Computationally guided high-throughput design of self-assembling drug nanoparticles

Nat Nanotechnol. 2021 Jun;16(6):725-733. doi: 10.1038/s41565-021-00870-y. Epub 2021 Mar 25.

Abstract

Nanoformulations of therapeutic drugs are transforming our ability to effectively deliver and treat a myriad of conditions. Often, however, they are complex to produce and exhibit low drug loading, except for nanoparticles formed via co-assembly of drugs and small molecular dyes, which display drug-loading capacities of up to 95%. There is currently no understanding of which of the millions of small-molecule combinations can result in the formation of these nanoparticles. Here we report the integration of machine learning with high-throughput experimentation to enable the rapid and large-scale identification of such nanoformulations. We identified 100 self-assembling drug nanoparticles from 2.1 million pairings, each including one of 788 candidate drugs and one of 2,686 approved excipients. We further characterized two nanoparticles, sorafenib-glycyrrhizin and terbinafine-taurocholic acid both ex vivo and in vivo. We anticipate that our platform can accelerate the development of safer and more efficacious nanoformulations with high drug-loading capacities for a wide range of therapeutics.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Candida albicans / drug effects
  • Computer Simulation
  • Drug Carriers / chemical synthesis
  • Drug Carriers / chemistry*
  • Drug Design
  • Drug Evaluation, Preclinical / methods
  • Dynamic Light Scattering
  • Excipients / chemistry
  • Female
  • Glycyrrhizic Acid / chemistry
  • High-Throughput Screening Assays / methods*
  • Humans
  • Machine Learning
  • Mice
  • Mice, Inbred Strains
  • Nanoparticles / chemistry*
  • Skin Absorption
  • Sorafenib / chemistry
  • Sorafenib / pharmacokinetics
  • Sorafenib / pharmacology*
  • Taurocholic Acid / chemistry
  • Terbinafine / chemistry
  • Terbinafine / pharmacology*
  • Tissue Distribution
  • Xenograft Model Antitumor Assays

Substances

  • Drug Carriers
  • Excipients
  • Taurocholic Acid
  • Glycyrrhizic Acid
  • Sorafenib
  • Terbinafine