Scaling CRISPR Cures to 500 Inborn Errors of Immunity and Beyod

   Fyodr Urnov – University of California, Berkeley

Advanced Clinical Therapies in Clinical Research at The Neuro

   Massimo Pandolfo + Ziv Gan-Or – The Neuro, McGill University, Montreal, QC

Multi-institutional Collaboration in Developing Personalized Treatments

   Navpreet Sangha + Stalinjeet Gill – SPG4 Hope Foundation

Base Editing in Rare Neurological Disorders

   Jagdeep Walia – Queens University, Kingston, ON

Non-Viral Genome Editing for Rare Genetic Disorders

   Yong-Hui Jiang – Yale School of Medicine, New Haven, CT

Gene Therapies for Oculopharyngeal Muscular Dystrophy (OPMD)

   Guy Rouleau – Chair of Neurology, McGill University, Montreal, QC

Neurosurgical Advances to Treat Genetic Brain Disorders

   Andres Lozano – University of Toronto, ON

Small extracellular vesicle communication to deliver RNA therapies

   Derrick Gibbings – University of Ottawa, ON

MRI-Guided Focused Ultrasound in the Delivery of Therapeutics to the Brain

   Isabelle Aubert – Sunnybrook Research Institute, Toronto, ON

Lessons learned in Therapeutic development for pediatric neuromuscular disorders

   Maryam Oskoui – CHU Ste-Justine Research Centre, Montreal, QC

The development of Onpattro for the treatment of polyneuropathy in individuals with HATTR

   Phillip Zamore – University of Massachusetts/ McGill University