Audentes Therapeutics has indefinitely delayed plans to seek regulatory approval for its rare disease gene therapy after the death of a third patient involved in a clinical trial of the drug.
This was the third death of a patient involved in the Audentes’ ASPIRO clinical trial, which is evaluating its AT132 gene therapy in patients with X-linked myotubular myopathy (XLTM), a rare neuromuscular disease.
XLTM mainly affects males and causes muscle weakness that ranges in severity from mild to life-threatening. In severe forms of the disease, the weakened muscle make breathing difficult and can lead to respiratory failure. The genetic disorder has a mortality rate of around 25% by the age of ten.
AT132 is an aden-associated virus (AAV) based gene therapy that is designed to deliver the MTM1 gene deficient in XLMTM. According to Audentes, preliminary findings show that the immediate cause of death of the third patient was gastrointestinal bleeding.
Read more: http://www.pmlive.com/pharma_news/audentes_rare_disease_gene_therapy_programme_indefinitely_delayed_after_third_patient_death_1347266