Desarrollo de fármacos biológicos

Biological therapeutics (biologics) are revolutionizing the treatment of cancer and immune inflammatory disease states. Initially, biologic drugs were limited to simple peptide molecules (e.g., insulin). However, in recent years, with the progression of innovative technological approaches, biologics comprised of monoclonal antibodies, fusion proteins, vaccines, cell and gene therapies have been created and deployed in the treatment of disease. To standardize practices in the development of these therapies, authorities from regulatory agencies and the pharmaceutical industry have established organizations, such as the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and the U.S. Pharmacopeia (USP), that issue guidelines to help ensure the production of safe, effective and high-quality medicines. These policies are not only important for novel innovator biologics, but also for the generation of biosimilar molecules. Biosimilars are biologics that are highly similar to and possess no clinically meaningful difference from a previously approved reference product. 

Because of the inherent complexity and variability of these therapeutics, robust characterization tools are essential throughout the drug discovery and development process to ensure on target mechanism of action, drug potency and biomanufacturing process consistency. As an example, cell based functional bioassays enable quantitative interrogation and validation of a biologic molecule’s target specificity (e.g., immune checkpoint receptor) or mechanistic endpoint (e.g. ADCC). In addition, reagents that facilitate streamlined antibody purification and site-specific enzymes for mass spectrometry-based analysis are crucial tools in biologics development workflows.