Inaugural ODC Rare Disease Research Symposium

October 16 | Children's Hospital of Philadelphia (CHOP)

This one-day symposium will bring together leading investigators, clinicians, translational scientists, and patient advocacy leaders from Penn, CHOP, and the broader rare disease community to discuss the latest advances in rare disease research and therapeutic development. The program will be organized around key therapeutic modalities: Gene Therapy, Gene Editing, RNA Therapeutics, and Non-Genetic Approaches to Rare Diseases, with dedicated sessions on trial design, and the role of the rare disease community in research. The day will close with a keynote by Dr. Kiran Musunuru on the future of rare disease medicine.

We anticipate a rich day of scientific exchange, with opportunities for discussion and networking aimed at accelerating collaborations and advancing therapeutic development for patients with rare diseases.

Space is limited, so we encourage you to register early. Please complete the registration form by October 1st, 2026

*agenda subject to change