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Susan Stevenson, executive director, and Craig Mickanin, director, of the Swiss multinational pharmaceutical company, Novartis, based in Basel, Switzerland, deliver an informative overview of the incredible advances in gene therapy and transformative technologies that are rapidly changing the techniques medical professionals utilize to fight disease. Sue Stevenson is an experienced director with an extensive history in the pharmaceuticals industry. Her focus is in biologics, life sciences, validation, and cell biology. She holds a BS in chemistry from Western Maryland College as well as a Ph.D. in biochemistry and molecular biology from the distinguished Wake Forest University.

Craig Mickanin holds a BS in biology and history from the prestigious William & Mary University, the second oldest college in the nation, and a well-respected cutting-edge research university. The Novartis Institutes for BioMedical Research (NIBR) is the innovation engine and the NIBR team collaborates across various scientific and organizational boundaries, working for therapeutic breakthroughs for all patients.

Novartis’s stated mission is to utilize science-based innovation to address the world’s most challenging healthcare issues. Novartis seeks to discover and develop breakthrough treatments and successful ways to deliver them to the world’s population in need of care. Research and development (R&D) are at Novartis’s core and a key component to their primary strategy. Stevenson describes Novartis’s mission goal as it relates to disease treatment and prevention, specifically regarding cell and gene therapy. She discusses a few of their gene therapy methods, including the process of removing some of a patient’s cells, to modify those cells to, in essence, fight a particular tumor type, and then reintroducing the cells into the patient’s body so that the cells can get to work destroying the tumor. This cell therapy is an example of ex vivo therapy. Stevenson also outlines some of their early development on injectable options for cell delivery and the clinical studies that are going on currently.

The biochemistry Ph.D. details their exciting work that she hopes will bring truly amazing medical advancements to patients worldwide, such as restoring blindness, restoring hearing, and curing cancer, as well as some other in vivo studies that are showing promise.

Mickanin discusses his early work and provides some insight into how technology advances have helped buoy the transformative aspect of the field. He discusses current stem cell studies and the extent of their use in the industry. Mickanin details some of the aspects of the CRISPR technology, which stands for Clustered Regularly Interspaced Short Palindromic Repeats. Mickanin states that the system allows researchers to induce double-stranded breaks into virtually any genome with specificity, and Novartis has been investing significant resources into the research. And Stevenson adds how their use of CRISPR/Cas9 technology will aid them in the process of introducing cells that can prevent disease or disease complications. She lays out the intricacies of the CRISPR/Cas9 and her hopes for its use ongoing, in future discovery and delivery of treatment options for patients.

Mickanin provides an overview of how ex vivo strategies and in vivo strategies are determined, and the issues that are involved when making a choice as to how to move forward with either for treatments. Stevenson discusses the expectations for the future and MIT studies that indicate perhaps as many as forty new gene therapy medicines may be released by the year 2022.

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