August 29, 2016

Stephane Bancel - Exponential Health - Digital pharma #gsummit

Stephane Bancel CEO, Moderna Theraputics

Stéphane Bancel joined Moderna Therapeutics in 2011 after learning of their novel platform developing messenger RNA therapeutics™, leaving behind his position as CEO of leading diagnostics company, bioMérieux. Under Mr. Bancel’s leadership, Moderna Therapeutics has brought together a team of industry leaders and raised significant venture funding. Stéphane Bancel is named as an inventor on over 45 patent filings in the field of messenger RNA technology, the breakthrough arena in which Moderna Therapeutics is developing therapies. Development of mRNA therapeutics creates the ability to produce therapeutic proteins in vivo, allowing for drug development in multiple disease areas including cardiovascular, oncology, and rare diseases.

What if mRNA could b e a digital medicine

mRNA as software or your cellular machinery
Human body as a protein factory

Protein based drugs have been slow and difficult
Gene therapy also is taking a long time to get developed and approved

could you go in between ?

It was thought that mRNA would last only minutes and cause flu like symptoms

But make a human a protein factory inside your cells

mRNA can be combined to do cocktails of antibodies

6 months from idea to a drug ready to go

mRNA is like software
24-120 months downto 6-24 months

Want to learn 10X faster

Build the 1st digital biotech company, invest IT and automation

create a network effect by signing partnerships






Core “Expression” Platform: messenger RNA Therapeutics™

mRNA
Modified messenger RNA (mRNA) Therapeutics™ can be encoded for virtually any known protein and designed to be taken up by the cells in specific tissues and organs. Our novel chemistry enables mRNA to elude the body’s innate immune response. Once delivered, like native mRNA in healthy individuals, mRNA Therapeutics™ act as cellular software directing ribosomes to express proteins or antibodies within targeted tissues – and have the power to catalyze the expression of hundreds to thousands of proteins for each mRNA molecule.

mRNA drugs drive therapeutic benefit by virtue of the proteins they encode. Because of mRNA’s ability to direct transient, dose-dependent intracellular expression of protein, we are witnessing the creation of a series of new drug modalities:

  • Secreted proteins
  • Antibodies
  • Combination therapy
  • Un-druggable intracellular pathways
  • Regenerative medicine
  • Vaccines

Through these modalities, our mRNA Therapeutics™ hold the potential to address a myriad of serious diseases that are not treatable through current approaches. To find out more, click here.

We have invested to create a new scale of drug discovery and development at Moderna. Our mRNA drugs can be made and dosed with unprecedented precision and speed, vastly reducing the time and costs of traditional drug development.

As a platform, mRNA Therapeutics™ have unique advantages for the development of novel mRNA drugs:

  • Native, wild-type proteins and antibodies are produced and studied in vivo, negating the need for extensive in vitro biology research;
  • mRNA drug candidates can be developed and tested in a matter of weeks, enabling an expedited process from concept to first-in-human studies;
  • All mRNA Therapeutics™ are made using the same reagents in the same cell-free production process, enabling rapid, cost-effective GMP manufacturing. This is possible due to the similarities among all messenger RNAs, which vary only in their RNA sequence.
In preclinical, in vivo, proof-of-concept studies, mRNA Therapeutics™ induced the production of dozens of effector proteins and have actually repaired and regenerated damaged tissue to achieve long-term therapeutic effects. What’s more, mRNA Therapeutics™ may address hundreds of diseases not treatable today. While current technologies target only secreted proteins (those that circulate in the bloodstream), mRNA Therapeutics™ can produce both secreted and intracellular proteins (those that exist inside cells), enabling the development of new medicines for disease targets that are untouchable with today’s injectable therapies.








Manufacturing and Automation

At Moderna, we are speeding the development of a series of new drug modalities, each with the potential to generate its own product development opportunities in many therapeutic areas. We are committed to developing the broadest possible array of drugs imaginable and to developing them simultaneously — to deliver on the promise of messenger RNA (mRNA) science for patients as quickly as possible.

We are investing in the build-out of highly automated production and manufacturing capabilities to power our discovery and development efforts at a scale and speed that is truly differentiating. Deploying the most advanced robotics technology, we are automating every step of the process — from filtering and purification, to the creation of each mRNA construct, to the rapid production and manufacture of those constructs for discovery and development.

In addition, we have pioneered a first-in-class drug discovery software solution which enables scientists working with Moderna to design, order, and optimize a drug in a matter of minutes, for delivery in weeks, from a cloud-based portal.

With this technology, we can achieve:

High levels of purification;
Unprecedented scale, with the capacity to produce dozens to hundreds of unique mRNA constructs per week, and thousands per year, for preclinical and clinical testing — with a cycle time of a handful of weeks;
Automated high throughput, enabling rapid scientific advances in our mRNA Therapeutics™ platform — such as a 1,000-fold increase in protein expression potency in a 12-month span; and
Unique levels of control and versatility, with the ability to adjust and course correct with speed, enabled by constant robotic reporting and data-generation.

At Moderna, we are on our way toward reducing the drug manufacturing cycle from weeks or months to just days — speeding our own mRNA scientific research efforts as well as those of our growing number of partners and ventures.



2 vaccines mRNA based in clinical trials

30 days to make a personalized cancer vaccine (working with Merck), in clinic next year

Rare genetic diseases

Regenerative medicine Grow back heart tissue after heart attack
Clinical trial in the fall in germany

FDA is improving by starting better balance risk and reward

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