634UA SG11202104634UA (en) | 2018-11-07 | 2019-11-06 | Codon-optimized abcb11 transgene for the treatment of progressive familial intrahepatic cholestasis type 2 (pfic2) |
Country Status (12)
Country | Link |
---|---|
US (1) | US20220002751A1 (en) |
EP (1) | EP3877000A1 (en) |
JP (1) | JP2022512930A (en) |
KR (1) | KR20210091736A (en) |
CN (1) | CN112955186A (en) |
AU (1) | AU2019375450A1 (en) |
BR (1) | BR112021008434A2 (en) |
CA (1) | CA3117274A1 (en) |
IL (1) | IL282973A (en) |
MX (1) | MX2021005435A (en) |
SG (1) | SG11202104634UA (en) |
WO (1) | WO2020094693A1 (en) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20240091382A1 (en) * | 2020-12-23 | 2024-03-21 | Vivet Therapeutics | Minimal bile acid inducible promoters for gene therapy |
GB202205606D0 (en) * | 2022-04-14 | 2022-06-01 | Genespire S R L | Lentiviral vector |
Family Cites Families (26)
Publication number | Priority date | Publication date | Assignee | Title |
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US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
WO1992001070A1 (en) | 1990-07-09 | 1992-01-23 | The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce | High efficiency packaging of mutant adeno-associated virus using amber suppressions |
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
JP3534749B2 (en) | 1991-08-20 | 2004-06-07 | アメリカ合衆国 | Adenovirus-mediated gene transfer into the gastrointestinal tract |
FR2836924B1 (en) | 2002-03-08 | 2005-01-14 | Vivalis | AVIAN CELL LINES USEFUL FOR THE PRODUCTION OF INTEREST SUBSTANCES |
WO2005010039A1 (en) * | 2003-07-31 | 2005-02-03 | Pfizer Products Inc. | Bsep polypeptide variants and uses thereof |
EP1528101A1 (en) | 2003-11-03 | 2005-05-04 | ProBioGen AG | Immortalized avian cell lines for virus production |
FR2884255B1 (en) | 2005-04-11 | 2010-11-05 | Vivalis | USE OF EBX AVIATION STEM CELL LINES FOR THE PRODUCTION OF INFLUENZA VACCINE |
CN101864392B (en) | 2005-12-13 | 2016-03-23 | 国立大学法人京都大学 | Nuclear reprogramming factor |
JP2007295929A (en) * | 2006-04-05 | 2007-11-15 | Geno Membrane:Kk | Canine bsep gene |
US8320244B2 (en) | 2006-06-30 | 2012-11-27 | Qualcomm Incorporated | Reservation based MAC protocol |
JP5813321B2 (en) | 2007-03-23 | 2015-11-17 | ウィスコンシン アラムニ リサーチ ファンデーション | Somatic cell reprogramming |
EP1985305A1 (en) | 2007-04-24 | 2008-10-29 | Vivalis | Duck embryonic derived stem cell lines for the production of viral vaccines |
EP1995309A1 (en) | 2007-05-21 | 2008-11-26 | Vivalis | Recombinant protein production in avian EBx® cells |
AU2013243947A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
CA2868391A1 (en) * | 2012-04-02 | 2013-10-10 | Stephane Bancel | Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same |
PT2968605T (en) | 2013-03-15 | 2022-09-22 | Univ North Carolina Chapel Hill | Methods and compositions for dual glycan binding aav vectors |
WO2014200816A1 (en) * | 2013-06-14 | 2014-12-18 | Biotranex, Llc | Method for measuring bile salt export transport and/or formation activity |
CA2960912A1 (en) * | 2014-09-16 | 2016-03-24 | Universitat Autonoma De Barcelona | Adeno-associated viral vectors for the gene therapy of metabolic diseases |
GB2560250A (en) | 2015-06-30 | 2018-09-05 | Ethris Gmbh | ATP-Binding cassette family coding polyribonucleotides and formulations thereof |
WO2017100551A1 (en) | 2015-12-09 | 2017-06-15 | Alexion Pharmaceuticals, Inc. | HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION |
EP3452101A2 (en) * | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
CN107812003B (en) * | 2017-11-23 | 2021-02-09 | 南京市儿童医院 | Application of VX-809 in preparation of medicine for treating progressive familial intrahepatic cholestasis |
CA3169063A1 (en) * | 2020-02-25 | 2021-09-02 | Leszek Lisowski | Adeno-associated virus capsid polypeptides and vectors |
US20240091382A1 (en) * | 2020-12-23 | 2024-03-21 | Vivet Therapeutics | Minimal bile acid inducible promoters for gene therapy |
EP4308173A2 (en) * | 2021-03-19 | 2024-01-24 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing pfic therapeutics |
-
2019
- 2019-11-06 SG SG11202104634UA patent/SG11202104634UA/en unknown
- 2019-11-06 BR BR112021008434-7A patent/BR112021008434A2/en not_active Application Discontinuation
- 2019-11-06 CN CN201980072814.7A patent/CN112955186A/en active Pending
- 2019-11-06 US US17/291,449 patent/US20220002751A1/en active Pending
- 2019-11-06 EP EP19795591.7A patent/EP3877000A1/en active Pending
- 2019-11-06 WO PCT/EP2019/080341 patent/WO2020094693A1/en unknown
- 2019-11-06 AU AU2019375450A patent/AU2019375450A1/en not_active Abandoned
- 2019-11-06 KR KR1020217017164A patent/KR20210091736A/en unknown
- 2019-11-06 JP JP2021524228A patent/JP2022512930A/en active Pending
- 2019-11-06 MX MX2021005435A patent/MX2021005435A/en unknown
- 2019-11-06 CA CA3117274A patent/CA3117274A1/en active Pending
-
2021
- 2021-05-05 IL IL282973A patent/IL282973A/en unknown
Also Published As
Publication number | Publication date |
---|---|
CA3117274A1 (en) | 2020-05-14 |
IL282973A (en) | 2021-06-30 |
US20220002751A1 (en) | 2022-01-06 |
KR20210091736A (en) | 2021-07-22 |
BR112021008434A2 (en) | 2021-09-28 |
CN112955186A (en) | 2021-06-11 |
WO2020094693A1 (en) | 2020-05-14 |
EP3877000A1 (en) | 2021-09-15 |
AU2019375450A1 (en) | 2021-06-17 |
MX2021005435A (en) | 2021-09-08 |
JP2022512930A (en) | 2022-02-07 |
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