Jeff Milton of BajaBio explains the proposed discovery and development process for a therapy targeting Partaap’s SPTLC2 mutation—from long-read sequencing and compound design to testing and the planned safety studies. 6 minutes 16 seconds. The programme and timeline described are proposed and may change as the work progresses.
What the video covers
Jeff Milton describes sequencing to identify potential targets, designing and making candidate compounds, and testing them in patient cells. He then outlines planned safety studies and clinical preparation. These are proposed development steps, not confirmation of an effective treatment or a guaranteed start date.