MX2021002198A - Secuencias de promotores optimizadas, construcciones de expresion libres de intrones y metodos de uso. - Google Patents
Secuencias de promotores optimizadas, construcciones de expresion libres de intrones y metodos de uso.Info
- Publication number
- MX2021002198A MX2021002198A MX2021002198A MX2021002198A MX2021002198A MX 2021002198 A MX2021002198 A MX 2021002198A MX 2021002198 A MX2021002198 A MX 2021002198A MX 2021002198 A MX2021002198 A MX 2021002198A MX 2021002198 A MX2021002198 A MX 2021002198A
- Authority
- MX
- Mexico
- Prior art keywords
- nucleic acid
- acid sequence
- regulatory element
- intron
- bdd
- Prior art date
Links
- 150000007523 nucleic acids Chemical group 0.000 abstract 7
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 5
- 230000001105 regulatory effect Effects 0.000 abstract 4
- 102000001690 Factor VIII Human genes 0.000 abstract 1
- 108010054218 Factor VIII Proteins 0.000 abstract 1
- 230000037430 deletion Effects 0.000 abstract 1
- 238000012217 deletion Methods 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
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
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- 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
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
-
- 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
- C07K14/775—Apolipopeptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/81—Protease inhibitors
- C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
- C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
- C07K14/8121—Serpins
- C07K14/8125—Alpha-1-antitrypsin
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Wood Science & Technology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Toxicology (AREA)
- Virology (AREA)
- Hematology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Dermatology (AREA)
- Immunology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862722547P | 2018-08-24 | 2018-08-24 | |
| US201862725096P | 2018-08-30 | 2018-08-30 | |
| US201862784116P | 2018-12-21 | 2018-12-21 | |
| PCT/US2019/048032 WO2020041773A1 (en) | 2018-08-24 | 2019-08-23 | Optimized promoter sequences, intron-free expression constructs and methods of use |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021002198A true MX2021002198A (es) | 2021-07-15 |
Family
ID=69591143
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021002198A MX2021002198A (es) | 2018-08-24 | 2019-08-23 | Secuencias de promotores optimizadas, construcciones de expresion libres de intrones y metodos de uso. |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12290574B2 (enExample) |
| EP (1) | EP3840775A4 (enExample) |
| JP (2) | JP2021533805A (enExample) |
| CN (1) | CN113226352B (enExample) |
| AU (2) | AU2019325688B2 (enExample) |
| BR (1) | BR112021003399A2 (enExample) |
| CA (1) | CA3111047A1 (enExample) |
| MX (1) | MX2021002198A (enExample) |
| TW (1) | TW202012425A (enExample) |
| WO (1) | WO2020041773A1 (enExample) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018281280B2 (en) | 2017-06-07 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| CN112040985A (zh) | 2018-02-07 | 2020-12-04 | 瑞泽恩制药公司 | 用于递送治疗性蛋白质的方法和组合物 |
| EP3793591A1 (en) | 2018-05-17 | 2021-03-24 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| EP4333886A4 (en) * | 2021-05-07 | 2025-11-12 | Generation Bio Co | NON-VIRAL DNA VECTORS FOR VACCINE ADMINISTRATION |
| WO2022236016A1 (en) * | 2021-05-07 | 2022-11-10 | Generation Bio Co. | Lyophilized non-viral dna vector compositions and uses thereof |
| AU2023261338A1 (en) * | 2022-04-28 | 2024-11-21 | Joint Stock Company "Biocad" | Codon-optimized nucleic acid encoding the fviii-bdd |
| CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
| EP4598590A4 (en) * | 2022-10-08 | 2026-05-27 | Lingyi Biotech Co Ltd | CONSTRUCTION TO IMPROVE GENE EXPRESSION |
| WO2025264490A1 (en) * | 2024-06-17 | 2025-12-26 | Spark Therapeutics, Inc. | Dna promoters |
| CN119242712A (zh) * | 2024-09-27 | 2025-01-03 | 南京师范大学 | 特异性靶向骨骼肌细胞的携带miRNA-486基因的腺相关病毒载体、药物组合物 |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20020065236A1 (en) | 1998-09-09 | 2002-05-30 | Yew Nelson S. | CpG reduced plasmids and viral vectors |
| BRPI0515819A (pt) * | 2004-12-01 | 2008-08-05 | Genzyme Corp | métodos para o fornecimento direcionado de material genético ao fìgado |
| US8008468B2 (en) * | 2005-02-16 | 2011-08-30 | Benitec, Inc. | RNAi expression constructs with liver-specific enhancer/promoter |
| WO2011109380A1 (en) | 2010-03-01 | 2011-09-09 | The Children's Hospital Of Philadelphia | Nucleic acids for targeting multiple regions of the hcv genome |
| US20140271550A1 (en) * | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
| JP6646282B2 (ja) * | 2014-04-24 | 2020-02-14 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | 多発性硬化症のためのａａｖ系遺伝子療法 |
| KR20180014051A (ko) * | 2015-05-28 | 2018-02-07 | 코넬 유니버시티 | 혈관 부종 치료제로서의 아데노-관련 바이러스 매개 c1e1의 전달 |
| EP4378487A3 (en) | 2015-06-23 | 2024-09-04 | The Children's Hospital of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| JP2018535929A (ja) * | 2015-09-24 | 2018-12-06 | バイオマリン ファーマシューティカル インコーポレイテッド | アデノ随伴ウイルス第ｖｉｉｉ因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤 |
| US10143760B2 (en) * | 2015-10-28 | 2018-12-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| PE20231949A1 (es) | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| EP3293260A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| KR102617502B1 (ko) | 2016-09-12 | 2023-12-26 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
| CA3086942A1 (en) | 2018-02-07 | 2019-08-15 | Genethon | Hybrid regulatory elements |
| BR112020015488A2 (pt) | 2018-02-08 | 2020-12-08 | Sangamo Therapeutics, Inc. | Nucleases alvo-específicas modificadas |
| SG11202011191WA (en) | 2018-05-16 | 2020-12-30 | Spark Therapeutics Inc | Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same |
| BR112021001498A2 (pt) | 2018-07-27 | 2021-04-27 | Regenxbio Inc. | vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
| CN113795575A (zh) | 2019-02-04 | 2021-12-14 | 自由行疗法有限公司 | 多核苷酸 |
| CA3106693A1 (en) | 2019-02-06 | 2020-08-13 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type i |
| EP3956354A1 (en) | 2019-04-19 | 2022-02-23 | Genethon | Gene therapy of fibroblast growth factor 23 related hypophosphatemic diseases |
| US20220195462A1 (en) | 2019-04-24 | 2022-06-23 | Regenxbio Inc. | Fully-human post-translationally modified antibody therapeutics |
| EP4004214A1 (en) | 2019-07-26 | 2022-06-01 | RegenxBio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
| US12662512B2 (en) | 2019-10-22 | 2026-06-23 | Genethon | Chimeric polypeptides and uses thereof |
| WO2021078834A1 (en) | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
| WO2021087366A1 (en) | 2019-11-01 | 2021-05-06 | Sangamo Therapeutics, Inc. | Compositions and methods for genome engineering |
| WO2021084276A2 (en) | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Factor viii construct |
| EP3913060A1 (en) | 2020-05-22 | 2021-11-24 | Genethon | Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy |
| GB202009741D0 (en) | 2020-06-25 | 2020-08-12 | Freeline Therapeutics Ltd | Polynucleotide |
| WO2022182986A1 (en) | 2021-02-26 | 2022-09-01 | Logicbio Therapeutics, Inc. | Manufacturing and use of recombinant aav vectors |
-
2019
- 2019-08-23 CN CN201980070435.4A patent/CN113226352B/zh active Active
- 2019-08-23 WO PCT/US2019/048032 patent/WO2020041773A1/en not_active Ceased
- 2019-08-23 AU AU2019325688A patent/AU2019325688B2/en active Active
- 2019-08-23 MX MX2021002198A patent/MX2021002198A/es unknown
- 2019-08-23 JP JP2021510015A patent/JP2021533805A/ja active Pending
- 2019-08-23 US US17/250,712 patent/US12290574B2/en active Active
- 2019-08-23 EP EP19852321.9A patent/EP3840775A4/en active Pending
- 2019-08-23 BR BR112021003399-8A patent/BR112021003399A2/pt unknown
- 2019-08-23 CA CA3111047A patent/CA3111047A1/en active Pending
- 2019-08-26 TW TW108130505A patent/TW202012425A/zh unknown
-
2024
- 2024-04-04 JP JP2024060919A patent/JP7874125B2/ja active Active
-
2025
- 2025-06-04 AU AU2025204161A patent/AU2025204161A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020041773A1 (en) | 2020-02-27 |
| JP7874125B2 (ja) | 2026-06-15 |
| AU2019325688A1 (en) | 2021-03-18 |
| JP2021533805A (ja) | 2021-12-09 |
| EP3840775A1 (en) | 2021-06-30 |
| AU2019325688B2 (en) | 2025-03-13 |
| TW202012425A (zh) | 2020-04-01 |
| US12290574B2 (en) | 2025-05-06 |
| BR112021003399A2 (pt) | 2021-05-18 |
| CA3111047A1 (en) | 2020-02-27 |
| US20220362408A1 (en) | 2022-11-17 |
| CN113226352A (zh) | 2021-08-06 |
| JP2024102065A (ja) | 2024-07-30 |
| EP3840775A4 (en) | 2022-07-06 |
| CN113226352B (zh) | 2025-08-26 |
| AU2025204161A1 (en) | 2025-06-26 |
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