Alnylam and its collaborators at Protiva Biotherapeutics, demonstrated silencing in primates of the gene for apolipoprotein B (apoB), a protein involved in cholesterol metabolism, with clinically significant efficacy as demonstrated by reductions in levels of cholesterol and low-density lipoproteins (LDL).
This peer-reviewed research, published in the scientific journal Nature, represents a major advance because it suggests that an RNAi therapeutic can be effective when delivered systemically using a dosage appropriate for application in future human clinical studies.
In the published research, Alnylam scientists and collaborators demonstrated potent silencing in primates of the gene for apoB, a disease-causing protein which to date has not been amenable to targeting with traditional small molecule, protein, or antibody therapies.
“We believe that these findings both advance the field of RNAi and expand the opportunity for RNAi therapeutics, as they represent a launching pad to extend beyond our current clinical efforts with direct RNAi therapeutics and address the broader potential of this promising technology with systemic RNAi,” said Dr John Maraganore, president and CEO of Alnylam Pharmaceuticals.
“These data give us confidence that with further optimization of our systemic RNAi platform we can move a systemic RNAi therapeutic candidate into human clinical trials as early as the next 18-24 months.”