Large Molecule Development

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Macromolecules, also known as biologics, are complex molecules typically derived from living organisms such as animals or microorganisms.

Macromolecules, also known as biologics, are complex molecules typically derived from living organisms such as animals or microorganisms. They differ from small molecules, which are chemically synthesized compounds with a defined structure.

Vaccines, blood and blood components, allergies, somatic cells, gene treatments, tissues, and recombinant therapeutic proteins are only a few examples of the wide range and diversity of biological products. Prompt use of biologics may provide the most effective means of treating a variety of diseases and conditions for which no other treatment options exist.

Examples of biologics considered large molecules

  • Monoclonal Antibodies (mAbs): Adalimumab (Humira), rituximab (Rituxan), and trastuzumab (Herceptin).
  • Recombinant Proteins: Erythropoietin (EPO), insulin, and growth factors like granulocyte colony-stimulating factor (G-CSF) and erythropoietin-stimulating agents (ESAs).
  • Fusion Proteins: Examples include etanercept (Enbrel), a fusion protein used to treat rheumatoid arthritis, and abatacept (Orencia), a fusion protein used for the treatment of autoimmune diseases.
  • Antibody-Drug Conjugates (ADCs): Ado-trastuzumab emtansine (Kadcyla) and brentuximab vedotin (Adcetris).
  • Therapeutic Enzymes: Examples include asparaginase used in the treatment of acute lymphoblastic leukemia (ALL) and recombinant coagulation factors used in the management of bleeding disorders.
  • Vaccines: Recombinant protein-based vaccines like the human papillomavirus (HPV) vaccine and the hepatitis B vaccine.
  • Gene Therapies: Viral vector-based gene therapies like Luxturna for inherited retinal diseases and Zolgensma for spinal muscular atrophy.
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