Biotherapeutics: From Small to Large Molecules and Cells

This book reviews biotherapeutics from a medicinal chemistry perspective. It covers proteins, nucleic acids, low molecular weight hormones, small peptides, extracellular vesicles, gene therapy, cell-based products, and tissue-engineered products. Expert contributors provide insights into the mechanisms of action and translational processes of biotherapeutics. Particular attention is given to the latest developments in therapeutic proteins and nucleic acids. Biotherapeutic formulation developments like encapsulation, structural modifications and nanovehiculization are also presented in this book.

Divided into 15 chapters, the book begins with basic concepts and definitions of biotherapeutics from a medicinal chemistry standpoint. The following chapters focus on therapeutic proteins, monoclonal antibodies, protein production, and structural modifications. Other chapters cover topics such as antisense oligonucleotides, aptamers, mRNA, gene therapy, as well as other biotherapeutics like low molecular weight hormones and small peptides. The book concludes with an overview of biotherapeutic formulations and an authoritative discussion on regulatory aspects.

Throughout the book, readers will learn how biotherapeutics, whether obtained through bioprocesses or not, impact bioresponses and can be utilized for therapeutic purposes. Given its breadth, the book appeals to researchers in medicinal chemistry and biotherapeutics, scholars of medicinal chemistry, students at all levels of biochemical studies, practitioners in the medical field, and anyone interested in biotherapeutics.

Victoria Calzada, MSc, PhD, is Assistant Professor at Faculty of Sciences, Universidad de la

República, Montevideo-Uruguay. In her current position, she is working with aptamers as in vivo molecular

imaging probes and as biotherapeutics. She is the principal investigator of the

national Aptamer group which includes students, national researchers and

international collaborators.

Dr. Calzada is a young researcher, with several peer-review publications in international

journals as well as participated as peer- review in grants and papers. Besides,

she has a sustained list of funding for her research program. Thus, since 2016

is distinguished with the Level 1 of the National Research System and she was

recently awarded the national L’OREAL-UNESCO award for Women in Science.

In addition, the Iberoamerican Network of Aptamers (REDIBA) is coordinated by Dr. Calzada since

2018.

Hugo Cerecetto received the Pharmacist degree from Universidad de la República and the Doctor of

Philosophy in Medicinal Chemistry from Universidad de Navarra (Pamplona,

Spain). He joined the Centro de Investigaciones Nucleares at Facultad de Ciencias-Universidad de la República

(Montevideo, Uruguay) as Área de Radiofarmacia responsible in 2013. Prior to coming to Facultad de Ciencias, he served as Professor of Organic Chemistry at the Facultad de Química-Universidad de la República (Montevideo, Uruguay). He has published extensively, almost 260 papers, in the area of medicinal chemistry and specialized R+D+i of new drugs.

In 2010, he was awarded the Elsevier-SCOPUS Prize for his publications. He has served on peer review panels and scientific advisory boards at Uruguayan and Ibero-American agencies. Dr. Cerecetto has had an active interest in graduate

education for many years.

Juan Pablo Tosar, PhD, is Assistant Professor and head of the Analytical Biochemistry Laboratory

at Facultad de Ciencias (Universidad de la República, Uruguay). He is also senior researcher at the Pasteur Institute of Montevideo, Uruguay. His research is focused on the design of new analytical techniques for the study of nucleic

acids, from electrochemical biosensors to next generation sequencing methods, with an emphasis on extracellular vesicles and extracellular RNA. His group has discovered extracellular ribosomes and extracellular nicked tRNAs, among other relevant scientific contributions. He is currently focused on understanding the mechanisms of RNA-mediated intercellular communication and the recognition of extracellular RNAs by innate immune sensors. He is also interested in exploring

new ways to use cell-free RNA molecules as both diagnostic and therapeutic agents.