Recovering What’s Lost in Translation
Description
When Daniel Fischer’s daughter Natasha was diagnosed with the rare genetic epilepsy, Dravet syndrome, his search for treatments eventually led him to tRNA therapies, an emerging area of genetic medicines that work to correct so-called nonsense mutations. Nonsense mutations prematurely cause the translation of a gene to stop before a protein is fully formed. What’s particularly compelling about the approach is that a single therapy has the potential to correct any nonsense mutation, regardless of the size of the gene or the gene in which the mutation occurs. We spoke to Fischer, CEO of Tevard, about his own journey as the parent of a child with a rare disease, how it led to his co-founding Tevard and its pursuit of tRNA therapies, and why this type of genetic medicine holds promise for so many people with rare diseases.
United States
