CA3232690A1 - Multi-motif dendrons and their supramolecular structures and uses thereof - Google Patents

Multi-motif dendrons and their supramolecular structures and uses thereof Download PDF

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Publication number
CA3232690A1
CA3232690A1 CA3232690A CA3232690A CA3232690A1 CA 3232690 A1 CA3232690 A1 CA 3232690A1 CA 3232690 A CA3232690 A CA 3232690A CA 3232690 A CA3232690 A CA 3232690A CA 3232690 A1 CA3232690 A1 CA 3232690A1
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Prior art keywords
dendron
composition
terminal group
peg
generation
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English (en)
French (fr)
Inventor
Omar Fizal KHAN
Grayson TILSTRA
Alanna Margaret MANNING
Yan Ming Anson LAU
Julien Couture-Senecal
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University of Toronto
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University of Toronto
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    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/10—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by nitrogen atoms not being part of nitro or nitroso groups
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    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08G—MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G83/00—Macromolecular compounds not provided for in groups C08G2/00 - C08G81/00
    • C08G83/002—Dendritic macromolecules
    • C08G83/003—Dendrimers
    • 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
    • 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
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    • C07—ORGANIC CHEMISTRY
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    • C07C275/00—Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups
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    • C07C323/23—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton
    • C07C323/39—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton at least one of the nitrogen atoms being part of any of the groups, X being a hetero atom, Y being any atom
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    • C07C327/06—Monothiocarboxylic acids having carbon atoms of thiocarboxyl groups bound to hydrogen atoms or to acyclic carbon atoms to hydrogen atoms or to carbon atoms of an acyclic saturated carbon skeleton
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    • C07D295/12—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms
    • C07D295/125—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/13—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • 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]
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • C—CHEMISTRY; METALLURGY
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    • 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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    • 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]

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  • Chemical & Material Sciences (AREA)
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  • Nanotechnology (AREA)
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  • Polymers & Plastics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Peptides Or Proteins (AREA)
CA3232690A 2021-11-29 2022-11-29 Multi-motif dendrons and their supramolecular structures and uses thereof Pending CA3232690A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163283588P 2021-11-29 2021-11-29
US63/283,588 2021-11-29
US202263398936P 2022-08-18 2022-08-18
US63/398,936 2022-08-18
PCT/CA2022/051745 WO2023092242A1 (en) 2021-11-29 2022-11-29 Multi-motif dendrons and their supramolecular structures and uses thereof

Publications (1)

Publication Number Publication Date
CA3232690A1 true CA3232690A1 (en) 2023-06-01

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CA3232690A Pending CA3232690A1 (en) 2021-11-29 2022-11-29 Multi-motif dendrons and their supramolecular structures and uses thereof

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US (2) US20240294714A1 (https=)
EP (1) EP4441030A4 (https=)
JP (1) JP2024543272A (https=)
KR (1) KR20240117084A (https=)
AU (1) AU2022396084A1 (https=)
CA (1) CA3232690A1 (https=)
IL (1) IL313225A (https=)
WO (1) WO2023092242A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117623978B (zh) * 2023-10-23 2026-03-20 山东大学 可生物降解的氨基酸衍生可电离脂质及其制备方法与应用
US12539279B2 (en) 2023-12-11 2026-02-03 The Governing Council Of The University Of Toronto Supramolecular ionizable lipid molecules with heteroatomic tuning for nucleic acid delivery

Family Cites Families (14)

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Publication number Priority date Publication date Assignee Title
AU2003279807A1 (en) * 2002-10-03 2004-04-23 The Board Of Trustees Of The University Of Arkansas Nanocrystals in ligand boxes exhibiting enhanced chemical, photochemical, and thermal stability, and methods of making the same
CN101220119A (zh) * 2008-01-23 2008-07-16 中国科学院化学研究所 一种超支化共聚物及其制备方法
US9102938B2 (en) * 2010-04-01 2015-08-11 Alnylam Pharmaceuticals, Inc. 2′ and 5′ modified monomers and oligonucleotides
CN104292129B (zh) * 2014-06-16 2018-03-02 中国石油化工集团公司 一种树枝状单体、采用该单体的处理剂及其制备方法
WO2017048789A1 (en) * 2015-09-14 2017-03-23 The Board Of Regents Of The University Of Texas System Lipocationic dendrimers and uses thereof
GB201600289D0 (en) * 2016-01-07 2016-02-24 Univ Liverpool Polydendrons
CA3040337A1 (en) * 2016-10-26 2018-05-03 Curevac Ag Lipid nanoparticle mrna vaccines
CN106519139A (zh) * 2016-11-10 2017-03-22 江苏苏博特新材料股份有限公司 一种早强型外加剂及其制备方法和应用
AU2021269137A1 (en) * 2020-05-08 2022-12-15 Orna Therapeutics, Inc. Circular RNA compositions and methods
WO2022168085A1 (en) * 2021-02-02 2022-08-11 Ramot At Tel-Aviv University Ltd. Lipids suitable for nucleic acid delivery
WO2022218295A1 (en) * 2021-04-13 2022-10-20 Chengdu Westgene Biopharm Co., Ltd Ionizable lipids and compositions for nucleic acid delivery
CN113402404B (zh) * 2021-04-30 2022-03-11 苏州科锐迈德生物医药科技有限公司 脂质化合物及包含其的脂质载体、核酸脂质纳米粒组合物和药物制剂
CN113264842B (zh) * 2021-07-21 2022-03-01 苏州科锐迈德生物医药科技有限公司 一种脂质化合物及包含其的脂质载体、核酸脂质纳米粒组合物和药物制剂
CN114262275B (zh) * 2021-12-15 2023-09-29 华中师范大学 一种高效低毒性dna及rna脂质递送载体

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Publication number Publication date
JP2024543272A (ja) 2024-11-20
WO2023092242A1 (en) 2023-06-01
EP4441030A1 (en) 2024-10-09
AU2022396084A1 (en) 2024-04-04
US20240294714A1 (en) 2024-09-05
KR20240117084A (ko) 2024-07-31
EP4441030A4 (en) 2025-10-22
IL313225A (en) 2024-07-01
US20240247109A1 (en) 2024-07-25

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