RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.
Shortly after birth, Kim Nye’s first daughter Tessa began suffering seizures. Though Nye gave birth to other children without significant health issues, when her fourth child Colton was born, he had the same symptoms as his oldest sister. The two siblings were eventually diagnosed with an ultra-rare genetic disorder and Nye began the TESS Research Foundation. The organization has been pursuing a gene therapy, which a biopharmaceutical company is now working to develop. In this second part of our series on gene therapies, we spoke to Nye about her own journey to becoming a rare disease advocate, how a gene therapy fit into her organization’s research agenda, and the considerations she is weighing before dosing her children with an experimental gene therapy.
