Biotherapeutics

Biotherapeutics are therapeutic agents derived from living organisms or biological systems, used to prevent, treat, or diagnose disease. They encompass a range of products including proteins, nucleic acids, vaccines, and cellular therapies. Biotherapeutics are typically produced by biotechnology methods such as recombinant DNA technology and mammalian or microbial cell culture, and they are often larger and more complex than traditional small-molecule drugs.

Major categories include monoclonal antibodies and other therapeutic proteins (such as insulin and clotting factors), vaccines that prime immune responses, gene therapies that introduce or modify genetic material, RNA-based therapies (for example siRNA and mRNA vaccines), and cell therapies (such as CAR-T cells and stem cell–based treatments).

Manufacture requires specialized bioprocessing with good manufacturing practice (GMP). This involves upstream production in controlled cell cultures, downstream purification, and stringent quality control. Regulatory oversight by agencies such as the U.S. Food and Drug Administration and the European Medicines Agency governs preclinical studies and phased clinical trials to assess safety and efficacy before approval.

Biotherapeutics offer highly specific mechanisms of action and therapeutic potential for complex diseases, but they pose challenges including high production costs, stability and storage requirements, potential immunogenicity, and variability between batches. Delivery is often parenteral, and access can be limited by pricing and healthcare infrastructure. Biosimilars, or near-identical versions of approved biologics, are increasingly used to address some cost concerns.

Advances in biotechnology continue to expand the biotherapeutics landscape with novel modalities such as multi-epitope antibodies, gene-editing–based approaches, and next-generation cellular therapies. Ongoing research aims to improve convenience, safety, and affordability while expanding indications across medicine.