Colony Forming Assay
When performed by highly trained, experienced personnel, the well validated colony forming cell (CFC) assay is predictive of these consequences. Further, data from this type of assay is often requested by regulatory bodies, including the FDA, before clinical trials can proceed. Drug classes that can be assessed include small molecules, monoclonal antibodies, bispecific antibodies, antibody-drug conjugates (ADCs), oligonucleotide therapeutics, CAR-T cells, novel biologic constructs, and others.
Cytokine Secretion & T Cell Activation
Utilizing our extensive experience designing and
performing primary blood cell-based assays, we
have developed a flexible assay platform to test
preclinical stage antibodies or other molecules for cytokine secretion. Here we present data
including readouts for both cytokine secretion
and T cell activation in response to several
stimulants added to PBMCs derived from fresh
whole blood.
Assess Off-Target Toxicity or Linker Stability
ADCs combine the benefits of both therapeutic antibodies and potent small molecule cytotoxic drugs. These moieties are connected through linkers that should be stable within systemic circulation but cleave within the antibody-targeted cells, to release highly specific drugs. With more complex structures come more biological challenges. We have assays to test for off-target toxicity to progenitor cells using the colony forming cell (CFC) assay or linker stability using a neutrophil differentiation assay (Zhao et al, 2017)
Screening Drugs to Treat Hemoglobinopathies
This serum-free assay platform offers a direct measurement of the HbF protein, using two methods in response to treatment with HU as shown, and additionally with other HbF inducers. This differs from other approaches looking at increases in transcript numbers or other indirect measurements. This in vitro assay will facilitate the ability to examine new potential drug therapies, both alone and in combination with HU, on HbF induction. This assay may be used to assess HU responsiveness in some SCD patients and to identify other patient-specific drug options.
Tips + Tricks
Working with PBMCs can be more difficult than cell lines. Part of this white paper hopes to present several pointers to aid the generation of viable and functional starting cell populations.
