LNPs for delivery of antisense therapeutics to skeletal muscle

Lipid nanoparticles for the efficient nuclear delivery of antisense oligonucleotide-based therapies to skeletal muscle tissue for the treatment of metabolic myopathies

Summary of research

At the moment, there are a limited number of metabolic myopathies (MM) for which effective therapy is available. One of the reasons for this is that it is difficult to deliver medications effectively to the muscles. In recent years, a lot of research has been conducted on a new type of therapy called antisense oligonucleotides (ASOs), which is a promising strategy for treating MM. However, the challenge with this therapy is that the uptake in muscles needs to be improved. In this project, we are developing technology based on lipid nanoparticles (LNPs), which are also used for delivering mRNA vaccines, to improve the uptake of this therapy into the skeletal muscles of MM patients. An additional advantage of this form of LNP therapy is that, with some adjustments, it can also target other tissues, which could significantly expand the range of metabolic disorders for which this therapy is suitable.

Awarded grant, amount: Metakids Catalyst Grant, €20.000