IL307874A - All-in-one DENDRIMER-based lipid particles enable precise gene editing mediated by HDR IN VIVO - Google Patents
All-in-one DENDRIMER-based lipid particles enable precise gene editing mediated by HDR IN VIVOInfo
- Publication number
- IL307874A IL307874A IL307874A IL30787423A IL307874A IL 307874 A IL307874 A IL 307874A IL 307874 A IL307874 A IL 307874A IL 30787423 A IL30787423 A IL 30787423A IL 307874 A IL307874 A IL 307874A
- Authority
- IL
- Israel
- Prior art keywords
- dendrimer
- vivo
- gene editing
- mediated gene
- lipid nanoparticles
- Prior art date
Links
- 239000000412 dendrimer Substances 0.000 title 1
- 229920000736 dendritic polymer Polymers 0.000 title 1
- 238000010362 genome editing Methods 0.000 title 1
- 238000001727 in vivo Methods 0.000 title 1
- 150000002632 lipids Chemical class 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
- 239000002105 nanoparticle Substances 0.000 title 1
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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- 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
- 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
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing 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
- C12N15/11—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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- 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
- 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)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Crystallography & Structural Chemistry (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163178453P | 2021-04-22 | 2021-04-22 | |
| PCT/US2022/026001 WO2022226344A1 (en) | 2021-04-22 | 2022-04-22 | All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL307874A true IL307874A (en) | 2023-12-01 |
Family
ID=83722696
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL307874A IL307874A (en) | 2021-04-22 | 2022-04-22 | All-in-one DENDRIMER-based lipid particles enable precise gene editing mediated by HDR IN VIVO |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20240207442A1 (https=) |
| EP (1) | EP4326886A4 (https=) |
| JP (1) | JP2024516164A (https=) |
| CN (1) | CN117500933A (https=) |
| AU (1) | AU2022262422A1 (https=) |
| CA (1) | CA3215509A1 (https=) |
| IL (1) | IL307874A (https=) |
| WO (1) | WO2022226344A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3349802B1 (en) * | 2015-09-14 | 2021-08-04 | The Board of Regents of the University of Texas System | Lipocationic dendrimers and uses thereof |
| JP2024511463A (ja) | 2021-03-22 | 2024-03-13 | リコード セラピューティクス，インク． | 細胞への標的送達のための組成物および方法 |
| US20250382640A1 (en) * | 2022-06-29 | 2025-12-18 | Juno Therapeutics, Inc. | Lipid nanoparticles for delivery of nucleic acids |
| US12364773B2 (en) | 2023-12-01 | 2025-07-22 | Recode Therapeutics, Inc. | Lipid nanoparticle compositions and uses thereof |
| CN120754284A (zh) * | 2025-07-30 | 2025-10-10 | 中国医学科学院基础医学研究所 | 一种用于肝脏靶向联合递送CRISPR-Cas9系统和单链DNA修复模板的脂质纳米颗粒 |
Family Cites Families (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| US5367066A (en) | 1984-10-16 | 1994-11-22 | Chiron Corporation | Oligonucleotides with selectably cleavable and/or abasic sites |
| FR2575751B1 (fr) | 1985-01-08 | 1987-04-03 | Pasteur Institut | Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques |
| AU598946B2 (en) | 1987-06-24 | 1990-07-05 | Howard Florey Institute Of Experimental Physiology And Medicine | Nucleoside derivatives |
| US5175273A (en) | 1988-07-01 | 1992-12-29 | Genentech, Inc. | Nucleic acid intercalating agents |
| US5134066A (en) | 1989-08-29 | 1992-07-28 | Monsanto Company | Improved probes using nucleosides containing 3-dezauracil analogs |
| US5130302A (en) | 1989-12-20 | 1992-07-14 | Boron Bilogicals, Inc. | Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same |
| US5587470A (en) | 1990-01-11 | 1996-12-24 | Isis Pharmaceuticals, Inc. | 3-deazapurines |
| US5459255A (en) | 1990-01-11 | 1995-10-17 | Isis Pharmaceuticals, Inc. | N-2 substituted purines |
| US5681941A (en) | 1990-01-11 | 1997-10-28 | Isis Pharmaceuticals, Inc. | Substituted purines and oligonucleotide cross-linking |
| BR9106702A (pt) | 1990-07-27 | 1993-06-08 | Isis Pharmaceuticals Inc | Analogo de oligonucleotideos e processos para modular a producao de uma proteina por um organismo e para tratar um organismo |
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| US5594121A (en) | 1991-11-07 | 1997-01-14 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified purines |
| AU3222793A (en) | 1991-11-26 | 1993-06-28 | Gilead Sciences, Inc. | Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines |
| US5484908A (en) | 1991-11-26 | 1996-01-16 | Gilead Sciences, Inc. | Oligonucleotides containing 5-propynyl pyrimidines |
| TW393513B (en) | 1991-11-26 | 2000-06-11 | Isis Pharmaceuticals Inc | Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines |
| US5502177A (en) | 1993-09-17 | 1996-03-26 | Gilead Sciences, Inc. | Pyrimidine derivatives for labeled binding partners |
| US5457187A (en) | 1993-12-08 | 1995-10-10 | Board Of Regents University Of Nebraska | Oligonucleotides containing 5-fluorouracil |
| US5596091A (en) | 1994-03-18 | 1997-01-21 | The Regents Of The University Of California | Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
| ES2646630T3 (es) | 2008-11-07 | 2017-12-14 | Massachusetts Institute Of Technology | Lipidoides aminoalcohólicos y usos de los mismos |
| AP2012006053A0 (en) | 2009-06-02 | 2012-02-29 | Brian Charles Keller | Pure peg-lipid conjugates. |
| US8889394B2 (en) | 2009-09-07 | 2014-11-18 | Empire Technology Development Llc | Multiple domain proteins |
| US20140141070A1 (en) * | 2011-07-06 | 2014-05-22 | Andrew Geall | Liposomes having useful n:p ratio for delivery of rna molecules |
| EP3349802B1 (en) * | 2015-09-14 | 2021-08-04 | The Board of Regents of the University of Texas System | Lipocationic dendrimers and uses thereof |
| EP3538515B1 (en) * | 2016-11-08 | 2022-07-20 | Ramot at Tel-Aviv University Ltd. | Cationic lipids for nucleic acid delivery and preparation thereof |
| WO2019204226A1 (en) * | 2018-04-16 | 2019-10-24 | University Of Massachusetts | Compositions and methods for improved gene editing |
| US12357580B2 (en) * | 2018-06-19 | 2025-07-15 | The Board Of Regents Of The University Of Texas System | Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids |
| WO2020051220A1 (en) * | 2018-09-04 | 2020-03-12 | The Board of the Regents of the University of Texas System | Compositions and methods for organ specific delivery of nucleic acids |
| CN112996519B (zh) * | 2018-09-04 | 2025-02-28 | 德克萨斯大学系统董事会 | 用于核酸的器官特异性递送的组合物和方法 |
| CA3121191A1 (en) * | 2018-11-28 | 2020-06-04 | Crispr Therapeutics Ag | Optimized mrna encoding cas9 for use in lnps |
| WO2020186213A1 (en) * | 2019-03-14 | 2020-09-17 | The Broad Institute, Inc. | Novel nucleic acid modifiers |
| WO2020241679A1 (ja) * | 2019-05-30 | 2020-12-03 | 国立大学法人北海道大学 | 脂質ナノ粒子 |
| WO2022204043A1 (en) * | 2021-03-22 | 2022-09-29 | The Board Of Regents Of The University Of Texas System | Compositions and methods for targeted delivery to cells |
| JP2024511463A (ja) * | 2021-03-22 | 2024-03-13 | リコード セラピューティクス，インク． | 細胞への標的送達のための組成物および方法 |
| EP4313004A4 (en) * | 2021-03-23 | 2025-01-15 | Recode Therapeutics, Inc. | Compositions and methods for targeted systemic delivery to cells |
| US20250281580A1 (en) * | 2021-04-05 | 2025-09-11 | The Board Of Regents Of The University Of Texas System | Compositions, methods and uses for treating cystic fibrosis and related disorders |
| US20240261440A1 (en) * | 2021-04-05 | 2024-08-08 | The Board Of Regents Of The University Of Texas System | Compositions, methods and uses for treating cystic fibrosis and related disorders |
| WO2023215790A1 (en) * | 2022-05-03 | 2023-11-09 | The Board Of Regents Of The University Of Texas System | Co-delivery of inhibitory nucleic acids and genome editors for tumor therapy |
-
2022
- 2022-04-22 WO PCT/US2022/026001 patent/WO2022226344A1/en not_active Ceased
- 2022-04-22 CA CA3215509A patent/CA3215509A1/en active Pending
- 2022-04-22 JP JP2023564557A patent/JP2024516164A/ja active Pending
- 2022-04-22 CN CN202280042709.0A patent/CN117500933A/zh active Pending
- 2022-04-22 IL IL307874A patent/IL307874A/en unknown
- 2022-04-22 US US18/556,614 patent/US20240207442A1/en active Pending
- 2022-04-22 AU AU2022262422A patent/AU2022262422A1/en active Pending
- 2022-04-22 EP EP22792601.1A patent/EP4326886A4/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3215509A1 (en) | 2022-10-27 |
| AU2022262422A1 (en) | 2023-11-02 |
| CN117500933A (zh) | 2024-02-02 |
| JP2024516164A (ja) | 2024-04-12 |
| EP4326886A1 (en) | 2024-02-28 |
| EP4326886A4 (en) | 2025-04-30 |
| AU2022262422A9 (en) | 2023-11-16 |
| US20240207442A1 (en) | 2024-06-27 |
| WO2022226344A1 (en) | 2022-10-27 |
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