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MIT Technology Review interviewed Synthorx president and CEO, Court Turner, to discuss how the company is pursuing breakthrough medicines with proteins unlike any seen in nature.

Proteins are formed by stringing together amino acids in a specific order determined by our genetic code.  The genetic code of the majority of organisms encodes the same 20 natural amino acids.

A protein’s function is determined by its unique 3-D structure, which is guided by the sequence of different amino acids. Scientists have made thousands of new synthetic amino acids in the lab, but it is very difficult to efficiently incorporate them into functional proteins and to do so at scale.  That’s where Synthorx’s expanded genetic alpabet technology comes in.

In Synthorx’s platform, special bacteria use our synthetic DNA bases (X and Y) along with A, T, C and G.  By introducing X and Y into the genetic code, Synthorx has developed a method for working with 256 codons, which can now be used to add multiple synthetic amino acids into novel proteins.

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.

“A lot of those amino acids are pretty redundant,” Romesberg says. “And to a medicinal chemist who spends their career making drugs, that must look extremely limiting.”

Synthorx can produce a wide variety of unique proteins that couldn’t otherwise by synthesized using naturally occurring codons alone.We can use this platform to screen through new proteins to discover novel drugs with improved therapeutic potential. We can also add synthetic amino acids to existing proteins to improve function.


Read the full article in MIT Technology Review.

Contact us to discuss how Synthorx’s technology can help develop new protein therapeutics.