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Файл:Ординатура / Хирургия / Библиотека им академика М.И. Перельмана / Книга_5436_Библиотеки_им_академика_М_И_Перельмана.pdf
262
Alan Mendelson, JD [died October 8, 2021], was a founding partner at Latham &
Watkins LLP’s Silicon Valley ofce and cochaired the rm’s Emerging Companies
Practice and Life Sciences Industry Groups. He served as a member of the University
of California Board of Regents and the Boards of Trustees of the UC Berkeley
Foundation and The Scripps Research Institute, served on the board of the California
Life Sciences Association, and was the corporate secretary for many public and
private companies. Throughout his career, Mr. Mendelson mentored many life
sciences- focused attorneys and biotech executives. He received his AB from
University of California, Berkeley, and his JD from Harvard University.
Daria Mochly-Rosen, PhD, is a Professor of Chemical and Systems Biology and
the George D Smith Professor of Translational Medicine at Stanford University
School of Medicine. She is Founder and Co-Director of SPARK at Stanford and the
President of SPARK GLOBAL.She leads a multi-disciplinary research lab and has
developed a number of drug leads for human diseases with a particular interest in
mitochondrial biology and pathology. In 2003, her lab’s basic research led to the
founding of KAI Pharmaceuticals, where she served as CSO for one year and as
chair of the Scientic Advisory Committee and a member of the Board of Directors
after her return to academia. KAI was subsequently acquired by Amgen and KAI’s
drug, Parsabiv, has been approved. She also co-founded ALDEA Pharmaceuticals
(now licensed to Foresee) and MitonaTherapeutics. Dr. Mochly-Rosen previously
served as chair of her department and the Senior Associate Dean for Research and
continues to teach graduate-level classes at Stanford University School of Medicine.
Her leadership in translational research efforts in academia led to awards from
Accelerating Australia (2019), Cures Within Reach (2020), Xconomy Award (2020),
and California Life Sciences Pantheon award (2022). Dr. Mochly-Rosen received
her PhD in Chemical Immunology from the Weizmann Institute of Science and was
a postdoctoral biochemistry fellow at University of California, Berkeley.
Judy Mohr, PhD, JD, received her PhD in Chemical Engineering from the
University of Texas at Austin and her JD from Santa Clara University. She has
extensive experience working in patent law with an emphasis in pharmaceuticals.
Dr. Mohr is a partner at McDermott Will & Emery LLP (Silicon Valley Ofce). She
also supports the SPARK program in an advisory role.
Christopher M.Reilly, JD, is Senior Director, Legal at Lyft Inc. He previously
served as an associate at the Silicon Valley ofce of Latham & Watkins LLP and
received his JD from the University of Virginia School of Law.
Maria Grazia Roncarolo, MD, is the George D.Smith Professor in Stem Cell and
Regenerative Medicine, Professor of Pediatrics and of Medicine, director of the
Center for Denitive and Curative Medicine, and co-director of the Institute for
Author Biographies
263
Stem Cell Biology and Regenerative Medicine at Stanford University. A pediatric
immunologist by training, she earned her medical degree at the University of Turin.
She worked at DNAX Research Institute for Molecular and Cellular Biology, where
she contributed to the discovery of novel cytokines. As director of the Telethon
Institute for Gene Therapy at the San Raffaele Scientic Institute in Milan, Dr.
Roncarolo developed novel approaches in cell and gene therapy. Her work led to the
discovery of ex vivo gene therapies for genetic diseases of the immune system,
including ADA-SCID and WASP, and metabolic diseases such as metachromatic
leukodystrophy. The landmark stem cell gene therapy treatment for ADA-SCID was
the world’s rst to be approved by the European Medicines Agency (EMA) under
the brand name Strimvelis® in May 2016. She discovered a new class of T cells and
is leading clinical trials using cell therapeutics to prevent immune mediated dis-
eases. She is a co-founder of Graphite Bio, which is developing a new class of thera-
pies to correct genetic defects in people with serious and life-threatening diseases.
Dr. Roncarolo established the Stanford Center for Denitive and Curative Medicine
to cure patients with currently incurable diseases through the development of inno-
vative stem cell and gene-based therapies.
Werner Rubas, PhD, has 30 years of biotech and pharmaceutical industry experi-
ence and is currently VP of Preclinical Development at Sutro Biopharma. Dr. Rubas
was previously at Nektar Therapeutics, most recently as Executive Director in Non-
Clinical Pharmacokinetics and Pharmacodynamics. Prior to Nektar, Dr. Werner was
Associate Director of the Drug Metabolism and Pharmacokinetics group at Roche. He
has been a SPARK advisor at Stanford University since 2010 and teaches classes on
drug development at UC Berkeley Extension. Dr. Rubas earned his PhD from ETH
Zurich and received his pharmacy license from the School of Pharmacy at ETH Zurich.
Julie Saiki, PhD, is co-founder and Chief Operating Ofcer of Azora Therapeutics,
a clinical-stage biotechnology company developing novel small molecules for the
treatment of inammatory diseases with high unmet needs. Previously, she was a
management consultant at McKinsey & Company, where she worked in the phar-
maceutical and medical products practice advising big pharma, biotech, and health-
care companies on strategy and operations. Dr. Saiki has an MS in Medicine and
PhD in Chemical and Systems Biology from Stanford University and is a former
Fulbright recipient.
Steve Schow, PhD, is an Adjunct Professor in the Chemical and Systems Biology
Department at the Stanford University School of Medicine and has been a SPARK
Advisor since 2009. Dr. Schow was the Vice President of Research and Development
at Telik, Inc. until his retirement in 2014. He has more than 40 years of pharmaceuti-
cal, biotech, and agrichemical industrial R&D experience. His work in drug R&D
spans a wide range of medical indications, as well research on pest control agents.
Dr. Schow holds a doctorate in chemistry from University of California, San Diego,
Author Biographies
264
and completed postdoctoral training at University of California, Los Angeles, and
the University of Pennsylvania.
Michael Taylor, PhD, is the Founder and Principal at Non-Clinical Safety
Assessment, a consulting rm specializing in the development of drugs and medical
devices. Dr. Taylor has more than 20 years of R&D experience in the pharmaceuti-
cal industry and has served on executive teams. He is a board-certied toxicologist
and holds PhD and MS degrees in Toxicology from Utah State University with
postdoctoral training at the NIH and CNRS of France. He has been a SPARK advi-
sor for the past 6 years.
John Walker is a biotechnology veteran with over 40 years of experience in the
healthcare and biopharmaceutical industries. He received a BA in History from the
State University of NewYork at Buffalo and is a graduate of the Advanced Executive
Program, J.L. Kellogg Graduate School of Business at Northwestern University.
Mr. Walker has served on the Board of Directors of over three dozen life sciences
companies. He served on the Board of Lucille Packard Children’s Hospital and is
currently a Trustee at the University of Puget Sound and the Board of Packard
Children’s Health Alliance.
Mary Wang, PhD, is Senior Director of Strategic Planning at Scripps Research.
She received her BS from Massachusetts Institute of Technology and her PhD from
Northwestern University.
Jennifer L. Wilson, PhD, is an Assistant Professor in Bioengineering at University
of California, Los Angeles. Her research aims to use protein–protein interaction
models to anticipate drug effects and identify new targets for untreated diseases.
She has pursued training at FDA, within biotech (at Merrimack Pharmaceuticals,
Genentech) and with SPARK to increase the translational potential of her network
models. She received her BS in Biomedical Engineering from the University of
Virginia and her PhD in Biological Engineering from Massachusetts Institute of
Technology.
Haim Zaltzman, JD, is a Partner at Latham & Watkins LLP and chairs its San
Francisco Bay Area Finance Practice. He focuses primarily on healthcare and
technology- related private equity, growth equity, and emerging growth nancing
transactions. Mr. Zaltzman has also been featured on CNBC, Bloomberg, The
Washington Post, CFO Magazine, The Recorder, Law360.com, The Daily Journal,
the International Financial Law Review, and VCExperts.com for his debt expertise.
He received his BA from Stanford University, MA from Russian Academy of
Sciences, and JD from Harvard Law School.
Author Biographies
265
A
Absorption, distribution, metabolism, and
excretion (ADME), 13, 14, 21–23, 29,
30, 49, 51, 95, 117–123, 126, 132, 244
Active pharmaceutical ingredient (API), 14,
15, 30, 31, 132, 135–138, 140, 145,
152–154, 157, 179, 181
Angel investor, 230
Animal models, 13, 21, 23, 36, 52, 68, 83,
101–104, 106, 108, 111–116, 121, 133,
135, 136, 156, 164
Antimicrobial, 57, 58, 244, 247, 248, 250, 251
antibiotic, 244
antiparasitic, 245
Articial intelligence (AI), 12, 34, 38, 39,
120–121, 248
B
Biologic License Application (BLA), 11, 15,
30, 78, 144–147, 149, 158, 176
Biomarker, 15, 19, 34–37, 86–92, 116, 123,
125, 135, 169, 172, 175, 211, 212, 223
Breakthrough, 21, 147
C
Cell therapy, 12, 34, 75–78, 161–163, 199, 220
Chemistry, Manufacturing, and Controls
(CMC), 6, 15, 145, 147, 149, 154, 157,
158, 161, 162, 164, 248–250
Clinical trial, 2, 4, 5, 7, 22, 28, 34, 40, 41, 53,
56, 77, 78, 102, 117, 123, 126, 127,
143, 145, 147, 148, 151–153, 155, 164,
165, 171–178, 195–199, 201, 209, 211,
213, 216, 219, 223, 247–249
clinical trial design, 12, 19, 25,
144, 164–176
clinical trial protocol, 165
phase 0, 167
phase 1, 165
phase 2, 165
phase 3, 165
Clinical utility, 87, 89, 91–92, 215, 224
Clinical validity, 87–91
Common Technical Document (CTD), 146
Conict of interest (COI), 192, 194–198, 201
Contract manufacturing organization (CMO),
143–144, 153, 154
Contract research organization (CRO), 14, 27,
31, 203, 249
D
Diagnostic, vii, 4, 5, 7–9, 23, 33–92, 125, 186,
187, 216, 221, 223–226
DNA editing, 68, 70, 73, 74
Drug delivery, 17, 116, 129, 132
Drug product (DP), 12, 14–15, 22, 26, 34, 38,
144, 145, 152, 153, 157, 159–161, 199,
203, 204
Drug repurposing, 37–39
repurposed drug, 37
Drug resistant, 22, 243, 251
E
End in mind, vii, 5, 16–17, 24, 25, 28, 64
Endpoint, 12, 15, 19, 29, 77, 106, 108,
165–167, 169, 172–177, 216
clinical endpoint, 176
surrogate endpoint, 149
Index
© 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
266
F
Fast track, 15, 21, 147
Freedom to operate (FTO), 21–24, 40, 158,
187, 190
G
Gantt chart, 26–28, 30, 158
Gene editing, 67–72, 74, 75, 162
Gene therapy, 12, 72, 75–81, 161, 162, 219, 226
Good Laboratory Practice (GLP), 14, 15,
29–31, 41, 93, 134, 138
Good Manufacturing Practice (GMP), 14, 15,
30, 123, 138, 151–153, 156–158, 161,
162, 179, 180
H
High-throughput screening (HTS), 10, 13, 38,
42–48, 66, 126, 202, 244
Human equivalent dose (HED), 113, 114, 168
I
Initial public offering (IPO), 10, 31,
225–227, 238
Institutional Review Board (IRB), 11, 38–41,
144, 166, 178, 198–201
Intellectual property (IP), 3, 20, 21, 23, 25, 31,
42, 64, 104, 187–191, 193–195, 198,
199, 227, 229, 232, 237–239
Investigational New Drug application (IND),
14, 15, 20, 26, 29, 38, 40, 41, 55, 56,
77, 79, 81, 112, 144–147, 149, 153,
154, 158, 164, 165, 167, 189, 199–201
L
Lead optimization, 47–53, 126, 140
lead compound, 53
M
Machine learning, 39, 56–61
Market share, 217–219
Market size, 20, 22, 132, 177, 210–212, 215,
216, 221, 246, 247
Medicinal chemistry, 10, 27, 43, 47–48, 50,
52, 53, 126, 244, 248
Metabolism, 13, 16, 22, 41, 49, 51, 102, 103,
107, 117, 119–124, 126, 130, 131, 134,
136, 168, 169, 244
Monoclonal antibody (mAb), 11, 12, 21, 22,
62, 125, 135, 155, 159, 160
Multiple ascending dose (MAD), 165, 168,
169, 180
N
Natural products, 54–56, 247–249
Neglected tropical disease (NTD), 202–204, 238
New Drug Application (NDA), 11, 15, 55,
127, 144–147, 149, 176, 205
New molecular entity (NME), 134, 143, 149,
165, 168, 187, 205
Not-for-prot, 37, 42, 187, 202–206
O
Orphan drug, 21, 145, 205
P
Patent, 3, 10, 21–23, 37, 39, 40, 43, 69,
186–193, 217, 227
non-provisional patent, 188
patent application, 188
patent protection, 9, 24, 40, 42, 186–188,
191–193, 217, 238
provisional patent, 186
Personalized medicine, 34, 81, 210–212
Pharmacodynamics (PD), 22, 23, 34, 51–53,
55, 70, 77, 102, 108, 112, 113,
115–117, 122, 123, 132, 167,
168, 223
Pharmacogenomics (PGx), 102, 117, 120,
123–129, 211
Pharmacokinetics (PK), 13–15, 22, 26, 42,
51–53, 55, 58, 69, 77, 102, 105, 108,
111, 112, 117–124, 126, 128, 134, 136,
147, 165–169, 174, 223, 244
Pharmacology, vii, 25, 29, 34, 36, 37, 52, 53,
105, 110–117, 135, 144–147, 248
Preclinical safety, 135
Preclinical safety study, 29, 41, 56, 102,
133–137, 168
Predictive value, 13, 59, 88–90
Pre-IND meeting, 3, 14, 29, 147,
148, 166–167
Priority Review Voucher (PRV), 205
Product share, 215–218
Proof-of-concept (PoC), 3, 37, 63, 64, 67, 68
Public disclosure, 186, 188–189, 193
Q
Quality assurance (QA), 6, 144, 151, 154, 161
Quality control (QC), 14, 15, 144, 150–154
R
Reproducibility, 43, 44, 87, 108, 110
Return on investment, 22, 40, 86, 219, 227,
243, 247
Index
267
RNA editing, 12, 34, 67–74
Route of administration, 12, 14, 21, 22, 38–42,
102, 103, 122, 129–133, 135, 145, 169,
199, 217, 221
S
Side effect, 11, 15, 17–18, 38, 39, 41, 50, 57,
59, 73, 102, 121, 122, 130, 133, 137,
180, 208
Single ascending dose (SAD), 165, 168,
169, 181
Startup, 2, 7, 8, 10, 163, 186, 187, 191–194,
196, 198, 207, 210, 221, 225,
228–232, 244
Surrogate endpoint, 15, 19, 34, 149,
169–175, 181
T
Target product prole (TPP), vii, 1, 5, 11, 12,
14, 20–25, 28–31, 44, 64, 66, 204,
209–210, 212, 213, 239
Technology transfer, 81, 157, 185–237
Technology transfer ofce (TTO), 78, 186,
190–195, 239
Toxicity, 17, 21, 23, 24, 37, 50–52, 55, 57, 64,
69, 70, 73, 102, 104, 105, 120, 122,
126, 131, 134–137, 164, 165, 180, 235
Toxicology, vii, 14, 21, 25–27, 29, 51, 77, 121,
122, 134, 136, 145–147, 160, 167, 244,
248, 249
U
Unmet clinical need, 5, 7, 10, 17–19, 221, 244
V
Vaccine, 34, 82–86, 95, 134, 135, 154–156,
161, 163, 179, 202, 204, 205, 226, 239
Venture capital (VC), 8, 10, 29, 30, 32, 52,
166, 187, 203, 225–231
W
World Intellectual Property Organization
(WIPO), 190
Index