Joshua Frase Foundation

A week before the 2020 virtual American Society for Gene & Cell Therapy conference, a tragedy took place in the rare disease and gene therapy communities. On May 6, the gene therapy company Audentes (acquired last year by Astellas Pharma) notified the X-linked myotubular myopathy (XLMTM) community that a child in the ASPIRO trial (NCT03199469) had died from sepsis. An investigation began with the help of FDA regulators to determine whether this was related to treatment with a high-dose adeno-associated virus (AAV) therapy.¹