A Practical Guide to Drug Development in Academia

Daria Mochly-Rosen, Ph.D., is the George D. Smith Professor of Translational Medicine, a Professor in the Department of Chemical and Systems Biology at Stanford University School of Medicine, and Founder and Co-director of SPARK at Stanford. She received her Ph.D. from the Weizmann Institute of Science, and was a postdoctoral fellow of biochemistry at University of California, Berkeley. She joined Stanford University in 1993 and served as the chair of her department for four years and then Senior Associate Dean for Research for seven years. She leads a multidisciplinary laboratory that includes basic researchers and clinicians, which recently focused their efforts on neurodegenerative diseases. In 2003, her lab’s research led to the founding of KAI Pharmaceuticals, where she served as CSO for one year, and as chair of the Scientific Advisory Committee and a member of the Board of Directors after her return to academia. She also co-founded two other biotech companies. In addition, Dr. Mochly-Rosen served on a variety of review groups including the Peer Review Advisory Committee of the NIH and the Council of Councils of the NIH. Kevin Grimes, M.D., M.B.A., is a Professor of Chemical and Systems Biology at Stanford University School of Medicine and Co-director of SPARK at Stanford. He received his M.D. from Brown University and after his internship and residency at Stanford, he joined the Stanford faculty as Clinical Assistant Professor of Medicine. Dr. Grimes then received his M.B.A from Stanford University and served as a White House Fellow. He later worked in the medical device and biotechnology sectors before returning to academia. He currently teaches drug discovery and development and teaches and practices internal medicine at Stanford.

Preface

Acknowledgements

Contents

Chapter 1 - Getting Started

                1.1 Advancing New Treatments from Academia to Biopharma

                1.2 Overview of Drug Discovery and Development

                1.3 Assessing Clinical Need

                1.4 Target Product Profile

                1.5 Project Management and Project Planning

                Key Terms and Abbreviations

                References

Chapter 2 - Therapeutics and Diagnostics Discovery

                2.1 Finding and Validating Drug Targets

                2.2 Repurposing Drugs

                2.3 Developing Assays for High-Throughput Screening

                2.4 Medicinal Chemistry and Lead Optimization

                2.5 Natural Products

                2.6 Approaching Machine Learning in Drug Development

                2.7 Therapeutic Antibody Discovery

                2.8 siRNA, Gene Editing, and RNA Editing

                2.9 Cell and Gene Therapy

                2.10 Vaccine Development

                2.11 Diagnostic Biomarkers

Key Terms and Abbreviations

References

Chapter 3 On the Way to the Clinic

3.1 When to Begin Animal Studies

3.3 In Vivo Pharmacology: Multiple Roles in Drug Discovery

3.4 Pharmacokinetics and ADME

3.5 Pharmacogenomics

3.6 Route of Administration and Drug Formulation

Key Terms and Abbreviations

References

Chapter 4 Preparing for the Clinic

4.1 Regulatory Considerations in Product Development

4.3 Technical Development and Manufacturing of Biological Products

4.4 Clinical Trial Design

4.5 Phase 3 Studies

Key Terms and Abbreviations

References

Chapter 5 Technology Transfer and Commercialization

5.1 Intellectual Property

5.2 Working with the University Technology Transfer Office

5.3 Navigating Conflicts of Interest

5.4 Working with the University Compliance Office

5.5 Not-For-Profit Drug Development

5.7 Selecting the Market for Your Drug

5.8 Commercial Assessments

5.9 Making a Compelling Pitch to Potential Investors

5.10 Venture Capital Funding

5.12 Plan, Organize, Motivate, and Control

Key Terms and Abbreviations

References

Chapter 6 Concluding Thoughts

6.1    Addressing the Market Failure for Developing Novel Antibiotics

Key Terms and Abbreviations

References

Author Biographies