CO2023016031A2 - Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos - Google Patents
Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticosInfo
- Publication number
- CO2023016031A2 CO2023016031A2 CONC2023/0016031A CO2023016031A CO2023016031A2 CO 2023016031 A2 CO2023016031 A2 CO 2023016031A2 CO 2023016031 A CO2023016031 A CO 2023016031A CO 2023016031 A2 CO2023016031 A2 CO 2023016031A2
- Authority
- CO
- Colombia
- Prior art keywords
- nuclease
- pcsk9
- treatment
- nucleic acid
- acid sequence
- Prior art date
Links
- 101710163270 Nuclease Proteins 0.000 title abstract 4
- 208000026350 Inborn Genetic disease Diseases 0.000 title abstract 2
- 208000016361 genetic disease Diseases 0.000 title abstract 2
- 230000008685 targeting Effects 0.000 title abstract 2
- 230000002068 genetic effect Effects 0.000 title 1
- 238000001727 in vivo Methods 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 101001098868 Homo sapiens Proprotein convertase subtilisin/kexin type 9 Proteins 0.000 abstract 3
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 3
- 102100038955 Proprotein convertase subtilisin/kexin type 9 Human genes 0.000 abstract 3
- 150000007523 nucleic acids Chemical group 0.000 abstract 3
- 101150094724 PCSK9 gene Proteins 0.000 abstract 2
- 230000009977 dual effect Effects 0.000 abstract 2
- 238000010362 genome editing Methods 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/40—Systems of functionally co-operating vectors
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Se proporciona un sistema de vectores duales para el tratamiento de un trastorno genético. El sistema incluye (a) un vector de edición génica que comprende un casete de expresión que comprende una secuencia de ácido nucleico que codifica una nucleasa y secuencias reguladoras que dirigen la expresión de la nucleasa en una célula objetivo que comprende un gen PCSK9; y (b) un vector donante que comprende una secuencia de ácido nucleico que codifica un producto exógeno para su expresión desde el locus de PCSK9, donde la secuencia de ácido nucleico insertada no codifica PCSK9, donde el sistema comprende además secuencias que dirigen a la nucleasa específicamente hacia el locus del gen PCSK9 natural; y donde el PCSK9 natural de la célula objetivo se encuentra opcionalmente extirpado o reducido después de la dosificación con el sistema de vectores duales.
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163180603P | 2021-04-27 | 2021-04-27 | |
US202163242474P | 2021-09-09 | 2021-09-09 | |
US202163244205P | 2021-09-14 | 2021-09-14 | |
US202263301933P | 2022-01-21 | 2022-01-21 | |
US202263331385P | 2022-04-15 | 2022-04-15 | |
PCT/US2022/026483 WO2022232232A1 (en) | 2021-04-27 | 2022-04-27 | Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2023016031A2 true CO2023016031A2 (es) | 2023-12-11 |
Family
ID=83847293
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2023/0016031A CO2023016031A2 (es) | 2021-04-27 | 2023-11-23 | Composiciones y métodos para el direccionamiento genético in vivo mediado por nucleasas para el tratamiento de trastornos genéticos |
Country Status (9)
Country | Link |
---|---|
EP (1) | EP4330412A1 (es) |
KR (1) | KR20240001708A (es) |
AU (1) | AU2022266662A1 (es) |
BR (1) | BR112023021129A2 (es) |
CA (1) | CA3216285A1 (es) |
CO (1) | CO2023016031A2 (es) |
IL (1) | IL307958A (es) |
TW (1) | TW202304528A (es) |
WO (1) | WO2022232232A1 (es) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023140971A1 (en) * | 2022-01-21 | 2023-07-27 | The Trustees Of The University Ofpennsylvania | Methods for treatment of ornithine transcarbamylase (otc) deficiency |
CN116790604B (zh) * | 2023-08-18 | 2023-10-27 | 成都中科奥格生物科技有限公司 | 一种sgRNA、CRISPR/Cas9载体及其构建方法和用途 |
Family Cites Families (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP6851319B2 (ja) * | 2015-04-27 | 2021-03-31 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系 |
EP3714048A1 (en) * | 2017-11-22 | 2020-09-30 | Modernatx, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
-
2022
- 2022-04-27 IL IL307958A patent/IL307958A/en unknown
- 2022-04-27 EP EP22796614.0A patent/EP4330412A1/en active Pending
- 2022-04-27 AU AU2022266662A patent/AU2022266662A1/en active Pending
- 2022-04-27 BR BR112023021129A patent/BR112023021129A2/pt unknown
- 2022-04-27 CA CA3216285A patent/CA3216285A1/en active Pending
- 2022-04-27 KR KR1020237040431A patent/KR20240001708A/ko unknown
- 2022-04-27 TW TW111116003A patent/TW202304528A/zh unknown
- 2022-04-27 WO PCT/US2022/026483 patent/WO2022232232A1/en active Application Filing
-
2023
- 2023-11-23 CO CONC2023/0016031A patent/CO2023016031A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
EP4330412A1 (en) | 2024-03-06 |
AU2022266662A1 (en) | 2023-11-02 |
WO2022232232A1 (en) | 2022-11-03 |
AU2022266662A9 (en) | 2023-11-16 |
BR112023021129A2 (pt) | 2023-12-12 |
CA3216285A1 (en) | 2022-11-03 |
IL307958A (en) | 2023-12-01 |
TW202304528A (zh) | 2023-02-01 |
KR20240001708A (ko) | 2024-01-03 |
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