SLN-124

 

Update: 01 February 2019

Silence Therapeutics is working on the development of therapeutic RNA molecules that use the body’s natural mechanism of RNAi to stop (or silence) targeted / specific genes in the genome from producing disease-causing proteins (i.e. gene silencing).

RNAi is the natural regulation of gene expression achieve by the body’s cells through a complex system of increasing or decreasing the production of specific gene products.

SLN124 has shown to reduce serum iron levels and balance tissue iron distribution in mice with β-thalassaemia.

Having been granted orphan drug designation by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), Phase Ib trials are planned to begin in the second half of 2019. 

 

Sources:

https://www.silence-therapeutics.com/media/1874/190115-silence-sln124-orphan-drug-status-rns.pdf