Applying a Disruptive Technology to Biopharma

Our strategy to de-risk and advance the disruptive technology of mRNA involves a focus on what we refer to as "modalities." A modality is a technological solution set that can be deployed to create a family of medicines for different diseases within one therapeutic area, and often across therapeutic areas. Within each modality, we first pursue diseases where we believe the biological risks are low. Once we’ve demonstrated success, we then tackle increasingly more complicated diseases within that modality. And it’s here that the scale and speed of using mRNA as a drug starts to become evident.

De-risking the Portfolio

The first place we’ve seen success within a modality is in the vaccine space.  We began with intramuscular (IM) delivery of viral vaccines in influenza. We developed mRNA that encoded for a single antigenic protein for this single pathogen. Based on where the science led us, this presented the lowest hurdle from a biological standpoint.

Our ‘operating system’ now has all of the necessary components in place to develop a monovalent viral vaccine (meaning a vaccine directed at one pathogen. Our first two monovalent viral vaccines (both for Influenza subtype A viruses) are in clinical development, and we have dosed more than 300 healthy subjects to date.

We are now able to quickly move other monovalent viral vaccines from initial concept to development candidate nomination at an accelerated pace. We have two more vaccines that have entered the clinic, and we have several more that will enter clinical study in 2017. Pipeline >

Portfolio growth within modalities

With early technology hurdles  overcome, we are now using the IM vaccines modality to create vaccines for diseases that involve more complex biology risk. For instance, we are developing vaccines with multiple mRNAs that encode for a multi-protein complex that acts as a single antigen from a single virus, as well as combination vaccines with multiple mRNAs to protect against infection from two or more distinct viruses.

Additionally, we are advancing our mRNA-based personalized cancer vaccines, which are a highly complex application of our IM vaccines modality. Rather than prevent infection from a pathogen, we are harnessing the immune system to identify and attack cancer.

Development of new modalities

We have applied learnings and advancements from the IM vaccines modality to tackle modalities that pose greater technical hurdles.

We have made marked progress with our localized therapeutics modality, in which we inject mRNA directly into tissue to elicit protein expression. With this modality, we are advancing two immuno-oncology development candidates that are delivered into a tumor to help fight cancer, and a cardiovascular development candidate that one day may provide a unique regenerative treatment option for patients with heart failure or after a heart attack, as well as other ischemic vascular diseases.

Other modalities are earlier stage, as we continue to refine our technology in the discovery stage. These include intravenous (IV) systemic therapeutics, IV liver therapeutics and inhaled pulmonary therapeutics.

Once we have overcome the biology and technical risks in the initial application in other  modalities, we anticipate seeing the same vertical growth within those modalities.

Partnering with World-Class Therapeutic Experts

The partnerships we have established have been a key enabling factor in helping us explore and introduce new mRNA modalities. We have partnered with biopharma companies who are world renowned leaders in various therapeutic areas. By combining their expertise in a given therapeutic area with our expertise in mRNA science, we have been able to accelerate the promise of mRNA drugs across a broad set of applications and disease areas.