A No-Nonsense Effort to Develop a Therapy that Works Across Genetic Diseases

RARECast

Nonsense mutations prematurely end the translation of a gene into protein and can result in a serious deficiency. About 10 to 15 percent of inherited genetic diseases involve nonsense mutations. Alltrna is developing transfer RNA therapies designed to correct the problem in protein synthesis caused by these aberrations. What’s particularly exciting about the approach is that a single therapy has the potential to work across nonsense mutations regardless of the gene in which they occur. We spoke to Michelle Werner, CEO of Alltrna, about nonsense mutations, how the company’s transfer RNA therapies work, and why the approach has the potential to address so many diseases at once.