CA3179824A1 - Lipids and lipid nanoparticle formulations for delivery of nucleic acids - Google Patents
Lipids and lipid nanoparticle formulations for delivery of nucleic acidsInfo
- Publication number
- CA3179824A1 CA3179824A1 CA3179824A CA3179824A CA3179824A1 CA 3179824 A1 CA3179824 A1 CA 3179824A1 CA 3179824 A CA3179824 A CA 3179824A CA 3179824 A CA3179824 A CA 3179824A CA 3179824 A1 CA3179824 A1 CA 3179824A1
- Authority
- CA
- Canada
- Prior art keywords
- compound
- alkyl
- independently
- date
- carbon
- 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
- 150000002632 lipids Chemical class 0.000 title claims abstract description 161
- 239000000203 mixture Substances 0.000 title claims abstract description 116
- 239000002105 nanoparticle Substances 0.000 title claims abstract description 66
- 150000007523 nucleic acids Chemical class 0.000 title claims description 70
- 102000039446 nucleic acids Human genes 0.000 title claims description 68
- 108020004707 nucleic acids Proteins 0.000 title claims description 68
- 238000012384 transportation and delivery Methods 0.000 title abstract description 37
- 238000009472 formulation Methods 0.000 title abstract description 11
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- 238000000034 method Methods 0.000 claims abstract description 111
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- 150000003839 salts Chemical class 0.000 claims abstract description 31
- 229940124597 therapeutic agent Drugs 0.000 claims abstract description 31
- 238000002360 preparation method Methods 0.000 claims abstract description 23
- 108020004999 messenger RNA Proteins 0.000 claims description 94
- -1 n-octyl Chemical group 0.000 claims description 86
- 125000000217 alkyl group Chemical group 0.000 claims description 49
- 229910052799 carbon Inorganic materials 0.000 claims description 40
- 150000001721 carbon Chemical group 0.000 claims description 36
- 229920006395 saturated elastomer Polymers 0.000 claims description 33
- 239000011203 carbon fibre reinforced carbon Substances 0.000 claims description 32
- 125000000753 cycloalkyl group Chemical group 0.000 claims description 28
- 125000004400 (C1-C12) alkyl group Chemical group 0.000 claims description 20
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 17
- 229910052757 nitrogen Inorganic materials 0.000 claims description 15
- 125000004433 nitrogen atom Chemical group N* 0.000 claims description 9
- 125000006686 (C1-C24) alkyl group Chemical group 0.000 claims description 8
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- 125000004108 n-butyl group Chemical group [H]C([H])([H])C([H])([H])C([H])([H])C([H])([H])* 0.000 claims description 7
- 125000000999 tert-butyl group Chemical group [H]C([H])([H])C(*)(C([H])([H])[H])C([H])([H])[H] 0.000 claims description 6
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- 108020005544 Antisense RNA Proteins 0.000 claims description 5
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- 125000002373 5 membered heterocyclic group Chemical group 0.000 claims description 3
- 125000000959 isobutyl group Chemical group [H]C([H])([H])C([H])(C([H])([H])[H])C([H])([H])* 0.000 claims description 3
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| MA45036A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2 |
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