Target Identification and Validation

The process begins with identifying a molecular target associated with a disease or condition. This target is then validated to confirm its role in the disease process and its potential as a therapeutic target.

Lead Compound Identification

Once the target is validated, medicinal chemists design and synthesize molecules (lead compounds) that have the potential to interact with the target and modulate its activity.

In vitro Screening

The lead compounds are subjected to in vitro screening assays to assess their biological activity, selectivity, and toxicity. These assays may involve testing the compounds in cell cultures or biochemical assays to determine their efficacy and safety profiles.

Hit-to-Lead Optimization

Promising lead compounds are further optimized through medicinal chemistry to enhance their potency, selectivity, and pharmacokinetic properties. This iterative process involves synthesizing analogs of the lead compounds and testing them to identify the most promising candidates for further development.

In vivo Pharmacology Studies

Lead compounds that demonstrate favorable in vitro characteristics undergo in vivo pharmacology studies in animal models. These studies evaluate the compounds' efficacy, safety, pharmacokinetics, and toxicology profiles in living organisms, providing crucial data to support their advancement towards clinical trials.

Safety Pharmacology and Toxicology Studies

Comprehensive safety pharmacology and toxicology studies are conducted to assess the potential adverse effects of the lead compounds on vital organ systems and overall physiological function. Identifying any safety concerns and informing dose selection for subsequent clinical trials.

Dose Range Finding Studies

DRF studies are performed to determine the appropriate dose levels of the lead compound for clinical evaluation. Establishing dose-response relationship and identifying the therapeutic dose range that maximizes efficacy minimizing adverse effects.

IND Application Preparation

Based on the preclinical data generated, the sponsor prepares an IND application which includes comprehensive information on the lead compound, its pharmacological properties, manufacturing process, preclinical safety data, and proposed clinical trial protocols.

IND Submission and Regulatory Review

The IND application is submitted to the regulatory agency for review. Regulatory authorities assess the adequacy of the preclinical data and proposed clinical trial protocols to ensure patient safety and ethical conduct.

IND Approval and Clinical Trials Initiation

Upon successful review, the regulatory agency grants IND approval, allowing clinical trials in human. This marks the transition from preclinical development to clinical testing, with the goal of evaluating the safety and efficacy of the drug candidate in patients.