AR121709A1 - Terapia génica para tratar la acidemia propiónica - Google Patents
Terapia génica para tratar la acidemia propiónicaInfo
- Publication number
- AR121709A1 AR121709A1 ARP210100811A ARP210100811A AR121709A1 AR 121709 A1 AR121709 A1 AR 121709A1 AR P210100811 A ARP210100811 A AR P210100811A AR P210100811 A ARP210100811 A AR P210100811A AR 121709 A1 AR121709 A1 AR 121709A1
- Authority
- AR
- Argentina
- Prior art keywords
- gene therapy
- treat
- propionic acidemia
- raav
- propionyl
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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)
- C12Y604/01002—Acetyl-CoA carboxylase (6.4.1.2)
-
- 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
- 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
-
- 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
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Obesity (AREA)
- Animal Behavior & Ethology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Hematology (AREA)
- Public Health (AREA)
- Veterinary Medicine (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)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063002541P | 2020-03-31 | 2020-03-31 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR121709A1 true AR121709A1 (es) | 2022-06-29 |
Family
ID=75660311
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP210100811A AR121709A1 (es) | 2020-03-31 | 2021-03-31 | Terapia génica para tratar la acidemia propiónica |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230129893A1 (https=) |
| EP (1) | EP4127189A1 (https=) |
| JP (1) | JP2023520402A (https=) |
| AR (1) | AR121709A1 (https=) |
| TW (1) | TW202204624A (https=) |
| WO (1) | WO2021202532A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112021006052A2 (pt) | 2018-10-01 | 2021-09-08 | Ultragenyx Pharmaceutical Inc. | Terapia gênica para tratar acidemia propiônica |
| WO2022198079A1 (en) * | 2021-03-19 | 2022-09-22 | Mayo Foundation For Medical Education And Research | Methods and materials for treating propionic acidemia |
| WO2023130003A2 (en) * | 2021-12-29 | 2023-07-06 | 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 |
Family Cites Families (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6569662B1 (en) * | 2000-01-21 | 2003-05-27 | Hyseq, Inc. | Nucleic acids and polypeptides |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| SI1633772T1 (sl) | 2003-06-19 | 2016-06-30 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| 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 |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of 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 |
| ES2714007T3 (es) | 2007-04-09 | 2019-05-24 | Univ Florida | Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2675484B1 (en) | 2011-02-14 | 2018-05-30 | The Children's Hospital of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| CN105755044A (zh) | 2011-04-22 | 2016-07-13 | 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 |
| NZ701693A (en) | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| 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 |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| HUE035875T2 (hu) * | 2013-10-25 | 2018-06-28 | Psioxus Therapeutics Ltd | Heterológ géneket tartalmazó onkolitikus adenovírusok |
| EP3186366A2 (en) * | 2014-08-27 | 2017-07-05 | Psioxus Therapeutics Limited | A process for the production of adenovirus |
| 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 |
| WO2016179138A1 (en) * | 2015-05-03 | 2016-11-10 | The Regents Of The University Of Colorado | Propionyl-coa carboxylase compositions and uses thereof |
| EP3384034B1 (en) | 2015-12-02 | 2020-07-08 | 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 |
| US11191847B2 (en) * | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
| SG11201808812RA (en) | 2016-04-15 | 2018-11-29 | Univ Pennsylvania | Novel aav8 mutant capsids and compositions containing same |
| AU2017302013B2 (en) | 2016-07-29 | 2022-05-26 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| KR102738752B1 (ko) * | 2016-12-30 | 2024-12-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
| WO2018144709A2 (en) * | 2017-02-01 | 2018-08-09 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating citrullenemia |
| EP3585883A4 (en) | 2017-02-21 | 2021-04-14 | 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 |
| CA3065946A1 (en) | 2017-06-05 | 2018-12-13 | 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 |
| BR112021006052A2 (pt) | 2018-10-01 | 2021-09-08 | Ultragenyx Pharmaceutical Inc. | Terapia gênica para tratar acidemia propiônica |
| JP2023514204A (ja) * | 2020-02-14 | 2023-04-05 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | Ｃｄｋｌ５欠損障害を処置するための遺伝子治療 |
-
2021
- 2021-03-30 WO PCT/US2021/024892 patent/WO2021202532A1/en not_active Ceased
- 2021-03-30 JP JP2022559589A patent/JP2023520402A/ja active Pending
- 2021-03-30 EP EP21721256.2A patent/EP4127189A1/en active Pending
- 2021-03-30 US US17/906,902 patent/US20230129893A1/en active Pending
- 2021-03-31 AR ARP210100811A patent/AR121709A1/es not_active Application Discontinuation
- 2021-03-31 TW TW110111870A patent/TW202204624A/zh unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20230129893A1 (en) | 2023-04-27 |
| TW202204624A (zh) | 2022-02-01 |
| WO2021202532A1 (en) | 2021-10-07 |
| EP4127189A1 (en) | 2023-02-08 |
| JP2023520402A (ja) | 2023-05-17 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA | Abandonment or withdrawal |