Senior VP Patrrick Baumhof, CureVac

mRNA therapeutics for treatment of liver fibrosis

The focus of the talk will be development of an mRNA therapy for the injured liver by a novel transcription factor based concept

As a first step a formulation was identified that delivers mRNA to liver hepatocytes into injured liver with the help of report protein coding mRNA. As a next step a transcription factor was identified that is connected with the liver disease and that was supplemented by mRNA based therapy. In three different disease models the functionality of the concept was shown to be efficacious, returning a diseased live to nearly normal stage. Nanoparticular mRNA therapeutics are a new class of medicine that is offering new therapeutic applications.

References
Therapeutic HNF4A mRNA attenuates liver fibrosis in a preclinical model
Taihua Yang, Marion Poenisch, Rajendra Khanal, Frédéric Chevessier,, Michael Ott, Amar Deep Sharma
Journal of Hepatology 2021 vol. 75 j 1420–1433 DOI:https://doi.org/10.1016/j.jhep.2021.08.011

Lipid nanoparticle delivery of unmodified mRNAs encoding multiple monoclonal antibodies targeting poxviruses in rabbits
Eric M. Mucker,1 Carolin Thiele-Suess,2 Patrick Baumhof,2 and Jay W. Hooper1,∗

Unmodified mRNA in LNPs constitutes a competitive technology for prophylactic vaccines
Johannes Lutz, Sandra Lazzaro, Mohamed Habbeddine, Kim Ellen Schmidt, Patrick Baumhof, Barbara L. Mui, Ying K. Tam, Thomas D. Madden, Michael J. Hope, Regina Heidenreich, Mariola Fotin-Mleczek
NPJ Vaccines. 2017

Proximity Ligation Assays for In Situ Detection of Innate Immune Activation: Focus on In Vitro-Transcribed mRNA
Emmeline L. Blanchard, Kristin H. Loomis, Sushma M. Bhosle, Daryll Vanover, Patrick Baumhof, Bruno Pitard, Chiara Zurla, Philip J. Santangelo
Mol Ther Nucleic Acids. 2019 Mar 1; 14: 52–66