Drug Discovery and Genomics How the Sequencing of the Human Genome and Related Developments has Impacted Drug Discovery B i o p h a r m a c e u t i c a l s L t d. “Fortunes will be won and lost in the genome grab. The race to secure the sequence patents will be over in five Years.” The World in 2001. The New Economist on pharma- ceuticals. B i o p h a r m a c e u t i c a l s L t d. The Promise The Concern The Human Genome Project and related technologies has generated thousands of novel potential drug targets. Validating those targets and their drugability and generating therapeutic options are now the rate limiting steps in drug development. Bottom Line B i o p h a r m a c e u t i c a l s L t d. Topics What is Genomics ? What is the relationship between genes and disease? What are the steps in developing a drug? What impact has genomics had on the process of drug development? B i o p h a r m a c e u t i c a l s L t d. What is Genomics ? Study of information stored in the genome structural and functional information Structural genomics — the sequence Information is encoded linearly and digitally in four coding molecules-bases Three bases = codon = amino acid A number of codons strung together code for a gene which codes for a protein Functional genomics — what the genes do B i o p h a r m a c e u t i c a l s L t d. B i o p h a r m a c e u t i c a l s L t d. B i o p h a r m a c e u t i c a l s L t d. Comparative Sequence Sizes (Bases) (yeast chromosome 3) 350 Thousand Escherichia coli (bacterium) genome Million Largest yeast chromosome now mapped Million Entire yeast genome 15 Million Smallest human chromosome (Y) 50 Million Largest human chromosome (1) 250 Million Entire human genome 3 Billion Return to text B i o p h a r m a c e u t i c a l s L t d.
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