On a rainy winter’s day in Boston, MA, we took a journey in biotherapeutic peptide mapping with Guilong Charles Cheng of Alexion Pharmaceuticals, Inc. Join us on this journey and discover how advancements in Thermo Scientifics’ chromatography, mass spectrometry and software are accelerating confident decision making and contributing to greater hope for patients with rare diseases.
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare diseases. Their goal is to deliver medical breakthroughs where none currently exist. They are driven in the knowledge that people’s lives depend on their work.
Dr Cheng is a development Scientist at Alexion, where he leads a team which focuses on monitoring and identifying changes in protein therapeutics and assessing the impact these molecular attributes may have on their safety, quality and efficacy.
Proteins, or large molecule biotherapeutics, differ significantly from small molecule drugs, such as ibuprofen. Protein biotherapuetics are much more complex, their production being associated with an abundance of opportunity for drug-product-related impurities and variants. This heterogeneity has to be assessed and each variant requires thorough characterization.
To fully characterize a biotherapeutic molecule, a series of attributes need to be measured. Dr Cheng describes how advances in Thermo Scientifics’ hardware and software allow him to accurately assess these attributes. He currently uses Orbitrap technology together with the new Thermo Fisher Scientific BioPharma Finder software to address characterization of the intact protein, protein sub-units and the protein at its primary sequence level. In this way, Dr Cheng and his team help to ensure patients receive the safest and most effective treatment for their rare disease.
The field of rare diseases suffers from a deficit of medical and scientific knowledge, with cures for the majority of these types of disease still elusive. Companies like Alexion provide a beacon of hope to individuals affected by rare diseases, with biotherapeutics in particular demonstrating effectiveness against such diseases. Take eculizumabwhich was developed by Alexion to treat paroxysmal nocturnal hemoglobinuria (PNH), a life threatening auto-immune disease of the blood. Eculizumab, a recombinant humanized monoclonal antibody, selectively targets and inhibits the branch of the body’s immune system which is responsible for the aberrant destruction of red blood cells, which is characteristic of this disease.
The research undertaken at Alexion allows them to LEARN from yesterday, patients to LIVE for today and provides HOPE for a rare disease free future!