Lead Discovery & Optimization for Efficacy, PK, & Safety from edX

In this course about lead selection and lead optimization, students will learn methods for discovering and optimizing leads with the end goal of advancing a compound into the clinic. The three-week course starts with the processes for finding molecules with promising activity for initiation of a drug program. The second week covers the criteria used to select the best molecules as new leads for the optimization stage. In the final week, students will learn different methods for identifying structural changes that will optimize a lead’s efficacy and PK properties as well as reduce toxicity.

Students in the course should understand pharmacodynamics, drug activity, pharmacokinetics, and preclinical safety. Students should also be comfortable with algebraic expressions, including logarithmic and exponential functions. A fundamental understanding of the structure of organic molecules and their functional groups is also required. The course is designed in modules. Each module opens with a short video on a selected topic. The module continues with a short reading or exercise with an assessment activity. Each module will require approximately 1 hour to complete, and the course includes 15 modules. Two approaches to the course are recommended. One, a student might spend an hour per day on the course and complete all 15 modules in approximately three weeks. Two, a student with additional available time may be able to work through the entire course in just a single week or over an extended weekend.

Students signed up for the Audit Track have access to the instructional video materials. Students enrolled through the Verified Track have access to the instructional videos, the readings and activities, the assessments, as well as selected additional videos related to the primary instructional videos.

What's inside

Learning objectives

General approaches to finding compounds with activity on a drug target
Methods for confirming target engagement by an active compound
Techniques for communicating the pharmacophore of a series of active molecules

Criteria that help priorities biologically active compounds as potential leads
Types of information that are most often used to guide lead optimization decisions
Different functional groups that are often used to improve pk properties without affecting on-target activity in a molecule

General approaches to finding compounds with activity on a drug target
Methods for confirming target engagement by an active compound
Techniques for communicating the pharmacophore of a series of active molecules
Criteria that help priorities biologically active compounds as potential leads
Types of information that are most often used to guide lead optimization decisions
Different functional groups that are often used to improve pk properties without affecting on-target activity in a molecule

Syllabus

week 1

actives, hits, leads, and clinical compounds

screening drug space for biological activity

types of screening libraries

hit confirmation and hit validation

drug-target binding

pharmacophore determination

lead selection

lead optimization

lead-like vs. drug-like

properties, metrics, and parameters

x-ray crystallography and cocrystals

structural changes for lead optimization

matched pair analysis and single point modifications

isosteres and bioisosteres

purity and in vivo toxicity

week 2

week 3

Good to know

Know what's good

, what to watch for

, and possible dealbreakers

Teaches general approaches for finding compounds with activity on a drug target

Teaches methods to confirm target engagement by an active compound

Teaches techniques to communicate pharmacophore

Examines criteria that help prioritize biologically active compounds as potential leads

Examines types of information used to guide lead optimization decisions

Examines different functional groups often used to improve PK properties without affecting on-target activity in a molecule

Activities

Be better prepared before your course. Deepen your understanding during and after it. Supplement your coursework and achieve mastery of the topics covered in Lead Discovery & Optimization for Efficacy, PK, & Safety with these activities:

Review basic organic chemistry

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This will help you recall the essential concepts of organic chemistry, which are necessary for understanding the material covered in this course.

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Review your notes and textbook from your previous organic chemistry course.
Complete practice problems to test your understanding.

Review statistics and data analysis

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This will help you brush up on your statistical skills, which are essential for analyzing experimental data and making informed decisions in drug discovery.

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Review your notes and textbook from your previous statistics course.
Complete practice problems to test your understanding.

Join a study group to discuss course materials

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This will provide you with opportunities to engage with classmates, clarify concepts, and reinforce your understanding of the course material.

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Find a group of classmates to study with.
Meet regularly to discuss the course material.

One other activity

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Design and present a lead optimization strategy

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This will allow you to apply the concepts you've learned in the course to a real-world scenario, demonstrating your ability to design and communicate an effective lead optimization strategy.

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Identify a lead compound and define the desired properties.
Develop a plan for optimizing the lead compound.
Present your strategy to a group of peers or experts.

Career center

Learners who complete Lead Discovery & Optimization for Efficacy, PK, & Safety will develop knowledge and skills that may be useful to these careers:

Drug Discovery Scientist

If you are a Drug Discovery Scientist, much of your work surrounds the process of drug discovery and development. This course will be very useful to you in this field. Each module will teach you the processes for finding molecules with promising activity, selecting the best molecules for optimization, and identifying structural changes that will optimize a lead’s efficacy. This course is ideally suited to your career.

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Medicinal Chemist

As a Medicinal Chemist, you will be responsible for designing, synthesizing, and testing new drugs. This course will be an excellent fit for you as it will help you understand how to optimize a lead’s efficacy and PK properties as well as reduce toxicity. This course will help you build a solid foundation in lead discovery and optimization.

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Pharmacologist

With a unique focus on lead discovery and optimization, this course will be useful to you as a Pharmacologist. As a Pharmacologist, it is your job to study drugs and their effects on living things. In this field, it is critical to understand the efficacy and safety of medications. This course will also introduce you to the complexities of pharmacokinetics, the study of drug activity. Understanding how the body processes drugs is critical to pharmacology research.

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Pharmaceutical Scientist

While designed for those pursuing a career in drug development, this course will be helpful to you as a Pharmaceutical Scientist. Pharmaceutical scientists are responsible for the research, development, and production of pharmaceuticals. This course will provide you with techniques for communicating the pharmacophore of a series of active molecules, criteria that help prioritize biologically active compounds as potential leads, and different functional groups that are often used to improve PK properties. By understanding these concepts, you will be better prepared to make informed decisions about drug development.

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Toxicologist

As a toxicologist, your job is to assess the safety of chemicals and other substances. A major aspect of toxicology is understanding the potential toxicity of new drugs. This course will be useful to you as it will help you understand the preclinical safety of drug candidates. By understanding how to identify structural changes that will reduce toxicity, you will be better prepared to evaluate the safety of new drugs.

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Health Scientist

As a Health Scientist, your work is focused on improving the health of individuals and communities. This course may be useful to you, especially if you are interested in drug development or research. By understanding the process of lead discovery and optimization, you will be better prepared to evaluate the safety and efficacy of new drugs.

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Clinical Research Associate

As a Clinical Research Associate, it is your role to manage clinical trials. This course may be useful to you as it will help you build a foundation in the drug development process. By understanding how drugs are discovered, optimized, and tested, you will be better prepared to manage clinical trials.

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Biostatistician

As a Biostatistician, your work involves the application of statistics to biological data. This course may be useful to you as it will help you build a foundation in drug development. By understanding how drugs are discovered, optimized, and tested, you will be better prepared to analyze clinical trial data.

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Science Writer

Science Writers are responsible for communicating complex scientific information to a variety of audiences. This course may be useful to you as it will help you build a solid foundation in drug development. By understanding how drugs are discovered, optimized, and tested, you will be better prepared to write about these topics in a clear and concise way.

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Sales Representative

As a Sales Representative in the pharmaceutical industry, you are responsible for selling drugs and medical devices to healthcare professionals. This course may be useful to you as it will give you a deeper understanding of the drug development process. By understanding how drugs are discovered, optimized, and tested, you will be better able to answer questions from healthcare professionals and promote your products effectively.

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Medical Writer

As a Medical Writer, your job is to create high-quality medical content. This course may be useful to you as it will help you build a solid foundation in drug development. By understanding how drugs are discovered, optimized, and tested, you will be better prepared to write about these topics for a variety of audiences.

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Regulatory Affairs Specialist

As a Regulatory Affairs Specialist, your job is to ensure that drugs and medical devices meet regulatory requirements. This course may be useful to you as it will help you build a foundation in the drug development process. By understanding how drugs are discovered, optimized, and tested, you will be better prepared to review and approve regulatory submissions.

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Quality Assurance Auditor

As a Quality Assurance Auditor, your job is to ensure that drugs and medical devices are manufactured in accordance with good manufacturing practices. This course may be useful to you as it will help you build a foundation in the drug development process. By understanding how drugs are discovered, optimized, and tested, you will be better prepared to conduct quality audits.

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Manufacturing Scientist

As a Manufacturing Scientist, you are responsible for the development and scale-up of manufacturing processes for drugs and medical devices. This course may be useful to you as it will help you build a foundation in the drug development process. By understanding how drugs are discovered, optimized, and tested, you will be better prepared to design and optimize manufacturing processes.

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Clinical Data Manager

As a Clinical Data Manager, your job is to manage clinical trial data. This course may be useful to you as it will help you build a foundation in the drug development process. By understanding how drugs are discovered, optimized, and tested, you will be better prepared to manage clinical trial data.

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