Pre-Conference Workshop Day
8:30 am Workshop Check-In & Light Breakfast
9:30 am – 12:30 pm Workshop A
The Future: How Baby KJ’s Personalized CRISPR Therapy Will Facilitate Better Rare Disease Targeting
Synopsis
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
12:30 pm Lunch Break & Networking
1:30 pm – 4:30 pm Workshop B
Optimizing LNPs to Efficiently & Safely Reach Hepatic Tissues & Beyond
Synopsis
With delivery being an unwavering and multi-faceted challenge for gene therapy, continuous innovations are being made across viral and non-viral delivery vehicles. Whether these developments look to enhance targeting to the liver and beyond, limit immunogenicity, increase cargo capacity or facilitate delivery of molecules outside of DNA, it is vital to keep on the pulse of the latest design and engineering approaches to improve the reach of gene therapies.
This Workshop Will Gather Experts to:
- Reinstate the importance for safety, doses and efficacy to engineer LNP delivery vehicles to avoid the liver and target specific targeting to the tissues or organs of interest
- Understand that there are a number of ways to enhance extra-hepatic delivery, including decorating LNPs with ligands on the surface which facilitate binding to the desired target and the available instruments to characterize this decoration
- Reflect that it may require a combination of different approaches for LNPs to have efficient specific targeting