Movement Towards the Clinic: Preclinical Development of Gene Activation Programs with CRISPR-GNDM® Platform
Time: 12:00 pm
day: Day Two
Details:
- Demonstrate the efficiency and durability of gene activation by Cas-9 mediated in vivo epigenome editing
- Understand the impact of using a muscle tropic AAV capsid delivery system
- Highlight that despite sustained Cas-9 expression and limited immunogenicity, favorable toxicology data support the therapeutic safety profile
