The Complex Business Model for of the mRNA Therapeutics Sector of the Biotech Industry
Curator & Reporter: Aviva Lev-Ari, PhD, RN
Curator: Aviva Lev-Ari, PhD, RN
Curator & Reporter: Stephen J. Williams, Ph.D.
Reporter: Aviva Lev-Ari, PhD, RN
Curator: Aviva Lev-Ari, PhD, RN
The complexity of the technologies involved in development of mRNA Therapies is demonstrated by the Platform components and the ecosystem put in place by Moderna Therapeutics Inc., the leading venture in the mRNA Therapeutics Sector of the Biotech Industry
The Moderna Ecosystem
Messenger RNA (mRNA) Therapeutics™ hold the potential to transform medicine across multiple drug modalities and therapeutic areas. As the first mover and leading company in the space, our responsibility is to simultaneously advance promising internal development programs, while also mobilizing an entire ecosystem capable of propelling the field forward for patients.
We form wholly-owned ventures to focus dedicated resources and staff in key disease areas where high unmet medical needs demand safe, efficacious and innovative therapies.
- Onkaido, the first of Moderna’s ventures, formed as a wholly-owned subsidiary to develop mRNA drugs in oncology, is utilizing all of the tools and modalities developed at Moderna.
- Valera, Moderna’s second venture, is focused on the advancement of vaccines and therapeutics for the prevention and treatment of viral and bacterial infectious diseases.
- Elpidera, Moderna’s third venture, is focused exclusively on the advancement of mRNA-based treatments for rare diseases.
- Caperna, Moderna’s fourth venture, is focused exclusively on the advancement of personalized vaccines for the treatment of cancer.
We are incubating additional ventures in diverse therapeutic areas, leveraging a cross-section of relevant modalities.
Moderna is working with world leaders in key therapeutic areas where our mRNA Therapeutics™ can potentially have a profound impact on patients’ lives. We enjoy close and productive strategic agreements with:
- AstraZeneca — mRNA Therapeutics focused on cardiovascular, metabolic and renal diseases as well as cancer;
- Alexion Pharmaceuticals — mRNA Therapeutics for rare diseases;
- Merck — mRNA-based vaccines and passive immunity treatments against viral diseases, as well as mRNA-based personalized cancer vaccines for multiple types of cancer;
- Vertex — mRNA Therapeutics for the treatment of cystic fibrosis;
- DARPA (the Defense Advanced Research Projects Agency) — mRNA-based antibody-producing drugs to protect against known and unknown emerging infectious diseases and engineered biological threats;
- Bill & Melinda Gates Foundation — Combination of mRNA-based antibody therapeutics to help prevent HIV;
- Karolinska Institutet and Karolinska University Hospital — mRNA Therapeutics to treat serious diseases; and
- Institut Pasteur — mRNA-based drugs and vaccines to fight infectious diseases.
Building from our mRNA core expression platform, we have created a new scale of drug discovery and development that enables a series of new drug modalities. Each modality represents a distinct approach to using the mRNA platform to encode proteins that achieve a therapeutic benefit, enabling us to develop numerous drug candidates across a wide array of therapeutic areas.
Vaccines are substances that teach the immune system to rapidly recognize and destroy invading pathogens such as bacteria or viruses, preparing the body’s adaptive immunity for future exposure to the pathogen. Historically, vaccines have introduced immune-activating markers from pathogens into the body. Conversely, Moderna is developing mRNA-based vaccines that enable the body to produce and present immunogenic proteins to the immune system.
Moderna is also developing mRNA-based personalized cancer vaccines to prime the immune system to recognize cancer cells and mount a strong, tailored response to each individual patient’s cancer. Moderna’s technology allows for a rapid turn-around time in production of these unique mRNA vaccines.
Many diseases are caused by defects in proteins that function inside cells. Existing methods of protein-based therapy do not allow for proteins to reach the intracellular space, and as such are unable to replace the defective, disease-causing proteins within cells. Moderna’s platform allows for the development of mRNA therapies that can stimulate production of intracellular proteins as well as transmembrane proteins. This could potentially lead to a novel approach to treating a vast array of rare genetic and other diseases caused by intracellular protein defects.
Many targets for the treatment of cancer have been identified but their therapeutic potential has been limited by either the inability to access these targets, or by systemic toxicities. Moderna’s platform allows for localized expression of therapeutic proteins within the tumor microenvironment.
Antibodies are secreted proteins that bind to and inhibit specific targets. Moderna’s platform has the potential to stimulate the body’s own cells to produce specific antibodies that can bind to cellular targets.
Proteins are large, complex molecules that have many critical functions both inside and outside of cells. Moderna’s platform stimulates cells to produce and secrete proteins that can have a therapeutic benefit through systemic exposure.
Moderna’s mRNA Platform
At Moderna, we are pioneering the development of a new class of drugs made of messenger RNA (mRNA). This novel drug platform builds on the discovery that modified mRNA can direct the body’s cellular machinery to produce nearly any protein of interest, from native proteins to antibodies and other entirely novel protein constructs that can have therapeutic activity inside and outside of cells.
Our efforts are helping Moderna and the industry to flatten the mRNA learning curve across the full breadth of competencies needed to drive the platform forward, including chemistry, mRNA biology, formulation, process development, automation and high-throughput production, quality, and Good Manufacturing Practice (GMP) manufacturing.