CO2020002674A2 - Moléculas de ácido nucleico y sus usos - Google Patents
Moléculas de ácido nucleico y sus usosInfo
- Publication number
- CO2020002674A2 CO2020002674A2 CONC2020/0002674A CO2020002674A CO2020002674A2 CO 2020002674 A2 CO2020002674 A2 CO 2020002674A2 CO 2020002674 A CO2020002674 A CO 2020002674A CO 2020002674 A2 CO2020002674 A2 CO 2020002674A2
- Authority
- CO
- Colombia
- Prior art keywords
- itr
- nucleic acid
- acid molecules
- present disclosure
- polypeptide
- 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
- 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
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- 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)
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- 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
- 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
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/644—Coagulation factor IXa (3.4.21.22)
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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
- 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
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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
- 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
- C12N2750/14211—Erythrovirus, e.g. B19 virus
- C12N2750/14241—Use of virus, viral particle or viral elements as a vector
- C12N2750/14243—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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Hematology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente descripción proporciona moléculas de ácido nucleico que comprenden una primera repetición terminal invertida (ITR), una segunda ITR y un casete génico que codifica un ARNmi y/o una proteína terapéutica. En ciertas realizaciones, la proteína terapéutica comprende un factor de coagulación, por ejemplo, un polipéptido FVIII, un polipéptido FIX o un fragmento del mismo. En algunas realizaciones, la primera ITR y/o la segunda ITR es una ITR de un virus no adenoasociado (AAV). La presente descripción también proporciona métodos para tratar trastornos hemorrágicos, tales como hemofilia, que comprenden administrar al sujeto la molécula de ácido nucleico o un polipéptido codificado por el mismo.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762543297P | 2017-08-09 | 2017-08-09 | |
PCT/US2018/046110 WO2019032898A1 (en) | 2017-08-09 | 2018-08-09 | NUCLEIC ACID MOLECULES AND USES THEREOF |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2020002674A2 true CO2020002674A2 (es) | 2020-08-10 |
Family
ID=63638309
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2020/0002674A CO2020002674A2 (es) | 2017-08-09 | 2020-03-09 | Moléculas de ácido nucleico y sus usos |
Country Status (15)
Country | Link |
---|---|
US (1) | US20210163986A1 (es) |
EP (1) | EP3665289A1 (es) |
JP (2) | JP7374883B2 (es) |
KR (1) | KR20200035130A (es) |
CN (1) | CN111247251A (es) |
AU (1) | AU2018313921A1 (es) |
BR (1) | BR112020002394A2 (es) |
CA (1) | CA3072334A1 (es) |
CO (1) | CO2020002674A2 (es) |
IL (1) | IL272463A (es) |
MX (1) | MX2020001593A (es) |
PH (1) | PH12020500222A1 (es) |
SG (1) | SG11202000764RA (es) |
TW (1) | TW201920255A (es) |
WO (1) | WO2019032898A1 (es) |
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JP2021533753A (ja) * | 2018-08-09 | 2021-12-09 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用 |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
CA3129321A1 (en) * | 2019-02-15 | 2020-08-20 | Generation Bio Co. | Modulation of rep protein activity in closed-ended dna (cedna) production |
WO2020186150A2 (en) * | 2019-03-13 | 2020-09-17 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) therapeutics |
SG11202109850SA (en) * | 2019-03-13 | 2021-10-28 | Generation Bio Co | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
MX2021012540A (es) | 2019-04-17 | 2022-01-18 | Codiak Biosciences Inc | Composiciones de exosomas y virus adenoasociados (aav). |
CN110684798A (zh) * | 2019-09-03 | 2020-01-14 | 深圳新诺微环生物科技有限公司 | 肌肉靶向的微环dna基因治疗 |
WO2021078834A1 (en) * | 2019-10-22 | 2021-04-29 | Genethon | Chimeric acid-alpha glucosidase polypeptides and uses thereof |
CN111334528B (zh) * | 2020-03-20 | 2022-02-18 | 山东农业大学 | 水貂肠炎细小病毒全基因组感染性克隆及其构建方法和应用 |
EP4087391A1 (en) | 2020-04-09 | 2022-11-16 | University of Sheffield | Gene therapy treatment |
WO2022023284A1 (en) | 2020-07-27 | 2022-02-03 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
CA3189673A1 (en) | 2020-08-23 | 2022-03-03 | Ajay MAGHODIA | Modified baculovirus system for improved production of closed-ended dna (cedna) |
WO2023028440A2 (en) | 2021-08-23 | 2023-03-02 | Bioverativ Therapeutics Inc. | Baculovirus expression system |
WO2023028441A1 (en) * | 2021-08-23 | 2023-03-02 | Bioverativ Therapeutics Inc. | Engineered itr sequences and methods of use |
KR20230085335A (ko) | 2021-12-07 | 2023-06-14 | 서제표 | 사용자 맞춤형 심리상담 전문가 매칭 장치 |
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-
2018
- 2018-08-09 SG SG11202000764RA patent/SG11202000764RA/en unknown
- 2018-08-09 CN CN201880065324.XA patent/CN111247251A/zh active Pending
- 2018-08-09 MX MX2020001593A patent/MX2020001593A/es unknown
- 2018-08-09 EP EP18772954.6A patent/EP3665289A1/en active Pending
- 2018-08-09 US US16/636,583 patent/US20210163986A1/en active Pending
- 2018-08-09 KR KR1020207006705A patent/KR20200035130A/ko not_active Application Discontinuation
- 2018-08-09 WO PCT/US2018/046110 patent/WO2019032898A1/en unknown
- 2018-08-09 TW TW107127876A patent/TW201920255A/zh unknown
- 2018-08-09 CA CA3072334A patent/CA3072334A1/en active Pending
- 2018-08-09 AU AU2018313921A patent/AU2018313921A1/en active Pending
- 2018-08-09 BR BR112020002394-9A patent/BR112020002394A2/pt unknown
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EP3665289A1 (en) | 2020-06-17 |
CA3072334A1 (en) | 2019-02-14 |
RU2020109872A (ru) | 2021-09-14 |
US20210163986A1 (en) | 2021-06-03 |
KR20200035130A (ko) | 2020-04-01 |
WO2019032898A1 (en) | 2019-02-14 |
AU2018313921A1 (en) | 2020-03-12 |
JP2020530999A (ja) | 2020-11-05 |
CN111247251A (zh) | 2020-06-05 |
JP7374883B2 (ja) | 2023-11-07 |
BR112020002394A2 (pt) | 2020-07-28 |
MX2020001593A (es) | 2020-07-13 |
PH12020500222A1 (en) | 2020-11-09 |
RU2020109872A3 (es) | 2021-11-22 |
TW201920255A (zh) | 2019-06-01 |
JP2024010074A (ja) | 2024-01-23 |
SG11202000764RA (en) | 2020-02-27 |
IL272463A (en) | 2020-03-31 |
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