Novel Approach to Treating Rare Disease Using an AAV Vector to Deliver Gene Editing Tools to the Brain
Time: 2:00 pm
day: Conference Day Two
Details:
- Provide overview of the development of LETI-101, a preclinical stage AAV5-delivered Life Edit® nuclease and guide RNA for mutant allele-specific editing in Huntington’s Disease
- Share approach for assessing manufacturability of LETI-101 through linking preclinical and CMC efforts
- Discuss the importance of biotech “owning” the future CMC problems at an early stage of pharmaceutical development to de-risk clinical trials and commercialization
