Audentes Therapeutics announced positive interim data from the group of patients receiving the first dose of the gene therapy clinical trial ASPIRO. They reported
– significant improvements in neuromuscular function
– significant improvements in respiratory function
– multiple developmental milestones achieved at 12 weeks in the first treated patient

This is very positive and significant news. Audentes have however reminded the patient community that this is still preliminary data, and that they “will only have more complete information about the potential efficacy or safety of the investigational gene therapy product after all enrolled patients are dosed and the full scope of data is collected from the clinical trial.”

Audentes is currently reviewing the interim efficacy and safety data with an independent committee prior to initiating enrollment and dosing of the next group of patients. The second update on ASPIRO is planned for quarter 2 of 2018.

Read the Audentes letter to the patient community here

Read the Audentes 4th January press release here

See the 4th January presentation by Audentes announcing these preliminary results

A very important question that emerges from the patient community at this phase in a research study is the biotechnology company’s approach to early access to the potential drug. Audentes have updated their website on their current position on Expanded Access. Read here