MEDICINE DESIGN

medical design

We are interested in establishing alliances to develop and access novel:

Medicinal Chemistry Synthesis Technology

  • Synthetic technologies that can accelerate the delivery of API
  • Flow chemistry approaches that enable mg to kg scale
  • Efficient scale-up (kg) capabilities for photo redox chemistry
  • Chemical transformations via biocatalysis
  • Synthetic methodologies for late stage diversification
  • Synthetic methodology to access small conformationally constrained multifunctional templates
  • Monomers and building blocks in drug-like property space
  • High-throughput optimization capabilities for synthetic transformations and biocatalysis
  • Innovative capabilities for parallel medicinal chemistry

Medicinal Chemistry

  • Approaches to expand NCE target space – RNA splicing or translational modulation, orally bioavailable or CNS-penetrant protein degradation, non-CRBN and non-VHL mediated strategies for targeted protein degradation, covalent inhibition, modulation of protein-protein interactions and non-Ro5 compounds
  • Solute ligand carrier, transcription factor, deubiquitylating (DUB) enzyme, phosphatase, RNA binding protein, and biomolecular condensate modulator design and screening technolog
  • ies
  • AI/ML methodologies to predict novel protein or RNA structures
  • Membrane protein structural biology technologies and capabilities, including ion channels, GPCRs and solute carrier proteins
  • DNA-compatible synthetic protocols and DNA backbone modifications that expand currently available methodology for DNA-encoded libraries (DEL)
  • Computational methods for quantitative affinity prediction and molecular dynamics simulation
  • Technologies to identify hits through virtual screening of very large compound collections
  • Ligand-based multi-parametric generative design platforms
  • Systems/chemical biology technologies enabling mechanism determination for phenotypic screening hits
  • Morphological or transcriptional profiling technologies to enable hit expansion and pathway inferences for phenotypic screening
  • Functional genomic and iPSC technology to enable facile generation of disease relevant cell systems for phenotypic screening

Pharmacokinetics Dynamics and Metabolism (PDM)

  • Translation
    • Translational modeling and simulation approaches, systems pharmacology/PK-PD; deep knowledge of targets/pathways; increased confidence in target drug selection
  • Bioanalysis and biomarkers
    • Novel bioanalytical and cellular imaging techniques
    • Specific biomarkers of ADME DDI liability (both transporter and enzyme biomarkers)
    • Novel methodology for pull-down of tissue specific exosomes
  • Disposition and delivery of therapies
    • Novel commercially viable delivery technologies (oral and non-oral, including topical)
    • Biodistribution of nanoparticles at whole organ and cellular level
  • Targeting, prediction and modeling of transporter-mediated disposition and DDIs – small molecules
    • Quantitation and scaling of transporters for input into physiological PK models of tissue penetration and clearance including biliary clearance
    • Determination of intracellular/sub-cellular unbound concentrations of transported drugs
  • Novel approaches to achieving selective tissue distribution (including receptor mediated and transporter mediated strategies)
  • Transformative in vitro assays such as PK on a chip
  • Technologies that enable tissue specificity and therapeutic window enablement
  • PBPK to predict clinical DDI