The once-sleepy field of RNA interference drug development has picked up steam in the last few years, and rare lipid disorders are emerging as one of key regulatory testing grounds for product candidates that promise to intervene in the production of disease-causing proteins.
The well-studied biology of lipid synthesis identified opportunities for RNA therapeutics to act upstream of current dyslipidemia treatments by silencing messenger RNA to interrupt production of specific lipoproteins. Akcea Therapeutics Inc., the cardiometabolic affiliate of antisense specialist Ionis Pharmaceuticals Inc
Alnylam Pharmaceuticals Inc., meanwhile, is leading the way forward with a related RNA interference approach, small interfering RNA (siRNA). The company, which has already notched three breakthrough therapy designations and one approval for its siRNA portfolio, licensed its siRNA inclisiran to The Medicines Co