Technology and Strategy Roundtables will run from 12:00-12:45 with 15 minutes for a quick summary from each table at the front of the room. These will take place in the conference rooms: look for your table number to participate.
1. Raw material supply for the commercial manufacturing of cell and gene therapies
High precision genome editing is key when developing a genetic cure for congenital diseases.
To achieve high precision, optimization of the nuclease per target sequence is a must. Our approach to achieving high precision editing with no off-targets is to employ advanced protein engineering using directed evolution.
The approach we present here is novel and broad; it opens up unlimited opportunities for gene editing therapy in cases of dominant negative mutations, compound heterozygous mutations and haploinsufficiency.