Shaking up the industry, redefining RNA medicine


near-clinic programs


issued patents


pending patent applications

Send a Message

Translate Bio is a coalition of passionate individuals coming together to send a message: that the promise of RNA medicines can and will be actualized. We leverage our unparalleled RNA expertise, including in mRNA delivery and manufacturing, to create the industry’s most comprehensive RNA-based therapeutic platform. This platform has the potential to change the lives of people living with debilitating genetic diseases.

We are committed to building the leading portfolio of RNA medicines. Currently there are two near-clinic lead programs in cystic fibrosis and ornithine transcarbamylase (OTC) deficiency, a urea cycle disorder. Other programs in rare liver and lung disease are currently in early development.

In addition, we actively seek partnerships and licensing opportunities in areas of unmet need, including immuno-oncology, vaccines and metabolic, ophthalmologic and neurodegenerative diseases.

We are rapidly advancing RNA therapeutics. Here’s how:

  • Two Near-Clinic Programs: Lead programs in cystic fibrosis and ornithine transcarbamylase (OTC) deficiency, a urea cycle disorder, bring differentiated therapeutic value, offer significant commercial potential, and are both slated to enter the clinic in 2018.
  • Comprehensive RNA platform with mRNA focus: Delivery of mRNA to multiple tissues, in vivo proof of concept across a variety of disease models and a proprietary cGMP manufacturing process for unprecedented yield, quality and potency.
  • Broad Therapeutic Potential: Our platform for powering endogenous protein production applies to a broad range of diseases in which the body’s protein production is deficient. Currently we’re focused on a set of specific rare diseases, but the full potential of the platform extends beyond these programs.
  • Comprehensive Intellectual Property Protection: Over 10 US patents issued; over 250 applications pending, covering all technologies.
  • Strong Leadership and Capitalization: Translate Bio is powered by deep RNA expertise and broad biotech industry experience, with major progress towards entering lead programs into clinic.

Let’s Talk

We’re interested in partnering with other dedicated experts to transform RNA science into meaningful therapies