Silence Therapeutics plc (LON:SLN) Chief Executive Officer Ali Mortazavi caught up with DirectorsTalk to discuss in vivo CRISPR/Cas9 gene editing data
Q1: Ali, you published some interesting news out this morning regarding in vivo CRISPR/Cas9 gene editing. In layman’s terms can you talk us though the highlights?
A1: We’re able to mediate gene editing in vivo, meaning that we can make a cut in the DNA in a specific gene to disrupt its expression permanently, so far we have sustained data over more than 200 days. We have shown this in a biological system (mouse) and specifically targeting the liver. This is relevant for severe diseases where a specific gene is known to be problematic. The anticipated delivery challenges led to the perception that the gene editing field would be heavily limited to ex vivo applications, outside the body. Therefore, this provides strong proof-of-concept for in vivo gene editing.
Q2: What does this mean for Silence Therapeutics?
A2: It means that our liposomal delivery systems have experienced a renaissance in a way, as we’ve been able to tweak them and adapt them to large cargos to mediate CRISPR/Cas9, while simpler conjugated delivery (GalNAc) is our system of choice to deliver siRNA, which are smaller, to liver.
Q3: What does this mean for investors?
A3: There is a lot of interest in this area if you look at the valuations of CRISPR companies and there is potential for partnerships which we are already exploring.
Q4: So, what are the next steps?
A4: The next steps for Silence Therapeutics plc is to complete the dataset so we can continue to pursue collaborations, joint ventures and partnerships.
