CL2021001327A1 - Vectores virales recombinantes y ácidos nucleicos para producirlos - Google Patents
Vectores virales recombinantes y ácidos nucleicos para producirlosInfo
- Publication number
- CL2021001327A1 CL2021001327A1 CL2021001327A CL2021001327A CL2021001327A1 CL 2021001327 A1 CL2021001327 A1 CL 2021001327A1 CL 2021001327 A CL2021001327 A CL 2021001327A CL 2021001327 A CL2021001327 A CL 2021001327A CL 2021001327 A1 CL2021001327 A1 CL 2021001327A1
- Authority
- CL
- Chile
- Prior art keywords
- nucleic acids
- produce
- viral vectors
- recombinant viral
- cassettes
- Prior art date
Links
- 150000007523 nucleic acids Chemical class 0.000 title abstract 3
- 102000039446 nucleic acids Human genes 0.000 title abstract 3
- 108020004707 nucleic acids Proteins 0.000 title abstract 3
- 239000013603 viral vector Substances 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 239000013612 plasmid Substances 0.000 abstract 2
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
- 230000003612 virological effect Effects 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
-
- 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
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Psychology (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)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862770202P | 2018-11-21 | 2018-11-21 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2021001327A1 true CL2021001327A1 (es) | 2021-12-31 |
Family
ID=70773595
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2021001327A CL2021001327A1 (es) | 2018-11-21 | 2021-05-20 | Vectores virales recombinantes y ácidos nucleicos para producirlos |
Country Status (20)
| Country | Link |
|---|---|
| US (1) | US20210324418A1 (https=) |
| EP (1) | EP3883954A4 (https=) |
| JP (2) | JP7704998B2 (https=) |
| KR (1) | KR20210103469A (https=) |
| CN (1) | CN113302201A (https=) |
| AR (1) | AR117145A1 (https=) |
| AU (1) | AU2019385506A1 (https=) |
| BR (1) | BR112021009733A2 (https=) |
| CA (1) | CA3120289A1 (https=) |
| CL (1) | CL2021001327A1 (https=) |
| CO (1) | CO2021008120A2 (https=) |
| EA (1) | EA202191418A1 (https=) |
| EC (1) | ECSP21044840A (https=) |
| IL (1) | IL283344A (https=) |
| MX (1) | MX2021005997A (https=) |
| PE (1) | PE20211419A1 (https=) |
| PH (1) | PH12021551155A1 (https=) |
| SG (1) | SG11202105326WA (https=) |
| TW (1) | TW202039533A (https=) |
| WO (1) | WO2020106916A1 (https=) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN107686842A (zh) * | 2016-08-03 | 2018-02-13 | 南京大学 | 一种靶多核苷酸编辑方法及其应用 |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| US12173304B2 (en) * | 2020-07-08 | 2024-12-24 | Neuracle Genetics Inc. | Intron fragments |
| WO2022026410A2 (en) * | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| EP4222270A4 (en) * | 2020-09-29 | 2025-02-26 | Neuexcell Therapeutics Inc. | NEUROD1-VECTOR |
| JP2023543360A (ja) * | 2020-09-29 | 2023-10-13 | ニューエクセル・セラピューティクス・インコーポレイテッド | Ｄｌｘ２ベクター |
| WO2022072309A1 (en) * | 2020-09-29 | 2022-04-07 | NeuExcell Therapeutics Inc. | Ascl1 vector |
| WO2022072325A1 (en) * | 2020-09-29 | 2022-04-07 | NeuExcell Therapeutics Inc. | Neurod1 combination vector |
| WO2022087321A1 (en) * | 2020-10-21 | 2022-04-28 | Duke University | Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy |
| CA3203975A1 (en) | 2020-12-03 | 2022-06-09 | Battelle Memorial Institute | Polymer nanoparticle and dna nanostructure compositions and methods for non-viral delivery |
| WO2022147575A1 (en) * | 2021-01-04 | 2022-07-07 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treatment of friedreich's ataxia |
| CA3216359A1 (en) | 2021-04-07 | 2022-10-13 | Battelle Memorial Institute | Rapid design, build, test, and learn technologies for identifying and using non-viral carriers |
| IL311499A (en) * | 2021-09-17 | 2024-05-01 | Astellas Gene Therapies Inc | Frataxin gene therapy |
| CN119213133B (zh) | 2022-05-16 | 2026-01-06 | 上海天泽云泰生物医药有限公司 | 用于治疗i型戊二酸尿症的重组aav载体 |
| US20250361525A1 (en) * | 2022-06-08 | 2025-11-27 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| EP4598590A1 (en) * | 2022-10-08 | 2025-08-13 | Lingyi Biotech Co., Ltd. | Construct for enhancing gene expression |
| WO2024163678A2 (en) * | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| AU2024353375A1 (en) | 2023-09-29 | 2026-04-09 | Battelle Memorial Institute | Polymer nanoparticle compositions for in vivo expression of polypeptides |
| WO2025122954A1 (en) | 2023-12-08 | 2025-06-12 | Battelle Memorial Institute | Use of dna origami nanostructures for molecular information based data storage systems |
| CN117701638B (zh) * | 2023-12-18 | 2024-10-18 | 苏州新芽基因生物技术有限公司 | 一种抑制残留aav载体质粒和质粒骨架转录活性的方法 |
| WO2026019230A1 (ko) * | 2024-07-15 | 2026-01-22 | 삼성바이오에피스 주식회사 | 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템 |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2002081632A2 (en) * | 2001-04-04 | 2002-10-17 | The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services | Nucleic acids for transgene expression |
| US20090221620A1 (en) * | 2008-02-20 | 2009-09-03 | Celera Corporation | Gentic polymorphisms associated with stroke, methods of detection and uses thereof |
| AU2014255665B2 (en) * | 2013-04-18 | 2018-08-02 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| US20160256571A1 (en) * | 2013-09-30 | 2016-09-08 | Sanofi | Invention |
| US10947596B2 (en) | 2014-05-06 | 2021-03-16 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for identification, assessment, prevention, and treatment of cancer using NFS1 biomarkers and modulators |
| US11697825B2 (en) * | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| MX2017009336A (es) * | 2015-01-16 | 2017-11-15 | Voyager Therapeutics Inc | Polinucleótidos dirigidos al sistema nervioso central. |
| CR20170406A (es) * | 2015-02-10 | 2017-11-14 | Genzyme Corp | Arni variante |
| US11040113B2 (en) * | 2015-03-23 | 2021-06-22 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with Friedreich ataxia |
| WO2016164642A1 (en) * | 2015-04-08 | 2016-10-13 | The United States Of America, As Represented By The Secretary Of Health And Human Services | Viral gene therapy as treatment for cholesterol storage disease or disorder |
| BR112017024514A2 (pt) * | 2015-05-16 | 2018-07-24 | Genzyme Corporation | edição genética de mutações intrônicas profundas |
| MX390848B (es) * | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| WO2017023708A1 (en) * | 2015-07-31 | 2017-02-09 | California Institute Of Technology | Activity-dependent expression of nucleic acids |
| SG11201802408RA (en) * | 2015-11-05 | 2018-05-30 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
| MX2018007330A (es) * | 2015-12-15 | 2019-03-14 | Genzyme Corp | Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. |
| EP4484443A3 (en) * | 2016-06-29 | 2025-03-26 | CRISPR Therapeutics AG | Materials and methods for treatment of friedreich ataxia and other related disorders |
| CA3045335A1 (en) * | 2016-12-01 | 2018-06-07 | Universite Laval | Crispr-based treatment of friedreich ataxia |
| WO2018204764A1 (en) * | 2017-05-05 | 2018-11-08 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
| AU2018338728B2 (en) * | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
-
2019
- 2019-11-21 WO PCT/US2019/062531 patent/WO2020106916A1/en not_active Ceased
- 2019-11-21 EP EP19887003.2A patent/EP3883954A4/en active Pending
- 2019-11-21 PE PE2021000747A patent/PE20211419A1/es unknown
- 2019-11-21 MX MX2021005997A patent/MX2021005997A/es unknown
- 2019-11-21 KR KR1020217017068A patent/KR20210103469A/ko not_active Ceased
- 2019-11-21 CA CA3120289A patent/CA3120289A1/en active Pending
- 2019-11-21 JP JP2021529002A patent/JP7704998B2/ja active Active
- 2019-11-21 AR ARP190103423A patent/AR117145A1/es unknown
- 2019-11-21 AU AU2019385506A patent/AU2019385506A1/en active Pending
- 2019-11-21 BR BR112021009733A patent/BR112021009733A2/pt unknown
- 2019-11-21 CN CN201980088932.7A patent/CN113302201A/zh active Pending
- 2019-11-21 SG SG11202105326WA patent/SG11202105326WA/en unknown
- 2019-11-21 EA EA202191418A patent/EA202191418A1/ru unknown
- 2019-11-21 TW TW108142416A patent/TW202039533A/zh unknown
-
2021
- 2021-05-20 PH PH12021551155A patent/PH12021551155A1/en unknown
- 2021-05-20 CL CL2021001327A patent/CL2021001327A1/es unknown
- 2021-05-20 IL IL283344A patent/IL283344A/en unknown
- 2021-05-21 US US17/326,884 patent/US20210324418A1/en active Pending
- 2021-06-21 CO CONC2021/0008120A patent/CO2021008120A2/es unknown
- 2021-06-21 EC ECSENADI202144840A patent/ECSP21044840A/es unknown
-
2025
- 2025-03-05 JP JP2025034621A patent/JP2025102783A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CO2021008120A2 (es) | 2021-08-09 |
| AU2019385506A1 (en) | 2021-06-03 |
| SG11202105326WA (en) | 2021-06-29 |
| JP2025102783A (ja) | 2025-07-08 |
| PE20211419A1 (es) | 2021-08-03 |
| EP3883954A4 (en) | 2022-08-10 |
| MX2021005997A (es) | 2021-08-11 |
| JP7704998B2 (ja) | 2025-07-09 |
| PH12021551155A1 (en) | 2021-11-03 |
| BR112021009733A2 (pt) | 2022-01-04 |
| CN113302201A (zh) | 2021-08-24 |
| EP3883954A1 (en) | 2021-09-29 |
| EA202191418A1 (ru) | 2021-08-05 |
| US20210324418A1 (en) | 2021-10-21 |
| WO2020106916A1 (en) | 2020-05-28 |
| AR117145A1 (es) | 2021-07-14 |
| IL283344A (en) | 2021-07-29 |
| TW202039533A (zh) | 2020-11-01 |
| JP2022508182A (ja) | 2022-01-19 |
| ECSP21044840A (es) | 2021-09-30 |
| CA3120289A1 (en) | 2020-05-28 |
| KR20210103469A (ko) | 2021-08-23 |
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