DD_1 Flashcards
(31 cards)
What are the key stages of the drug development pipeline?
Target Identification → Lead Discovery → Preclinical Testing → Clinical Trials (Phases I-IV) → FDA Approval
What are common drug targets?
Membrane-bound receptors (GPCRs, ion channels), Enzymes, Intracellular proteins (e.g., kinases, transcription factors)
What are ‘undruggable’ targets?
Proteins lacking conventional binding pockets, tackled with PROTACs, molecular glues, or DNA-encoded libraries
How do small molecules differ from biologics?
Small molecules are chemically synthesized, can penetrate cells, and are often orally available. Biologics are large, complex proteins that target extracellular molecules and require injection.
What is High-Throughput Screening (HTS)?
A method to test thousands of compounds against a biological target rapidly
What are compound libraries?
Collections of potential drug molecules, including synthetic, natural products, and DNA-encoded libraries
What is structure-based drug discovery (SBDD)?
Uses 3D protein structures from X-ray crystallography, NMR, or Cryo-EM to design drugs
What is in silico screening?
Computationally docking molecules to predict how they interact with a target protein
What are GPCRs?
G Protein-Coupled Receptors, involved in neurotransmission, hormones, and sensory processes
What are the different types of GPCR ligands?
Agonists (activate), Antagonists (block), Allosteric Modulators (enhance or inhibit indirectly)
What is systems biology?
An integrative approach using genomics, proteomics, and metabolomics to identify drug targets
What is network pharmacology?
Analyzing drug interactions with multiple targets to develop safer, multi-target drugs
What are protease inhibitors?
Drugs that block enzymes breaking down proteins, e.g., HIV protease inhibitors, Paxlovid for COVID
What is the difference between covalent and reversible inhibitors?
Covalent inhibitors permanently bind to targets, while reversible inhibitors temporarily block enzyme activity
What are molecular glues?
Small molecules that induce protein-protein interactions, leading to targeted protein degradation
What are PROTACs?
Proteolysis-Targeting Chimeras, molecules that tag target proteins for degradation
What is drug repurposing?
Finding new uses for existing drugs, e.g., Imatinib for leukemia and gastrointestinal stromal tumors (GIST)
What are the key stages of the drug development pipeline?
Target Identification → Lead Discovery → Preclinical Testing → Clinical Trials (Phases I-IV) → FDA Approval
How do small molecule drugs differ from biologics?
Small Molecules: Low weight, chemically synthesized, cell-permeable, often oral.
Biologics: Large, complex, made in living cells, injectable, highly specific.
What is a lead compound?
A prototype drug with promising characteristics but requiring further modification.
What is a prodrug?
An inactive drug that becomes active inside the body.
What is the main goal of Phase I clinical trials?
Safety testing in healthy volunteers.
What is tested in Phase II clinical trials?
Efficacy in a small group of patients.
What happens in Phase III clinical trials?
Large-scale patient testing for safety and efficacy.
