Our partners at DTx Pharma continue to make great progress on suppressing the overexpression of PMP22, which causes CMT1A. DTx Pharma’s proprietary technology has the potential to render RNA-based therapeutics such as ASO and siRNA more potent, safer and longer-acting and to move this technology closer to the clinic for CMT1A patients.

The first milestone in the project has been successfully completed. DTx screened a series of siRNAs in cultured cells and identified a number that strongly decrease the expression of PMP22. Furthermore, their proprietary technology is very successful in increasing the delivery of siRNAs to cultured cells. The next step is to determine how well these siRNAs that target PMP22 can be delivered to the peripheral nervous system of mice. Additional experiments will also be performed using ASOs.

Despite the COVID-19 pandemic, the project is on track and the community can expect further updates in mid-2020.