Chair: Yael Weiss, MD, PhD, Vice President Business Development, Ultragenyx
11:50-12:00 Yael Weiss, MD, PhD, Vice President Business Development, Ultragenyx: “Opening Remarks”
12:00-12:15 Julia Hawkins, Partner, LocalGlobe: “The Journey of a Parent to a Child with a Rare Genetic Condition”
12:15-12:35 Tal Zaks, MD, PhD, Chief Medical Officer Moderna Therapeutics: “Moderna mRNA Therapies for Rare Genetic Diseases”
12:35-12:50 BatSheva Kerem, Founder, SpliSense: “Antisense Oligonucleotide Based Treatment for Cystic Fibrosis”
12:50-13:05 Robert Ward, CEO, Eloxx Pharmaceuticals: “About Eloxx Pharmaceuticals”
13:05-13:20 Yael Weiss, MD, PhD, Vice President Business Development, Ultragenyx: “From Small Molecules to Gene Therapy and Everything in Between”
13:20-13:35 Prof. Nissim Benvenisty, MD, PhD, New Stem Ltd: “Human Haploid Pluripotent Stem Cells”
13:35-13:50 Aleks Schein, ART BioScience: “mRNA Therapeutics for the Treatment of Rare Diseases”
13:50-15:20 Lunch Break
15:20-15:35 Natalie Yivgi Ohana, PhD, Founder & Chief Scientist, Minovia Therapeutics: “Mitochondria Augmentation Therapy for Rare Mitochondrial Diseases”
15:35-15:50 Or Kakhlon, Department of Neurology, Hadassah Medical Organization: “Using Small Molecules to Treat Glycogen Storage Disorders: New Mechanistic and Translational Insights”
15:50-16:05 Chris Adams, PhD, MBA, Chief Executive Officer, Cydan II Inc: “Rare Disease Company Formation”
16:05-16:20 Daniel Fischer, Founder at Tevard Biosciences: “A Parent on the Race to Find a Therapy for Dravet Syndrome”
16:20-16:35 Tauhid Ali, PhD, Vice President and Head of TAK-celerator, Takeda: “A Rare Disease Unit within Big Pharma”
16:35-17:15 Panel: Rare Diseases – Not a Rare Success. Developing Therapies for Rare Genetic Diseases
Moderator: Yael Weiss, MD, PhD, Vice President Business Development, Ultragenyx
Panelists:
Julia Hawkins, Partner, LocalGlobe
Tauhid Ali, PhD, Vice President and Head of TAK-celerator, Takeda
Chris Adams, PhD, MBA, Chief Executive Officer, Cydan II Inc.
Daniel Fischer, Founder at Tevard Biosciences
