News and press releases about the Joshua Frase Foundation and its efforts behind CNM/MTM

Tue, Jul 07, 2020

Gene Therapy: It’s Time to Talk about High-Dose AAV

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