GENERAL INTEREST>>>>
To: David A. Kincade (266 )
From: Casaubon Thursday, May 13 1999 10:28AM ET
Reply # of 270
Historically, big pharma tested extracts of EVERYTHING against cell cultures, to randomly identify organic agents capable of arresting cell growth. Then, the compounds structure was identified (arduous work) and modifications were made to try and optimize the activity and pharmicodynamics (how long the compound stays in the blood, what the tissue distribution is like, the metabolic fate, absorption profile, etc)
Now, we try and identify proteins associated with a disease state, and interfere with the pathway somehow. Sometimes blocking production of the protein, other times inhibiting the action of the protein, and sometimes even increases the production of a desired protein. Gene therapy attempts to regulate the production of proteins, by acting on DNA or RNA, the machinery necessary to produce proteins. Vertex primarily is involved in identifying relevant proteins to a particular disease state, determines the structure of the protein, and attempts to purposefully design organic molecules (not amino acid based, which is why we aren't biotech) to engage the protein, associated with the disease state, in a desired manner.
Companies that try to influence a disease state by introducing proteins, either directly (injection) or through gene therapy, are classical Biotech companies. Companies which try to moderate a disease state, by interfering with the way a protein acts, usually via orally available organic molecules (not proteins), are more classically defined as drug companies. The distinction is rather big, because typically proteins are hard to deliver and usually suffer from poor pharmicodynamics (proteins can get chewed up by enzymes fairly easily), whereas organic molecules are usually more robust, and can be administered orally rather than injection (the primary mode of administration for proteins).
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