Gene Therapy Situation Gets More Awkward With Another Death In Astellas XLMTM Study
Patient Received Lower Dose Of AT132 After Initial Hold Lifted
An expert pointed out that XLMTM patients are already at high risk, but if linked to therapy toxicity, the death could doom the ASPIRO trial.
You may also be interested in...
Kate is focused on delivering gene therapies for two rare, neuromuscular diseases by deploying AAV technology that it says will target muscle tissue and enable regulated therapeutic payloads.
Despite facing two clinical holds for its gene therapy candidates, Astellas is committed to proceeding with R&D projects in this modality. Outgoing CEO Kenji Yasukawa says technical challenges have motivated the firm's recent deals with academia and bioventures, and also provided updates on other key pipeline assets in the second part of this exclusive interview with Scrip.
JAMA reports on the COVID-19 Prevention Network’s efforts. Also, Inhibikase said the US FDA lifted a hold on its drug for Parkinson’s disease, while Astellas Pharma said the agency lifted a clinical hold on its gene therapy for Pompe disease. And Trefoil, Theriva Biologics and Nektar initiated new trials.