UCSF Rare Disease Symposium
Catalyzing Innovation and Bridging Gaps in Rare Diseases
Program
In recognition of the over 300 million people worldwide with a rare disease, this symposium highlights translational research and innovative start-ups focused on rare eye diseases and epilepsies.
Panel 1
AGENDA
8:30 am – 9:00 am – Check-in with Light Breakfast
9:00 am – 9:15 am – Welcome and Introductions
9:15 am – 9:40 am – “Chasing My Cure” – David Fajgenbaum (UPenn) in conversation with Jim Levine
9:40 am – 11:25 am – Morning Case Studies (Rare Eye Diseases)
9:40 am – 10:00 am – Journey as a Patient and Researcher – Van Duesterberg (UCSF)
10:00 am – 10:30 am – Re-writing RNA for Stargardt Disease – Mike Ehlers (Apple Tree Partners)
10:30 am – 10:55 am – More than Target Practice: Resurrecting Two Drugs – Gary Hammer (Univ. of Michigan)
10:55 am – 11:25 am – Rare Eye Disease Startup Panel Discussion – Daniel Chung (Sparing Vision), Lukas Scheibler (Apellis)
11:25 am – 12:00 pm – Gene Therapy for ART-SCID: An Ultra Rare Disease – Morton Cowan, Jennifer Puck, and Cammie Edwards (UCSF)
12:00 pm – 12:45 pm – Lunch Break
12:45 pm – 1:00 pm – In Conversation – Sparsh Shah (Musician, Speaker, Patient) and Roopa Ramamoorthi (UCSF)
1:00 pm – 1:30 pm – Early Afternoon Keynote – Kathryn North (Murdoch Childrens Research Institute)
1:30 pm – 3:00 pm – Afternoon Case Studies (Rare Epilepsies)
1:30 pm – 2:00 pm – Rare Epilepsies Keynote – Dan Lowenstein (UCSF)
2:00 pm – 2:20 pm – Startup Spotlight
2:20 pm – 3:00 pm – Rare Epilepsies Panel Discussion – Scott Baraban (Epygenix), Navneet Matharu (Regel Therapeutics)
3:00 pm – 3:30 pm – Machine Learning for Drug Discovery – Daphne Koller (Insitro)
3:30 pm – 3:45 pm – Concluding Remarks
4:00 pm – Social Hour