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Developing cancer and infectious disease vaccines using mRNA

CureVac GmbH is a biopharmaceutical company developing prophylactic vaccines for infectious diseases and therapeutic cancer vaccines. These are based on the application of the company’s mRNA-technology platform, RNActive, which modifies and stabilises mRNA to make it suitable for medical purposes. The technology platform allows the large-scale GMP production of mRNA vaccines for any possible target at feasible costs.

candidates. The company has established and is advancing its leadership in RNA technologies, and has a broad intellectual property base built on its RNA technology platform, RNActive. CureVac is developing its own pipeline as well as drug candidates through partnerships. CureVac is developing an entirely new class of therapies based on a fundamental new understanding of the medical potential of messenger (m)RNA as a vaccine. The company’s current focus is on the development of therapeutic and prophylactic mRNA vaccines and RNA-based adjuvants for the treatment of cancer and infectious diseases. The RNAdjuvants are comprised of modified, non-coding, long and complexed RNA molecules and have already been shown in a preclinical setting to increase the efficacy of a large number of different vaccines, including protein- and peptide-based vaccines without toxic effects. CureVac’s founders were the first to show


eadquartered in Tübingen, Germany, CureVac GmbH is an integrated biopharmaceutical company with a pipeline of promising drug

that injected mRNA can induce a robust and specific T-cell immune response, and RNActive vaccines combine both the antigenic and adjuvant properties of mRNAs. As such, both CureVac’s vaccines and adjuvants are delivered via intradermal injection. “We have the demonstrated know-how to advance our mRNA vaccines through preclinical and clinical development, therapeutically in oncology, prophylactic and infectious diseases. By making mRNA available for therapeutic purposes, we are working to lay the foundations for a novel class of drugs,” says Ingmar Hoerr, co- founder and CEO of CureVac.. CureVac’s technology was developed from the research conducted by Ingmar Hoerr and colleagues in the laboratories of Professor Hans-Georg Rammensee and Professor Günther Jung at the University of Tübingen in Germany. Ingmar Hoerr founded CureVac with Florian von der Mülbe, and postdoctoral fellow Steve Pascolo during his PhD work at the University of Tübingen. At a time when there was no scientific interest in the use of mRNA for medical applications, Hoerr and his colleagues were the first to show that

complexed mRNA could stimulate a strong specific T-cell response when delivered by direct injection into the skin. This groundbreaking finding led to CureVac’s first patents and the company was founded in 2000, securing significant seed financing and government grants. CureVac has about 100 employees and has acquired some €145 million in four private equity rounds. The completion of an €80 million financing round with its main investor dievini Hopp BioTech holding GmbH & Co KG was announced in September 2012[MS1].

Schematic of the intradermal application of RNActive vaccines. 34 sp2 Inter-Active September/October 2012

Resourceful, non-toxic molecules RNA is a family of molecules comprising many members with different functions, and an important class of biomolecules in nature. mRNA has the ability to transmit genetic information, a key process in the development and evolution of living organisms and the molecules are the blueprints of protein products, as they are comprised of 200 to 10,000 nucleotides that are translated by ribosomes into proteins. “mRNA are resourceful, non-toxic molecules that address the shortcomings of recombinant virus or DNA-based vaccination therapies,” says Ingmar Hoerr, co-founder and CEO. “RNA-vaccines do not contain promoter elements as found in DNA vaccines, which are mostly virus-derived, and vaccination with DNA has several disadvantages including the possible integration of the DNA into the host genome as the result of homologous recombination or a random event. This can lead to inactivation or activation of genes, may affect regulatory elements, and possibly induce oncogenesis. In addition, long-term and/or uncontrolled expression of the transgene or induction of pathogenic anti-DNA antibodies can occur. “CureVac’s RNActive vaccines are comprised of modified and formulated mRNA that is translated into the target antigen, and

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