There are many companies looking to develop new treatments for Huntington's disease. Alnylam are looking at gene silencing medicines. Read on to find out more about what this is.
Alnylam has pioneered RNA interference (RNAi) therapeutics, which are a type of gene-silencing medicine. RNAi therapeutics treat diseases in a different way - by silencing the genes that cause or contribute to them. RNA interference (RNAi) is a naturally occurring process already at work inside your body which regulates how genes make new proteins. Proteins play a critical role in almost everything your body does, but sometimes, they can cause or contribute to disease. RNAi ’silences’ the production of unwanted proteins to treat disease at its source.
Alnylam has already developed medicines using this technology in other disease areas and are now investigating a potential new medicine for the possibility of developing one for treatment of Huntington’s disease. The company is conducting a Phase 1b clinical trial of ALN-HTT02, an investigational RNAi therapeutic that targets exon 1 of HTT with the aim of reducing all forms of mutant Huntingtin protein. The trial will assess the safety, tolerability, and target engagement, and is currently recruiting adult patients in the UK, Canada and Germany.
Alnylam looks forward to continuing to work with the Huntington Disease Association and the wider patient community as they progress our development program.