Lipid nanoparticles (LNPs) for RNA medicine and Tolero-LNPs
Lipid nanoparticles protect novel RNA-based drugs and deliver them to their sites of action in the body. ISAR Bioscience aims to reduce side effects and to make them more powerful.
Novel RNA-based vaccines and therapies will greatly enrich medicine in the coming years. RNA-based technologies have become widely known through the development of COVID-19 vaccines. To protect RNAs, they are usually “packaged” in a kind of fat droplets called lipid nanoparticles (LNPs). LNPs protect the RNAs which they contain from damage, and they help deliver them to their target sites in the body.
Although adverse effects have been rare with COVID-19 vaccines, there is clearly room for improvement in the tolerability and efficacy of LNP packaging. ISAR Bioscience, as coordinator of the large research consortium “BASE-Lipid,” aims to further exploit this potential. Our goal is to make better LNPs available and also to avoid the need for foreign licenses. Together with our consortium partners, we have filed patent applications for several novel LNPs.
Another novel development of ISAR Bioscience is represented by Tolero-LNPs. These are novel RNA drugs based on innovative, patent-protected LNPs. They contain novel lipids that not only package LNPs efficiently and stably and release them in specific organs, but which are also biodegradable and particularly well tolerated. Tolero-LNPs contain and use specific mRNAs to generate immune tolerance, thereby enabling the treatment of autoimmune diseases, which we are initially developing for Graves’ disease of the thyroid (and eye). Tolero-LNPs show favorable effects in the activation of dendritic cells from patients with this disease.
The Paul Ehrlich Institute and the Federal Institute for Drugs and Medical Devices, the competent regulatory authorities, certified ISAR Bioscience in a hearing at the end of 2024 that its Tolero-LNPs are well on the way to becoming a therapy for autoimmune diseases. We will now continue production in accordance with good manufacturing practice (GMP) and initiate further preclinical studies and toxicological tests. Our goal is to conduct clinical trials as soon as possible.
Patent
Weber C, Czaplewska JA, Ulrich S. Schubert US, Klüpfel J, Steffens E, Holthoff HP, Martin Ungerer M, Gutmann MO, Gerolimetto G, Meinel L, Wang X, Michael Dirauf M, Benjamin Friebe B (2024) Copolymers and lipid compositions for lipid nanoparticles. Patent application EP 24156905.2
