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C1
Correction to: Therapeutics
andDiagnostics Discovery
DariaMochly-Rosen, KevinGrimes, RamiN.Hannoush,
BruceKoch, GretchenEhrenkaufer, DanielA.Erlanson,
JulieSaiki, JenniferL.Wilson, ShelleyForceAldred,
AdrianaA.Garcia, JinBillyLi, RosaBacchetta,
MariaGraziaRoncarolo, Alma- MartinaCepika,
HarryGreenberg, StevenN.Goodman,
andMichaelA.Kohn
The updated version of this chapter can be found at
https://doi.org/10.1007/978- 3- 031- 34724- 5_2
Correction to:
Chapter 2 in:D. Mochly-Rosen, K. Grimes (eds.), A Practical
Guide to Drug Development in Academia,
https://doi.org/10.1007/978- 3- 031- 34724- 5_2
The original version of Chapter 2, “Therapeutics and Diagnostics Discovery,”
was previously published without Dr. Gretchen Ehrenkaufer being credited as the
chapter author. The book has been updated with these changes.
© The Author(s), under exclusive license to Springer Nature Switzerland AG 2024
D. Mochly-Rosen, K. Grimes (eds.), A Practical Guide to Drug Development in
Academia, https://doi.org/10.1007/978-3-031-34724-5_7
253
Author Biographies
Shelley Force Aldred, PhD, is a serial entrepreneur and experienced drug devel-
oper. Dr. Force Aldred is CEO and co-founder of Rondo Therapeutics, a biopharma-
ceutical company developing bispecic therapeutic antibodies for oncology
indications. Previously, she served as VP for Preclinical Development at Teneobio
Inc., a highly successful multispecic therapeutic antibody company. In the past,
Dr. Force Aldred was COO and co-founder of SwitchGear Genomics, a venture-
backed functional genomics platform company. Prior to founding SwitchGear
Genomics, Dr. Force Aldred was a Scientic Director on Stanford’s ENCODE
Project and received her PhD from Stanford University.
Rosa Bacchetta, MD, is an Associate Professor of Pediatrics at Stanford University
and has long-standing experience investigating the mechanisms of immunological
tolerance, specically in pediatric patients after hematopoietic stem cell transplan-
tation, and those with genetic diseases of the immune system. She completed her
pediatrics residency at the University of Turin, then received training in molecular
and cellular immunology at the DNAX Research Institute of Molecular and Cellular
Biology. She then worked for fteen years at the San Raffaele Scientic Institute
(HSR-TIGET), where she focused on dissecting the genetic and immunological
basis of primary immune-regulatory diseases that might be treated by gene therapy.
Dr. Bacchetta’s research focuses on dissecting the role of FOXP3 and Treg cells in
human immune responses, with a goal of establishing cell and gene transfer-based
therapies for IPEX Syndrome. She is sponsoring a phase 1 gene therapy trial using
autologous Treg-like cells to treat IPEX patients.
Mark Backer, PhD, received a BS in Chemistry from Stanford University and a
PhD in Chemical Engineering from the University of Washington. He has worked
to develop biopharmaceuticals since joining Genentech as its seventh employee in
1978 and has participated in the development of seven commercial products. He is
currently General Manager of Alava Biopharm Partners, a consulting group focused
© The Editor(s) (if applicable) and The Author(s), under exclusive license to Springer
Nature Switzerland AG 2023
D. Mochly-Rosen, K. Grimes (eds.), A Practical Guide to Drug Development in Academia,
https://doi.org/10.1007/978-3-031-34724-5
254
on CMC and regulatory support for product developers. He also supports the
SPARK program in an advisory role.
Glenn Begley, MBBS (MD-equivalent), PhD, is an oncologist and hematopathol-
ogist whose academic and commercial roles include board level and senior posi-
tions in Australia, the US and the UK.He currently serves as Co-Founder and Head
of Drug Discovery at Parthenon Therapeutics and as Senior Scientic Consultant to
Certara and to BridGene Biosciences. He was CEO of BioCurate, an Australia-
based joint venture between the University of Melbourne and Monash University.
Previously, he was Chief Scientic Ofcer at Akriveia Therapeutics, Global Head of
Hematology and Oncology Research at Amgen, Senior Vice President and CSO at
TetraLogic Pharmaceuticals, and executive director of the Western Australian
Institute of Medical Research. His early research rst described human G-CSF, and
in later clinical studies he rst pioneered the use of G-CSF-“mobilized” blood stem
cells (so-called “stem cell transplantation”). He received his PhD from the University
of Melbourne and was elected Fellow of the Australian Academy of Health and
Medical Sciences.
Rebecca Begley, PhD, is an Executive Director of Project Management at Terns
Pharmaceuticals. Prior to that, she worked at Gilead Sciences, Inc., as a Clinical
Pharmacologist and Regulatory Project Manager, and at KAI Pharmaceuticals as a
Scientist and Project Manager. She received her undergraduate degree from Barnard
College and her PhD from Stanford University in 2004.
Terrence F. Blaschke, MD, is Emeritus Professor of Medicine and Molecular
Pharmacology at Stanford University School of Medicine. His early research
included the study of drugs used in HIV-infected patients, which lead to a particular
interest in the access and adherence to these drugs in low- and middle-income coun-
tries. At Stanford University, he served roles including Associate Dean for Medical
Student Advising and Associate Director, Stanford General Clinical Research
Center. After becoming emeritus, he joined the Bill and Melinda Gates Foundation
as a Senior Program Ofcer, Global Health Discovery and Translational Sciences,
and has published extensively on medication adherence.
Robert F. Booth, PhD, has more than 30 years of experience in drug discovery
and development in both large and small biopharmaceutical companies, both in the
US and in Europe. He also has experience in business development, venture capital,
and in academia. In the recent past, he has served as co-founder and Chairman of the
boards of Curasen Therapeutics (a SPARK project) and Ab Initio Biotherapeutics
and more recently as Chairman of Myoforte Inc. (another SPARK project). Prior to
those roles, he was founder and CEO of Virobay Inc., Operating Partner at TPG
Biotech, Chief Scientic Ofcer at Celera Genomics, and Senior Vice President at
Author Biographies
255
Roche. He currently serves as a board member and on the scientic advisory boards
of several biotech companies. He received his BSc and PhD in biochemistry from
the University of London.
Peter Boyd, JD, MBA, is a healthcare entrepreneur with a background in busi-
ness, science, and law. He was a co-founding member of Harpoon Medical where
he negotiated and managed all aspects of a complex structured nancing and the
subsequent acquisition by Edwards Lifesciences. He worked as an associate until
2012in the Silicon Valley ofce of Latham & Watkins LLP, where he represented
startup companies and venture capitalists. Mr. Boyd earned his BS in Biology from
the University of North Carolina at Chapel Hill and a joint JD–MBA degree from
the University of Virginia.
Jennifer Swanton Brown, RN, CCRP is Director of Clinical Research Quality
and Assistant Dean for Compliance, Regulatory and Quality in the ofce of the
Senior Associate Dean for Research, Stanford School of Medicine.
Alma-Martina Cepika, MD, PhD, is a physician scientist with an extensive back-
ground in human immunology and genomics. She is an Instructor of Pediatrics at
Stanford University School of Medicineand a current recipient of several awards in
the eld of cell and gene therapies, including the SPARK Translational Research
Grant, the National Blood Foundation Early Career Research Grant, and the
American Society for Gene + Cell Therapy Career Development Award. She com-
pleted her postdoctoral training at the Baylor Institute for Immunology Research
and obtained her MD degree at the University of Zagreb School of Medicine in
Croatia. There, she also completed an international master’s course in Leadership
and Management of Health Services, founded in collaboration with the London
School of Economics and Political Sciences. She received an early exposure to bio-
pharma during her PhD at Croatia’s Institute of Immunology, which was a leading
developer of vaccines, anti-venoms, and other medicinal products for south-
east Europe.
Leon Chen, PhD, MBA, is a Partner at The Column Group, focused on investing
and new company formation. Prior to joining TCG, he was a venture partner at
OrbiMed Advisors and a Partner at Skyline Ventures. He was a cofounder of KAI
Pharmaceuticals and has held founder or early startup roles in multiple biotech
companies including Eikon Therapeutics, Escape Bio, Adicet Bio, and TranscripTx.
As a biotech entrepreneur, he has experience in drug discovery and building a
research organization and has taken drugs from the laboratory into clinical develop-
ment. Dr. Chen has a PhD in Molecular Pharmacology from Stanford University
School of Medicine and an MBA from the Stanford Graduate School of Business.
Author Biographies
256
Eugenio L. de Hostos, PhD, MBA, is Senior Director at the Global Health portfo-
lio at Calibr, the drug discovery and development division of Scripps Research.
Previously, Dr. de Hostos was Director in the Drug Development Program at PATH
and The Institute for OneWorld Health. He received his BS from Yale University,
his MBA from Presidio Graduate School, and his PhD from Stanford University.
Emily Egeler, PhD, is Director of Regulatory Operations and Procedures for the
Center for Cancer Cell Therapy at Stanford University School of Medicine, where
she supports investigator-initiated studies with novel cell therapies. Her previous
roles include Regulatory Specialist with the Cancer Clinical Trials Ofce and
Program Coordinator for the SPARK Translational Research Program, both at
Stanford University School of Medicine. She received her PhD in Chemical and
Systems Biology from Stanford University.
Gretchen Ehrenkaufer, PhD, did her doctoral work at University of California,
San Francisco in the lab of Dr. Thomas Kornberg, where she studied Hedgehog
signaling in Drosophila development. After graduating with a PhD in cell biology,
she began infectious disease work in Dr. Upi Singh’s lab at Stanford University,
studying the protozoan parasite Entamoeba histolytica that causes amoebic dysen-
tery. While in Dr. Singh’s lab, she worked on many diverse projects, including
investigating the transcriptional changes and molecular mechanisms of amebic
development, and analysis of small RNA populations. Her involvement with SPARK
began in 2016 as part of a project to develop new therapeutics to treat amoebic
infection. This work has resulted in multiple publications and identied promising
drug candidates, including several with pan-parasitic activity and others that are
FDA approved or in late-clinical stages. She has been a Program Manager at SPARK
since 2021.
Daniel A. Erlanson, PhD, is VP of Innovation and Discovery at Frontier
Medicines. Before that, he co-founded Carmot Therapeutics, whose Chemotype
Evolution technology he co-developed. Dr. Erlanson received his BA in Chemistry
at Carleton College and his PhD in Chemistry at Harvard University in the labora-
tory of Gregory L.Verdine. He was an NIH postdoctoral fellow with James A.Wells
at Genentech. From there, he joined Sunesis Pharmaceuticals at its inception, and
went on to develop fragment-based technologies and to lead medicinal chemistry
programs. As well as co-editing two books on fragment-based drug discovery, Dr.
Erlanson is editor of Practical Fragments (http://practicalfragments.blogspot.com/).
Lyn Frumkin, MD, PhD, previously worked at biotechnology companies Amgen,
Inc. and ICOS Corp., where he provided broad expertise, including leading clinical
teams conducting global programs for the successful approval of pivotal programs
Author Biographies
257
in North America, Europe, and Japan. Dr. Frumkin was a Consulting Professor at
Stanford University School of Medicine and has been an advisor to SPARK since
2010. He is also a Fulbright awardee, which allows service as an expert consultant
on programs related to transitional medicine/global health at foreign academic insti-
tutions. Dr. Frumkin received his MD-PhD from the University of Washington with
postgraduate training in internal medicine at Stanford University and subsequent
subspecialty training in Neurology and Infectious Diseases/Virology.
Nicholas Gaich is the founder and CEO of Nick Gaich and Associates, a rm dedi-
cated to providing executive coaching, leadership development, strategic planning,
and operational performance. He has more than 46 years of experience with exper-
tise ranging from executive coaching, organizational development, change manage-
ment, supply chain management, customer service, hospital service line economics,
clinical research administration, business development, and marketing. Mr. Gaich
also serves as the CEO/President of the Morgan Hill Chamber of Commerce. Mr.
Gaich retired in 2012 as Assistant Dean of Clinical and Translational Research
Operations, Stanford Center for Clinical and Translational Research and Education
at Stanford University School of Medicine. He also held a two-year appointment on
the Consortium Management Group at the National Center for Research Resources,
National Institutes of Health. He received his BBA with an emphasis in Healthcare
Administration from National University of San Diego.
Adriana A. Garcia, PhD, is an Academic Professional at SPARK at Stanford.
Prior to that, she received her PhD in Chemical and Systems Biology from Stanford
University and a BS in Biochemistry from San Francisco State University. Her doc-
toral work investigated the stabilization of protein oligomers as a therapeutic strat-
egy for a common enzymopathy known to cause hemolytic anemia.
Steven N. Goodman, MD, MHS, PhD, is Associate Dean for Clinical and
Translational Research and Professor of Epidemiology and of Medicine at Stanford.
He directs the Stanford Program on Research Rigor and Reproducibility (SPORR),
whose aim is to increase the adoption of best scientic practices throughout the
Stanford School of Medicine. He leads a variety of training programs in epidemiol-
ogy and clinical research, and his research is in scientic and statistical inference,
with connections to research, policy, and ethics. Outside of Stanford, Dr. Goodman
serves as chair of the PCORI Methodology Committee and is senior statistical edi-
tor of the Annals of Internal Medicine and scientic advisor to the national Blue
Cross-Blue Shield technology assessment program. He was awarded the Spinoza
Chair in Medicine from the University of Amsterdam for his work in inference, the
Lilienfeld award from the American College of Epidemiology for his lifetime con-
tributions to the eld of epidemiology, and in 2020 was elected to the National
Academy of Medicine.
Author Biographies
258
Julie Papanek Grant, MBA, is a venture capitalist and pharmaceutical executive.
She has incubated, founded, and invested in a number of new pharmaceutical com-
panies as a General Partner at Canaan. Prior to joining Canaan, she worked across
Development and Commercial functions at Genentech and subsequently Roche.
After graduating from Yale University with a BS in Molecular Biophysics and
Biochemistry, she received an MPhil in BioScience Enterprise from Cambridge
University and an MBA from theStanford Graduate School of Business.
Harry Greenberg, MD, is the Joseph D. Grant Professor of Medicine and
Microbiology and Immunology and an Associate Dean for Research at Stanford
University School of Medicine. He has been an active NIH-funded investigator for
almost 40 years during which time his studies have focused primarily on viruses
that infect the GI tract, liver, or respiratory tree. Dr. Greenberg was the Chief
Scientic Ofcer at biotechnology company Aviron (now MedImmune Vaccines),
where he played an important role in bringing the live attenuated inuenza vaccine
to licensure. He was also a long-term advisor to Bharat, an Indian Vaccine company
that has developed novel vaccines for low-income countries such as a new rotavirus
vaccine and a novel and highly effective Salmonella Typhi conjugate vaccine.
Kevin Grimes, MD, MBA, is a Professor of Chemical and Systems Biology at
Stanford University and Co-Director of SPARK at Stanford. He received his MD
from Brown University. Dr. Grimes began his career as a Clinical Assistant Professor
of Medicine at Stanford, where his primary duties included the teaching and prac-
tice of internal medicine. Dr. Grimes received a Hartford Foundation Fellowship to
study health economics and obtained an MBA at the Stanford Graduate School of
Business. He was subsequently selected as a White House Fellow and assigned to
the Department of Defense, where he served as Special Assistant to the Secretary.
He spent fteen years in industry, working in the medical device, life science con-
sulting, and biotechnology sectors prior to returning to Stanford to co-direct
SPARK.Dr. Grimes also teaches graduate student courses on drug discovery and
development and continues to teach and practice internal medicine. He has received
the David Rytand Award for Excellence in Clinical Teaching and the Faculty Award
for Excellence in Graduate Teaching.
Grace Hancock, PhD, is Program Manager for the Global Health portfolio at
Calibr, the drug discovery and development division of Scripps Research. She
received her BS from the Johns Hopkins University and her PhD from University of
California, Los Angeles.
Rami N. Hannoush, PhD, spent fteen years at Genentech where he was a Senior
Group Leader, and he led both early- and late-stage discovery biology programs in
Ophthalmology, Immunology, and Oncology, with a focus on drug discovery and
Author Biographies
259
development, as well as target discovery and validation. His laboratory in transla-
tional biology at Genentech focused on new drug modalities and understanding the
role of protein–protein interactions in stem cell signaling pathways for cellular repro-
gramming and tissue regeneration, and his group made important contributions in the
elds of Wnt signaling and protein lipidation. He obtained his PhD in Chemistry
from McGill University and did his postdoctoral training in chemical biology and
cell biology at HarvardUniversity. He has received numerous academic and profes-
sional awards, authored more than 60 peer-reviewed scientic publications, held sev-
eral patents, and served on multiple scientic editorial boards as well as advisory
boards for organizations spanning industry and academia. He is also currently an
adjunct Professor of Chemical and Systems Biology at Stanford University.
Karin Immergluck, PhD, is the Executive Director of the Stanford Ofce of
Technology Licensing and Industrial Contracts Ofce. Prior to joining Stanford, she
was the Executive Director of the Ofce of Technology Management at University
of California, San Francisco. She obtained her PhD in Molecular Developmental
Genetics from the University of Zurich.
Carol D.Karp is the Chief Regulatory Ofcer for Prothena Biosciences. She pre-
viously held leadership roles with Johnson & Johnson, CV Therapeutics, Esperion,
and VIVUS.Her experience in the biotechnology/pharmaceutical sector spans the
global development and commercialization of products to address unmet needs for
therapeutic areas including neurodegenerative, cardiovascular, renal, metabolic,
immunologic, and addiction diseases and disorders. Ms. Karp has been a SPARK
advisor since 2009. She earned her BA in Biology from the University of Rochester,
where she currently serves as Vice Chair of the Board of Trustees.
J. Jekkie Kim, JD, MD, MBA, LLM is a Partner at Latham & Watkins LLP.Dr.
Kim advises healthcare, life sciences, and technology companies and their investors
on a range of intellectual property transactional matters. She draws on her back-
ground as a medical doctor and has developed a particular focus on digital health,
agricultural technology, and cross-border transactions that involve markets such as
China. She received an MD from College of Medicine, Yonsei University, JD from
Boston University, MBA from Case Western Reserve University, and LLM from
Yonsei University Department of Medical Law and Ethics.
Nina Kjellson is a tenured venture capitalist with over 25 years of biotechnology
and digital health investing experience. She is a General Partner at Canaan Partners
and was previously a General Partner at InterWest Partners. Prior to InterWest, she
was an Associate at Bay City Capital, a life sciences merchant bank and an Analyst
at Oracle Partners, a healthcare hedge fund. She began her career conducting
research for the Kaiser Family Foundation. She is a Health Innovation Fellow at the
Author Biographies
260
Aspen Institute and a member of the Leadership Council of the Health Innovation
Center at Oliver Wyman. She is a member of the boards of directors of Girle Effect,
Essential Access Health and Life Science Cares’ Bay Area chapter. She has a BA in
Human Biology from Stanford University.
Bruce Koch, PhD, received his PhD in Cell and Developmental Biology from
Harvard University and completed his postdoctoral studies at University of
California, Berkeley. He joined the discovery research group at Syntex rising to the
position of Director of Discovery Technologies at Roche Pharmaceuticals. He is
currently the Director of the Stanford’s ChEM-H/Chemical and Systems Biology
High-Throughput Screening Knowledge Center at Stanford University.
Michael A. Kohn is Professor Emeritus of Epidemiology and Biostatistics at
University of California, San Francisco and was an attending emergency physician
at Mills-Peninsula Medical Center in Burlingame until May 2018. He teaches in
UCSF’s Training in Clinical Research (TICR) Program on research methods, clini-
cal epidemiology, and database management. He also provides consultations to
clinical investigators who need help with study design, sample size calculations,
data management, and statistical analysis. His primary research interest is diagnos-
tic testing. He co-authored the clinical epidemiology textbook Evidence-Based
Diagnosis, is a contributing author to Hulley etal., “Designing Clinical Research,”
and created the UCSF Clinical & Translational Science Institute (CTSI) online sam-
ple size calculators (www.sample- size.net). Dr. Kohn is also Chief Medical Ofcer
of QuesGen Inc. He received an AB from Stanford University, an MPP from Harvard
Kennedy School, and an MD from Stanford and trained in emergency medicine
(board certied) at the Denver Afliated Program.
Jin Billy Li, PhD, is an Associate Professor of Genetics at Stanford University. He
received his bachelor’s and master’s degrees at Tsinghua University and his PhD
degree from Washington University in St. Louis. After his postdoctoral training at
Harvard Medical School, he started his laboratory at Stanford in 2010 where he has
focused on studying RNA editing mediated by ADAR enzymes. His laboratory
focuses on two aspects of ADAR RNA editing. One is the biological function of RNA
editing to evade dsRNA-mediated autoimmunity, which has led to new approaches to
treating cancer and inammatory diseases. The other is to harness the ADAR enzyme
for site-directed RNA base editing that overcomes the challenges of CRISPR/Cas-
based DNA editing and holds great potential for treating rare and common diseases.
Robert Lum, PhD, is a veteran in drug development with experience in both large
and small pharmaceutical companies. He has served in a variety of positions, includ-
ing Senior Director, Pharmaceutical Operations at Concentric Analgesics; Senior
Director,Technical Operations at Aquinox; Director of Process Development at
Author Biographies
261
Pzer (formerly Medivation); Executive Director, Process Development and
Manufacturing at Geron Corp.; Vice President, Technology and Preclinical
Development at Telik, Inc.; Assistant Director of Medicinal Chemistry at CV
Therapeutics; Senior Scientist at Arris Pharmaceutical; and Assistant Senior
Investigator at SmithKline Beecham. He received his BS in Chemistry from
University of California, Berkeley, and his PhD in Organic Chemistry from
Massachusetts Institute of Technology.
Collen Masimirembwa, PhD, is the founder, President, and CEO of the African
Institute of Biomedical Science and Technology (AiBST). He has over 25 years of
experience in pharmacokinetics and pharmacogenomics for drug discovery and
development, ten of them as a Principal Scientist at AstraZeneca Pharmaceutical
company. He is President of the African Pharmacogenomics Consortium and chairs
the Global Pharmacogenomics Research Network. His pioneering work in pharma-
cogenomics in Africa has won him many awards including the Human Genome
Organisation Award and the Bill and Melinda Gates Foundation Calestous Juma
Science Leadership Award to expand pharmacogenomic research across Africa. He
is also the Director of SPARK GLOBAL Africa.
Ted McCluskey, MD, PhD, has over 25 years of experience in biotechnology,
pharmaceutical, and diagnostic development designing, implementing, and analyz-
ing clinical trials. He has worked in multiple disease areas and participated in four
product approvals. Past positions include Chief Medical Ofcer and VP Clinical at
AVIIR, Inc., Senior Director of Clinical Research at Johnson & Johnson/Scios Inc.,
and Senior Medical Director at Genentech/Roche. He is an interventional cardiolo-
gist and was previously on the faculty at University of Cincinnati, trained at the
Cleveland Clinic and University of California, San Francisco, and received his MD
and PhD from Washington University in St. Louis. Dr. McCluskey was also for-
merly active in Sand Hill Angels, having served as its President and on the Board of
Directors. He has been an advisor at SPARK since 2011.
Dirk Mendel, PhD, is a veteran in drug development with close to 30 years of
experience in large and small biotech and biopharma companies. He has worked in
a variety of therapeutic areas, most notably virology and oncology, and has contrib-
uted signicantly to the development of therapeutic small molecules, antibodies,
and peptides, including 7 that have made it to market. He has worked in all phases
of drug development, from early research through clinical proof-of-concept and
regulatory approval, and has spent the past 20 years focusing on the translational
aspects of drug development, leading translational efforts from lead optimization
through clinical proof-of-concept to understand patient identication and stratica-
tion, and identifying and understanding the relationships between compound expo-
sure and activity/toxicity to enable clinical candidate selection and facilitate early
clinical development. Dr. Mendel received his BS in Engineering from Stanford
University and his PhD in Physiology from Dartmouth Medical School.
Author Biographies