CL2023001593A1 - Composiciones y métodos para dirigirse específicamente a bcl11a - Google Patents
Composiciones y métodos para dirigirse específicamente a bcl11aInfo
- Publication number
- CL2023001593A1 CL2023001593A1 CL2023001593A CL2023001593A CL2023001593A1 CL 2023001593 A1 CL2023001593 A1 CL 2023001593A1 CL 2023001593 A CL2023001593 A CL 2023001593A CL 2023001593 A CL2023001593 A CL 2023001593A CL 2023001593 A1 CL2023001593 A1 CL 2023001593A1
- Authority
- CL
- Chile
- Prior art keywords
- systems
- specifically target
- compositions
- methods
- nucleic acids
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 abstract 3
- 102000039446 nucleic acids Human genes 0.000 abstract 3
- 150000007523 nucleic acids Chemical class 0.000 abstract 3
- 101100493741 Homo sapiens BCL11A gene Proteins 0.000 abstract 2
- 108091033409 CRISPR Proteins 0.000 abstract 1
- 238000010354 CRISPR gene editing Methods 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000034737 hemoglobinopathy Diseases 0.000 abstract 1
- 229920001184 polypeptide Polymers 0.000 abstract 1
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 1
- 102000004196 processed proteins & peptides Human genes 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/14—Blood; Artificial blood
- A61K35/18—Erythrocytes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/34—Muscles; Smooth muscle cells; Heart; Cardiac stem cells; Myoblasts; Myocytes; Cardiomyocytes
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/48—Reproductive organs
- A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
- A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
-
- 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
- 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
-
- 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
-
- 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
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- 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 [RNase]; Deoxyribonucleases [DNase]
-
- 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
-
- 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 [CRISPR]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Cell Biology (AREA)
- General Health & Medical Sciences (AREA)
- Developmental Biology & Embryology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Reproductive Health (AREA)
- Hematology (AREA)
- Gynecology & Obstetrics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Mycology (AREA)
- Cardiology (AREA)
- Vascular Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063120885P | 2020-12-03 | 2020-12-03 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2023001593A1 true CL2023001593A1 (es) | 2023-12-15 |
Family
ID=81854906
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2023001593A CL2023001593A1 (es) | 2020-12-03 | 2023-06-02 | Composiciones y métodos para dirigirse específicamente a bcl11a |
| CL2024001577A CL2024001577A1 (es) | 2020-12-03 | 2024-05-27 | Composiciones y métodos para dirigirse específicamente a bcl11a |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2024001577A CL2024001577A1 (es) | 2020-12-03 | 2024-05-27 | Composiciones y métodos para dirigirse específicamente a bcl11a |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20240026386A1 (https=) |
| EP (1) | EP4255502A4 (https=) |
| JP (1) | JP2023552379A (https=) |
| KR (1) | KR20230129230A (https=) |
| CN (1) | CN116801913A (https=) |
| AU (1) | AU2021391783A1 (https=) |
| BR (1) | BR112023010717A2 (https=) |
| CA (1) | CA3200815A1 (https=) |
| CL (2) | CL2023001593A1 (https=) |
| CO (1) | CO2023007900A2 (https=) |
| GB (1) | GB2616795B (https=) |
| IL (1) | IL303359A (https=) |
| MX (1) | MX2023006565A (https=) |
| PE (1) | PE20231177A1 (https=) |
| WO (1) | WO2022120094A2 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20230128289A (ko) | 2020-12-03 | 2023-09-04 | 스크라이브 테라퓨틱스 인크. | 조작된 클래스 2 유형 v crispr 시스템 |
| WO2022261149A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| WO2023235888A2 (en) | 2022-06-03 | 2023-12-07 | Scribe Therapeutics Inc. | COMPOSITIONS AND METHODS FOR CpG DEPLETION |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| TW202413643A (zh) | 2022-06-07 | 2024-04-01 | 美商斯奎柏治療公司 | 用於靶向pcsk9的組合物及方法 |
| WO2023240027A1 (en) | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Particle delivery systems |
| US20250361525A1 (en) | 2022-06-08 | 2025-11-27 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2017252023B2 (en) * | 2016-04-18 | 2024-05-02 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| MX2019003674A (es) * | 2016-09-30 | 2021-01-08 | Univ California | Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas. |
| CN110168084A (zh) * | 2016-12-05 | 2019-08-23 | 爱迪塔斯医药公司 | 用于内源和来源DNA的单发指导RNA(ogRNA)靶向的系统和方法 |
| US20190367924A1 (en) * | 2017-02-17 | 2019-12-05 | Temple University - Of The Commonwealth System Of Higher Education | Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5 |
| CN109722414A (zh) * | 2017-10-27 | 2019-05-07 | 博雅辑因（北京）生物科技有限公司 | 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基 |
| WO2019213273A1 (en) * | 2018-05-01 | 2019-11-07 | The Children's Medical Center Corporation | Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9 |
| JP2021532815A (ja) * | 2018-08-07 | 2021-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 新規Ｃａｓ１２ｂ酵素およびシステム |
-
2021
- 2021-12-02 AU AU2021391783A patent/AU2021391783A1/en active Pending
- 2021-12-02 EP EP21901487.5A patent/EP4255502A4/en active Pending
- 2021-12-02 IL IL303359A patent/IL303359A/en unknown
- 2021-12-02 WO PCT/US2021/061672 patent/WO2022120094A2/en not_active Ceased
- 2021-12-02 CN CN202180092086.3A patent/CN116801913A/zh active Pending
- 2021-12-02 GB GB2309871.8A patent/GB2616795B/en active Active
- 2021-12-02 MX MX2023006565A patent/MX2023006565A/es unknown
- 2021-12-02 CA CA3200815A patent/CA3200815A1/en active Pending
- 2021-12-02 US US18/255,172 patent/US20240026386A1/en active Pending
- 2021-12-02 PE PE2023001811A patent/PE20231177A1/es unknown
- 2021-12-02 BR BR112023010717A patent/BR112023010717A2/pt unknown
- 2021-12-02 JP JP2023533861A patent/JP2023552379A/ja active Pending
- 2021-12-02 KR KR1020237022391A patent/KR20230129230A/ko active Pending
-
2023
- 2023-06-02 CL CL2023001593A patent/CL2023001593A1/es unknown
- 2023-06-16 CO CONC2023/0007900A patent/CO2023007900A2/es unknown
-
2024
- 2024-05-27 CL CL2024001577A patent/CL2024001577A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA3200815A1 (en) | 2022-06-09 |
| CO2023007900A2 (es) | 2023-06-30 |
| KR20230129230A (ko) | 2023-09-07 |
| EP4255502A2 (en) | 2023-10-11 |
| US20240026386A1 (en) | 2024-01-25 |
| EP4255502A4 (en) | 2025-07-16 |
| GB2616795A (en) | 2023-09-20 |
| WO2022120094A2 (en) | 2022-06-09 |
| PE20231177A1 (es) | 2023-08-01 |
| JP2023552379A (ja) | 2023-12-15 |
| GB2616795B (en) | 2025-08-13 |
| GB202309871D0 (en) | 2023-08-16 |
| BR112023010717A2 (pt) | 2023-10-03 |
| WO2022120094A3 (en) | 2022-08-25 |
| AU2021391783A1 (en) | 2023-06-22 |
| MX2023006565A (es) | 2023-08-07 |
| IL303359A (en) | 2023-08-01 |
| CL2024001577A1 (es) | 2024-10-18 |
| CN116801913A (zh) | 2023-09-22 |
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