1 November 2018

Building bridges to realise biological pharmaceutical therapies

Enrico Mastrobattista appointed Professor of Pharmaceutical Biotechnology and Delivery

Dr. Enrico Mastrobattista has been appointed Professor of Pharmaceutical Biotechnology and Delivery at Utrecht University’s Faculty of Science as of November 1st. His Chair will focus on the design, formulation and delivery of novel biotherapeutics and advanced therapies. Mastrobattista’s expertise is rather unique, in that he combines technical pharmaceutical knowledge on advanced biotherapeutics as well as on the controlled delivery systems necessary to get them inside the right cells.

Imagine you have end-stage cancer that no longer responds to conventional chemotherapy. By taking a small biopsy from the tumour, a personalized synthetic vaccine can be made that will stimulate your immune cells to recognize and destroy the cancer cells throughout the body. Or you are a haemophilia patient suffering from severe bleeding. Instead of having to inject very expensive recombinant or plasma-derived clotting factors every day to stop the bleeding, a single injection with a nanomedicine that targets and corrects the genetic defect in the clotting factor will be enough to cure the disease. This is the future Enrico Mastrobattista is working on.

Very promising

A large number of drug products that currently enter the market are cells or drugs manufactured in or extracted from a biological system such as a cell or an entire organism. These so-called biopharmaceuticals and advanced therapy medicinal products (ATMPs) seem very promising, but both the production of these drugs and their delivery to the right places in the body, still pose many challenges. Enrico Mastrobattista is seen as one of few pharmaceutical scientists who has the knowledge and experience to contribute to breakthroughs in the field.

A glimpse of the challenging search for the 'magic bullet': a delivery system for gene therapy which exclusively targets the ‘bad’ cells.

Very challenging

Many biological drugs rely on sophisticated delivery systems to reach their intended target site in the human body. For example, CRISPR/Cas has emerged as a powerful tool to correct specific mutations or knock out entire disease-causing genes, but it is useless without a delivery system that enables safe, intracellular delivery of the CRISPR/Cas components. Dr. Mastrobattista is leveraging his extensive expertise on nanomedicines to design synthetic delivery systems for gene editing tools, including CRISPR/Cas9. Furthermore, he is developing strategies that enable intracellular delivery of proteins for therapeutic applications.

Bridges

Mastrobattista is qualified as someone who is capable of building the bridges necessary to overcome these challenges. “By now, we have over 20 years of experience in experimenting with biopharmaceuticals and finding solutions to the delivery problems for these complex molecules. The prospects of this wholly new pharmaceutical concept are very, very promising. But it has also become clear that we can only succeed in realising them if we are able to integrate the most advanced fundamental and applied research”, he explains. “From the molecular and cellular level in the fundamental life sciences, to nanomaterials sciences and pharmaceutical and medical research.”

We can only succeed if we are able to integrate the most advanced fundamental and applied research

Future

The pharmaceutical future Mastrobattista envisions is still a long way off. “But I am convinced that someday we will be able to modify patients’ cells in such a way that this leads to a direct cure or that they will produce biotherapeutics at will and at concentrations that can be tailored to the individual patient’s needs.”

Enrico Mastrobattista

A medical biologist by training, Enrico Mastrobattista obtained his PhD in Advanced Drug Delivery. He then spent over two years as a Marie Curie postdoctoral fellow in the MRC-Laboratory of Molecular Biology in Cambridge. Since his return to Utrecht University, he has published over 100 scientific articles and several book chapters on pharmaceutical biotechnology. Mastrobattista also holds several patents and served as the scientific coordinator at IMI COMPACT, a large public-private partnership on finding solutions for the delivery problem of biopharmaceuticals.

Prix Galien

In 2008 he obtained a Vidi grant for his research on directed evolution of artificial viruses for gene delivery. In 2013 he was awarded the Prix Galien Research Award for his work on drug delivery. Earlier this year he received a Vici grant of 1.5 million Euros, for his research proposal on restoring genetic defects using CRISPR/Cas9 in nanoparticles. Since 2013, Mastrobattista has been a board member of the Netherlands Society of Gene & Cell Therapy (nvgct.nl).

More about Enrico Mastrobattista