Term
| Finding a target protein that is associated with a disease |
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Definition
| target identification --> target validation --> lead generation --> lead optimization --> clinical candidate |
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Term
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Definition
| study of the human genome to understand relationship of genes to physiology & dx |
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Term
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Definition
| inactivation of a single mouse gene to understand its function |
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Term
| Target Knock-Out in Cells |
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Definition
Antibodies;
Anti-Sense Oligonucleotides;
RNA interference; |
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Term
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Definition
| molecules that interact with the drug target |
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Term
| High Throughput Screening |
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Definition
| automated process for testting 100,000s of compounds for drug leads |
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Term
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Definition
natural products;
commercial compounds;
compound repository; |
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Term
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Definition
| synthesizing & testing analogs of lead compound to find one with all properties desired in final drug molecule |
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Term
| Vascular Endothelial Growth Factor (VEGF) |
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Definition
pro-angiogenic growth factor;
angiogenic activity primarily mediated by KDR (receptor tyrosine kinase expressed predominately in endothelial cells);
KDR signaling |
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Term
| First In Human (FIH) Clinical Studies: Phase I |
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Definition
Tested on Refractory Patients (oncology only);
Reason: safety, biological effects, metabolism, kinetics, drug interactions'
Careful dose escalation in 20-30 pts |
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Term
| Phase II Clinical Studies: Efficacy |
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Definition
Multicenter trial using MTD determined in Phase I;
Who: selected pts based on Phase I;
Why: therapeutic efficacy, dose range, kinetics, metabolism;
Dose range, Phase I determined in 200-300 pts; |
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Term
| Phase III Clinical Studies: Safety & Efficacy; |
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Definition
Large multicenter trial;
Who: selected pts based on Phase II;
Why: Safety & Efficacy - better than standard of care, prolonged survival, prolonged "progression free" survival, quality of life better than standard of care;
Dose & population determined in Phase II, 1000s of pts, ability to detect ADRs that occur <1 per 100 pts; |
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