Scientific Platform

A transformative approach starts within

Healthy Expression

Translate Bio is dedicated to transforming RNA science into the healthy expression of proteins. Our medicines power endogenous protein production and therefore combat disease from within.

We are currently developing ways to use the body’s own mechanisms to fight a wide range of diseases for which there are currently no truly effective treatments.

mRNA Ingenuity
Our team of scientists has developed novel therapeutic messenger RNA (mRNA) designed to enable the in vivo production of both intracellular, membrane-associated and secreted proteins. In other words, our technology enables a patient’s own cells to produce the proteins needed to treat or prevent diseases that cannot be addressed with current technologies.

Figure 1. An mRNA treatment, introduced into the body, is translated by the ribosome into a much-needed protein.

Inside our cells, mRNA supplies the biological instructions for producing protein. Proteins play a vital role in our health. Many diseases are caused by the over- or underproduction of critical proteins due to a missing or mutated piece of the genetic code. Our mRNA medicines are designed to deliver a new set of instructions, which cells can directly translate into protein (Figure 1).

In some cases, the goal is to replace a missing or defective protein completely, while in others, supplementing an underproduced protein is all that is required to restore healthy expression.

Best-in-Class Delivery and Manufacturing
Translate Bio leads the industry in addressing two historical roadblocks to mRNA therapeutics development: delivery and manufacturing.

Figure 2. A Delivery methods, like this PEG-and-lipid nanoparticle, protects the mRNA medicine until it arrives at a therapeutic destination.

For nearly a decade, our team of scientists has worked to overcome delivery challenges with our exclusive lipid licenses, proprietary custom lipids, formulation designs and optimized coding sequences for each route of administration (Figure 2). Furthermore, our proprietary cGMP manufacturing process produces unprecedented yield, quality, and potency.

With our best-in-class formulation and delivery technology, as well as significant advantages in manufacturing scalability and quality, we are equipped to rapidly develop and deliver new, safe and effective medicines.

Oligonucleotides – Another Modality for Healthy Expression
Translate Bio was founded based on technology developed in the laboratory of Jeannie T. Lee, MD, Ph.D., a medical researcher at Massachusetts General Hospital. Dr. Lee discovered and explained a key role of long non-coding RNA in epigenetic regulation and its role in developmental biology and disease.

Today, Translate Bio continues to build on Dr. Lee’s novel findings with early work in oligonucleotide therapeutics aimed, as with our mRNA, at upregulating gene expression and protein regulation for therapeutic benefit. Oligonucleotides have unique attributes in their manufacture, delivery, mechanism of action and duration of effect that may provide us with another valuable approach in our ongoing fight against genetic diseases.

Emerging Applications of Our RNA Technology Platforms
The Translate Bio team is exploring additional applications for our groundbreaking mRNA technology, including:

  • Gene editing utilizing a nonviral, non-integrative delivery method that allows for safer, repeated administration of therapies
  • Endogenous production of therapeutic antibodies with fewer negative immune responses and easier administration
  • Rapid, flexible development of vaccines for immuno-oncology and infectious diseases

Areas of Focus

Learn where our mRNA technology is having an impact