biyofarmasötik

Biyofarmasötik (biopharmaceuticals) are medicines produced using biotechnology, including recombinant proteins, monoclonal antibodies, vaccines, enzymes, and gene or cell therapies. They differ from conventional small-molecule drugs in their use of living cells or biological processes.

Production and technology: Most products are produced in living systems such as bacteria, yeast, or mammalian cell lines using genetic engineering, fermentation, and downstream purification. Manufacturing emphasizes Good Manufacturing Practice (GMP), rigorous characterization, and reproducibility to ensure consistent quality between batches.

Applications: Used to treat cancer, autoimmune diseases, rare genetic disorders, and infectious diseases. Examples include insulin produced by recombinant DNA, monoclonal antibodies for cancer and inflammatory diseases, enzyme replacement therapies, vaccines, and newer cell-based therapies like CAR-T, as well as gene therapies delivered by viral vectors.

Regulation and safety: Development follows preclinical studies and phased clinical trials, with extensive safety and efficacy data required. Biosimilars, similar but not identical to originators, require a regulatory pathway demonstrating similarity in structure, function, and clinical effect. Post-marketing surveillance monitors adverse events and manufacturing consistency; immunogenicity and cold-chain requirements are important considerations.

Market and challenges: Biopharmaceutical manufacturing is complex and costly, with high barriers to entry and large-scale facilities. Intellectual property and ethical considerations, access and affordability, and supply chain integrity shape the field. Ongoing research explores more efficient expression systems, advanced purification, personalized biopharmaceuticals, and novel vectors for gene therapy.