KR20240038705A - 간 유전자의 생체 내 편집 방법 - Google Patents
간 유전자의 생체 내 편집 방법 Download PDFInfo
- Publication number
- KR20240038705A KR20240038705A KR1020247002171A KR20247002171A KR20240038705A KR 20240038705 A KR20240038705 A KR 20240038705A KR 1020247002171 A KR1020247002171 A KR 1020247002171A KR 20247002171 A KR20247002171 A KR 20247002171A KR 20240038705 A KR20240038705 A KR 20240038705A
- Authority
- KR
- South Korea
- Prior art keywords
- ttr
- guide rna
- cas nuclease
- administration
- gene
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/42—Oxazoles
- A61K31/423—Oxazoles condensed with carbocyclic rings
-
- 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
- A61K31/60—Salicylic acid; Derivatives thereof
- A61K31/603—Salicylic acid; Derivatives thereof having further aromatic rings, e.g. diflunisal
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
- A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
- A61K47/6927—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
- A61K47/6929—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- 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
-
- 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
-
- 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
- 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]
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Nanotechnology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Optics & Photonics (AREA)
- Dermatology (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163202744P | 2021-06-22 | 2021-06-22 | |
| US63/202,744 | 2021-06-22 | ||
| US202163202812P | 2021-06-25 | 2021-06-25 | |
| US63/202,812 | 2021-06-25 | ||
| US202163263466P | 2021-11-03 | 2021-11-03 | |
| US63/263,466 | 2021-11-03 | ||
| US202163264435P | 2021-11-22 | 2021-11-22 | |
| US63/264,435 | 2021-11-22 | ||
| US202263314878P | 2022-02-28 | 2022-02-28 | |
| US63/314,878 | 2022-02-28 | ||
| PCT/US2022/034454 WO2022271780A1 (en) | 2021-06-22 | 2022-06-22 | Methods for in vivo editing of a liver gene |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20240038705A true KR20240038705A (ko) | 2024-03-25 |
Family
ID=82655316
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247002171A Pending KR20240038705A (ko) | 2021-06-22 | 2022-06-22 | 간 유전자의 생체 내 편집 방법 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20250269062A1 (https=) |
| EP (1) | EP4359531A1 (https=) |
| JP (1) | JP2024527525A (https=) |
| KR (1) | KR20240038705A (https=) |
| AU (1) | AU2022296523A1 (https=) |
| CA (1) | CA3224995A1 (https=) |
| IL (1) | IL309055A (https=) |
| MX (1) | MX2023015213A (https=) |
| TW (1) | TW202327626A (https=) |
| WO (1) | WO2022271780A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| JP3131222B2 (ja) | 1991-12-24 | 2001-01-31 | アイシス・ファーマシューティカルス・インコーポレーテッド | ギャップを有する2′修飾オリゴヌクレオチド |
| JPH10500310A (ja) | 1994-05-19 | 1998-01-13 | ダコ アクティーゼルスカブ | 淋菌及びトラコーマ クラミジアの検出のためのpna プローブ |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| KR20230156800A (ko) | 2015-03-03 | 2023-11-14 | 더 제너럴 하스피탈 코포레이션 | 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제 |
| CN120842296A (zh) | 2015-09-21 | 2025-10-28 | 垂林克生物技术有限公司 | 用于合成5’-加帽rna的组合物和方法 |
| LT3436077T (lt) | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| BR112019015220B1 (pt) | 2017-09-29 | 2023-10-03 | CF&I Steel L.P. D/B/A EVRAZ Rocky Mountain Steel | Método para unir trilhos de aço com entrada de calor por solda controlada |
| EP3688161A1 (en) | 2017-09-29 | 2020-08-05 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating attr amyloidosis |
| US20230140670A1 (en) | 2017-09-29 | 2023-05-04 | Intellia Therapeutics, Inc. | Formulations |
| US20220009878A1 (en) * | 2018-10-02 | 2022-01-13 | Intellia Therapeutics, Inc. | Ionizable amine lipids |
| AU2020248337A1 (en) * | 2019-03-28 | 2021-11-04 | Intellia Therapeutics, Inc. | Compositions and methods for ttr gene editing and treating ATTR amyloidosis comprising a corticosteroid or use thereof |
| JP7631215B2 (ja) * | 2019-03-28 | 2025-02-18 | インテリア セラピューティクス,インコーポレイテッド | Ttrガイドrnaと、rnaガイドdna結合剤をコードするポリヌクレオチドとを含む組成物および方法 |
| WO2021046265A1 (en) * | 2019-09-06 | 2021-03-11 | Generation Bio Co. | Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof |
| EP4073249A1 (en) * | 2019-12-11 | 2022-10-19 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
-
2022
- 2022-06-22 KR KR1020247002171A patent/KR20240038705A/ko active Pending
- 2022-06-22 WO PCT/US2022/034454 patent/WO2022271780A1/en not_active Ceased
- 2022-06-22 CA CA3224995A patent/CA3224995A1/en active Pending
- 2022-06-22 TW TW111123229A patent/TW202327626A/zh unknown
- 2022-06-22 AU AU2022296523A patent/AU2022296523A1/en active Pending
- 2022-06-22 EP EP22744574.9A patent/EP4359531A1/en active Pending
- 2022-06-22 JP JP2023579195A patent/JP2024527525A/ja active Pending
- 2022-06-22 IL IL309055A patent/IL309055A/en unknown
- 2022-06-22 US US18/572,155 patent/US20250269062A1/en active Pending
- 2022-06-22 MX MX2023015213A patent/MX2023015213A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20250269062A1 (en) | 2025-08-28 |
| CA3224995A1 (en) | 2022-12-29 |
| TW202327626A (zh) | 2023-07-16 |
| MX2023015213A (es) | 2024-03-19 |
| IL309055A (en) | 2024-02-01 |
| WO2022271780A1 (en) | 2022-12-29 |
| EP4359531A1 (en) | 2024-05-01 |
| JP2024527525A (ja) | 2024-07-25 |
| AU2022296523A1 (en) | 2023-12-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
Patent event date: 20240119 Patent event code: PA01051R01D Comment text: International Patent Application |
|
| PG1501 | Laying open of application | ||
| A201 | Request for examination | ||
| PA0201 | Request for examination |
Patent event code: PA02012R01D Patent event date: 20250623 Comment text: Request for Examination of Application |