For the first time, scientists at The Scripps Research Institute and Synthorx, guided by Floyd Romesberg, Ph.D., have developed the first semi-synthetic organism that can store and retrieve increased genetic information.
The genetic code for almost all life uses four DNA “letters” (A, T, G, C). These letters are arranged into genes that encode proteins that carry out most of life’s functions.
The semi-synthetic organism was made to maintain, replicate, transcribe, and translate a synthetic DNA base pair (called X and Y) in order to incorporate various non-natural amino acids (nnAAs) into a full-length protein. Adding two more DNA bases to expand the genetic alphabet can be used to make novel proteins for improved therapeutics.
In addition, this research, published in Nature, breaks through technical barriers to creating more diverse proteins for improved drug characteristics as well as enabling cost-effective scale-up for drug development.
Synthorx is applying this breakthrough as a drug development platform to improve the properties of new and existing protein therapeutics. 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 Synthorx to create entirely new proteins with greatly enhanced properties enabling a new generation of tailored protein therapeutics.