Synthesis of Ester, Carbonate, and Carbamate-derived Novel Biodegradable Ionizable Lipids from Methyl Ricinoleate or Methyl 12-Hydroxystearate and its Applications

Provided herein are compounds, such as compounds of Formula (I), and pharmaceutically acceptable salts, hydrates, solvates, polymorphs, co-crystals, tautomers, stereoisomers, and isotopically labeled derivatives thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of polynucleotides, such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic disease, proliferative disease, hematological disease, neurological disease, liver disease, spleen disease, lung disease, painful condition, psychiatric disorder, musculoskeletal disease, a metabolic disorder, inflammatory disease, or autoimmune disease).

Researchers

Daniel Anderson / Jacob Witten / rajith manan / Akiva Gordon / Bowen Li / Luke Rhym

Departments: Department of Chemical Engineering, David H Koch Institute for Integrative Cancer Res, Chemical Engineering
Technology Areas: Chemicals & Materials: Polymers / Drug Discovery and Research Tools: Genomics & Proteomics / Therapeutics: Nucleic Acids, Vaccines

  • synthesis of ester, carbonate, and carbamate-derived novel biodegradable ionizable lipids from methyl ricinoleate or methyl 12-hydroxystearate and its
    United States of America | Published application

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