Bioanalysis Zone

Monitoring Drug Efficacy: Quantitation of Transthyretin Mutants using Thermo Scientific™ MSIA™ microcolumns and UHPLC-HRMS



Misfolding and aggregation of serum wild-type (wt) and mutant transthyretin protein (TTR) is the cause of Transthyretin Amyloidosis (ATTR), a severe and fatal disease characterized by abnormal deposits of TTR protein in the form of amyloid in the body’s organs and tissues. Currently new drugs are being developed which decrease the amount of TTR production as a mechanism to potentially slow or stop disease progression. Therefore, quantitation of TTR levels in serum is crucial in monitoring the pharmacodynamic efficacy of therapeutic measures. Conventional immunoturbometric or ELISA based assays of TTR do not differentiate between wt and mutant TTR. Presented is the application of MSIA for the simple and effective simultaneous quantification of wt and mutant TTR in human serum for monitoring the efficacy of a new therapeutic antisense drug.

What will you learn?  

  • Application of the Mass Spectrometric Immunaossay (MSIA) Workflow
  • The power of combining MSIA Microcolumn sample preparation with HRAM Detection
  • Measuring Antisense ODN activity through the monitoring of TTR expression via MSIA
  • Emerging therapeutic approaches for TTR amyloidosis, use of Antisense ODN

Who should attend?

  • CRO Directors
  • Bioanalytical Directors
  • Lab Managers
  • Pharmaceutical Senior Scientists




Hua Wang
Research Chemist
Charles River Laboratories

Dr. Hua Wang is a Research Chemist at Charles River laboratories, Ashland. He is the key scientist at Bioanalytical Chemistry Department in CRL Ash to develop LC-MS assays for large molecule bioanalysis. Dr. Wang received his Ph.D. in chemistry from Case Western Reserve University. Before joining CRL, he was a scientist at Sanofi Genzyme in MA.




Dr. Eric Niederkofler
Research & Development Manager
Thermo Fisher Scientific

Dr. Eric Niederkofler is an R&D Manager with Thermo Fisher Scientific over seeing products related to Thermo Scientific Mass Spectrometric Immunoassay (MSIA). His focus is in the development of products and applications supporting hybrid LC-MS approaches for the bioanalysis of biologics and protein biomarkers in the fields of Pharma/Biopharma and Translational/Clinical Research.

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