Amsterdam Molecular Therapeutics (AMT) has revealed positive preclinical data from a study using an AAV gene therapy product to lower cholesterol.
The data showed that a single dose of the gene therapy, which carries a short hairpin RNA to silence Apolipoprotein B100 (ApoB100), resulted in a reduction of serum cholesterol of approximately 80%, without any signs of toxicity.
AMT’s CEO Jorn Aldag said that a long-lasting, significant reduction of serum cholesterol can be achieved with just a single dose of the company’s cholesterol-targeting gene therapy.
“This preliminary study suggests that AMT’s technology may have overcome one of the major problems of shRNA therapies, namely efficient and non-toxic intracellular delivery,” said Aldag.
In the study, AMT used its proprietary AAV-based platform to efficiently deliver a shRNA that silences both human and mouse Apolipoprotein B100 (ApoB100).
ApoB100 is the structural protein of low density lipoprotein (LDL) particles that carry cholesterol.