SG11202002263TA - Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency - Google Patents
Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiencyInfo
- Publication number
- SG11202002263TA SG11202002263TA SG11202002263TA SG11202002263TA SG11202002263TA SG 11202002263T A SG11202002263T A SG 11202002263TA SG 11202002263T A SG11202002263T A SG 11202002263TA SG 11202002263T A SG11202002263T A SG 11202002263TA SG 11202002263T A SG11202002263T A SG 11202002263TA
- Authority
- SG
- Singapore
- Prior art keywords
- pklr
- treat
- delivery
- lentiviral vectors
- pyruvate kinase
- Prior art date
Links
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
- C12N15/63—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
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
-
- 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
-
- 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
-
- 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)
- C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
-
- 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)
- C12Y207/02—Phosphotransferases with a carboxy group as acceptor (2.7.2)
- C12Y207/02003—Phosphoglycerate kinase (2.7.2.3)
-
- 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
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762573037P | 2017-10-16 | 2017-10-16 | |
| PCT/US2018/056136 WO2019079338A1 (en) | 2017-10-16 | 2018-10-16 | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11202002263TA true SG11202002263TA (en) | 2020-04-29 |
Family
ID=66174611
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11202002263TA SG11202002263TA (en) | 2017-10-16 | 2018-10-16 | Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US11642422B2 (en) |
| EP (1) | EP3697452A4 (en) |
| JP (2) | JP7403461B2 (en) |
| KR (1) | KR20200116076A (en) |
| CN (1) | CN111163810A (en) |
| AU (1) | AU2018352583A1 (en) |
| BR (1) | BR112020007444A2 (en) |
| CA (1) | CA3076253A1 (en) |
| IL (1) | IL273737A (en) |
| MX (1) | MX2020003954A (en) |
| SG (1) | SG11202002263TA (en) |
| WO (1) | WO2019079338A1 (en) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109641063B (en) | 2016-04-20 | 2022-11-15 | 能源环境和技术研究中心O.A., M.P. | Compositions and methods for enhancing gene expression of PKLR |
| CN110904102A (en) * | 2018-09-18 | 2020-03-24 | 中国科学院上海生命科学研究院 | Promoter for recombinant protein expression |
| CA3185267A1 (en) | 2020-08-07 | 2022-02-10 | Spacecraft Seven, Llc | Plakophilin-2 (pkp2) gene therapy using aav vector |
| CN111733187B (en) * | 2020-08-26 | 2020-11-06 | 和元生物技术（上海）股份有限公司 | WPRE mutant virus vector and application thereof |
| KR102540747B1 (en) | 2020-11-30 | 2023-06-08 | 제이케이생명과학 주식회사 | Manufacturing method of drink |
| CN117441023A (en) * | 2021-05-13 | 2024-01-23 | 能源环境和技术研究中心O.A.,M.P. | Lentiviral vector and use thereof |
| CN114898802B (en) * | 2022-07-14 | 2022-09-30 | 臻和(北京)生物科技有限公司 | Terminal sequence frequency distribution characteristic determination method, evaluation method and device based on plasma free DNA methylation sequencing data |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE3853886T2 (en) | 1987-09-17 | 1995-10-05 | Massachusetts Inst Technology | HUMAN, ERYTHROID-SPECIFIC TRANSCRIBUTION HANCER. |
| FR2705361B1 (en) | 1993-05-18 | 1995-08-04 | Centre Nat Rech Scient | Viral vectors and use in gene therapy. |
| US6027721A (en) | 1996-05-20 | 2000-02-22 | Cytotherapeutics, Inc. | Device and method for encapsulated gene therapy |
| US6136597A (en) | 1997-09-18 | 2000-10-24 | The Salk Institute For Biological Studies | RNA export element |
| US6607879B1 (en) | 1998-02-09 | 2003-08-19 | Incyte Corporation | Compositions for the detection of blood cell and immunological response gene expression |
| US7994278B1 (en) | 1999-08-06 | 2011-08-09 | Nobel Biosciences Llc | Biologically active polypeptides derived from a novel early stage pregnancy factor designated maternin (MA) |
| CA2382832C (en) | 1999-10-12 | 2012-06-26 | Institut Pasteur | Lentiviral triplex dna, and vectors and recombinant cells containing lentiviral triplex dna |
| DE19960113A1 (en) | 1999-12-08 | 2001-06-13 | Wita Proteomics Ag | Means and procedures for the diagnosis of Lyme disease and Lyme disease vaccine |
| US6627442B1 (en) | 2000-08-31 | 2003-09-30 | Virxsys Corporation | Methods for stable transduction of cells with hiv-derived viral vectors |
| GB0024550D0 (en) | 2000-10-06 | 2000-11-22 | Oxford Biomedica Ltd | |
| US7575924B2 (en) | 2000-11-13 | 2009-08-18 | Research Development Foundation | Methods and compositions relating to improved lentiviral vectors and their applications |
| US7629153B2 (en) | 2001-08-02 | 2009-12-08 | Research Development Foundation | Methods and compositions relating to improved lentiviral vector production systems |
| RU2305708C2 (en) | 2001-10-02 | 2007-09-10 | Энститю Клейтон Де Ля Решерш | Recombinant lentiviral vector, host cell transduced with lentiviral vector, methods for transduction and uses thereof |
| AU2003226973A1 (en) | 2002-02-04 | 2003-09-02 | Develogen Aktiengesellschaft Fur Entwicklungsbiologische Forschung | Proteins involved in the regulation of energy homeostatis |
| DK1616012T3 (en) | 2003-04-24 | 2008-04-21 | San Raffaele Centro Fond | Lentiviral vectors bearing synthetic bidirectional promoters and their applications |
| US20070048285A1 (en) | 2005-08-24 | 2007-03-01 | Christopher Baum | Self-inactivating retroviral vector |
| ES2325711B1 (en) | 2007-04-17 | 2010-06-17 | Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas (Ciemat) | EXPRESSION VECTORS UNDERSTANDING THE PROMOTER OF THE HUMAN PKLR GENE AND ITS USE FOR THE ELABORATION OF PHARMACEUTICAL COMPOSITIONS INTENDED FOR SOMATIC GENE THERAPY WITH SPECIFIC EXPRESSION IN ERYTHROID CELLS. |
| WO2008136670A2 (en) | 2007-05-02 | 2008-11-13 | Erasmus University Medical Center Rotterdam | Improved methods and means for lentiviral gene delivery |
| JP5456689B2 (en) | 2007-12-07 | 2014-04-02 | ミルテンイ バイオテック ゲーエムベーハー | A centrifuge that separates a sample into at least two components |
| DE102010003419B4 (en) | 2010-03-30 | 2019-09-12 | Evonik Degussa Gmbh | Process for the fermentative production of L-ornithine |
| US9192651B2 (en) | 2012-04-02 | 2015-11-24 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of secreted proteins |
| KR20150105635A (en) | 2012-12-12 | 2015-09-17 | 더 브로드 인스티튜트, 인코퍼레이티드 | Crispr-cas component systems, methods and compositions for sequence manipulation |
| GB201318347D0 (en) | 2013-10-16 | 2013-11-27 | Ucl Business Plc | Retroviral vectors |
| CN104020298A (en) | 2014-06-12 | 2014-09-03 | 中国科学院化学研究所 | Self-powered microtubule-kinesin transport system and preparation method thereof |
| US10350245B2 (en) | 2015-01-21 | 2019-07-16 | Fred Hutchinson Cancer Research Center | Point-of-care and/or portable platform for gene therapy |
| AU2016348782A1 (en) | 2015-11-05 | 2018-05-31 | Cellectis | Process of gene-editing of cells isolated from a subject suffering from a metabolic disease affecting the erythroid lineage, cells obtained by said process and uses thereof. |
| CN109641063B (en) | 2016-04-20 | 2022-11-15 | 能源环境和技术研究中心O.A., M.P. | Compositions and methods for enhancing gene expression of PKLR |
-
2018
- 2018-10-16 AU AU2018352583A patent/AU2018352583A1/en active Pending
- 2018-10-16 SG SG11202002263TA patent/SG11202002263TA/en unknown
- 2018-10-16 CN CN201880064224.5A patent/CN111163810A/en active Pending
- 2018-10-16 MX MX2020003954A patent/MX2020003954A/en unknown
- 2018-10-16 CA CA3076253A patent/CA3076253A1/en active Pending
- 2018-10-16 JP JP2020542062A patent/JP7403461B2/en active Active
- 2018-10-16 EP EP18869037.4A patent/EP3697452A4/en active Pending
- 2018-10-16 US US16/755,033 patent/US11642422B2/en active Active
- 2018-10-16 WO PCT/US2018/056136 patent/WO2019079338A1/en unknown
- 2018-10-16 BR BR112020007444-6A patent/BR112020007444A2/en unknown
- 2018-10-16 KR KR1020207013555A patent/KR20200116076A/en not_active Application Discontinuation
-
2020
- 2020-04-01 IL IL273737A patent/IL273737A/en unknown
-
2023
- 2023-03-08 US US18/180,760 patent/US20230355807A1/en active Pending
- 2023-12-12 JP JP2023208939A patent/JP2024037845A/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| MX2020003954A (en) | 2020-10-05 |
| RU2020114948A3 (en) | 2021-12-24 |
| WO2019079338A1 (en) | 2019-04-25 |
| JP2021500920A (en) | 2021-01-14 |
| KR20200116076A (en) | 2020-10-08 |
| BR112020007444A2 (en) | 2020-10-20 |
| AU2018352583A1 (en) | 2020-04-09 |
| US20230355807A1 (en) | 2023-11-09 |
| US11642422B2 (en) | 2023-05-09 |
| RU2020114948A (en) | 2021-10-28 |
| EP3697452A4 (en) | 2021-11-24 |
| CA3076253A1 (en) | 2019-04-25 |
| IL273737A (en) | 2020-05-31 |
| CN111163810A (en) | 2020-05-15 |
| JP7403461B2 (en) | 2023-12-22 |
| US20210187126A1 (en) | 2021-06-24 |
| EP3697452A1 (en) | 2020-08-26 |
| JP2024037845A (en) | 2024-03-19 |
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