Brad Ringeisen
Seminars
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
- Evaluate partnerships between pharma and biotech companies and the impact academia can have on the development of genome editing therapies
- Discuss the ideal criteria for company collaborations and investment to improve discovery efficiency
- Highlight key strategies from successful collaborations to direct and refine future collaborative efforts
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
