Creating a new generation of targeted therapeutics

Recombinant therapeutic proteins have had a dramatic impact on medicine and the treatment of numerous diseases. These include insulin for diabetes, antibodies, cytokines and interferons for cancer immunotherapy, human growth hormones for growth deficiencies, and several enzymes and blood factors for heart attacks and strokes.

While these advances have improved lives and changed how new therapeutics are developed, many protein therapeutics have biophysical and pharmacological limitations.

In order to overcome these limitations, therapeutic proteins are frequently chemically modified to enhance their pharmacological properties. The ability to selectively replace amino acids in proteins with a wide variety of non-natural amino acids should allow us create entirely new proteins with greatly enhanced properties enabling a new generation of tailored protein therapeutics.

An expanded genetic alphabet can encode for more diverse and novel proteins

Proteins are molecules that contain long chains formed by the stringing together of amino acid building blocks that are drawn from a standard repertoire that is the same for all living cells. A protein’s function is determined by its unique 3-D structure, which is guided by the sequence of different amino acids. The genetic code of the majority of organisms encodes the same 20 natural amino acids.

A wide diversity of non-natural amino acids can be created with useful characteristics that are not found in the 20 natural amino acids. These characteristics can be leveraged to improve delivery, potency, efficacy, selectivity and efficiency. To create more diverse protein therapeutics we need to incorporate non-natural amino acids.

Non-natural amino acids are synthesized in vitro and represent an extensive range of structural and electronic properties not found in the common 20 amino acids. With Synthorx’s technology platform, we will be able to site-specifically incorporate multiple non-natural amino acids into proteins using Synthorx’s base pair, X and Y.

Synthorx can also combine many of these modifications within a single product to develop novel classes of therapeutics. By increasing the number of building blocks used to create proteins, exciting new opportunities in protein therapeutics can be realized.

Are you looking to create new protein therapeutics? Let’s talk.