Optimizing CasPhi for MB-111: A Multi-Component Engineering Problem
Time: 12:00 pm
day: Day One AM
Details:
- Explore how MB-111, a single-dose, in vivo gene editing therapy to treat Familial Chylomicronemia Syndrome, Persistent Chylomicronemia, and Severe Hypertriglyceridemia, inactivates APOC3, resulting in durable lowering of triglycerides
- Understand that at the core of MB-111 is Mammoth Biosciences’ proprietary engineered CasPhi system, a hypercompact CRISPR nuclease derived from phage
- Investigate how engineering CasPhi required balancing an array of parameters including nuclease and guide RNA potency, fidelity and stability
