Biodegradable Lipids and Formulations for Intramuscular, In Vitro, and Ex Vivo Transfection of mRNA

Provided herein are compounds, such as compounds of Formula (I), and pharmaceutically acceptable salts thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of agents, including polynucleotides such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, spleen diseases, lung diseases, painful conditions, psychiatric disorders, musculoskeletal diseases, metabolic disorders, inflammatory diseases, and autoimmune diseases). Also provided herein are methods of synthesis of compounds of Formulae (I) and (II).

Researchers

Daniel Anderson / Kaelan Reed / Akash Gupta / Arnab Rudra

Departments: Department of Chemical Engineering, Chemical Engineering, David H Koch Institute for Integrative Cancer Res
Technology Areas: Biotechnology: DNA & RNA Editing / Chemicals & Materials: Catalysis & Synthesis, Nanotechnology & Nanomaterials / Drug Delivery: Microparticles & Nanoparticles / Drug Discovery and Research Tools: Genomics & Proteomics / Therapeutics: Cell Based Therapy

  • biodegradable lipids and formulations for intramuscular, in vitro, and ex vivo transfection of mrna
    United States of America | Published application

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