Improving the Process of Rare Disease Treatment Development Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President, Ultragenyx Pharmaceutical, Inc.

Progress Toward Molecular Based Therapies for Neuromuscular Disease Jerry R. Mendell, M.D. Director, Center for Gene Therapy The Research Institute at Nationwide Children’s Hospital

Development of a 2-hydroxypropyl- β-cyclodextrin therapeutic trial for Niemann-Pick disease, type C1 Forbes D. Porter M.D., Ph.D. Senior Investigator, Program Head and Clinical Director, NICHD, NIH

Development of a Novel RNAi Therapeutic, Patisiran, for the Treatment of TTRmediated Familial Amyloidotic Polyneuropathy (FAP) Akshay K. Vaishnaw, M.D., Ph.D. Executive Vice-President and Chief Medical Officer, Alnylam Pharmaceuticals, Inc.

Exploration of AAV-Mediated Gene therapies for Inherited Ocular Disorders Gwyneth Jane Farrar, Ph.D. Professor of Genetics Smurfit Institute of Genetics Trinity College, Dublin

Gene Therapy for Haemophilia B UCL/St Jude's Trial Update at 4 Years Edward G.D. Tuddenham, M.D. Emeritus Professor of Haemophilia, UCL Katherine Dormandy Haemophilia Centre Royal Free Hospital