Drug discovery Our approach to drug discovery builds the basis of UCB’s future, so we continuously monitor disruptive technologies, keep pace with evolving science and expand our therapeutic modality platforms so that our researchers have access to state-of-the-art capabilities enabling them to pioneer new approaches to drug discovery. New Chemical Entities Small Molecules Defined as any organic compound with a low molecular weight, small molecules have the ability to regulate biological processes, can yield oral bioavailability, achieve wide tissue distribution and can be suitable for targets inside cells. They can also be used to remove disease-causing proteins inside the cell, using targeted degradation.With today’s advancements in science and technology, new possibilities for innovative and creative new approaches to discovering small molecule drugs are opening up. This includes using computer-aided drug design (CADD) with artificial intelligence (AI). Peptides Peptides are made up of amino acids linked by a peptide bond, and they exhibit properties that can open up a new landscape of opportunities to treat disease.UCB’s ExtremeDiversity™ platform enables the discovery of synthetic macrocyclic peptides and is based on messenger ribonucleic acid (mRNA) display. Macrocyclic peptides combine properties such as the diversity, specificity and high affinity of therapeutic antibodies with some of the attractive pharmacological properties of small molecules. New Biological Entities Monoclonal Antibodies Monoclonal antibodies are bioengineered proteins that act like human antibodies in the immune system. They target proteins with exceptional specificity and can be engineered to target multiple proteins (bispecific antibodies), combine with linker molecules (antibody drug conjugates), and can be reduced in size to bring unique advantageous pharmacological properties (fragment antibodies).Monoclonal antibody drugs can be used against targets that are outside cells or on the cell surface, but because of their size, they generally can’t reach targets inside cells. Gene Therapies Adeno-Associated Viral (AAV) Vectors Gene therapy uses modified viruses or other technologies to deliver therapeutic genes to cells or tissues to address genetic diseases at their source. They can work by several mechanisms: they can replace a gene causing a medical problem with a healthy copy of the gene, add genes that help fight against or treat disease, or turn off the disease-causing gene(s).One of the most exciting advances in modern medicine has been the discovery of how AAV can be used as an effective delivery system for therapeutic genetic material into living tissues. AAV gene therapy has broad therapeutic implications for a vast array of severe diseases.
