Where Science Meets Silence: Innovations in Rare Diseases
đź“… February 13, 2026 8:30am-6:00pm PST
đź“Ť William J. Rutter Center (Robertson Auditorium) at UCSF Mission Bay (1675 Owens St, San Francisco, CA 94158)
Register Free
Program
The Rare Disease Symposium 2026 is hosted by the UCSF Catalyst Program and Institute for Human Genetics, in partnership with Foundation Ipsen. Rare diseases affect more than 350 million people worldwide. With a focus on the interdisciplinary development of new diagnostics, new therapeutics, and social advances for patients and their families affected by a rare disease, we hope all participants come away from this symposium with an expanded understanding of the field and the inspiration to make an impact.
Agenda
Time | Who | Title | Topic | |
Check-in with Light Breakfast | 8:30 | |||
Welcome & Opening Remarks | 9:00 | Roopa Ramamoorthi | Director, Catalyst Program, UCSF | Event logistics overview Catalyst Program and Innovation Ventures |
James Levine | President, Foundation Ipsen | Foundation Ipsen | ||
Opening Fireside Chat (tentative) | 9:15 | Gini Deshpande | CEO, NuMedii | Atul Butte’s legacy in rare cancer and “scalable privilege” |
Guy Kawasaki | Chief Evangelist, Canva | Innovation and experience with living with Meniere’s disease | ||
Rare Disease Genetics Overview | 10:00 | Nadav Ahituv | Director, Institute for Human Genetics, UCSF | Rare diseases with hearing loss overview (e.g., Stickler, Usher’s; of 7,000 rare diseases, ~400 involve hearing loss) |
Morning Panel: Drug Discovery in Rare Diseases | 10:30 | Fyodor Urnov | Director, CRISPR Cures Center | $20M initiative for pediatric rare diseases |
Aaron Tward | Chief Scientific Officer, Akouos Former UCSF Catalyst awardee | Gene therapy for hearing loss | ||
Dylan Chan or Russ Dahl | Professor of Otolaryngology, UCSF CSO, Jacaranda Biosciences | |||
Luke Gruenert | Director, Strategic Innovation, Chiesi Global Rare Diseases | |||
Photography & Human Perspective in Rare Disease | 12:00 | Rick Guidotti | Founder and CEO, Positive Exposure | Film Screening: Angelman in Frame film: Angels on Earth |
Lunch | 12:30 | |||
Afternoon Keynote (tentative) | 1:30 | James Sabry | Chief Business Officer and EVP, BioMarin | |
Patient Story (Prelude to Panel) | 2:00 | Patient speaker TBD | Personal journey with rare disease hearing loss | |
Afternoon Panel: New Technologies & Diagnosis for Hearing Loss | 2:20 | Dylan Chan | Professor of Otolaryngology, UCSF; cochlear implants expert | Pediatric hearing loss and genetics of hearing loss |
Konstantina Stankovic | Otorhinolaryngologist, Stanford | Cochlear imaging technology Revolutionary diagnostics currently available | ||
Nancy Young | Otolaryngologist, Lurie Children’s Hospital Chicago/Northwestern | AI powered personalized pediatric care | ||
Closing Patient Keynote | 4:00 | Rebecca Alexander | Author, Motivational Speaker, Psychotherapist, Mental Health Educator, Disability Rights Advocate, and Fitness Coach | Living with Usher syndrome — hearing and vision loss |
Concluding Remarks | 4:20 | Nadav Ahituv | Director, Institute for Human Genetics, UCSF | Highlighting key take aways |
Reception | 4:30 |