AveXis has announced that they have completed enrollment in a Phase 1 study testing systemic delivery of gene therapy for SMA.
The trial has enrolled a total of 15 patients who met enrollment criteria of diagnosis of SMA Type 1 before six months of age, with two copies of the SMN2 backup gene, as determined by genetic testing. The trial includes two dosing cohorts:
- Cohort 1 includes three patients dosed at (6.7 X1013 vg/kg), aged six to seven months at time of dosing;
- Cohort 2 includes 12 patients dosed at (2.0 X1014 vg/kg), aged one to eight months at time of dosing.
“We are pleased to have reached this important initial clinical milestone in our effort to bring AVXS -101 to patients who suffer from SMA Type 1, a devastating disease for which there are currently no FDA-approved therapies,” said Sean Nolan, President and Chief Executive Officer of AveXis. “The data from this initial trial will inform future clinical studies in Type 1 and potentially other types of SMA. We look forward to reviewing the data from this study over the coming year as we continue the development of AVXS-101.”