Background and aim: Osteoarthritis (OA) is characterized by chronic joint pain and functional impairment and imposes a huge burden on the individual patient and health care systems. Current treatments relieve symptoms, but do not counteract disease progression. Intra-articular (in-joint) gene therapy-like mRNA therapy offers a promising highly innovative solution for the treatment of OA.
Despite their great promise, most of the current OA gene therapies rely on viral vectors for transfection, which are associated with high safety risks and costs, and have low transfection rates due to poor penetration in the extracellular matrices of joint tissues. This project proposes a novel approach, using polymer nanoparticle-based delivery of stabilized mRNA candidates. The patented nanotechnology of 20MED is combined with the proprietary stabilized non-immunogenic mRNA technology of ETHRIS, to deliver a non-viral mRNA-based ‘transcript therapy’ for injection into the joint. The preclinical efficacy will be tested in ex-vivo joint bioreactors and rat disease models by ARI and Paracelsus Medical University. The aim is to generate preclinical data with proof of concept for the novel mRNA transcript therapy.
Eurostars; ARI Funding EUR 200,000; Period 2019-2022.
Engbersen J, 20Med Therapeutics, NL
Planck C, Ethris Gmbh, DE
Traweger A, Paracelsus Medical University, AT