A Practical Guide to Drug Development in Academia

Daria Mochly-Rosen, Ph.D., is the George D. Smith Professor of Translational Medicine, the Senior Associate Dean for Research, and a Professor in the Department of Chemical and Systems Biology at Stanford University School of Medicine and the Co-director of SPARK. She received her Ph.D. from the Weizmann Institute of Science in Israel, and was a postdoctoral fellow in the department of biochemistry at UC Berkeley. She joined Stanford University in 1993 and served as the chair of her department for four years. In 2003, her lab’s basic research led to the founding of KAI Pharmaceuticals, where she served as the CSO for one year, and as the chair of the Scientific Advisory Committee and a member of the Board of Directors after her return to academia. She also founded ALDEA Pharmaceuticals in 2011. In addition, Dr. Mochly-Rosen served on a variety of review groups including the Peer Review Advisory Committee of the NIH and on the Council of Councils of the NIH.  Kevin Grimes, M.D., M.B.A., received his M.D. from Brown University and his M.B.A. from Stanford University. After his training, he joined the Stanford faculty as an academic internist. He later worked in the medical device and biotechnology sectors before returning to academia. He is currently the Co-director of the SPARK Translational Research Program and Associate Professor of Chemical and Systems Biology at Stanford, where he also teaches drug discovery and development and the practice of internal medicine.

Chapter 1: Getting Started

1.1 Advancing New Treatments to the Clinic within Academia

Daria Mochly-Rosen

1.2 Overview of Drug Discovery and Development

Kevin Grimes

1.3 Assessing Clinical Need

Kevin Grimes

1.4 Target Product Profile (TPP)

Robert Lum

1.5 Project Management and Project Planning

Rebecca Begeley and Daria Mochly-Rosen

Chapter 2: Discovery and Preclinical Work

2.1 Robustness of Preclinical Studies

Daria Mochly-Rosen

2.2 Repurposing Drugs

Kevin Grimes

2.3 Developing Assays for High Throughput Screening (HTS)

Bruce Koch

2.4 Medicinal Chemistry and Lead Optimization

Daniel A. Erlanson

2.5 Vaccine Development

Harry Greenberg

2.6 When to Begin Animal Studies

Daria Mochly-Rosen

2.7 In vivo pharmacology: Multiple Roles in Drug Discovery

Simeon Taylor

2.8 Pharmacokinetics and ADME Properties

Werner Rubas and Emily Egeler

2.9 Route of Administration and Drug Formulation

Terrence F. Blaschke

2.10 Preclinical Safety Studies

Michael Taylor and Kevin Grimes

Chapter 3: Preparing for the Clinic

3.1 Regulatory Considerations in Product Development

Carol Karp

3.2 Manufacturing and Quality Control

Susan Wade

3.3 Technical Development and Manufacturing of Biological Products

Mark Backer

3.4 Clinical Trial Design

Ted McCluskey

3.5 Overview of Clinical Trials

Ted McCluskey

Chapter 4: Transferring Technology

4.1 Intellectual Property

Judy Mohr

4.2 Working with the University Technology Transfer Office

Katharine Ku

4.3 Avoiding Conflicts of Interest

Emily Egeler

4.4 Working with the University Compliance Office

Jennifer Swanton Brown, Nicholas Gaich, and Steven Alexander

Chapter 5: Commercialization and Entrepreneurship

5.1 Selecting the Market for Your Drug

Lilliane Brunner Halbach

5.2 Commercial Assessments

Julie Papanek

5.3 Making a Compelling Pitch to Potential Investors

Leon Chen

5.4 Venture Capital Funding

Kevin Kinsella

5.5 Not-For-Profit Drug Development

Eugenio L. de Hostos

5.6 Legal Aspects of a Start-up Biotechnology Company

Alan C. Mendelson, Peter E. Boyd, and Christopher M. Reilly

5.7 Founder Preferred Stock

Scott M. Iyama and Stephen J. Venuto

5.8 Plan, Organize, Motivate and Control

John Walker

Chapter 6: Concluding Thoughts

6.1 A Call to Action: Changing How We Pursue Drug Discovery and Development

Steven Schow