The Unlimited Possibilities of mRNA
As a molecule found within all forms of cellular life, messenger RNA (mRNA) is central to biology itself. It quite literally finds itself the messenger between DNA, the body’s genetic blueprint, and proteins, the molecules responsible for the structure, function and regulation of the body.
For a long time, mRNA was considered unstable and was relegated to the shadow of its sister molecule DNA—at least, until our founder Dr. Ingmar Hoerr discovered in the late 1990s that mRNA could be stabilized for potential use as a therapeutic molecule. Flash forward to today—CureVac’s mRNA technology has enabled the human body to produce its own medicine.
Convinced that mRNA has unparalleled potential as a drug, CureVac has embarked on an exciting mission and pioneered unexplored territory. With mRNA in our sight, the CureVac method was developed from the cumulation of many years of work, research and nucleotide analysis—all in the name of overcoming the instability of mRNA molecules. We analyzed millions of naturally occurring sequences to develop an incomparable insight into the endogenous RNA language. These findings enabled us to develop an extensive in-house nucleotide sequence library that allows us to optimally assemble the various pieces of the mRNA puzzle for the desired therapeutic use without having to rely on additional chemical modifications in the RNA.
Each CureVac product can be thought of as a tailored molecular creation in which we customize the 5’ and 3’ untranslated regions and the open reading frame to ensure translation of the mRNA sequence produces the ideal levels of protein creation in the body. All the different parts of a CureVac-customized mRNA molecule create a packaged product specially optimized for different indications.
And because so many diseases—from cancers to genetic conditions to infectious diseases—can be traced back to proteins, mRNA becomes a truly remarkable therapeutic with virtually infinite possibilities. Here are just a few areas in which we are making progress:
Our approach to mRNA-based cancer treatments enhances antigenic properties of proteins, helping to trigger the patient’s immune system to attack cancer cells. We use a special transport system, which simulates a viral infection in the body and thus optimally addresses the immune system. Our therapeutic pipeline consists of several mRNA molecules encoding specific tumor-associated antigens, which are recognized by the body and fought with the usual antiviral immune response
In addition, we attack the cancer cells directly with our RNA and specifically modify the tumor tissue environment to align the immune system with the tumor (immune modulation). We have promising Phase 1 clinical data for our potential intratumoral (tumor injection) therapies in melanomas, head and neck skin cancers (HNSCC) and adenoid cystic carcinomas (ACC).
Our latest venture is optimizing mRNA molecules to trigger production of antibodies without triggering an immune response in the patient – unlike many available immunotherapy and biologic treatments. Our antibody work has potential to protect against viruses and toxins and can be applied in many disease indications including cancer, cardiovascular diseases, infectious diseases and autoimmune diseases.
Molecular Therapies for Rare Diseases
Many rare genetic diseases are based on defective or missing intracellular or transmembrane proteins. These cannot be treated with recombinantly produced proteins, as the intracellular administration of proteins has not yet been satisfactorily resolved. We have made progress in adapting our mRNA approaches to produce high amounts of missing or defective protein directly in the cell. This allows us to address some of the most difficult rare diseases where patients have little or no other treatment options. We have preclinical data on several different molecular therapy candidates for selected indications, including hepatic genetic diseases.
Our prophylactic vaccines represent a novel technology which can enable production of safe, efficacious and cost-effective vaccines for a range of infectious diseases, including influenza and rabies. And by partnering with international non-profits, our impact can have a truly global reach.