BR112021024124A2 - Expressão otimizada de fenilalanina hidroxilase - Google Patents
Expressão otimizada de fenilalanina hidroxilaseInfo
- Publication number
- BR112021024124A2 BR112021024124A2 BR112021024124A BR112021024124A BR112021024124A2 BR 112021024124 A2 BR112021024124 A2 BR 112021024124A2 BR 112021024124 A BR112021024124 A BR 112021024124A BR 112021024124 A BR112021024124 A BR 112021024124A BR 112021024124 A2 BR112021024124 A2 BR 112021024124A2
- Authority
- BR
- Brazil
- Prior art keywords
- phenylalanine hydroxylase
- lentiviral
- optimized expression
- expression
- optimized
- Prior art date
Links
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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
-
- 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
- A61K38/44—Oxidoreductases (1)
-
- 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
- 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
- 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
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15023—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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—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
- 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
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
- C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962855506P | 2019-05-31 | 2019-05-31 | |
| PCT/US2020/035584 WO2020243717A1 (en) | 2019-05-31 | 2020-06-01 | Optimized phenylalanine hydroxylase expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112021024124A2 true BR112021024124A2 (pt) | 2022-01-11 |
Family
ID=73553945
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112021024124A BR112021024124A2 (pt) | 2019-05-31 | 2020-06-01 | Expressão otimizada de fenilalanina hidroxilase |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20220162643A1 (enExample) |
| EP (1) | EP3976076A4 (enExample) |
| JP (1) | JP2022535745A (enExample) |
| KR (1) | KR20220068954A (enExample) |
| CN (1) | CN113905768A (enExample) |
| AU (1) | AU2020283069A1 (enExample) |
| BR (1) | BR112021024124A2 (enExample) |
| CA (1) | CA3137698A1 (enExample) |
| IL (1) | IL288400A (enExample) |
| WO (1) | WO2020243717A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010045659A1 (en) | 2008-10-17 | 2010-04-22 | American Gene Technologies International Inc. | Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules |
| US11980663B2 (en) | 2015-07-08 | 2024-05-14 | American Gene Technologies International Inc. | HIV pre-immunization and immunotherapy |
| US10137144B2 (en) | 2016-01-15 | 2018-11-27 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta T-cells |
| IL297238B2 (en) | 2016-01-15 | 2024-08-01 | American Gene Tech Int Inc | Methods and preparations for gamma-delta T-cell activation |
| JP7153332B2 (ja) | 2016-02-08 | 2022-10-14 | アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド | Ｈｉｖワクチン接種および免疫療法 |
| ES2911448T3 (es) | 2016-03-09 | 2022-05-19 | American Gene Tech Int Inc | Vectores combinados y métodos para el tratamiento del cáncer |
| WO2017213697A1 (en) | 2016-06-08 | 2017-12-14 | American Gene Technologies International Inc. | Non-integrating viral delivery system and methods related thereto |
| CA3028982A1 (en) | 2016-07-08 | 2018-01-11 | American Gene Technologies International Inc. | Hiv pre-immunization and immunotherapy |
| WO2018017882A1 (en) | 2016-07-21 | 2018-01-25 | American Gene Technologies International Inc. | Viral vectors for treating parkinson's disease |
| US11820999B2 (en) | 2017-04-03 | 2023-11-21 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| US12559769B2 (en) | 2017-06-16 | 2026-02-24 | American Gene Technologies International Inc. | Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells |
| US11939601B2 (en) | 2017-11-22 | 2024-03-26 | Modernatx, Inc. | Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria |
| MX2023003019A (es) * | 2020-09-16 | 2023-05-08 | Generation Bio Co | Vectores de adn de extremo cerrado y usos de estos para expresar fenilalanina hidroxilasa (pah). |
| EP4402267A4 (en) * | 2021-09-16 | 2025-11-26 | Generation Bio Co | Liver-specific expression cassettes, vectors, and their uses for expressing therapeutic proteins |
| CN115772503B (zh) * | 2022-08-16 | 2023-10-17 | 广东省南山医药创新研究院 | 一种表达pah的基因修饰细胞药物及其制备方法与应用 |
| IL327036A (en) * | 2023-09-19 | 2026-05-01 | Poseida Therapeutics Inc | Compositions and methods for combining viral vectors |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013151664A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| EP3574923A1 (en) * | 2013-10-22 | 2019-12-04 | Translate Bio, Inc. | Mrna therapy for phenylketonuria |
| CN115094062A (zh) * | 2015-04-16 | 2022-09-23 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
| WO2017106326A1 (en) * | 2015-12-14 | 2017-06-22 | The Trustees Of The University Of Pennsylvania | Aav-anti pcsk9 antibody constructs and uses thereof |
| AU2017248189B2 (en) * | 2016-04-08 | 2021-04-29 | Translate Bio, Inc. | Multimeric coding nucleic acid and uses thereof |
| EP3562494A4 (en) * | 2016-12-30 | 2020-08-19 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS |
| US11820999B2 (en) * | 2017-04-03 | 2023-11-21 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| KR20200057766A (ko) * | 2017-10-02 | 2020-05-26 | 아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드 | 페닐케톤뇨증을 치료하기 위한 프로모터 및 인핸서 조합을 포함하는 벡터 |
-
2020
- 2020-06-01 BR BR112021024124A patent/BR112021024124A2/pt unknown
- 2020-06-01 WO PCT/US2020/035584 patent/WO2020243717A1/en not_active Ceased
- 2020-06-01 US US17/610,111 patent/US20220162643A1/en active Pending
- 2020-06-01 CN CN202080040373.5A patent/CN113905768A/zh active Pending
- 2020-06-01 JP JP2021570364A patent/JP2022535745A/ja active Pending
- 2020-06-01 KR KR1020217042390A patent/KR20220068954A/ko active Pending
- 2020-06-01 EP EP20814445.1A patent/EP3976076A4/en active Pending
- 2020-06-01 CA CA3137698A patent/CA3137698A1/en active Pending
- 2020-06-01 AU AU2020283069A patent/AU2020283069A1/en not_active Abandoned
-
2021
- 2021-11-25 IL IL288400A patent/IL288400A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020243717A1 (en) | 2020-12-03 |
| AU2020283069A8 (en) | 2022-05-12 |
| US20220162643A1 (en) | 2022-05-26 |
| CA3137698A1 (en) | 2020-12-03 |
| AU2020283069A1 (en) | 2022-01-06 |
| KR20220068954A (ko) | 2022-05-26 |
| EP3976076A4 (en) | 2023-06-07 |
| EP3976076A1 (en) | 2022-04-06 |
| IL288400A (en) | 2022-01-01 |
| CN113905768A (zh) | 2022-01-07 |
| JP2022535745A (ja) | 2022-08-10 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B08F | Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette] |
Free format text: REFERENTE A 6A ANUIDADE. |