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Jenny Stanford Series on Nanomedicine
Series Editor
Raj Bawa

Titles in the Series

Vol. 1 Vol. 4
Handbook of Clinical The Road from Nanomedicine to
Nanomedicine: Nanoparticles, Precision Medicine
Imaging, Therapy, and Clinical Shaker A. Mousa, Raj Bawa, and
Applications Gerald F. AudeƩe
Raj Bawa, Gerald F. Audette, 2020
and Israel Rubinstein, eds. 978-981-4800-59-4 (Hardcover)
2016 978-0-429-29501-0 (eBook)
978-981-4669-20-7 (Hardcover)
Vol. 5
978-981-4669-21-4 (eBook)
Current Issues in Medicine,
Pharma, and Biotech
Vol. 2
Raj Bawa and Gerald F. AudeƩe,
Handbook of Clinical
eds.
Nanomedicine: Law, Business,
2021
Regulation, Safety, and Risk
Raj Bawa, ed., Gerald F. AudeƩe
Vol. 6
and Brian E. Reese, asst. eds.
Drug Delivery at the Nanoscale:
2016
A Guide for ScienƟsts, Physicians,
978-981-4669-22-1 (Hardcover)
and Lawyers
978-981-4669-23-8 (eBook)
Raj Bawa, ed.
2022
Vol. 3
Immune Aspects of
Biopharmaceuticals and
Nanomedicines
Raj Bawa, János Szebeni,
Thomas J. Webster, and
Gerald F. AudeƩe, eds.
2018
978-981-4774-52-9 (Hardcover)
978-0-203-73153-6 (eBook)
 Jenny Stanford Series on Nanomedicine Vol. 4

The Road
from Nanomedicine
to Precision Medicine
Part A

edited by
Shaker A. Mousa, PhD, MBA
Chairman & Executive Vice President, The Pharmaceutical Research Institute,
Rensselaer, New York, USA
Professor of Pharmacology, Albany College of Pharmacy and Health Sciences,
Albany, New York, USA

Raj Bawa, MS, PhD

Patent Agent, Bawa Biotech LLC, Ashburn, Virginia, USA
Vice President and Chief IP Officer, Guanine Inc., Rensselaer, New York, USA
Scientific Advisor, Teva Pharmaceutical Industries, Ltd., Israel

Gerald F. Audette, PhD

Associate Dean of Faculty, Faculty of Science,
Associate Professor, Department of Chemistry,
York University, Toronto, Canada
Published by

Jenny Stanford Publishing Pte. Ltd.

Level 34, Centennial Tower
3 Temasek Avenue
Singapore 039190
Email: [email protected]
Web: www.jennystanford.com

Note from the Series Editor and the Publisher

Extensive efforts have been made to make the information provided herein as
accurate and as up-to-date as possible. However, the accuracy and completeness of
this information cannot be guaranteed. It is important to note that knowledge and
best practices in the various fields represented in this book (medicine, drug delivery,
nanomedicine, precision medicine, genomics, bioinformatics, biologics, tissue
engineering, patent law, FDA law, regulatory science, toxicology, pharmaceutical
sciences, etc.) are constantly evolving. As new research and experience broaden
our knowledge base, changes in research methods, legal and business practices,
diagnostics, assays, tools and techniques, medical formulations, and/or treatments
may become necessary. Therefore, it is imperative that the reader does not solely
rely on the information presented herein and always carefully reviews product
labels, warnings, data, and directions before using or consuming any formulation,
device or drug product. For additional information about a product, please contact
the manufacturer, FDA, inventor, physician, pharmacist, or other licensed health-
care professional, as appropriate. Similarly, careful evaluation of any procedures,
manufacturing steps, legal ideas, medical protocols, regulatory guidances, or assays
described herein is warranted. To the fullest extent of the law, neither the publisher
nor the editors or authors make any representations or warranties, express or
implied, with respect to the information presented in this book, for its use or
misuse, or interpretation thereof. In this regard, they assume no liability for any
injury and/or damage to persons or property as a matter of product liability,
negligence, or otherwise.

A catalogue record for this book is available from the Library of Congress and the
British Library.

The Road from Nanomedicine to Precision Medicine

Copyright © 2020 Jenny Stanford Publishing Pte. Ltd. All rights reserved. This book,
or parts thereof, may not be reproduced in any form or by any means, electronic or
mechanical, including photocopying, recording or by any information storage and
retrieval system now known or to be invented, without written permission from
the publisher. For photocopying of material in this volume, please pay a copying
fee through the Copyright Clearance Center, Inc., 222 Rosewood Drive, Danvers, MA
01923, USA. In this case, permission to photocopy is not required from the publisher.

ISBN 978-981-4800-59-4 (Set)

ISBN 978-0-429-29501-0 (Set) (eBook)
ISBN 978-981-4800-98-3 (Part A) (Hardcover)
This book is dedicated to my wife and children for their
love, support, and encouragement.

—Shaker A. Mousa

Dedicated with love to the three most amazing women

in my life:
my mother, my wife, and my mother-in-law.

—Raj Bawa

This book is dedicated to my wife and son, for their love

and support.

—Gerald F. Audette
 Cover Image

A depiction of nano-robots in the human cardiovascular system.

Such nano-combination products (NCPs), currently at the R&D
phase, could be commercialized in the coming decades. They could
serve multiple functions of diagnostic evaluation, drug delivery,
and assessment of therapy—all within the same nanoengineered
combination product. As technological advances continue to
merge product types (drug, biologic, or device), I expect that
more products will fall into the category of combination products.
The FDA and the EMA are certain to struggle with appropriate
regulatory pathways for such highly integrated combination
products at the interface of the three product domains.
—Dr. Raj Bawa, Series Editor
 Also Read
Handbook of Clinical Nanomedicine.
Vol. 1. Nanoparticles, Imaging, Therapy, and
Clinical Applications
Raj Bawa, PhD, Gerald F. AudeƩe, PhD, and Israel
Rubinstein, MD (Editors)
978-981-4669-20-7 (Hardcover), 978-981-4669-21-4 (eBook)
1662 pages

This handbook (55 chapters) provides a comprehensive roadmap

of basic research in nanomedicine as well as clinical applica ons.
However, unlike other texts in nanomedicine, it not only highlights
current advances in diagnos cs and therapeu cs but also explores
related issues like nomenclature, historical developments, regulatory
aspects, nanosimilars and 3D nanofabrica on. While bridging the gap
between basic biomedical research, engineering, medicine and law, the
handbook provides a thorough understanding of nano’s poten al to
address (i) medical problems from both the pa ent and health provider’s perspec ve, and (ii) current
applica ons and their poten al in a healthcare se ng.

“Dr. Bawa and his team have meƟculously gathered the disƟlled experience of world-class
researchers, clinicians and business leaders addressing the most salient issues confronted in
product concept development and translaƟon.”
Gregory Lanza, MD, PhD
Professor of Medicine and Oliver M. Langenberg Dis nguished Professor
Washington University Medical School, USA

“This is an outstanding, comprehensive volume that crosscuts disciplines and topics fiƫng
individuals from a variety of fields looking to become knowledgeable in medical nanotech research
and its translaƟon from the bench to the bedside.”
Shaker A. Mousa, PhD, MBA
Vice Provost and Professor of Pharmacology
Albany College of Pharmacy and Health Sciences, USA

“Masterful! This handbook will have a welcome place in the hands of students, educators, clinicians
and experienced scienƟsts alike. In a rapidly evolving arena, the authors have harnessed the field
and its future by highlighƟng both current and future needs in diagnosis and therapies. Bravo!”
Howard E. Gendelman, MD
Margaret R. Larson Professor and Chair
University of Nebraska Medical Center, USA

“It is refreshing to see a handbook that does not merely focus on preclinical aspects or exaggerated
projecƟons of nanomedicine. Unlike other books, this handbook not only highlights current
advances in diagnosƟcs and therapies but also addresses criƟcal issues like terminology, regulatory
aspects and personalized medicine.”
Gert Storm, PhD
Professor of Pharmaceutics
Utrecht University, The Netherlands
 Handbook of Clinical Nanomedicine.
Vol. 2. Law, Business, RegulaƟon,
Safety, and Risk
Raj Bawa, PhD (Editor), Gerald F. AudeƩe, PhD, and Brian
E. Reese, PhD, MBA, JD (Assistant Editors)
978-981-4669-22-1 (Hardcover), 978-981-4669-23-8 (eBook)
1448 pages
This unique handbook (60 chapters) examines the en re “product life
cycle,” from the crea on of nanomedical products to their final market
introduc on. While focusing on cri cal issues relevant to nanoproduct
development and transla onal ac vi es, it tackles topics such as
regulatory science, patent law, FDA law, ethics, personalized medicine,
risk analysis, toxicology, nano-characteriza on and commercializa on
ac vi es. A separate sec on provides fascina ng perspec ves and
editorials from leading experts in this complex interdisciplinary field.
“The disƟnguished editors have secured contribuƟons from the leading experts in nanomedicine
law, business, regulaƟon and policy. This handbook represents possibly the most comprehensive and
advanced collecƟons of materials on these criƟcal topics. An invaluable standard resource.”
Gregory N. Mandel, JD
Peter J. Liacouras Professor of Law and Associate Dean
Temple University Beasley School of Law, USA
“This is an outstanding volume for those looking to become familiar with nanotechnology research
and its translaƟon from the bench to market. Way ahead of the compeƟƟon, a standard reference
on any shelf.”
Shaker A. Mousa, PhD, MBA
Vice Provost and Professor of Pharmacology
Albany College of Pharmacy, USA
“The editors have gathered the disƟlled experience of leaders addressing the most salient issues
confronted in R&D and translaƟon. Knowledge is power, parƟcularly in nanotechnology translaƟon,
and this handbook is an essenƟal guide that illustrates and clarifies our way to commercial
success.”
Gregory Lanza, MD, PhD
Professor of Medicine and Oliver M. Langenberg Dis nguished Professor
Washington University Medical School, USA
“The Ɵtle of the handbook reflects its broad-ranging contents. The intellectual property chapters
alone are worthy of their own handbook. Dr. Bawa and his coeditors should be congratulated for
gathering the important wriƟngs on nanotech law, business and commercializaƟon.”
Richard J. Apley, JD
Chief Patent Officer
Litman Law Offices/Becker & Poliakoff, USA
“It is clear that this handbook will serve the interdisciplinary community involved in nanomedicine,
pharma and biotech in a highly comprehensive way. It not only covers basic and clinical aspects
but the oŌen missing, yet criƟcally important, topics of safety, risk, regulaƟon, IP and licensing. The
secƟon Ɵtled ‘PerspecƟves and Editorials’ is superb.”
Yechezkel (Chezy) Barenholz, PhD
Professor Emeritus of Biochemistry and Daniel Miller Professor of Cancer Research
Hebrew University-Hadassah Medical School, Israel
 Immune Aspects of Biopharmaceuticals
and Nanomedicines

Raj Bawa, János Szebeni, Thomas J. Webster and Gerald F.

AudeƩe (Editors)
978-981-4774-52-9 (Hardback), 978-0-203-73153-6 (eBook)
1038 pages

The enormous advances in the immunologic aspects of biotherapeu cs

and nanomedicines in the past two decades has necessitated an
authorita ve and comprehensive reference source that can be relied upon
by immunologists, biomedical researchers, clinicians, pharmaceu cal
companies, regulators, venture capitalists, and policy makers alike. This
text provides a thorough understanding of immunology, therapeu c
poten al, clinical applica ons, adverse reac ons, and approaches to
overcoming immunotoxicity of biotherapeu cs and nanomedicines. It
also tackles cri cal, yet o en overlooked topics such as immune aspects of nano-bio interac ons,
current FDA regulatory guidances, complement ac va on-related pseudoallergy (CARPA), advances
in nanovaccines, and immunogenicity tes ng of protein therapeu cs.

“This outstanding volume represents a review of the various effects of biopharmaceu cals and
nanomedicines on the immune system: immunotherapy, vaccines, and drug delivery; challenges
and overcoming transla onal barriers stemming from immunotoxicity; strategies to designing
more immunologically friendly formula ons.”
África González-Fernández, PhD, MD
Professor of Immunology and President of the Spanish Society of Immunology,
University of Vigo, Spain

“For those who are specialists, and for those interested in a broader understanding of biologics
and nanomedicines, this is a superb book, with interna onally accomplished contributors. It serves
both as a reference and as a prac cal guide to the newest advances in these important fields.
Highly recommended!”
Carl R. Alving, MD
Emeritus Senior Scien st, Walter Reed Army Ins tute of Research, Silver Spring, Maryland, USA

“A skillfully produced book that addresses an o en-missed topic: immune aspects of biologicals
and nanoscale therapeu cs, with an emphasis on clinical relevance and applica ons.”
Rajiv R. Mohan, PhD
Professor and Ruth M. Kraeuchi Missouri Endowed Chair Professor,
University of Missouri, Columbia, USA

“An indispensable masterpiece! It represents a rich source of informa on on interac ons of

biologics and nanodrugs with the immune system—all cri cal for medical applica ons. Volume 3,
once again, achieves the series’ high standards.”
László Rosivall, MD, PhD, DSc Med, Med habil.
Széchenyi Prize Laureate and Professor, Faculty of Medicine, Semmelweis University,
Budapest, Hungary
The American Society for Nanomedicine (ASNM) (https://www.
nanomedus.org) is a nonprofit, professional medical organization
based in Ashburn, Virginia, USA. It was founded in 2008 by Dr. Raj
Bawa of Bawa Biotech LLC and Dr. Esther Chang of Georgetown
Medical Center. The ASNM comprises members drawn from diverse
fields, including medicine, law, nanotechnology, pharma, biotech,
engineering, and biomedical sciences with the common goal of
advancing nanomedicine research to benefit global health. These
goals are achieved through an open forum of ideas and collaborative
efforts as well as close cooperation with our partner organizations.
Since its inception, the ASNM has organized major international
conferences.
Specifically, the vision of the ASNM includes
• promoting research related to all aspects of nanomedicine
and providing a forum through scientific meetings for the
presentation of basic, clinical, and population-based research;
• promoting and facilitating the formal training of physicians, basic
medical scientists, engineers, molecular biologists, statisticians,
and allied healthcare providers in nano-related medical research
and education;
• encouraging primary and secondary preventive measures and
nano-based technologies to reduce the incidences of various
diseases;
• facilitating the establishment of programs and policies that can
better serve early diagnosis.
The European Foundation for Clinical Nanomedicine (https://www.
clinam.org), founded in 2007 by Beat Löffler and Patrick Hunziker,
is an organization based in Basel, Switzerland. Its primary mission
is to advance medicine to the benefit of individuals and society
through the application of nanoscience and targeted medicine.
Aiming at prevention, diagnosis, and therapy, it supports clinically
focused research and the interaction and information flow between
clinicians, researchers, and the public. The major goal is to support
the development and application of nanomedicine and targeted
medicine and having in scope all nanomedicine-related fields.
The foundation runs a lab, creates an annual summit for
clinical nanomedicine, and established the European Journal of
Nanomedicine (now PRNANO online). The 12th CLINAM Summit
will be held May 17–20, 2020, and is titled “Clinical Nanomedicine
and the Impact of Digitalization and Artificial Intelligence for
Precision Medicine—The Technologies for Diagnosis and & Therapy
in Personalized Medicine.” The Summit traditionally brings
together over 500 participants from more than 40 countries.
CLINAM founded the European Society for Nanomedicine
(ESNAM), which has more than 1,000 members today. ESNAM was
the driving force for the formation of the International Society
for Nanomedicine, (ISNM) which brings together members from
Japan, Korea, USA, Canada, Europe, South America, Australia,
Africa, and India. CLINAM organizes worldwide summer schools.
The next one is planned in Asia for the autumn of 2020. During
the summit, CLINAM also hosts satellite meetings. For the past five
years, the International Pharmaceutical Regulators Programme
(IPRP), a meeting for global regulatory authorities, is also held.
This group uses the CLINAM platform to provide statements on
the global cooperation to come to an optimal framework for
regulatory matters pertaining to nanomedicine and precision
medicine.
 About the Editors
Shaker A. Mousa, PhD, MBA, FACC, FACB, FAHA,
FNAI, is an endowed chair, tenured professor of
pharmacology at Albany College of Pharmacy
in Albany, New York, USA. He also serves as
the executive vice president and chair of the
Pharmaceutical Research Institute in Rensselaer,
New York, USA. Dr. Mousa is the founder of several
spin-off pharmaceutical and biotechnology companies. Previously,
for two decades, he was a senior principal research scientist and
a research fellow at DuPont Pharmaceuticals and Imaging Co.,
DuPont Merck, and DuPont Pharmaceuticals Company. He is a
recipient of several national and international awards, including
the 2017 Kuwait Foundation for Advancement in Sciences Laureate
for Applied Sciences in Medicine.
Among Dr. Mousa’s professional accomplishments are his
contributions to several patents, speci ically to the discovery and
development of novel anti-platelet, anti-thrombotic therapies,
noninvasive myocardial perfusion, and thrombus imaging agents.
His work is reported in over 1,000 peer-reviewed publications
and he holds over 400 US and international patents. He contributed
to the discovery and development of the following products/
clinical candidates: Cardiolite® (Tc-99m sestamibi, non-invasive
myocardial perfusion imaging agent), Marluma (for breast cancer
detection), DMP444 (Tc-99m platelet GPIIb/IIIa antagonist
for non-invasive thrombus imaging agent for venous and arterial
thromboembolic disorders), Roxi iban (DMP754, oral anti-
platelet/anti-thrombotic agent for the prevention and treatment of
coronary, carotid and peripheral artery thromboembolic
disorders). Dr. Mousa is involved in the discovery of novel site
directed anti-αv/β3 for tumor radiotherapy, imaging, and novel
pharmacological aspects of heparins and non-anticoagulant
heparins. His main research interests are in drug discovery and
drug development arena as well as bringing novel concepts from
the bench-to-the-bedside and vice versa via key enabling
xvi About the Editors

technologies/strategies, including nanotechnology, biotechnology,

stem cell, and pharmacotherapy.
Dr. Mousa received his PhD in pharmacology from Ohio
State University (USA), completed postdoctoral research in
cardiovascular pharmacology at the University of Kentucky (USA),
and earned his MBA from Widener University (USA). He is an
elected Fellow of the American College of Cardiology (FACC), a
Fellow of the National Academy of Clinical Biochemistry (FACB),
and fellow of the American Heart Association (FAHA). In 2018,
he was elected as a fellow of the National Academy of Inventors
(FNAI). He is a member of several national and international
societies and serves on several NIH study sections, US Department
of Defense, and other funding agencies. In addition, he is the
editor-in-chief of Biomedicines and serves on the editorial boards
of several high impact scientific and medical journals.

Raj Bawa, MS, PhD, is president of Bawa Biotech

LLC, a biotech/pharma consultancy and patent
law firm based in Ashburn, Virginia, USA that he
founded in 2002. Trained as a biochemist and
microbiologist, he is an inventor, entrepreneur,
professor, and registered patent agent licensed to
practice before the US Patent & Trademark Office. He
is currently a scientific advisor to Teva Pharmaceutical Industries,
Ltd. (Israel), a visiting research scholar at the Pharmaceutical
Research Institute of Albany College of Pharmacy (Albany, New
York), and vice president and chief intellectual property officer at
Guanine, Inc. (Rensselaer, New York). He has previously served as
a principal investigator of SBIRs as well as a reviewer for both the
NIH and NSF. Currently, he is principal investigator of a CDC grant
to develop an assay for Carbapenem-resistant Enterobacteriaceae.
In the 1990s, Dr. Bawa held various positions at the US Patent &
Trademark Office, including primary examiner from 1996–2002.
Previously, he was an adjunct professor at Rensselaer Polytechnic
Institute in Troy, New York from 1998–2018, where he received
his doctoral degree in three years (biophysics/biochemistry).
He is a life member of Sigma Xi, co-chair of the nanotech and
precision medicine committees of the American Bar Association
(2015–20) and founding director of the American Society for
Nanomedicine (founded in 2008). He has authored over 100