RNA Therapeutics

Translating the message

What Is RNA?

Short for ribonucleic acid, RNA is arguably the basis of life on earth, allowing organisms to pass genetic information from one generation to the next. RNA is present in all living cells, and its most fundamental role is as a messenger, carrying instructions from DNA on how to create proteins. This type of RNA is known as mRNA (messenger RNA).

RNA in Action
RNA plays many critical roles in the coding, decoding, regulation and expression of genes.

Transcription is the first step in gene expression, when a specific segment of DNA is transcribed into mRNA. When mRNA is transported out of the cytoplasm, the translation process begins. There, the mRNA is decoded, translating each triplet of its nucleic acids (called codons) into amino acids. As the ribosome assembles amino acids, they fold into the encoded protein.

RNA and Disease
There are many ways transcription and translation can go wrong. In fact, a primary cause of many chronic illnesses—from cancers to autoimmune disease—is a dysregulation of gene expression and the resulting overproduction or underproduction of proteins.

To treat these types of disease, RNA therapies have a uniquely powerful ability to regulate protein expression and restore health.

While RNA therapeutics work on their own, they can also work in combination with existing treatments, presenting an exciting and transformative approach to developing new options for people living with diseases with no current treatments.

Our RNA Medicines
Translate Bio is developing transformative RNA therapies to treat diseases caused by the dysregulation of gene expression. Currently, we are developing new approaches to treat a wide range of diseases and access new targets not currently addressable by existing approaches and modalities.

By using mRNA, the body’s own endogenous machinery for controlling protein production, we’re on a quest to combat disease from within.

en·dog·e·nous (enˈdäjənəs/) adj.
  1. proceeding from within; derived internally.
  2. Biology. growing or developing from within; originating within.

Scientific Platform

A transformative approach to RNA therapeutics