Making Sense of Nonsense Mutations

Nonsense mutations, genetic mutations that abruptly halt the construction of a specific protein before it is complete, underlies a wide range of rare, genetic diseases. Eloxx Pharmaceuticals is developing therapeutics that address this type of error by restoring production of the complete protein. We spoke to Bob Ward, CEO of Eloxx, about nonsense mutations, the company’s library of small molecule compounds that target the protein production mechanism, and why this approach may offer a way to get at hundreds of rare diseases.