The Science

Restoring full-length functional proteins

A nonsense mutation is a point mutation in a sequence of the messenger RNA (mRNA) that results in a premature stop codon (PTC). As a result, the protein translation process is terminated and a truncated, incomplete, and usually nonfunctional protein is the final product. Since these proteins are responsible for many essential processes in the body, a nonsense mutation can cause a deadly genetic disease.

Eloxx compounds demonstrate the ability to restore full-length functional proteins in genetic diseases. Eloxx compounds bind to the ribosome, facilitating read-through of premature stop codons and enabling translation of full-length functional proteins.

What is a non-sense mutation?

The ribosome translates the genetic code
The translation process produces full-length proteins
Non-sense mutations create premature termination that leads to truncated proteins

How does Eloxx’s science work?

Eloxx compound (ELX-02) facilitate read-through of premature stop codons
Eloxx enables the creation of full length proteins