EXPERTISE AND CAPABILITIES

The R&D areas we support are indicated below:

• Design and synthesis of novel lipids (e.g. cationic, pegylated)
• Radiolabelled and stable-isotope lipids to support ADME studies
• Synthetic process technology transfer to Contract Manufacturing Organization (CMO)

• Formulation development and optimization using scalable components and processes
• Process characterization and optimization
• Manufacturing process technical transfer to cGMP CMO

• Analytical methods development and transfer, including lipid and nucleic acid-specific analyses
• Biophysical characterization including LNP size and size distribution encapsulation, surface charge and pKa, cryo-electron microscopy, ³¹P-NMR
• Bioanalytical method development and analyses for LNP components

• Pharmacodynamic studies for protein replacement therapeutics
  • Reporter gene (e.g. luciferase, green fluorescent protein) expression in vivo by fluorescent/luminescent live-imaging
  • Reporter tissue/cell gene expression ex vivo by flow cytometry, fluorescence microscopy, or fluorescence/luminescence spectroscopy
  • Transgene expression by immunohistochemistry/immunocytochemistry
• Safety/tolerability studies
  • Clinical pathology/histopathology
  • Immune response characterization (e.g. complement, cytokines/chemokines)
• Bioanalytical
  • Nucleic acid therapeutic
  • LNP components

• Focuses on successful management of the program to meet partner objectives
  • Coordination and execution of Partner Work Plan
  • Monitoring budget, resources and timelines

• Catalyses a strengthened partnership alliance to ensure excellent alignment on deliverables
  • Regular communication with partner project/program management team to ensure alignment
  • Management of objectives, agendas, material preparation, attendees and logistics for semi-annual face-to-face meetings