The Future: How Baby KJ’s Personalized CRISPR Therapy Will Facilitate Better Rare Disease Targeting
Time: 9:35 am
day: Pre-Conference Workshop Day
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. With the baby KJ team swiftly adapting base editing technology, rapidly co-ordinating regulatory approval and redefining clinical strategies, this session offers an unmissable insight into how this pioneering approach to precision medicine has created a transformative model for targeting rare diseases more effectively and efficiently in the future.
This Workshop Will Gather Experts to:
- Explore the base editing approaches, regulatory interactions and preclinical and clinical strategies which were quickly adapted to treat a young baby with CPS1 deficiency
- Reflect on how these strategies enabled the fast-tracked progress of this treatment to the patient to build optimized preclinical and clinical plans and accelerate therapeutic delivery
- Emphasize how this new type of personalized medicine paves the way for ‘CRISPR-on-demand’ to enable better rare disease coverage
