Morning Workshop: Expanding Rare Disease Targeting Using Lessons Learned from Baby KJ’s Personalized CRISPR Therapy
Time: 9:30 am
day: Day Two
Details:
Following the groundbreaking work to launch the world's first personalized CRISPR therapy to treat baby KJ, a new blueprint for rare disease treatment is forming. This interactive workshop with the baby KJ team offers unmissable insights into how this pioneering approach to precision medicine has created a transformative model for targeting rare diseases more effectively and efficiently in the future.
- Explore the potential of applying base editing and other approaches to future cases treating rare diseases
- Lessons on how to fast track the progress of treatment to patients; weighing risk-benefit while building optimized preclinical and clinical treatment plans and accelerating therapeutic delivery
- Emphasizing new strategies for rare disease targeting, and learning how to build on past experiences to enable the targeting of many rare diseases
