Medicinal Chemistry

1. Drug Discovery Process
   1. Target Identification and Validation
      1. Understanding disease mechanisms
         1. Genomic studies
         2. Proteomic studies
         3. Pathway analysis
      2. Identifying biological targets
         1. Enzymes
         2. Receptors
         3. Ion channels
         4. Nucleic acids
      3. Target validation approaches
         1. Genetic techniques
            1. Knockout models
            2. RNA interference
         2. Pharmacological methods
            1. Use of small molecules
            2. Antibody-based validation
      4. Challenges in target validation
         1. Biological complexity
         2. Target accessibility
         3. Off-target effects
   2. Hit Identification
      1. High Throughput Screening (HTS)
         1. Automation of screening processes
         2. Assay development
         3. Data analysis and interpretation
      2. Virtual Screening
         1. Computational techniques
            1. Molecular docking
            2. Molecular dynamics
         2. Database mining
         3. Focused vs. random screening
      3. Importance of hit identification
         1. Novel chemical entities
         2. Broad chemical diversity
      4. Challenges in hit identification
         1. False positives/negatives
         2. Assay reliability
   3. Lead Optimization
      1. Optimization strategies
         1. Enhancing potency
         2. Improving selectivity
         3. Reducing toxicity
      2. Structure-activity relationships (SAR)
         1. Iterative optimization
         2. Analogue synthesis
      3. Balancing pharmacokinetic properties
         1. Absorption and distribution
         2. Metabolic stability
      4. Multidisciplinary collaboration
         1. Chemists
         2. Biologists
         3. Pharmacologists
      5. Challenges in lead optimization
         1. Complexity of biological systems
         2. Off-target interactions
   4. Candidate Selection
      1. Preclinical evaluation
         1. Pharmacokinetics and pharmacodynamics (PK/PD)
         2. Toxicology studies
            1. Acute toxicity
            2. Chronic toxicity
         3. Safety pharmacology
      2. Criteria for candidate selection
         1. Therapeutic window
         2. Dosage forms and feasibilities
      3. Risk assessment and management
         1. Identifying potential issues early
         2. Developing mitigation strategies
      4. Regulatory considerations
         1. Meeting preclinical regulatory requirements
         2. Preparing for clinical trials
      5. Transitioning from discovery to development
         1. Documentation and reporting
         2. Project management strategies