AR116569A1 - Terapia génica para tratar la acidemia propiónica - Google Patents
Terapia génica para tratar la acidemia propiónicaInfo
- Publication number
- AR116569A1 AR116569A1 ARP190102785A ARP190102785A AR116569A1 AR 116569 A1 AR116569 A1 AR 116569A1 AR P190102785 A ARP190102785 A AR P190102785A AR P190102785 A ARP190102785 A AR P190102785A AR 116569 A1 AR116569 A1 AR 116569A1
- Authority
- AR
- Argentina
- Prior art keywords
- gene therapy
- treat
- propionic acidemia
- propionyl
- subunit
- Prior art date
Links
Classifications
-
- 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
- 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
- A61K38/53—Ligases (6)
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- 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
-
- 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
- C12N9/93—Ligases (6)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y604/00—Ligases forming carbon-carbon bonds (6.4)
- C12Y604/01—Ligases forming carbon-carbon bonds (6.4.1)
- C12Y604/01003—Propionyl-CoA carboxylase (6.4.1.3)
-
- 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
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- 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
-
- 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
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Manufacturing & Machinery (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862739471P | 2018-10-01 | 2018-10-01 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR116569A1 true AR116569A1 (es) | 2021-05-19 |
Family
ID=70055045
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP190102785A AR116569A1 (es) | 2018-10-01 | 2019-10-01 | Terapia génica para tratar la acidemia propiónica |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US12472268B2 (OSRAM) |
| EP (1) | EP3861131A4 (OSRAM) |
| JP (1) | JP2022513318A (OSRAM) |
| KR (1) | KR20210082460A (OSRAM) |
| CN (1) | CN112955557A (OSRAM) |
| AR (1) | AR116569A1 (OSRAM) |
| AU (1) | AU2019354296A1 (OSRAM) |
| BR (1) | BR112021006052A2 (OSRAM) |
| CA (1) | CA3113975A1 (OSRAM) |
| TW (1) | TW202028472A (OSRAM) |
| WO (1) | WO2020072451A1 (OSRAM) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020264353A1 (en) * | 2019-06-27 | 2020-12-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha |
| US20220325266A1 (en) * | 2019-10-15 | 2022-10-13 | The United States Of America,As Represented By The Secretary,Department Of Health And Human Services | Synthetic genes for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase beta |
| US20230129893A1 (en) | 2020-03-31 | 2023-04-27 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| US20240175050A1 (en) * | 2021-03-19 | 2024-05-30 | Mayo Foundation For Medical Education And Research | Methods and materials for treating propionic acidemia |
| EP4457351A2 (en) * | 2021-12-29 | 2024-11-06 | The United States of America, as represented by the Secretary, Department of Health and Human Services | Improved gene therapy constructs for the treatment of propionic acidemia caused by mutations in propionyl-coa carboxylase alpha |
| CN118166004B (zh) * | 2024-05-14 | 2024-12-03 | 北京剂泰医药科技有限公司 | 用于编码人PCCA或PCCB蛋白的mRNA及其用途 |
Family Cites Families (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2975413T3 (es) * | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| PL2357189T3 (pl) | 2003-06-19 | 2017-08-31 | Genzyme Corporation | Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
| HUE030719T2 (en) | 2007-04-09 | 2017-05-29 | Univ Florida | Raav vector compositions having tyrosine-modified capsid proteins and methods for use |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US10392632B2 (en) | 2011-02-14 | 2019-08-27 | The Children's Hospital Of Philadelphia | AAV8 vector with enhanced functional activity and methods of use thereof |
| CN107012171B (zh) | 2011-04-22 | 2021-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| JP6342886B2 (ja) | 2012-04-18 | 2018-06-13 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法 |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| US10266845B2 (en) | 2013-02-08 | 2019-04-23 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| US20140303901A1 (en) * | 2013-04-08 | 2014-10-09 | Ilan Sadeh | Method and system for predicting a disease |
| EP2986290A4 (en) * | 2013-04-19 | 2017-01-04 | Thomas Jefferson University | Caveolin-1 related methods for treating glioblastoma with temozolomide |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| EP3291829A1 (en) * | 2015-05-03 | 2018-03-14 | The Regents of the University of Colorado, a body corporate | Propionyl-coa carboxylase compositions and uses thereof |
| RU2021132397A (ru) | 2015-05-16 | 2022-02-24 | Джензим Корпорейшн | Генное редактирование глубоких интронных мутаций |
| CN115141836B (zh) * | 2015-10-28 | 2024-08-27 | 桑格摩生物治疗股份有限公司 | 肝特异性构建体、因子viii表达盒及其使用方法 |
| WO2017096164A1 (en) | 2015-12-02 | 2017-06-08 | The Board Of Trustees Of The Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| JP6947739B2 (ja) | 2016-02-16 | 2021-10-13 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | 既存のヒト中和抗体に対して耐性である新規組換えアデノ随伴ウイルスカプシド |
| WO2017165859A1 (en) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| CA3019126A1 (en) * | 2016-03-30 | 2017-10-05 | Spark Therapeutics, Inc. | Cell line for recombinant protein and/or viral vector production |
| CA3019423A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
| US11191847B2 (en) * | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
| WO2017192761A1 (en) * | 2016-05-03 | 2017-11-09 | The Regents Of The University Of Colorado, A Body Corporate | Propionyl-coa carboxylase compositions and uses thereof |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| EP4700118A3 (en) | 2016-07-29 | 2026-04-22 | The Regents of the University of California | Adeno-associated virus virions with varint capsid and methods of use thereof |
| EP3562514B1 (en) | 2016-12-30 | 2026-04-29 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating wilson's disease |
| BR112019013576A2 (pt) | 2016-12-30 | 2020-02-04 | The Trustees Of The University Of Pennsylvania | terapia genica para o tratamento da fenilcetonuria |
| WO2018144709A2 (en) | 2017-02-01 | 2018-08-09 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating citrullenemia |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| WO2018222503A1 (en) | 2017-05-31 | 2018-12-06 | The Regents Of The University Of California | Adeno-associated virus with variant capsid and methods of use thereof |
| AU2018280116B2 (en) | 2017-06-05 | 2025-03-27 | Research Institute At Nationwide Children's Hospital | Enhanced modified viral capsid proteins |
| IL276859B2 (en) | 2018-02-27 | 2025-12-01 | Univ Pennsylvania | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
| US20230129893A1 (en) | 2020-03-31 | 2023-04-27 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
-
2019
- 2019-10-01 KR KR1020217012982A patent/KR20210082460A/ko not_active Ceased
- 2019-10-01 EP EP19869846.6A patent/EP3861131A4/en active Pending
- 2019-10-01 TW TW108135582A patent/TW202028472A/zh unknown
- 2019-10-01 JP JP2021542078A patent/JP2022513318A/ja active Pending
- 2019-10-01 BR BR112021006052-9A patent/BR112021006052A2/pt not_active IP Right Cessation
- 2019-10-01 CA CA3113975A patent/CA3113975A1/en active Pending
- 2019-10-01 AR ARP190102785A patent/AR116569A1/es not_active Application Discontinuation
- 2019-10-01 WO PCT/US2019/054003 patent/WO2020072451A1/en not_active Ceased
- 2019-10-01 AU AU2019354296A patent/AU2019354296A1/en not_active Abandoned
- 2019-10-01 US US17/278,847 patent/US12472268B2/en active Active
- 2019-10-01 CN CN201980070896.1A patent/CN112955557A/zh active Pending
-
2025
- 2025-10-21 US US19/365,040 patent/US20260041788A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US12472268B2 (en) | 2025-11-18 |
| US20260041788A1 (en) | 2026-02-12 |
| WO2020072451A1 (en) | 2020-04-09 |
| TW202028472A (zh) | 2020-08-01 |
| WO2020072451A8 (en) | 2021-05-14 |
| EP3861131A1 (en) | 2021-08-11 |
| AU2019354296A1 (en) | 2021-04-29 |
| EP3861131A4 (en) | 2022-06-15 |
| CN112955557A (zh) | 2021-06-11 |
| US20210283272A1 (en) | 2021-09-16 |
| CA3113975A1 (en) | 2020-04-09 |
| BR112021006052A2 (pt) | 2021-09-08 |
| KR20210082460A (ko) | 2021-07-05 |
| JP2022513318A (ja) | 2022-02-07 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA | Abandonment or withdrawal |