Biologically-based medicines have transformed the lives of millions of patients with disabling and life-threatening diseases. These biological products – including therapeutic proteins and peptides as well as nucleic acid therapeutics such as oligonucleotides, mRNA products, and gene therapies – have the unprecedented potential to treat diseases caused by recessive gene disorders, acquired genetic diseases, and some viral infections.
Oligonucleotide therapies use short, single- or double-stranded DNA or RNA molecules to enhance or repress the expression of target RNA in order to treat or manage a wide range of diseases. Growing interest in oligonucleotides is driven by an increased focus on and demand for personalized medicine that requires easy, cost-effective scale-up from research amounts to commercial needs.
Large RNA molecules including messenger RNA (mRNA) have emerged as an important new class of therapeutics that can enable the body to make the proteins it need to prevent, treat, or cure diseases, as recently demonstrated by vaccines based on mRNA sequences encoding for a modified version of the SARS-CoV-2 spike protein.
As mRNAs are delicate molecules, analytical characterization of mRNA therapeutics presents unique challenges that require new technologies and solutions to improve the stability and achieve efficient and safe delivery of mRNA.
Adeno-associated virus (AAV) gene therapy seeks to modify or manipulate the expression of a gene in order to treat or cure diseases by fixing their underlying cause. AAVs are the main biopharmaceutical viral vectors for use in gene delivery for the treatment of a variety of human diseases. Full characterization, including sequencing and post-translational modification (PTM) identification of viral proteins, is required to mitigate immunogenicity and ensure the safety, quality, and efficacy of AAV products.
Biotherapeutic proteins are pharmaceutical drug products produced using biological systems and are often used to treat diseases like cancer, autoimmune disorders, or infectious diseases by modulating cellular signaling processes. They encompass various biologically-based drug products such as monoclonal antibodies, fusion proteins, antibody–drug conjugates, bispecific antibodies, and cell and gene therapy products.
As the pipeline for biotherapeutics and the number of related clinical trials continues to expand, the demand for biotherapeutics has increased significantly and the need for reliable process development and scalable manufacturing solutions has emerged.
High-performance liquid chromatography (HPLC) is the method of choice if high-purity products are desired. It is an ideal fit in a scalable manufacturing workstream, is less labor-intensive compared with other orthogonal methods, and offers the possibility of automation.
We will talk more about the specific applications needed for biopharmaceutical process monitoring by addressing the various challenges in process development and manufacturing.
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