[SPL-01] Semi-Plenary Lecture 01

[SPL-01-1] The Therapeutic Landscape for Amyotrophic Lateral Sclerosis

^○Lucie Bruijn (Translational Medicine, Novartis Gene Therapies)

I have worked in the neurodegenerative field for the past 30 years. After completing a Pharmacology Degree, I pursued a career in neuroscience at the University of London, UK. During my post-doctoral fellowship, I developed a SOD1 mouse model of Amyotrophic Lateral Sclerosis (ALS) which demonstrated that protein aggregation is a prominent feature in human disease. This has led to the development of multiple therapeutic strategies focused on protein aggregation and more importantly formed the basis of the use of antisense technology to lower SOD1 protein. As Chief Scientist for The ALS Association I built a translational program bridging the gap between industry and academia, establishing initiatives for drug development, clinical trials and biomarkers,. I worked closely with the FDA in establishing the FDA guidelines for ALS. These initiatives led to the development of the first antisense therapy for neurodegenerative diseases. As Scientific Advisory to the Healey Center at MGH, I helped establish the first platform trials in ALS. As advisor to the Dementia Discovery Fund I supported the first investments for drug development in ALS . In my current role at Novartis I work in the Gene therapy translational medicine team to develop gene therapy strategies for ALS.