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Protein Therapeutics: a summary and pharmacological classification

Protein Therapeutics: a summary and pharmacological classification. Benjamin Leader, Quentin J. Baca and David E. Golan. Yiben Wang 11/16/11. Protein Therapeutics: -25,000-40,000 different genes in the human genome -Alternative splicing of genes -Post-translational modifications

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Protein Therapeutics: a summary and pharmacological classification

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  1. Protein Therapeutics: a summary and pharmacological classification Benjamin Leader, Quentin J. Baca and David E. Golan Yiben Wang 11/16/11

  2. Protein Therapeutics: -25,000-40,000 different genes in the human genome -Alternative splicing of genes -Post-translational modifications -Very high number of functionally distinct proteins Disease View: -Immense challenge: -One protein is mutated or abnormal -Present in high or low concentrations Therapeutic View: -Tremendous opportunity 130 different proteins or peptides – FDA approval

  3. Advantages of protein therapeutics: • Proteins – highly specific and complex set of functions • -Chemical compounds can’t mimick • Specificity • -Less chance of interference; reduce adverse effects • Natural production • -Well tolerated; no immune response • Diseases – gene is deleted or mutated • -Replacement treatment – no gene therapy (Unavailable) • Faster FDA approval time • -2003: 1980-2002  33 therapeutic proteins, 294 small-molecule drugs (>1year) • Unique form and function • -Companies – patent protection

  4. Functional classification of protein therapeutics: Group I -Protein therapeutics with enzymatic or regulatory activity Group II -Protein therapeutics with special targeting activity Group III -Protein vaccines Group IV -Protein diagnostics

  5. Group I: Protein therapeutics with enzymatic or regulatory activity Ia: Replacing a protein that is deficient or abnormal Ib: Augmenting an existing pathway Ic: Providing a novel function or activity

  6. Group II: Protein therapeutics with special targeting IIa: Interfering with a molecule or organism IIb: Delivering other compounds or proteins

  7. Group III: Protein vaccines IIIa: Protecting against a deleterious foreign agent IIIb: Treating an autoimmune disease IIIc: Treating cancer

  8. Group IV: Protein diagnostics

  9. Challenges for protein therapeutics: • Protein solubility, route of administration, distribution, and stability. • Immune response. • Physiologically active – post-translational modifications. • Costs • 5. Ethics

  10. Recombinant human proteins: -FDA – biotechnology medicines: -Monoclonal antibodies -Natural interferons -Vaccines -Hormones -Modified natural enzymes -Cell therapies More work needs to be done.

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